Blood Safety Transcripts

DEPARTMENT OF HEALTH AND HUMAN SERVICES
ADVISORY COMMITTEE ON BLOOD SAFETY AND AVAILABILITY

Fourteenth Meeting

WHAT SHOULD BE THE DEPARTMENT'S CURRENT AND FUTURE
ACTIONS UNDER THE BLOOD ACTION PLAN
TO MONITOR THE AVAILABILITY OF BLOOD PRODUCTS
IN THE UNITED STATES

Volume II

8:05 a.m.

Friday, April 20, 2001

Hyatt Regency Capitol Hill Hotel

400 New Jersey Avenue, N.W.

Washington, D.C. 20001

P A R T I C I P A N T S

Larry Allen
Michael P. Busch, M.D., Ph.D.
Rajen K. Dalal
Richard J. Davey, M.D.
Jay Epstein, M.D.
G. Michael Fitzpatrick
Ronald Gilcher, M.D.
Edward D. Gomperts, M.D.
Fernando Guerra, M.D.
Paul F. Haas, Ph.D.
William Hoots, M.D.
Harvey Klein, M.D.
John Kuhn, M.D.
Karen Shoos Lipton, J.D.
Lola Lopes, Ph.D
Gargi Pahuja
John Penner, M.D.
John Walsh
Jerry Winkelstein, M.D.
Lawrence McMurtry
Stephen D. Nightingale, M.D.
Mary E. Chamberland, M.D.
David Snyder, Rph, D.D.S.
Virginia Wannamaker

AGENDA ITEM

Statement of Issue - Stephen Nightingale, M.D.

Current Efforts to Monitor the Blood Supply

- Marian Sullivan, MS

- Edward Wallace, Ph.D.

- James Greer, Ph.D.

National Blood Data Resource Center

FDA Perspective on Monitoring the Blood

Supply - Richard Lewis, Ph.D., FDA

Current Monitoring of the Supply of Plasma

Derivatives - Jan Bult, Plasma Protein

Therapeutics Association

FDA Perspectives on Monitoring the Plasma Derivative Supply - Mark Weinstein, Ph.D.

A Distributor's Perspective on Monitoring

of the Blood and Plasma Derivatives Supplies

- Patrick Schmidt, FFF Enterprises and

Lifetree Technologies, Inc.

How Can Shortages and Potential Shortages of

Blood Products and Plasma Derivatives Best Be

Identified and Monitored? A Consumer Panel

Jason Bablak, J.D., Immune Deficiency

Foundation

Val Bias, National Hemophilia Foundation

Nancye Buelow, Alpha-1 Alliance

Jan Hamilton, Hemophilia Federation

of America

What Parameters of Blood Product Supply

Should Be Monitored? How should these

Parameters be Analyzed? Where should

Responsibility for this Activity be Placed?

An Industry Panel

Christopher Hillyer, M.D., American

Association of Blood Banks

Celso Bianco, M.D., America's Blood Centers

Jacquelyn Fredrick, American Red Cross

Public Comment

Committee Discussion and Recommendations

Adjourn

DR. NIGHTINGALE: Good morning and welcome to the second day of the 14th meeting of the Advisory Committee on Blood Safety and Availability. My name is Stephen Nightingale, and I have ten minutes allocate for the statement of the issue and I will take one.

The issue before the committee today is: What should the department's current and future actions under the Blood Action Plan be to monitor the availability of blood products in the United States? This is a technically very complex issue. It is also an issue about which there has been some very legitimate disagreement.

We are not seeking scientific closure today. What we are seeking today is the broadest range of input possible. The plan, as I enunciated yesterday for this, as for all past meetings, is to solicit public input, solicit the views of the committee, go back after the meeting and make sure that all the views of the stakeholders, interested parties, whatever you wish to call us collectively, are incorporated into a draft document which is then circulated within the Public Health Service. When consensus is reached on that draft document, it is presented to the Secretary for action, and I appreciate very much the willingness of all of you to participate in this process.

The secondary issue today is an equally simple one, perhaps even simpler. We need to get out of here early. The reason we need to get out of here early is not because this issue is not important, because there are lot of other issues of interest to the blood community that are also important. And when I said I thank you all for being here, I thank you for sharing your valuable time on this issue, and in return I recognize your interest in other issues before the community and we are trying our best to accommodate them both.

Dr. Gomperts?

DR. GOMPERTS: Thank you, Dr. Nightingale. Welcome.

In order to meet the needs of the business of the committee today, what I'm trying to plan to do is to take a late lunch; in other words, we'll aim to go through 1:00, 1:30, see how things are going, take a couple of breaks because there is a lot on the table. So we'll play as it comes from the point of view of breaks and so on.

The first presentation is Ms. Mary Sullivan, National Blood Data Resource Center, on the current efforts to monitor blood supply.

MS. SULLIVAN: Good morning. The National Blood Data Resource Center would like to thank Dr. Nightingale and the committee for bringing this important issue to the table and for allowing us to have the floor for a while this morning. I'll be the first of three speakers from the NBDRC, and I only have one slide, and that's it.

I want to very briefly highlight for you some of our recent activities at the Data Center. As most of you know, we were founded by the AABB as a wholly owned subsidiary in 1997. We are in independent, not-for-profit corporation with a separate governing board. Our mission is strictly blood-related data collection and analysis.

Since we opened our doors less than four years ago, we have completed two nationwide blood collection and utilization surveys, which cumulatively were completed by more than 2,500 institutions that transfuse and/or collect blood, including every U.S. blood center. These surveys have been financed entirely by our generous charter members, our institutional and individual members, our customers, the AABB, and most recently by a loan from the National Blood Foundation.

As you know, we have always shared these data willingly with this committee, and we plan to continue to do so, and the other advisory committees as well. And I'm proud to announce that next week we will release the report of our 2000 nationwide blood collection and utilization survey, although we realize that it may be our last report of this scope if we can't secure sustaining external funding for this biennial effort.

The databases that have resulted from these nationwide surveys serve as a valuable and largely untapped resource that provides a statistical foundation for many of our activities, such as the validation of blood data collected via other methods, the development of representative samples and other samples as appropriate of blood centers and hospitals for monitoring and for other research purposes, for weighting and adjustment of data from such samples, and these data also serve as accurate national denominator data for analyses of data from other sources.

At this point in time, the NBDRC databases collectively exceed one gigabyte, and these are all data that directly characterize blood services and related activities in the United States.

In January of last year, we began to collect monthly monitoring data for the National Heart, Lung, and Blood Institute, as called for by the Blood Action Plan. We began year one of an anticipated three-year study by collecting data from a statistically representative sample of blood centers and planned for the collection of detailed utilization data from hospitals by the second half of year one.

Dr. Jim Greer from the Data Center will describe in much greater detail in a moment the blood center sample and the variables that have been collected.

The NHLBI, with the benefit of Dr. McCurdy's guidance and expertise, determined the variables to be included in the monthly data from the blood center sample and initially assumed the primary responsibility for the analysis and interpretation of these data, which they purchased from us in unlinked format. These data were usually delivered to NHLBI within three weeks of the close of each month.

We did add one variable, however, to the set which we at the Data Center particularly wanted to capture, and that is, monthly U.K. deferrals. And we presented our analysis of these data last year at the TSE Advisory Committee meeting in June.

It's important for you to know that we do compensate all of the blood centers that provide the monthly data in order to ensure their compliance with the due dates and in many cases to provide compensation for their initial programming modifications that may have been necessary for them to create the data reports that meet our specifications.

However, late in year one of this monitoring project and without having begun the collection of utilization data from hospitals, the NHLBI decided to discontinue their involvement in monitoring of the blood supply. Since then--actually, since January 1st of this year, the NBDRC has continued to collect the blood center data in the absence of any financial agreement so as not to interrupt the time series and jeopardize potential analyses of these valuable data.

We began to analyze the data from year one intensively, as I said, just within the last three to four months, and Jim will discuss these in a moment.

But before I turn the program over to Jim, I would like to tell you one of our additional goals at the NBDRC is ultimately to provide information about the blood supply that is not only timely and accurate and of importance to the industry, but that will provide real benefit to the medical community at large and that will be understandable to the public.

In this regard, I'm pleased to announce that we have been awarded a modest grant from the Abbott Laboratories Fund for preliminary development and pilot testing of a blood availability index--more correctly, a set of indices, which we hope will be broadly disseminated and provide a relative assessment of the strength of the blood supply on a routine basis similar to the consumer price indices.

This project assumes the continued and centralized collection of timely supply data as well as utilization data and would not be feasible without them. We're very excited about this prospect, and we hope that this goal can be fulfilled.

We urge the committee to support a consistent and sufficient source of funding to ensure the collection of accurate and representative blood data, and at this point I'll stop and introduce Dr. Jim Greer, senior analyst at the National Blood Data Resource Center.

Thank you.

DR. GREER: Good morning, and I'd like to express my appreciation to be able to present this data today.  What I'm going to do in my presentation today, I'm going to cover six topics.

First, I'm going to briefly characterize the sample and information which we have been gathering on a monthly basis.

Secondly, I'm going to present plots of these monthly data, focusing on the units of blood and on red blood cells distributed, as well as the inventories.

Third, I'm going to examine this same information, again, primarily focusing on distribution and inventory, but organized by two alternative lenses to look at the data: one by geographical region in the country and, secondly, if time permits, by the size of the blood centers, that is, the size of the blood centers in terms of their annual volume of collections.

Fourth, I'm going to look at the trends in these data.

Fifth, I'm going to offer some tentative conclusions.

And, sixth, I'm going to identify what we envision to be our next steps.

The project and the sample, there are 26 blood centers that provide us data on a monthly basis, and I'd like to emphasize all the data you see this morning is going to be from all the 26 centers. These represent a very substantial share of total blood collections in the United States, 34 percent. This is a representative sample. It's a representative sample of all blood centers that collect 25,000 units or more of whole blood annually.

Just parenthetically, we estimate there are 28 blood centers in 1998 and 1999 that collect less than 25,000. Indeed, there are 18 blood centers that collect less than 15,000 units of blood a year.

We collect on a monthly basis a variety of information. We collect information on the units of blood, the units of whole blood and red blood cells released for distribution, as well as platelets from whole blood and platelet pheresis. We have a biweekly inventory, and I emphasize, in other words, we have twice as many data points for inventory than we do for any of our other information; information on imports and exports as well as outdates, data on units or ordered versus units shipped, as well as delayed provision of blood. And starting in January of this year, we started to collect information on the collection of whole blood.

I'd like to emphasize that nearly all of this information is collected by blood group and type, although today I'm going to be exclusively presenting just totals.

The reason we're starting to analyze these data now is that literally in terms of the time series, sufficient data has now been collected, and at this point we're really permitted to talk about tentative findings.

Now we really get to the meat of the matter. At the risk of eliciting a collective moan, I'm going to take--I want to take you back to the memory of your very first statistics course. If your instructor was anything like mine, you heard a mantra of: look at your data, look at your data, look at your data. Today we're going to be largely looking at this data.

What you see here is a plot of the units of whole blood and red blood cells released for distribution from January 2000 through March 2001, in other words, just last month. The scale on the left is in thousands of units, and the scale here is generally between around 300,000 to 350,000 per month.

The highest levels of distribution occurred in March 2000, 366,000 units; August of 2000, again, 366,000 units; and January of 2001, 372,000 units. The lowest levels of distributions occurred in April 2000, which are about 328,000 units, and in December of 2000, about 338,000 units. Nearly half, I'd like to emphasize--I've done means tests, paired t-tests, to test for statistical significance. Nearly half of these monthly changes are significant from month to month. Those that were significant are between January and February, February and March, March and April, and April and May, as well as November and December, December and January 2001, as well as January 2001 and February 2001.

This is a slide which shows the inventories of whole blood and red blood cells January 2000 through, I'd like to note with some pride, April 4, 2001. The last point on this data, in other words, is just barely two weeks old.

The magnitude, I'd like to note, is significantly smaller. Whereas, you saw a magnitude in units of distribution between around 300,000 to 350,000 on a monthly basis, this is around--this varies between about 30,000 to 60,000.

The inventories here were largest in the second week of April, 54,000 units, and in the second week of November, 59,000 units. Inventories were most diminished during the summer months of 2000.

Again, I looked at statistically significant changes between these biweekly periods, and generally what I found is between May and June 2000 there were statistically significant changes taking place as well as in October, November, and December when the inventories were recovering.

That is a bit difficult to see, but what it is is a combination of U.S. Public Health Service regions, and we've combined them into five. Why did we combine them? Bluntly, some regions had too few blood centers to reasonably sample--to do a random sample. Also, NBDRC wished to collect and analyze data which was sensitive to geographical differences in the United States, and to do this we had to combine Public Health Service regions to get a sufficient number of cases in each region.

There are, as you can quickly see, we have--the blood centers participating are distributed not equally but well distributed throughout the United States.

I admit this is a bit of a cluttered and messy graph. However, what I want to alert you to is that the top line is the units released for distribution in the Northeast and the Midatlantic region. That's a combined region, the Northeast and the Midatlantic. Bluntly, what this graph shows is that the units of distribution--or I should say that the Northeast and the Midatlantic were very important in terms of the total number of units distributed. Much more revealing is when we look at the inventory. This is looking at the inventory exactly in the same way. The top line, again, is the Northeast and the Midatlantic region combined. And what it shows here is that the variation in inventories in the Northeast and Midatlantic are graphically important. Little variation occurs in other regions of the country, but the dramatic increases and decreases, declines over the national inventory, that is, which we saw previously, are largely driven by the situation in the Northeast and the Midatlantic regions.

We chose one other way to look at these same data, and that is in terms of the size of the blood centers providing us information. Generally, I divided the sample, and this is my criteria, it's NBDRC's criteria, divided essentially into relatively small blood centers that collect 75,000 units or less per year of blood; medium, which collect up to 150,000; and then the large centers, which collect over 150,000.

This is the plot of the distribution by the size of the blood centers, and logically enough, the top line represents the level of distribution of large blood centers, and they are predominantly important. Probably more revealing is when you look at inventory.

Again, the top line there is the inventories of blood centers in the Midatlantic and the Northeast.

Again, what you see here is the clear graphic importance of large blood centers. I misspoke there. Sorry. The top line are large blood centers. And, again, here we see the graphic importance of large blood centers in inventory.

I plotted additionally a trend line for--the top graph there is a trend line for distribution. The bottom is the regression trend line for inventories. I don't think I am off base by saying that those are very flat trends.

What I want to reiterate is that if you look--as we have looked at these data, it is very clear there are significant changes that take place month to month. You can see it graphically. I've also told you--although I can show you but I don't plan to--that there are patterns of statistically significant changes as well. There's a great deal of variance in this data in terms of distribution and in terms of inventory, and also there I'm breaking it down by whole blood, red blood cells, platelets from whole blood, and platelets pheresis.

In other words, what we see is that there are graphic changes that take place each month, or biweekly, in the case of inventory. We have tested and identified several of these monthly or biweekly changes are statistically significant, and we've also noted that there is a variance in the data. Yet these findings do not jibe with those very flat trend lines.

In other words, what I'm trying to say is the richness which you find, if you look at the variation that takes place on a monthly or on a biweekly basis, it is utterly lost if you look only at the trend lines.

I'm only going to offer a few very tentative conclusions, and they're tentative because we've only been doing the analysis for a relatively brief period of time, and we're breaking into new ground where we don't really know what to expect to find.

Generally, we can say that there are observable changes that occur month to month. I can also say that we've done statistical tests, and so that there are some significant differences in changes that take place month to month; but, nonetheless, the regression lines show little change.

Furthermore, if you look at it in terms of the size of the blood centers, the distribution or the actual patterning of the distribution is relatively unaffected by size, but inventory changes are most pronounced in the very largest blood centers. And if you look at these same data regionally, inventory changes are most pronounced in the blood centers located in New England and the Midatlantic regions, and the low inventories that we noted in January through February and June through September coincide with well-publicized blood shortages.

Oh, that's very difficult to read. Before I talk about what our future plans or additional analyses we intend to do, I kind of have two sidebars. One of those is that NBDRC plans to put some sampling of these data in tabular form as well as in graphic form on our website, which is www.nbdrc.org. And, additionally, we hopefully plan to add, as Marian pointed out in her introduction, utilization data from a sample of hospitals to complement the work we've done on supply.

We have three areas we plan to move forward in. One is to use the information, the very detailed, very rich data from the 2000 survey, to validate the findings--the data we're getting from our blood centers and also to be able to find out--to figure out ways to weight that data to extrapolate to national estimates.

Secondly, we plan to use time series analysis, particularly to identify trends. ARIMA, by the way, is autoregressive integrated moving averages, which is essentially a very fancy way of saying we're going to try to identify a structure in the time series of the data so you can start to adjust for seasonal changes which repeat themselves, obviously, year to year.

And, lastly, what we want to engage in is multivariate analysis where we're really trying to identify the relationship between the supply variables we're already finding and also to identify the relationship between supply variables and utilization information as it comes in.

I guess I'd also like to say at the very end here that NBDRC openly seeks the input of practitioners in the field and those who have already conducted analysis concerning the supply and utilization of blood and blood products. We're not trying to--we understand that we're engaging in new ground here, and we see this as an opportunity to bring many people together to collectively do this analysis for everyone's benefit.

What I'd like to do now is to introduce Dr. Wallace, who is the Chair of the Board of the National Blood Data Resource Center, for his comments.

Thank you.

DR. WALLACE: Thank you, Jim.

As some of you know, I've been engaged in doing a variety of different kinds of data collection and analysis in the blood field for over 35 years. What's happened, of course, is that as you get older, they push you further and further up, until finally you end up being a chairman of something.

Doug Serger (ph) and I did a whole series of national surveys over the period from--well, actually, we began with the first one in 1971, but the main ones that we did were during the 1980s and 1990s, finishing in '94. And at the same time, we also did a series of studies on blood utilization by DRGs and found some very interesting patterns that I would like to end up with discussing with you.

But today what I've been asked to do is to kind of stand back and for the National Center to give you an overview of what we in the center believe should be the kinds of information that a committee such as this, with the responsibilities that it has, what this committee should be seeking in the way of information about what is going on in the blood supply in the United States.

So what I would like to do to begin with is to combine the first two questions that your Secretary has asked us to address, which are, of course: What parameters should be monitored by this committee? And, second of all, what analysis should be presented to the committee? And then, finally, after doing that, I would like to talk very briefly about where the responsibility for this activity should lie.

Now, in discussing the blood supply today, I realize that we have both a plasma sector and we have the cellular sector. I'd like to limit my remarks to the cellular sector, that is, to the whole blood and the various derivatives that are obtained from volunteer blood donors. And I would like to begin, in talking about the adequacy of that supply, by looking at the balance between transfusion demand on the one side and collection supply on the other.

Now, that balance is very important, because if transfusion demand becomes too high relative to supply, as we all know, we have shortages popping up in different areas around the United States. On the other hand, if supply gets out of balance with demand, then what we have is an unacceptable level of outdates in the system as a whole. And we've seen both in the past, and we would like as a result of this kind of monitoring to bring those two--the demand and supply--much closer into balance.

First of all, what I'd like to do is to talk about transfusion demand. Now, the reason for that is that transfusion demand is what drives this whole system. And that demand comes from over 4,500 hospitals in the United States; 96 percent of the transfusion of red blood cells, some 90 percent or more of the platelets, all take place in the hospitals. A small proportion, of course, does take place in the blood centers.

Now, what makes transfusion demand so important is that not only does it drive the system, but also there can be abrupt changes in demand, sudden, what we call transition points, turning points, that can suddenly bring about a major change in the entire system.

And, interestingly enough, over the period from 1987 to 1997, we had a steady decline in allogeneic red cell transfusions in the United States that was paralleled by a similar decline in allogeneic red blood cells collections. So that between 1987 and 1997, there was a constant decline. But what happened was that the allogeneic collections lagged behind the transfusion demand so that by 1997, the margin between total supply and demand had reduced to about 5.4 percent of total supply, whereas two years earlier it had been almost twice that.

Well, what was interesting was at that point there was a sudden and abrupt shift, and between 1997 and 1999, allogeneic red cell transfusion demand increased over 10 percent. Fortunately, with that kind of a turning point, the information that was available to the system, first in terms of prior warnings about this going to occur, but also in terms of the feedback information that came from the hospitals to the blood centers, the blood centers were able as a result of that to increase their collections almost proportionate to that. Otherwise, we would have had some major shortages here in the United States.

So it's very important to have this kind of feedback information from the demand side into the supply side and to have it in advance so that the supply side can respond to these demands. And, of course, as you know, most of that increase in demand was a result of a major increase in transplantation activity.

Now, in selecting parameters to monitor as far as the committee is concerned, it seems to me that they are fairly straightforward. What we would like to know is what is going on as far as demand for components, both in terms of red cells, platelets, and plasma, and we would like to know as far as red cells are concerned, what is going on as far as type as well as the component demand itself. And what we're going to have to do is to get that information from the hospitals.

Well, one of the problems, those of you who have worked with data in the hospitals, is that when you go to the hospitals and you tell them the kind of information that you want, the kind of data, the first thing that you find out is, well, yeah, we can do this, but we can't do that. And so what you find out is that choices have to be made.

If you're interested solely in annual data, as we have done with the national surveys, then this isn't a problem. Most hospitals are able to produce that information, sometimes eventually, but they do it. And as a consequence, you get out your national surveys, although sometimes there's a considerable delay in doing that.

But when you ask them to do it monthly, the first response is: Well, you know, we can't do that. And so what you realize is that in order to do it monthly, what you have to do is to assist them. And you have to assist them both in terms of changing their systems in order to do it, and at the same time, you're going to have to provide them with some kind of material resources that will help them do that, particularly today when all of the hospitals are pleading poverty.

If you, on the other hand, ask them, well, we want to go a little further, what we really want to know is we would like to know how transfusion demand is broken down by DRGs, the first thing you get is: Oh, no, this we can't do.

Now, I say that because it can be done, but we found out in doing studies along these lines that in order to do it, what you had to do was you had to put together a record structure for the hospital, tell them exactly what information you wanted, you realize very quickly that that information had to be drawn from three or four or five different kinds of information systems in the hospital, and that they had to build all kinds of bridges. And this is the reason why they're unable to do it.

In studies we did about seven or eight years ago, we started out with 30 major university centers. We sent them that record structure, asked them can you do this, six came back and said we can. A couple of them called and said, Would you come down and help us do it? And the answer was, well, we don't have the time to do it. So we ended up with six out of 30.

So what I'm saying is if you want monthly data on the standard parameters, yes, if you will help the hospitals. If you want DRG data, that's going to be a totally different kind of an undertaking. And I'll turn to that finally in the end, but I think it's an important thing to do.

Now, as far as going about getting information from the hospitals, let me just give you some background for national survey. At that time we decided, or at least I did, that all this enumeration of thousands of hospitals doesn't make much sense. It's kind of like the census arguing that we could do this better with sampling than we could with enumeration, although politically enumeration seems to be the thing.

And so we tried an experiment. We said, all right, we'll take about 100 hospitals that affiliated with the AABB, and what we will do is we will develop that as a stratified random sample, and we'll also do the enumeration. And then when we get done, what we'll do is we'll compare the two and see how it works.

Well, interestingly enough, the sample worked--I think it may have worked a little better, but actually it worked within about 2 percent of what we got on the enumeration. And as I say, I think it may have worked a little better because we really didn't understand from the enumeration that well how to make the adjustments for that other 12 percent. And I think we did a better job of the overall estimation with a sample of about 100 hospitals.

So what I would say is, in order to do this, what you need is a sample--that is, to get transfusion demand, what you need is a sample, but it has to be stratified, it has to be very carefully put together, and you're going to have to recognize that you're going to have to help the hospitals in terms of their data-gathering systems, because what's going to happen is, for some of them, if you just go and ask them, yes, they can change their systems. If you go and ask others, what they're going to do is they're going to take some individual and say, You're it, you're the one that collects the information.

And so if you have an automated information system and it's been adapted to the needs that you've sent for, then what you're going to get is prompt and valid kinds of responses, and hopefully also it will come in over the Internet so that things can be done very quickly.

On the other hand, if what you do is you get hospitals that have set up some individual, you may get the information, you may not. It may come in when you want it or it may not. And so what you have is a real confounding kind of a problem. So, therefore, in setting up the sample, it's very, very important to work with the hospitals to make sure they can do what they say they can do, and then go ahead and assist them in doing that.

Now, what I would like to do is to turn very briefly also to the side of supply, because if we're collecting transfusion information and we're collecting it in terms of components, and we're also collecting information in terms of the frequency with which the hospitals say, no, we were not able to supply the full amount of blood that was required for all of our surgery, and we get the information from them about things such as, well, how frequently did this happen and at the same time what components were in short supply, both by type of component--or by component and by type, what we need to do then is to have a way of matching that against what's going on on the supply side.

Now, the supply side in the United States, as we all know, is very different from the demand side. You've got 4,500 hospitals out there on the one side, and what you've got is 145 blood centers that are collecting 92 percent of all of the whole blood and about 90 percent of the platelets and the plasma in the United States, that is, that's used for transfusion and not, obviously, for processing.

And, in addition, what we have are two major groupings of centers, and they just about split the center total. So if you're thinking about monitoring supply, obviously what one wants to do is to monitor, as we have been doing here, the supply coming from the blood centers.

Now, in designing a system, though, for monitoring, what we need to do is that we need to make sure that several center organizations are appropriately represented in any sample that we develop. But, in addition, what we need to do is to recognize that location is an important element as far as supply is concerned, because location, and particularly those centers in major metropolitan areas where it is difficult to get substantial participation of the population of those areas into the collection process--and it's costly as well--those are the areas so frequently where problems can arise, particularly during seasonal variations.

And, finally, what we need to do, as far as the supply is concerned, is, like with transfusion data, we need to have it by months, because if we're going to match the two and begin to look at the whole system as it works, what we have to do is to have the data that is working together.

Now, as far as analyzing the data, it's pretty straightforward. We like to know what the beginning inventories are for all the components. We'd like to know what collections are. We'd like to know what distributions are. And we would also like to know what those ending inventories are, and we'd like to know it by type as well as by component.

In addition, it would be worthwhile knowing the number of units of each component that were not--that the center was unable to fill in terms of the hospital orders. Now, I know this isn't a great kind of measure of shortage, and I know from past experience that there are a lot of hospitals that, when a center gets an order from them, they take a look at the order and say, well, they got a wall of blood out there already, why is it that all they want to do is age the blood that we send out there? But looked at in the aggregate and looked at over time, this can be a useful indicator of some potential problems that may exist on the supply side, particularly if you can match that with the kind of information on transfusion type of demand shortages in terms of any postponements of elective surgery. So these are indications, and they have to be taken as indications.

Now, what is really important, though, as you get the information from the transfusion side, the demand information, and you get the supply information, you have to put it together. And in putting it together, I would say the important thing here is that those who put it together have to have a good understanding of how this system works. They have to understand how the hospitals operate. They have to understand also about the centers. And they have to be able to interpret this information, and interpret it in a summary fashion, so that you as a committee get it in a summary fashion, in a way in which you can then understand it and be able as a result of this to make some kinds of decisions on your own.

So being presented the information in great detail, you know, tabular form or graphic form or what have you, is all very interesting. But I think if you're like me, you say, well, so what? You know, what does this all mean? What do we want to do with it?

And so what I would point out is what's extremely important is, yes, gather the monitoring information, but you need to work together with those who are gathering the information and doing the analysis. But one of the committee's designees has got to be working with that group in order to pull together the information and be able to summarize it, and summarize it usefully for the committee and its functions.

Now, finally, what I'd like to do is to talk very briefly about where responsibility for this activity should lie. Well, first of all, of course, and clearly, the responsibility lies with this committee. I assume that the committee is not going to function as a whole and, therefore, it will designate one or more representatives to handle the responsibility.

Now, beyond that, the responsibility also lies, on the one hand, with the hospitals that are involved in the sample in terms of providing the information that is required and providing it in a timely fashion. And the center is the same thing, so that this information comes in, is able to be analyzed, and you get it in a current fashion. It's not very useful if you're looking at kind of seasonal variations and you're three or four months behind time.

Finally, I would point out that it's important that this be done for the committee by an independent agency, independent of the various sectors that operate in the blood system. And I say this because any one of the sectors has an unintended bias. If you look at it from the center supply side, you're going to have a certain bias because you're seeing only half of the equation. If you're looking at it from the demand side, you're seeing the other half of the equation.

So it's important to get the input from both sides, but it's also important, as the committee should be functioning, to look at it as a whole and to be able to synthesize it.

So I would argue that what we need--or what you need, not me, is an independent agency that can function for you and work with you and work with your designee to put this information together for the committee and put it together in a way which is summarized to meet the committee needs and not in a great deal of detail.

I'll make a pitch right now by saying I think the National Center is the kind of agency that you should be considering. There are others, but I think we, among those, are capable of doing this kind of work.

Now, finally, what I'd like to do, though, before I finish, is kind of go back to something that has been very important to me, and I think it ought to in time be important to you. I'm not sure that this particular topic falls within the province of this committee. I would hope that it would. And what I am talking about here is monitoring responsible usage of this resource. By responsible usage, what I mean is how is the resource used by the hospitals, by the physicians and surgeons in the hospitals, with respect to patient needs.

We've done some studies along these lines, particularly with respect to cardiac bypasses and with respect to knee and hip replacements. And what we found was very interesting. We found that amongst a group of very, very advanced third-level hospitals that there were wide variations in the way in which the resource was used. In some instances, they were very, very careful in terms of the use of the resources, perhaps even too careful. In other instances, two and three times as much blood was used for the same procedures.

And when we brought this information together and we sat down with the representatives of the hospitals and we pointed out to them these variations, they were very surprised. They weren't aware that this kind of difference existed amongst their colleagues in other hospitals. And it existed even within the hospitals amongst their colleagues in the hospital.

But what we did find was that, given the information, they responded; then there was a considerable shrinkage in the variation with the sense that this is being used is--this resource, this very valuable resource, is being used in a more efficient sense.

Now, in order to do this, you have to go and look at the use of the resource at the level of the procedure or the medical process that's taking place--in other words, at the level of the DRG. It's a very difficult thing to do, and I'm not sure, as I say, that it falls within the province of this committee. But I think it's something that should be of concern to all of us if we're involved in the blood area, because blood and blood components, while they may be described from time to time as a commodity by people who are looking at them from a strictly economic point of view, these are human tissue, provided by volunteers, they're expected to be used responsibly for humanitarian purposes, and I think it's an obligation of all of us in the area, in the system, to try our best to see that that's what takes place.

Thank you.

[Applause.]

DR. GOMPERTS: Dr. Epstein?

DR. EPSTEIN: Dr. Wallace, before you escape, could I perhaps ask you a question?

DR. WALLACE: Certainly.

DR. EPSTEIN: I enjoyed your presentation. I've enjoyed your papers. It's a pleasure to meet you.

What troubles me is whether we have predictive power. Obviously, if we have delayed reporting of, you know, two-year surveys or two-year annual reporting of annual surveys, it's all in retrospect. And here we're talking about trying to do something more on line. But what bothers me the most is that we haven't seemed to have figured out what kind of predictors there are for when we're going to get in trouble. We seem to only predict trouble after we're already in trouble. And the key thing here seems to be the quest for benchmarks or measures or triggers or predictors that would have a prospective value in the system.

So could you comment on the feasibility and your thought of what that would take?

DR. WALLACE: Well, obviously, what we want to do is to look at the demand side because that's where the changes are going to take place. If it's a steady state system, as you saw there in those trends, then the seasonal variations are what really are a concern. But those are relatively easy to predict, not necessarily in terms of the total amount, but with a reasonable inventory buffer, those are relatively easy to take care of.

Where the real problem lies is in the sudden changes in technology, sudden changes in procedures. There's an interesting paper coming out--I would recommend it to all of you. You may not be too interested in the fact that it deals with cost, although maybe you are if you're in the center business because cost is an extremely important thing. But what it is is a study of 60 university hospitals, and what they've done is that they have looked at the cost of blood per DRG.

Unfortunately, they weren't able to get down to blood utilization, but cost was a pretty good surrogate for what we would be interested in, which is utilization.

And two things came out of that. First of all, they found that there were 34 DRGs that really drove the entire transfusion demands for blood. Only 34. And of those, 10 accounted for a very high proportion of the total.

And if you looked at the list of 10, what you found was that the top five or six, of which transplantation took place in, I think, three or four of them, were the real driving factors in terms of pushing up the demand.

So if you want a short-term kind of monitoring system that would enable you to see these turning points or to see sudden increases, then I would say what you need is a monitoring system that would monitor a set of those critical DRGs and see what's happening and compare that with what has happened, because transfusion is much, much more level than collection. You don't have quite the variation in it.

And so you would be able to look at it from month to month or cumulatively three months to three months and get a good, early-on indication of what's likely to happen as far as blood transfusion demand for blood. That would be my way of doing it.

DR. GOMPERTS: Dr. Davey?

DR. DAVEY: Dr. Wallace, thank you. That was an excellent overview for us.

A question which I think will come up during the day is issues of compliance, especially in the demand side. I think you mentioned, if I recall it, six out of 30-some hospitals were willing to provide the data that you required. And you mentioned that on the carrot side you could give them the supplies and the tools that they needed.

Any other comments about how to encourage compliance? Do we need to have a stick side as well as a carrot side to get the right data on board?

DR. WALLACE: It isn't the stick and carrot situation. What it is is that if you go back and look at the way hospitals put together their information systems, you had a pharmacy developing one information system; you had the accounting group developing another; you had the medical records group developing another one. You had the surgery group developing one; you had the blood system developing one. And the information is scattered all around. And they haven't been very effective in pulling it all together into kind of a meta-system that would handle the whole.

Brigham I know right now, because I've been working with them for a number of years, is trying to do that, and they're succeeding. But it's not an easy thing to do.

So when we approached them, they were not unwilling to do it. They said, We can't do it. And, in fact, one of the six we had a pediatrician who actually volunteered to go down and to sit down every month and to take the information from one system and manually type it into the other system so we would have the full record.

That's the problem. And what you have to do is that you have to sit down with them, those who can do it, sit down with them and work it out with them so that they can adapt their systems to what you need, because their systems are not synthesized, they're not a whole.

DR. GOMPERTS: Dr. Guerra?

DR. GUERRA: A couple of questions for either Dr. Wallace or Dr. Greer. On one of the slides, there's the indication that on the demand side, which I think is greatest in the Northeast and the Midatlantic region of the country, it's also a region that I think in recent years has been losing population, and other regions of the country have been steadily increasing. So that that would be a disproportionate demand, unless, you know, you have some way to link the age distribution of the blood to, you know, age groups and--or I guess some of it is perhaps done by DRG. But what might serve as an explanation for that?

DR. GREER: I believe that in the Northeast and the Midatlantic is a very large concentration of both large and substantial hospitals. You know, the hospital complex in Boston is very substantial, as in New York, as in Philadelphia, as in Pittsburgh. In large part, that drives that substantial demand. Independently of the fact that you have relative population loss compared to other areas of the country, actually you have a hospital that will actually have catchment areas which are not limited to their local geography, are actually state, regional, and even national catchment areas in some cases.

DR. GUERRA: Or international areas, I imagine.

DR. GREER: Yes.

DR. GUERRA: And I guess that probably a lot of the supply of blood probably goes to serve many patients.

DR. GREER: In some task force that I'm on, I have some familiarity working with ICD-9 data where it actually had provided the zip code of the patient, and we were astonished to find in some hospitals that they literally were having--they literally caught patients from hundreds of zip codes, in some cases thousands of zip codes. Actually, you know, very substantial catchment areas.

DR. GUERRA: Is it possible to link some of that data to utilization by age groups?

DR. GREER: I think so, but I'm engaged in high speculation at that point in time. I think some of the ICD-9 data has some basic demographics, but I'm not sure how specific it is in terms of age, whether, you know, you actually have age 48 or you have, you know, age groupings, say under 10, 10 to 25, that sort of thing. Possible, but I'm uncertain.

DR. GUERRA: But there's nothing at least in your data that would indicate that, you know, 30 or 40 percent of the supply is going to populations over 75 or 80 years of age for--

DR. GREER: Not--since we're exclusively at this point in time looking at supply, we only have supply data. You know, that would be entirely speculative. I have no basis to say what age group this blood and blood products are going to.

DR. GOMPERTS: Dana?

DR. KUHN: I wanted to ask just three, I think, pretty short, practical questions here, and I want to thank you for your presentation and concern for collecting data.

The first question I wanted to ask, being that you are an independent non-profit, is: How much funding is needed to run the NBDRC's data collection program for the 34 percent of the U.S. blood centers? I think that's a very important question to ask, especially if we're to entertain how we are going to continue to look for an entity to do this kind of a program. That's the first question.

The second one is: Who collects the data from the other 66 percent of the U.S. blood centers?

And then the third question is--or maybe this is a naive question. But why wouldn't all blood centers want to use the NBDRC as a central aggregate data collection entity?

DR. GREER: I can only answer one of those questions, and Marian can take the other two.

I can answer the second question: Who collects the data from the other 66? No one, to my knowledge, although they may--there may be independent organizations that do so, but I'm unaware.

MS. SULLIVAN: I can tell you that the monthly monitoring project exclusively for the blood centers, which we undertook last year and, as I said, have continued in the first four months of this year and hope to continue, runs at a cost of just about $190,000 annually. And more than half of that, almost two-thirds of that is used for the compensation of the blood centers, as I mentioned. And that's strictly for that piece, and not for other activities that we may have suggested.

In our biennial surveys, the 1998 survey and the 2000 survey, we did capture every U.S. blood center, and that's exactly how we know that our monthly sample collectively is responsible for 34 percent of the total supply.

There are other data collection mechanisms. Obviously, ABC and ARC do capture data from their members, respectively. We overlap on the centers from which they're collecting data. They're not--it's not three separate groups collecting data from different groups of blood centers. We certainly overlap with both of those.

And in answer to your last question as to why all blood centers would not want to have their data centrally captured and analyzed and fed back to them in a useful and beneficial way by the Data Center, I can't possibly imagine why anyone wouldn't want that.

DR. GOMPERTS: Thank you.

DR. CHAMBERLAND: I had a question probably for Dr. Greer. When you showed the trends by region in the Northeast, the aggregated Northeast/Midatlantic, I believe, there were seven centers, I believe, that were captured in that region. And I was curious: Were they all equally drivers in the trends in demand and inventory, whatever? Or were there outliers in that group that really were responsible for that overall, those overall trends?

DR. GREER: I have not looked at it in that level of detail yet. That's a good line of inquiry. Particularly, I'd be concerned whether there are only one or two or three, for instance, that are doing that.

I suspect that, indeed, there are outliers there, but that's a next step in analysis.

DR. CHAMBERLAND: Okay. Thanks.

DR. GOMPERTS: I have a question for Dr. Wallace and perhaps Ms. Sullivan. To what extent is the health insurance industry interested in this data?

DR. WALLACE: I don't know. We have obviously made a number of inquiries of various and sundry organizations that we think should be interested. We have received very polite responses, but we have not had anyone come forth and say--other than, you know, we'll buy a copy of a report. But a copy of the report doesn't do much to finance this kind of undertaking.

So to answer your question, we haven't had any what we would call welcome response from the health insurance industry yet. I hope we would get some.

DR. GOMPERTS: Thank you.

MR. DALAL: To any of the speakers, is there data available in other countries, European countries, Canada, Japan, on collections, inventories, and availability?

MS. SULLIVAN: We may not be the best group to answer that question. We frequently receive inquiries, similar inquiries, and generally respond that we know that the WHO blood program does capture some data such as that. And, actually, yesterday in the presentations, I was quite impressed with the volume of data that we saw presented regarding blood availability in both developed and developing countries, which I had never seen presented before. So it actually gave me a feeling that there's more data out there than we typically see from around the world. But that's about the most I can say with respect to that.

Dr. McCurdy?

DR. GOMPERTS: Dr. Busch--oh, Paul?

DR. McCURDY: Perhaps Mike was going to say the same thing. There's a group in Finland that has done a study throughout a number of countries, particularly developed countries, and they have a fair amount of data that I've seen presented, not yet published, but there are such things as the majority of developed countries collect between 30 and 40 units of blood per 1,000 population per year. There are a small number that collect more than 40, even 50 to 60 units per year, and there are some that collect less than that.

They have also noted the differences between hospitals and, indeed, between individuals and hospitals, and my recollection is that this depends primarily on where the surgeon involved trained, that he carries with him habits of usage and that accounts for at least some of the variability.

DR. GOMPERTS: Thank you.

Mike?

DR. BUSCH: I've never taken an economics class in my life, but this seems like a lot of macro-, microeconomics distinctions. We're talking about the annual surveys or biennial surveys that I think Dr. Wallace's group had done and that the AABB data center took over. It seemed to be extremely valuable and give us these historical trends, and seemed to be able to also dissect, at least would be capable of dissecting within a year trans-seasonal trends of this nature. And I think what you've attempted to do with these more frequent surveys is to get at a much more online program that could theoretically, especially if you could begin to capture the usage side, could theoretically give us a tool to predict shortages and react proactively.

But I think we all know that at the blood center level, there's major focus on this on a daily basis. There's inventory control meetings every single day in every blood center that are trying to figure out do they have enough blood, what should they collect. And it would seem that you're trying to do something at a national level that has been a great struggle at the individual center level to deal with. And to me, if this were to be pursued, it would be more logical to try to capitalize on what's going on at the center level and help them perhaps tap into the regional hospitals that they're serving, assist the centers at developing the capacity to better predict shortage issues, because right now my sense is they're not able to do that. They don't have the link, appropriate link that you've described to the DRGs, to the usage side, that at a regional level where the blood is being used and needed, they're able to respond to that. And if that could be established, if you could then be able to capture that through the centers, it would seem much more simple than you folks going to the hospitals delinked from the centers.

Then my other question is: As you're getting into what you're getting into, the actual monitoring of utilization and production, I suspect--I don't know, but I suspect you're beginning to receive problems related to the competitive issues within the blood center. And I'm very curious as to whether you're getting cooperation or not from, for example, the large blood programs like the Red Cross or BSI, whether they are--some of this issue is a reaction to the fact that you're now tapping into the bread and butter of how these blood centers operate and in reality compete with one another.

DR. WALLACE: Let me respond to the first, and then I'll let Marian talk to you about the competitive situation.

Yes, you're perfectly correct. From the blood center point of view, it seems to me that it would be very useful for them, first of all, to identify the hospitals that are key to them, because that's where the change--and then within the hospitals be able to monitor those critical DRGs. And if we could set up that kind of a system and then synthesize it for the country as a whole, that would be a wonderful system. I would strongly support anything like that.

It might be worthwhile trying it on a pilot basis to see could that be done in maybe one or two or three major centers. And if so, then it would become a model that could be then spread across the country. So it's an excellent idea.

But I'll let Marian talk to you about the competitive problems.

MS. SULLIVAN: As an independent organization attempting to capture these data, the Data Center hopes that we can successfully kind of transcend the struggles and the difficulties that may be occurring, and the competition, within the blood industry. And I think, I believe that we have really done that successfully with the tremendous cooperation of all U.S. blood centers. And I think that the response rates on our various surveys, some of which we haven't even discussed here today, really speak to that. There are no groups, no clusters, no individual blood centers which have been uncooperative and declined participation either in the monthly program or in our nationwide surveys, except there were two small community blood centers that did not participate in the 2000 survey.

We very carefully protect the confidentiality and the identities of all of the centers that provide data to us, so we don't usually name names. But if anyone wants to know who the non-cooperative blood centers are, I'm always happy to name their names. But I won't. But in answer to that question, we get great cooperation from the blood centers.

DR. GOMPERTS: Ron?

DR. GILCHER: I think there are really four key words here: utilization, demand, supply, and inventory management. And my question is relating to have you done any work with the Joint Commission, because, in theory, the infrastructure is in place on the utilization side.

Now, I can tell you as a blood center director the problems that exist when I try to monitor utilization committees. But every single hospital has a blood utilization committee which, in theory, monitors the usage of blood by the total amount and also by the use by each physician and the kind of surgery, for example, that they perform, or the oncologist or whatever. So, in theory, that data is there.

Have you done any work in that regard? Because we clearly find at our blood center that we have hospitals that will attempt to horde certain blood types, and we set with them--we work with them, and we set the inventory level for each hospital. But in spite of that, in spite of what they agree on, we find them hording certain blood types. For example, O negatives, classic problem in the whole country.

Have you done any work with Joint Commission to look at utilization? Because I think that's a piece that is there, at least some of that data. The infrastructure is in place.

DR. WALLACE: We haven't done anything with the Joint Commission, but we did do a considerable bit of work earlier on with the comprehensive group that was up at the University of Michigan, CPHA group that collected the information on utilization. But they collected the whole array of activity, as some of you may remember. I think they're still in operation somewhere.

What we found was that from an aggregate point of view, the reporting of activities that went on in the hospital was very good except for certain areas, and blood utilization was one of the poorest.

The reason we knew was we were doing studies on cardiac bypass, and we knew from the studies approximately what the utilization patterns were in terms of the means and the variances. And then we took the CPHA data on the same DRGs, and we compared them, and we found that the CPHA data was significantly lower, indicating that the hospitals were not reporting the blood utilization like they were other activities. And, in fact, we just had to drop the whole kind of a study.

Perhaps the utilization data is better, but our latest experience with data, which is with the American Hospital Association, indicated to us that the American Hospital Association and their data gathering is having the same problems as CPHA.

The only area that might be worthwhile looking into in an aggregate point of view is HCFA, because, of course, they get the information from the standpoint of setting the reimbursement rates. But the problem there is, of course, the enormous amount of data. It's not pooled together. And so, as far as I know, nobody has been successful in doing this, but it's always worth a try.

DR. GOMPERTS: Steve?

DR. NIGHTINGALE: May I make a comment? We agree with you, Dr. Wallace, that it is worth a try. And because we agree with you, we are delighted to announce that we have secured from HCFA the half-time services of Dr. Barbara Silverman.

Dr. Silverman, would you stand up, please? This is Dr. Silverman's first day on this detail.

[Applause.]

DR. NIGHTINGALE: Dr. Silverman is an epidemiologist with the Health Care Financing Administration, and they have graciously loaned us her talent for a six-month period to do an exploratory analysis of what data can be extracted from the HCFA massive database. We're not looking for answers. We're looking for questions. Dr. Silverman can be reached, obviously, in the audience today, and I would encourage anyone in the audience who has specific issues that they'd like her to address to talk to her. She will only be with us half-time for the next six months, so she can be reached through the following telephone number: 202-690-5560.

Also, since she will only be here part-time, Ms. Virginia Wannamaker, who is with us full-time, is also on generous loan from the Health Care Financing Administration. Ms. Wannamaker, Ginny, would you--I saw you back there. That's her. If the line in front of Barbara is too long, she's also an expert.

We recognize this concern, and we're trying to meet it, and any help that the audience, the community as a whole can give us in maximizing the utilization of these individuals will be most appreciated.

DR. GOMPERTS: Thank you. There are two other questions. John?

DR. PENNER: Just a reinforcement. Ron's point is well taken. I sit on the transfusion committee and run one. I know the amount of blood that is used by every doctor in that hospital on a monthly basis. I know what the total is.  And that information, it seems to me, it's exactly what you're after, and if you could check the transfusion committee's reports, which have to be provided to JCAH every year, and notice that each monthly evaluation has to be there, if it isn't, then there are serious repercussions. That data is just sitting there. It's ready to be pulled out, I think, from every hospital in the country. And it would seem to me to be a fairly simple approach to pick that up.

Then maybe one other comment, and that is, year in and year out you have a very flat line, as you said, and with the exception of perhaps a few of the technical variations, perhaps, with marrow transplantation and tissue transplants that there are these little blips, but pretty much it goes along with the training of the physician at the time he came out of his residency or her residency. And that is very consistent year in and year out, because it very seldom is bothered by any educational impact, unless one works hard at it. And we've worked hard at it as part of a major funded study that we did a number of years ago, and so you can't impact that. But if you don't, then it continues for the life of that physician. So that you can look at this year and you can figure next year is not going to be much different than this year and the following year is going to be the same, that the use of blood in trauma or by the hematology-oncology group or by the surgeons is going to continue to follow that pattern because it's not going to be much of a change unless there's an educational change.

DR. GOMPERTS: Thank you.

MS. LIPTON: My only comment, Mike, when you were talking about can you put this down to the center level, and I think you probably could. I think what confounds the issue is when you really aren't the sole supplier for a particular hospital, and I think we're going to hear more about that later. But I think that's where it gets to be very, very difficult to try to use the centers to track the information.

DR. GOMPERTS: Other comments? Dr. Goodman?

DR. GOODMAN: Jesse Goodman from CBER. I'd just like to ask particularly the people who have spoken what their sense is or results have been with more of a sentinel event kind of approach, because you're looking at a large amount of aggregate data here, and what we're really concerned about is these gaps between supply and demand. And the models used, for instance, in adverse event surveillance or an infectious disease surveillance have been pretty useful for detecting early numbers of events which occur more frequently than one would expect in background.

So while the comment that I had heard that, well, yeah, you know, somebody doesn't release the blood because they know somebody's not going to use it, and this looks like there wasn't blood available, that can be subjected to pretty careful analysis and you can develop a nosology, a terminology of these kinds of events.

So I'm just sort of wondering whether those models could be applied, because there is a lot of local data, but part of what you're trying to get at here is when regional or national problems develop. And what we see is, for instance, one physician may see an unusual case of a respiratory illness and make nothing of it, and they may be unaware that seven other physicians in that region have seen similar cases. And we don't want to wait until it's in the newspaper that there's an epidemic of Legionnaire's disease. You want to have informatics that pick up those seven cases at a time when those seven individuals aren't aware of it.

So I think if data are collected through these kinds of mechanisms and it's not known whether this kind of approach could work, that may be one thing worth studying. But perhaps there already is experience with this, so that was sort of the question part of it.

DR. GOMPERTS: Thank you. Any response to that?

DR. NIGHTINGALE: I do, because I might be able to say this a little quicker than Dr. Greer can, because I know less statistics than he does. But I think that what you can get out of time series, but only after you've got a second--through two cycles, through December 2000, as you're looking for five basic mathematical events. You're looking for cycles, you're looking for trends, you're looking for discontinuities, you're looking for clusters, and you're looking for non-linearities. And once you find those, the trick is to figure out what they mean.

Dr. Greer, did I get close?

DR. GREER: Yes.

DR. GOMPERTS: Dr. Chamberland?

DR. CHAMBERLAND: Agreed, but in keeping with Dr. Goodman's comments, I think any sort of system, surveillance system that is designed has to have the capacity for early warning, because while, by and large, these trends appear, you know, from your 15 months and then the surveys that have been done on an ongoing basis, may appear in certain ways predictable.

I think Dr. Wallace's comment about we have to be prepared for the unexpected or the outlier, and I think all of us sitting here today know that we're sitting on the verge of what may be a real significant impact on blood safety, and that is the impending deferral criteria for BSE, for new variant CJD. There's been a series of advisory committee meetings, and we know that there is much discussion underway as to how these deferral criteria will take shape and what impact they will have on blood availability.

So I think, you know, we're sitting right now on a situation that's going to quickly evolve, and I think we want to be prepared to capture that in a very timely fashion and not after the fact, you know, be able to say, yeah, that was a 6 percent, 5 percent, whatever. We want to be able to respond.

So I agree with you in part, Steve, but I think there also has to be that early warning, that sentinel ability.

DR. GOMPERTS: Thank you. Thanks for that focus.

FLOOR QUESTION: I just had one quick comment. If you're really looking for--

DR. GOMPERTS: Please come to the microphone.

FLOOR QUESTION: I'm sorry. Bob [inaudible], New York Blood Center. I just--if you're looking for a really good data system, look at this, you might want to look at the Veterans' Health System, because they have a very, very advanced clinical database that I'm sure all this is captured, and I don't see anybody here from the VA, so they probably wouldn't really understand it. But that's a good system to look at.

DR. GOMPERTS: Thank you.

Jay?

DR. EPSTEIN: Following up on Dr. Chamberland's point, which, of course, is a driving concern, I would like to ask the previous speakers what's their view of the elasticity of supply, both acutely and chronically. Because I think in many ways the whole issue of anxiety over donor loss is driven by perception rather than scientific fact about elasticity of the donor base. And I just wonder if you could give us some comments from a historical point of view.

DR. WALLACE: Well, short term--by that I mean if events occur that require substantial increments in supply short term--the system is quite elastic. It's been able both in terms of expansion, and expansion quickly through emergency kinds of recruitment, to collect additional amounts of blood that are required. It's in the longer term, I think, that many of us have concerns as we look at the donor base and we see it aging and we see that the proportion of the population that is continuing to be engaged in donation is shrinking in relationship to the volume that is required.

Whether that is a real problem in the longer term is hard to detect because, of course, places like Great Britain and all have followed a rather different system where they actually have panels of donors that they can draw on in a much more disciplined kind of fashion. And so they have actually a lower percentage of the population that is participating, but they're participating on a much more frequent, repeat donor basis.

So at least this was the experience that I had with them in the past. Now, they may have changed more recently, and some of you may be aware of that.

But to answer your question about the elasticity over the longer term, that's going to be an interesting problem, I think, because what we are not seeing is quite the filling in of the lower numbers of donors in the lower age brackets to compensate for loss of the aging donor population.

DR. GOMPERTS: Thank you.

Ron?

DR. GILCHER: I'd like to comment on the elasticity chain. It's clearly there, and it was demonstrated in the recent past, as you know, in Oklahoma City, with respect to both the bombing and their tornado, and it really was a very nice experiment in a sense, because we had this incredible turnout of donors in both situations, and there is what I call the give threshold. And that threshold clearly moves up and down, and we monitored the donors who came in both scenarios, and, interestingly, there is a certain percentage of those donors who turned out who did not show up again until the tornado occurred, which was four years later, and they came out again. And it was--their threshold is obviously different.

So the donors are there. The elasticity is there to respond, as long as it is perceived by the donors that that need is an urgent situation, and they will respond.

DR. GOMPERTS: Thank you.

MS. SULLIVAN: I would like to add just a little more to what Dr. Wallace and Dr. Gilcher have said from the historical perspective from our nationwide survey results and to throw a few more statistics at you, if you don't mind.

In 1989, allogeneic collections totaled 13.6 million units, and the margin between collections and transfusions was nearly 1.9 million at that time. That was 13.8 percent of the supply.

We have been carefully tracking that margin, and I wish I had a slide with me today to show you graphically how that looks. But by 1997, the margin between collections and demand had shrunk into 862,000 units, 7.2 percent of the supply, which at that time was nearly 12--the allogeneic supply was nearly 12 million. At that time, we projected that, given a set of five assumptions--assuming that blood collections continued to decrease at the same rate as they had been in recent years at that time and that transfusion demand continued to increase at the same rate, and some other things were unchanged--that eventually the margin would disappear completely. And our extrapolations led us to believe that if those assumptions were true, that could have happened by last year.

Happily, what happened is that blood centers in this country--and it was exclusively blood centers--really rose to that and recognizing the significant increases in transfusion demand over the last few years, collected 10 percent more allogeneic units in 1999 than they had in 1997. So even though the transfusion rate or the--not the rate, but even though allogeneic transfusions increased more than 8 percent in that two year period, collections increased around 10 percent, thus preventing the disappearance of the margin. So right now--actually the available allogeneic supply in 1999, the margin was just under one million units. So I hope that adds a little bit to the picture that you have.

DR. GOMPERTS: Thank you for the presentations as well as the excellent discussion.

We will move on now with Dr. Richard Lewis, FDA perspective on monitoring of blood supply.

DR. LEWIS: Thank you, Dr. Gomperts, Dr. Nightingale, and members of the committee. In an attempt to get a jump on technology, I've had my first slide up for the last 40 minutes.

[Laughter.]

DR. LEWIS: Probably for the only time in my life it can be said I need no introduction.

Part of the mission of the FDA is to assure the safety of biological products, and among those biological products is blood. And in evaluating these products, quite often we find ourselves in a position of doing a risk/benefit analysis. Dr. Epstein referred to that yesterday in response to a question about synthetic oxygen-carrying products, and we see that quite often in many other products, including blood, and that they have to be evaluated in the context of their use.

The availability of products is not strictly part of our mission, but we view it as part of a safety issue. And the ultimate decision of FDA-regulated products is the public health.

In order to make an informed decision about product safety, a full impact of that decision needs to evaluated. Thus, in developing deferral policies for blood donors, an important component of evaluating that impact is the availability of blood and blood products.

We have addressed prior supply concerns, including plasma derivatives, which Dr. Weinstein will talk about later this morning, and monitoring those. We've looked at availability issues in blood testing devices, among other things, in the context of compliance. And we will continue to probably look at availability. In fact, we've developed a medical necessity policy to evaluate products in times of shortage.

Historically, in recognition of the importance of the blood supply in the face of the initial variant CJD deferral recommendations, Dr. Satcher in June of 1999 convened a working group to make recommendations regarding the predicted changes in supply as a result of that deferral. That particular working group was composed of members of the Public Health Service, including people from CDC, HCFA, HRSA, FDA, as well as representatives from the Blood Products Advisory Committee, who included members of the community as well as representatives from the Department of Defense.

The result of the early meetings of that committee was to propose five different activities to help address possible supply shortages. The primary recommendation that we're talking about today was to monitor the blood supply. In addition, it was recommended to encourage more donations by eligible donors, to improve donor relations as a part of recruitment and retention, to remove some safe--to remove restrictions from safe donation, and to address economic issues that may be facing the blood industry. And this particular committee has discussed that last issue in previous meetings.

The first step in monitoring the blood supply was the contract with NBDRC that you've heard about this morning. The initial commitment was that NHLBI would sponsor that contract and evaluate it on a yearly basis. And during that time, other decisions would be made about who would take responsibility for that particular monitoring, and that brings us to the discussion this morning.

To address some of the other issues, FDA sponsored workshops on recruiting practices. If one is to believe the evaluations that we received, that was a well-received workshop that presented those particular practices throughout the country that worked very well in recruiting people. We addressed the donor history questionnaire in a workshop, and this was part of an ongoing effort of a working group that's now led by the American Association of Blood Banks to evaluate the donor history questionnaire to attempt to simplify it and to evaluate each of the questions in here.

This is an initiative that we thought was important for a number of years, and as a result of resource needs, the FDA itself was not able to undertake this. And, in fact, it's still probably on a wide scale. It's still not--we're not able to address the overall questionnaire. But I think that this task force is doing an excellent job and has taken on an important task in evaluating it question by question.

We've also worked at the FDA on guidance development. There are guidances in the works for computer-assisted and self-administered donor interviews. We've separated this particular guidance from a guidance that we anticipate will result from the Uniform Donor History Questionnaire Working Group because we have already accepted supplements to licenses to allow blood centers to use computer-assisted questionnaires and self-administered donor interviews, and this guidance documents the considerations that we used in approving those supplements.

A draft guidance has been issued--and we hope to finalize that soon--for hemochromatosis patient donation to include this particular resource and benefit those particular patients.

One of the major comments that we heard throughout our recruiting workshop was that the industry needed some guidance on donor incentives. The question of appropriate donor incentives had always been a compliance issue, and our Office of Compliance took the lead on developing a draft compliance policy guide on donor incentives, and that's available like our own guidances on the FDA website.

To go back to the question of availability, availability is the main issue for FDA, and it is the safety issue. And as Dr. Wallace has pointed out and the following committee discussion addressed, there's many components of availability. The availability itself is difficult to measure. We look at reports of shortages such as canceled surgeries, urgent blood drives, as well as other attempts to measure various components of availability, that is, both the demand side and the supply side.

Not to belabor this, from my naive view, demand is composed of a number of various characteristics, not only the use of the inventories at the transfusion centers, the amount of resale from one particular center to another, and certainly many other components. And the supply side is also affected by how many donors, how many regular donors donate more frequently than others, how deferrals reflect the availability of donors, as well as recruiting efforts. All of this is, I think, addressed in Dr. Epstein's question about how elastic is the entire distribution chain.

To conclude, let me say that FDA is in favor of gathering information that can be used as a part of our decisionmaking. We would like to know what components there are and the availability of various components. We've brought this question here, and Dr. Nightingale and Dr. Gomperts will lead that discussion later this morning to address these issues of what data should be collected, and then the ultimate decision of who should take the role of overseeing this entire program and whether or not it's a PHS component or whether it resides outside of our service.

Thank you.

[Applause.]

DR. GOMPERTS: Any comments or questions for Dr. Lewis?

[No response.]

DR. GOMPERTS: There being none, we will move ahead. The next presentation is Mr. Jan Bult, Plasma Protein Therapeutics Association, a presentation on the current monitoring of the supply of plasma derivatives.

MR. BULT: Thank you, Mr. Chairman, members of the committee. My name is Jan Bult. I'm the president of PPTA, and it's a privilege being here to present our three-year results of a data collection effort that we announced, at this committee, as a matter of fact, three years ago.

What I would like to do as introduction is mention the fact that we are a global organization. We do represent 11 member companies all over the world; however, in the United States, we have six companies that are active in the North American marketplace. That should be reminded because that doesn't mean that we have information for all companies available because they're not active in the States.

Now, I mention to you the presentation that we made in 1998, and that was on April 27th, almost three years ago, when we were in the middle of an IVIG shortage. Some of you will remember the very lively meeting. Certainly I do.

At that meeting, we made a commitment, and what I did, I quoted the commitment that we made at that meeting, and what we said is that we commit to continue our data collection effort that we started. We will collect and make public information on production data every three months, quarterly, so that all parties will be able to understand the current production trends. At that moment we anticipated that this information will allow us to better understand, predict, and respond to the threat of any future short supply situations. Later in my presentation I will give you our perspective how that worked.

Now, let me tell you what kind of products we collect information about. As you can see in this list, we have seven groups of products. We collect information on albumin in different formulations, recombinant Factor VIII, high purity Factor VIII, and immunoglobulins. That information is made available to the entire public, and I'll tell you later in more detail how we do that.

We also see three groups of products: intermediate purity Factor VIII, Factor IX pure, and Factor IX complex. That information is collected. However, it is not made available, and the reason for that is--and, again, I will explain that later in more detail, but it is important that we maintain a confidentiality for the individual providers. We are a small industry, so we want to find a way to publish data that guarantees confidentiality. Therefore, the rule that we have is that there have to be at least three providers to make information public.

Now, the current data providers, as you can see from this list, we have five member companies. Those are the companies that are printed in blue. But we also have two non-member companies. The American Red Cross and Novartis, which, of course, are significant providers of products to the American market, are part of the system. And that gives you a very accurate picture.

How do we guarantee confidentiality? In the previous discussion, the issue already came up. What happens when you have a sole provider of blood products in a hospital. We are facing similar situations. And the way we deal with that is that we have used a third party. We use a third party, which is Georgetown Economic Services. It's a company here in Washington, D.C., and that organization receives all the company-specific data. The data doesn't come to our office. It goes straight to Georgetown Economic Services. And that organization aggregates the data that we can use for publication.

I already mentioned to you that we need to have at least three providers in order to guarantee confidentiality. Two would be a problem for competitive reasons. Three is the minimum that we use. However, what we do is we submit all the company individual data to FDA, so FDA has a full picture what's happening in North America, and they get it every month.

Now, if we talk about confidentiality, it is one thing, but I think it's more important to talk about methodology. How does the system work? As I mentioned before, we submit the data to the third party, Georgetown Economic Services. Of course, we use a standard operational procedure so that we make sure it's all done in a standardized fashion. We want to have the data completed by the middle of each month. It's very important to recognize--earlier on I said that we made a commitment to have quarterly data. We improved the system very rapidly, and we are now on a monthly data-gathering effort. And in order to make it work, we need to have the data in by the 15th of each month.

The next step is that Georgetown Economics is then going to aggregate the data, and then what we do is we provide the--we allow the data providers a two-day period to verify their company individual data. So they don't get the full picture. They get their own data back to make sure that we have accurate reporting.

Then what we do is we provide FDA with all company individual data, as mentioned before, and around the 25th or in a couple of days we will be able to publish the results of the month of March.

Now, of course, if you go in more detail, the standard operational procedure is a pretty detailed document, but just to give you an indication about the timing, what we do is we provide the companies with the questionnaire, and it's the same questionnaire that we use every month. But then we check around the 10th of each month whether there's an anticipated problem: sickness, illness. You need to have a contact person. And as you can imagine, with a small industry, a small number of participants, we want to make sure that we have the full picture. So if there's any anticipated problem, then we will contact the company to make sure that they are participating.

Up to now, we have never had any problem. The companies are very willing and very cooperative. So we've never had to use this rule.

As I mentioned, around the middle of each month we need to have the data. The third party reviews and checks for any anomaly and asks for clarification. I told you about the verification period. And then we aggregate the data, give it to FDA, and make it available to the public.

Now, if we talk about public, what are we talking about? We send it every month to the consumer organizations. They get it actively from us, and as you can see, it's the Alpha One Foundation, Alpha 1 National Association, Committee of Ten Thousand, the Hemophilia Federation of America, Immune Deficiency Foundation, and the National Hemophilia Foundation. They get it every month, and we make sure that all the consumer organizations that are interested get it in this manner.

In addition to that, we provide the government--we send it to the Department of HHS. It goes to Dr. Nightingale. It goes to FDA, Dr. Weinstein. And as I mentioned, in addition, they get the individual data.

To make sure that the entire public is able to see the information, it's on our website, and we have two addresses that allow everybody to look into details of this data collection effort.

Now, what is the kind of information that we collect? We have the total beginning inventory of each individual month. That's what we collect. Then what we do is we ask data for an IVIG emergency supply. For those of you in that discussion three years ago, that was one of the initiatives that we developed to have an emergency supply, and that's measured.

We also have the information about the distribution in the U.S., and then we also ask for the impact of recalls or withdrawals when applicable. In '98 we had the situation that there were recalls because of the CJD situation, and it was very important to know what the impact was. So since that moment, we asked the question. However, in the last couple of years, the response was zero. So I question whether we should continue with that question because we have other systems in place to inform the community about recalls. But that's not the topic for today.

Now, Dr. Nightingale asked me to say a couple of words on what you can do on any data gathering on future supply and what legal constraints are in place. I have two slides on that.

The first one is that--and that's a no-brainer--each association, of course, is free to use publicly available information. Well, it doesn't always mean that everybody sees what's published all over the world, but we make a serious effort to know what's going on and use that information. But what you should not forget is we are dealing with a very concentrated industry. We have, as I told you in the beginning, worldwide 11 manufacturers, and here in North America we have currently seven members, six of them active in the marketplace.

Now, for certain products, we are down to only three manufacturers, and that means it's even more concentrated. And that mean we have to respect antitrust laws, and the smaller the industry, the more rigid the application of the law is. So we are--of course, we won't violate the law, but we need to respect what we can do.

If we talk about future supply, I put it here and I'm going to repeat it again. Unpredictable means unpredictable. Sometimes I have the impression that some people think that everything can be known. Well, that's not true, and especially when you are dealing with a small industry. If something unpredictable happens, it has an impact. And we've seen it, and we see it today.

The other comment is that industry is only one part of the entire chain. What I mean by that is especially when you want to measure inventory, we can measure what we have. But we don't know what is available in other parts of the distribution chain. Even we don't know what's in the fridge of the patients.

Can I get a glass of water, please?

Now, first of all, I'm not a lawyer so I hope that you're not going to challenge me in very detailed questions about this one. But I think it's important to list some of the constraints that we have to respect.

We are not allowed as an association--and, again, I remind you, we are a small industry. We have competitors on the table, and we're not allowed to facilitate information exchange among members which are focusing on the future situation. Of course, we are free to talk about what has happened and what is the retrospective data, but about future issues it's very difficult.

Now, you can think you can be very creative and find ways to have public announcements and organize meetings and do it that way. It doesn't work. It doesn't work because there are statements that say these disclosures could be viewed as a means of signaling competitors so they can make plans based upon the activities of the other manufacturers. And we cannot do that.

The other thing that you should know is that the antitrust laws do not cover pricing issues alone. They do deal with other issues, and I wrote down what we are not allowed to do. We cannot talk about how to limit production, how to reduce inventories. Well, we had a discussion today about inventories. I just want to make you aware that we are at the edge what we can do from a legal point of view. We cannot coordinate output, allocate capacity. That's one of the things that you hear these days: Can we sit together, can we find a way to know from each other what's going to happen in the future? We as an association are not allowed to do that.

Quotas, the discontinuation of particular products, or limit supply, those are the things that we're not allowed to do.

Now, my message to you is not to tell you what the barrier is. I'm not here to tell you why we can't do certain things. I'm here to tell you that we're willing to explore alternative ways to address the issues that are important to you. But we need to respect the law, and you cannot expect us to violate the law. And certainly in my position, I'm not going to do it.

Let me give you some examples of what we've done in the last three years. We started in '98 with the question about immunoglobulins. So what you see here is the result of three-year data-gathering effort, where you see the distribution of immunoglobulins in North America since '98. Those are monthly numbers, and everybody knows it's very difficult to make any analysis based on monthly figures. But what I wanted to do was be very transparent and give you a full picture of what we have done.

Now, of course, there are different ways to look at these numbers. You can plot it in a different manner and compare the yearly distribution. So you can see that there is--whether there is a trend or not. I'm very careful in making any conclusion, drawing any conclusion out of this. But at least it gives you a full picture of what has happened.

Now, what I would like to mention is that one of the things that we did is we started to talk about a so-called ratio. What we did was we divided the inventory at the beginning of each month with the distribution of that month, and if that ratio is one, what it basically means is that we have an inventory of about one month, and that gives you an indication.

Now, if you look at the ratio since '98, you can see that overall we're still in short supply. If we have a ratio that's lower than one, we consider to be in short supply. Now, what that means is that nobody would accept that there's going to be a large inventory in a situation of short supply. And what this graph tells you is that, indeed, we try to find ways to bring products to the marketplace as rapidly as possible, don't build it up, and bring it to the patients where it's needed. Again, you can plot it in a different manner, but that's just detail.

Now, let's talk about recombinant Factor VIII, another issue for several organizations in the room today, extremely important because we are facing a shortage right now.

Now, what you see here, since '98, this is the distribution of recombinant Factor VIII in North America. Again, if you plot it in this manner, you can see that there's some monthly fluctuations, but what we're seeing right now is, if you look at the light blue color, which is for the year 2001, you can see that we started in January with about 65 million units, and in February it was down to, let's say, 55, something around that. I expect that the March data are going to be much lower because of the current supply situation, and that is a problem, because now we are back to the situation where we have only two providers. And the rule that we introduced was we're only going to make data available when we have at least three.

So there are two alternatives that you can consider. One is you can aggregate the numbers for recombinant Factor VIII and high purity to at least be sure that you are able to provide the full picture of the Factor VIII. The disadvantage is that when you do it, you will not be able to give the exact picture for plasma-derived Factor VIII. And in conversations with consumer organizations this week, we learned that that's a big problem, and they would really appreciate it if we find a way to continue the publication of high purity Factor VIII as a single group rather than combining.

As a matter of fact, later on today, I will have a conference call with our board to talk about it to see how we can accommodate that request.

Now, the other solution may be much better, and it is that you look to all manufacturers in the marketplace. There is a third manufacturer called Genetics Institute, and currently they are not part of the system. But I can tell you based upon ongoing dialogue--and my last conversation with Genetics Institute was yesterday--they agreed that it could make this statement. Genetics Institute is seriously willing to consider participation in this data collection effort. We are going to sit down very, very soon face to face to talk about the practicalities. I know that a similar meeting will take place with FDA. They are very positive about it, and we will try to resolve this as rapidly as possible.

MR. BULT: And if they are going to participate, we are back to three providers, and we are able to disclose all information. I think it is tremendous progress.

The ratio I explained to you, that what's happening, as you can see, for Recombinant Factor VIII now we are around the situation that we have one month of supply or probably less.

Now, you can do the same picture for the high purity, the plasma-derived Factor VIII. Again, the distribution in the last three years, and as I mentioned on other occasions, you see similar patterns, but what you see here is that there is a bigger inventory. You can see that the ratio tells you that we are, for plasma-derived Factor VIII, in a situation that is about three months of inventory. Well, that may be the ideal situation, depending where you're coming from, but at least it gives you some guarantee that if there is a monthly fluctuation that you have a cushion that you can work with and that doesn't exist for the other two products that I showed to you.

I have not, in this presentation, put in the data on albumin and other products, but I felt that this may be helpful to you to understand what kind of information we are getting, how it works.

Now, another important factor is how can we use this system in other parts of the world? Now, with the SOP that we developed and with the help of the third party, we started to use the same procedure and the same third party for collection on European data. Since the urgency for information of Factor VIII was so huge, we moved very rapidly, and on our website you can see information about Factor VIII and you can see in the blue column the quantity of plasma-derived Factor VIII, and in the red column it is the--I see a typo--it is plasmatic Factor VIII and not VII, and recombinant Factor VIII as well.

I know that some of the consumer organizations have asked us to find ways to introduce this collection effort in other parts. We have done it for Europe, but let me tell you one of the difficulties that you are facing in Europe. And as some of you know, and certainly you can hear, I am a European. Europe doesn't mean Europe. Here in the States you have a very good system, a functioning system, where you're able to collect data as a country. In Europe, you have individual countries, different rules, different languages, different cultures. Don't think that there is a Europe like you have in the States.

Let me start asking the definition of Europe? What is Europe? How many countries are part of Europe? Is that the European Union? Does it include Switzerland? Is it Council of Europe? So you have all kind of practical problems that you have to deal with. And therefore what we decided was to collect information about the 15 member states of the European Union because that is the majority of the information that's available. It's just a logistical problem, but I think an important problem to realize when you start data collection efforts in other parts of the world.

Now, I would like to go back to the commitment that I made on April 27th. We have said that we want to continue a data collection effort. I hope that I have been able to show you that we did it. We said that we would make available the production data, and I have shown you the results of three years. We said we were going to do it every quarter, we improved it. We are now back--we improved it to monthly data gathering. And yes it does give you production trend.

Another part of the commitment for us, are we able to better understand, predict, and respond to supply situations? I have a question mark around it. When we started, of course, that was our hope. But as you've seen from the numbers, it is very difficult to predict future supply. I do believe that the monitoring that we have in place works excellent. I think it works, and the results that you've seen in the last few years, really gives me proof of that.

However, when you are working in such a concentrated industry and when something unpredictable happens, then you are facing a different situation.

Now, that brings me to the question can shortages be predicted? Well, I repeat it one more time. Unpredictable means unpredictable. It is unrealistic to think that we will be able to give an accurate picture of things that may happen in the future. We do our best, but it's not always possible.

Then what you need to realize is what is happening when you have a small versus large industry. If you collect data from hundreds or thousands of institutions, of course the impact when something goes wrong with one or two is not as dramatic when something goes wrong in our industry. Therefore, that is a factor that needs to be considered.

I mentioned before, when we find a ratio of inventory divided by distribution less than one, we consider that as a situation of being in short supply, and we need to find ways to have a rapid delivery to the marketplace.

I think that the summary, if I look at the benefits, and I am looking at it from a consumer perspective, I think from the timeliness, monthly versus quarterly data is a very good thing. We've done it. It's very accurate because we are talking about real numbers. It's not speculation, real numbers. The transparency, I told you how we distribute the information and it's available on the website. Is it full disclosure? Absolutely. We have members and nonmembers participating in the system. I just told you about the willingness of Genetics Institute to participate, which gives us even a better picture.

As far as the confidentiality, that is very important because with a small industry, it is important that you find a way to guarantee confidentiality to get full participation, and we find a way to do that. I do believe that we are providing valuable information which has led to public recognition of the efforts that we have made.

I would like to finish by thanking all of the companies that have provided all of the data. I would like to thank Georgetown Economic Services for their tremendous work, and I would like to thank Brenda Norman, who is a data coordinator in our office who worked very hard to make this information available to you.

Thank you.

[Applause.]

DR. GOMPERTS: Are there any comments or questions for Mr. Bult?

Karen?

MS. LIPTON: So my big question is how much does all of this cost, and who pays for it? I mean, and including you just mentioned that it sounds like you have a coordinator in your office.

MR. BULT: Yes. You know, when you ask questions about money to a Dutchman, they're always sensitive, right?

[Laughter.]

MS. LIPTON: But you're always honest.

[Laughter.]

MR. BULT: I am. I am. Yes, I am.

The total cost is in the neighborhood of $40,000 a year. That includes the service of a third party, as well as our staff efforts. And the costs are paid by the data providers. It's a self-funding initiative.

MS. LIPTON: Forty thousand dollars, including the person in your office?

MR. BULT: Correct. So it's part time. It's not full time, it's part time.

DR. GOMPERTS: Other questions?

MR. WALSH: I'd like to thank you for your comments, and on behalf of the consumers, express our appreciation for the data that's been released and reported on a monthly basis.

I'd also like to thank you for your objectivity and directness with respect to the limitations of the PPTA's ability to collect data. One of the most important components of data to the consumer is what can we expect in the future? What treatment algorithms do we have to evaluate based upon availability of product, whether it's delaying surgeries or delaying holidays or stockpiling product to use during acute exacerbations.

So I think, based on the limitations that you've identified, do you think that there's a different system or there could be a coordinated effort? Do you think that because you're a trade organization and not able, based upon some of the antitrust issues, to be able to look at predictive data; there's a different process or a different structure that might be helpful?

MR. BULT: Well, one of the things that we discussed early on with involved parties is can we come together with a small group that's going to explore different alternatives, including the legal barriers that we are facing right now. I think there are limitations on what you can do. Of course, if you look at the demand side, you can look at the development in patients, the shift in, for example, in the Factor VIII supply, the shift in bleeding treatment rather than preventive treatment, what's happening. The shift that you see in the entire world is important. So you can see a trend.

If you look at immunoglobulins, we have seen an increase, I would say an average increase every year of about 10 percent in demand. I mean, those are numbers that you can work with. And I do know that individual companies are working to expand their capacity. They are building new plants. As everybody knows, building a new plant is not a thing that can happen overnight, but those are the things, in the long term, that you can do.

I think it's unrealistic to expect that companies will be able to put on the table all of their business plans, for example, to tell exactly what's going to happen in the world. You need to realize that companies have an obligation to provide patients in the entire world, and not just in North America or Europe or Japan. It is a global responsibility. But I can tell you we are really willing to explore other alternatives to have a better grip on future supply.

But as long as you have a concentrated industry, with a small number of manufacturers, and you are facing unpredictable issues, it's going to be a problem.

DR. GOMPERTS: Thank you. Other comments, questions?

DR. GUERRA: Is there any data that would allow you to link the number of affected individuals with a variety of conditions for which these products are used and the demand for those? I mean, is there a registry, for example, through some of the different associations?

MR. BULT: Well, we don't have that information. But I do know that, for example, an organization like IDF did a survey. I know they have a statement later on today. Maybe that is a good organization to ask this question to.

DR. GOMPERTS: John?

DR. PENNER: Just an interesting note, with respect to this committee, is last year we recommended or urged the hemophilia organizations to consider strongly converting all of the hemophiliac patients to a recombinant form of Factor VIII, as opposed to the plasma derived. As you can see, we have come to grips with that problem.

The deficiency of the recombinant Factor VIII now has put us into a position where product pricing has now gone about twice what it was before, and the shortage is a very, very distinct one. And we are faced with the other problem that once one has patients on the recombinant form, you cannot switch them back to the plasma-derived form without a great deal of concern.

MR. BULT: Can I comment on that? Because I think it's important to recognize that we all are aware of the current shortage with recombinant Factor VIII. And in our statements that we've made, we really applaud the efforts of the hemophilia community to work on contingency plans, find ways to look at scheduling of elective surgery, for example, but including the switch of recombinant to plasma-derived Factor VIII.

And I do understand the concerns, where you're coming from. I do also know, in practice, patients have been switching. I am really very happy that we can say that with all of the other initiatives that we have made in the last years, that the safety of those recombinant- and plasma-derived is really excellent. So, from that point of view, we have no concern, but there is more than that. There is emotion, there is the fact that you have to convince patients to switch and go back. I am fully aware of that.

We are working with our entire industry as hard as we can to address the current problems, to make sure that we are as open and transparent as we can be, and if there's any concern, I'm open for any question of hemophilia organization. I've done it in the past. I will continue to do it in the future.

DR. GOMPERTS: Thank you.

Yes?

DR. BUSCH: Yes, just the concept that unpredictable is unpredictable I think, you know, part of our goal is to try to bring better tools, better monitoring to be able to predict what's possible. And over time I think we improve that, and I think you've demonstrated that.

The one example of the recombinant--I'm not aware of the event that appears to have triggered this. It sounds like one manufacturer went off-line, for some reason, which is why you couldn't continue to monitor. A lot of these issues historically have been either regulatory or manufacturing problem issues that somebody knew beforehand that this was coming down the line, and these things could have been anticipated.

So I guess, for my interests, what happened, and why wasn't that predictable from your mind, the event that led to the recombinant Factor VIII?

MR. BULT: In the situation that we're facing right now, is that one manufacturer announced/released that there is a reduced--that release of recombinant Factor VIII is stopped for a 90-day period because of issues that came out of inspections. The issues needed to be addressed. It had to deal with a lot of proven documentation in the entire manufacturing process. There was a bacterial contamination that is public information that I can share with you that was unknown at the moment of the inspection.

I don't know the specific details. I can only refer to the public available information, and the company has done everything possible to make sure that the safety and efficacy of the product is not effective. As a result, release has been stopped and is expected to return very soon in small quantities and in normalization in about three months. That was a public statement that has been made.

Now, was that predictable? No. I think the good thing is that with all of the redundancy in place and the systems in place, the company was able to identify the problem and to address it. And, of course, if it can be predictive, it would be nice, but this is a good example of why those things cannot be predicted.

What can be predicted, to give you another example, is that if a company has to schedule maintenance, that's a good thing. You need to schedule maintenance. Of course, that is going to have an impact on the output of that plant. And it's good management practice to make sure that you plan in advance, and you make sure that you're covered for that period. Well, if those things happen at the same time when an unexpected situation occurs, then of course you can have amplification of the results, but I think that's very--pretty detailed of what's going on right now.

DR. GOMPERTS: Other comments?

There is one comment from the floor. Professor?

DR. ALAIN: Yes, I apologize to intervene being European, and I am not going to dispute the definition of Europe.

[Laughter.]

DR. ALAIN: My question is about the predictability of events. One that seems to me very distinct that might have considerable impact on the supply in the United States is the possibility of other countries than the U.K. to ban the plasma usage because their own plasma usage because of the variant CJD. And I know, for instance, that France has been considering it.

I wanted to ask what are the contingency plans? What's predicted? What's going to happen? What's going to be done if this occurs? Which is quite, in my view, predictable.

MR. BULT: What is happening right now in different parts of the world is that there is increasing concern about the collection of blood and plasma related to BSE issues, especially in Europe and in new variant Creutzfeldt Jakob disease in some parts.

We need to realize, No. 1, a lot of the decisions nowadays are political. It's not always based on pure science. There is a lot of politics involved here. The fact that two ministers in Germany lost their jobs because of some, let's say, questionable management of the BSE crisis is a very clear example.

What we need to do, as an industry, is we need to make sure that we have full dialogue with the regulatory agencies all over the world to make sure that we have a good understanding, similar understanding about what's going on right now. Let me give you one example.

When you look at the list of countries that, and their risk categories, there is a group of categories that list countries where you have risk, and the highest category is United Kingdom, Portugal, and I think it's Ireland, if I do it by head. And then you have Class III, and it goes up.

Now, if you look at the definition, every country that has more than 100 cases of BSE is in the highest category. Well, United Kingdom has 185,000 cases. Portugal has 600, and they are in the same category, and everything is far below. So I think we need to have a very clear understanding of what it means and how the risks should be associated.

Now, I have seen examples already where countries have a problem when or ask questions when products are manufactured from European plasma. I think that is something that we have to fight against. We need to go, and to dialogue, and to have a scientific discussion of what's going on. Everybody knows that the plasma for U.K. is deferred. Everybody knows that France had a discussion about what they would do with their own plasma. The French plasma is not deferred. The French government has said they have a preference, if possible, to look into other ways of distribution, but they have not banned their own plasma.

And what we are doing as an industry, we are in dialogue with the regulatory agencies. We are trying to do whatever we can to be as transparent as possible. We talk about our initiatives, what we do in our process to make sure that the risk that is still perceived as a theoretical risk is even further reduced. And I do believe that the bottom line is dialogue, dialogue, dialogue, and make sure that there's an exchange of scientific information and that we're all learning from the same science.

DR. GOMPERTS: Thank you.

DR. PENNER: Just a quick question. I know you can't do anything more than speculate, but on easing of the recombinant Factor VIII and IVIG shortages, do you have an idea when one might see some easing of these shortages?

MR. BULT: Well, first of all, I never speculate, and I'm not going to do it here as well. What I can do is I can refer to public statements that have been made. As far as the situation where recombinant Factor VIII is concerned, the company that was in question here made statements that they expect a release this month within a four-week period and a normalization of their release in 90 days. That is a public statement. If there is any change, we have to talk with that individual manufacturer.

As far as IVIG is concerned, companies are building additional capacity. We are bringing products to the marketplace as rapidly as we can, and it's a function of time. Building additional capacity is not an issue that can be addressed overnight.

DR. PENNER: Time in years or are we talking about a year or two or are we talking about five years or are we talking about six months?

MR. BULT: As I said before, I'm not going to speculate because I got the same questions four years ago, and I think that the experience so far has shown that if I would have made any prediction at that moment, that I would be in trouble now.

DR. PENNER: But you would expect those programs that are coming online are going to be coming online with new manufacturing in the next year, that you know of?

MR. BULT: Yes, that is true. What I know is that, for example, companies that have been out of the marketplace last year, and that situation occurred in the United States, back, so that's going to be a factor and that should result in more availability of products.

DR. GOMPERTS: Okay. Thank you. Thank you, Mr. Bult.

We will take a 10-minute break. Thank you.

[Recess.]

DR. PENNER: Dr. Weinstein? Thank you.

DR. WEINSTEIN: I'd like to thank the committee for giving me the opportunity to talk about FDA's perspective on monitoring the distribution of plasma derivatives.

I will talk about the reasons for monitoring distribution. I will describe current monitoring efforts, and I will discuss some of the good and bad points of the current system. Finally, I will mention some additional data that might be helpful to have, along with concerns in obtaining the added information.

The first question to consider is why monitor the supply of derivatives. As my colleague, Dr. Lewis, has already mentioned, the primary reason is because the availability of product is a safety issue, and distribution data is one element in helping to ensure that product is available.

In considering shortages, one of the most pressing needs that the FDA has is to be able to tell when the shortage is underway and when it has ended. Short-term regional spot shortages occur occasionally and cannot always be distinguished from the beginning of a severe crisis.

The need for a good monitoring system became very apparent in November and December of 1997, when we were suddenly confronted with an acute shortage of IGIV. The right kind of distribution data can give us hard evidence that a shortage is likely to occur or is underway.

Distribution data enables us to compare present distribution to historical output from a single company and from the industry as a whole. Had we known that inventories and distribution of product were being severely eroded in 1997, we might have been able to take action to avert or at least moderate the crisis that occurred at that time.

Distribution data can be used to establish that in a specific situation, product availability ought to be considered as a major element when evaluating the release of a product that is under regulatory scrutiny. We can then weigh the risk to public health of the potential product shortage versus the risk of a regulatory issue under consideration.

Distribution data helps us to assess whether short- and long-term efforts to increase distribution are working. We can tell, for example, how much the licensure of a new product or a new plant or a production suite will potentially raise the overall level of product on the market.

This quantitative estimate of impact can help us to prioritize our efforts when we have a choice among several different activities that could increase distribution.

Finally, distribution data can help us to assess whether changes in policy have affected distribution. For example, distribution data can help us to evaluate how the change in our CJD policy in September of 1998 affected the supply of plasma derivatives.

Now what is the current situation of data reporting to the FDA? In November of 1998, CBER required manufacturers to report monthly distribution figures of plasma derivatives and analogous recombinant products to the FDA. Subsequently, industry and FDA agreed that Georgetown Economic Services would collect the data. Aggregate data from all manufacturers of a given product is publicly available through PPTA, and FDA receives individual company data, as Jan Bult has described to you. This information is approximately a month old at the time of receipt of the information.

We also receive the first day of the month inventory and emergency supply data, which are voluntarily included in both sets of information.

Now, this graph shows a representative example of the publicly-available data from PPTA. Shown here is the recombinant Factor VIII. You see first day of the month inventory and distribution data. This is for, say, November of 1999. Note that the areas that I have labeled shortage and no shortage. This includes the time period of December of 1999 and January of 2000, when there was no shortage.

If you look at the distribution data in September of 2000 and October of 2000, this is a period that we did have a shortage, as reported by the consumers and distributors of product. And so we see that, in fact, this data does not clearly show us when a shortage is occurring or when it is likely to occur. One may speculate, for example, that the demand in the winter months for recombinant Factor VIII might have been less than the demand in the fall or in the summer, late summer and early fall. And so this element of availability of distribution does not necessarily give you an indication that a shortage is actually occurring.

Now, possibly a more frequent collection of data that has been requested by the PPTA will give us a better indication of shortage for the historical record. More frequent collection of data should decrease the averaging effect that we get from aggregating an entire month distribution into one number.

What is the value of the present distribution data? The data has proved valuable in giving us an accurate figure for total distribution. We don't have to speculate from indirect sources about yearly or average monthly distribution. We can also know what is the range of variation in distribution.

One place where this information is useful is in assessing the potential impact of obtaining new sources of product. The broad overall trend in distribution that can be derived from these data tells us whether product distribution is improving or not over a long period of time. Accurate information about an individual company's market share helps us to assess the degree into which a disruption of that company's distribution will affect the total market. Information about the ratio of first day inventory to monthly distribution can provide some indication of the buffering capacity in inventory that can moderate production disruption.

Additional data is needed to manage and monitor an acute crisis in distribution. In this situation, the information is usually acquired by an all-out effort by FDA's staff, who make numerous calls to manufacturers, distributors, health care providers, and patient groups over a very short period of time. In these situations, real-time data and near-future projections are needed.

We need to know the amount and source of immediately available product to advise consumers about potential alternative sources of material. We also have to have periodic updates from manufacturers to find out whether the projections for their product distributions are, in fact, meeting their goals. Knowing the average amount of product normally available derived from our monthly updates is helpful in the situation because it gives us an estimate of the shortfall that needs to be made up.

Now here are some additional suggestions for distribution data that theoretically might be good to have. These include very current data, rather than month-old information about product availability, from manufacturers would give us a better idea of the real-time market situation. Information about demand would give an indication about the degree to which actual output is falling short of desired levels.

Demand data from manufacturers perhaps could be based on the amount of product involved and filled in back orders. This information could be collected over time to provide data for trending analysis.

It might be valuable to have a statistical sampling of product availability from sentinel distributors, consignees, home care providers, hospitals and pharmacies. The sampling sites would depend on the nature of a product. I have already mentioned the importance of having data to tell the consumer where to access product and whether a manufacturer is achieving projected distribution goals.

In the realm of predicting a shortage, FDA does not routinely have information about whether a manufacturer has slowed production or has decided to decrease release of product in the U.S. market. We may learn about such an occurrence months after it has been underway. Such information would be valuable to have as an early warning of potential shortage. Manufacturers could proactively report the occurrence and estimated duration of distribution slowdowns.

Finally, it would be helpful to know how much product is being used for various therapies. Such information could aid in focusing our attention on reducing those inappropriate off-label uses that consume a lot of product.

Now, however, before we can begin to think of collecting future data, we should consider a number of critical factors, and these include who is to collect and evaluate the situation, should the information be public, what are the legal and practical implications? What are the unintended consequences? What are the antitrust issues? What are the costs, and is the outcome worth the effort?

Just one example of such collection of data and how it might affect demand. If projections about future production are made publicly available, they may raise expectations that cannot be realized because of unforeseen setbacks or, if projections are for less product availability, hording and speculation may further decrease availability.

Finally, resources are needed to answer these questions before we begin to pursue further expansion of data collection.

Thank you for your attention.

[Applause.]

DR. GOMPERTS: Any comments or questions?

Karen?

MS. LIPTON: I just have a quick question, and actually maybe Jan is the better person to ask. But you talk about this one-month inventory. How did you decide what was a reasonable level of inventory? And this goes back to something that Ron was saying about you get agreed-upon inventories. I wonder or doubt probably that they are really uniform because it sounds like you customize it per hospital. But how did you agree that one month was the appropriate--

DR. WEINSTEIN: Well, actually, inventory information is a one-day inventory. It's the first day of the month inventory, and this is just a snapshot in time of what a company has at that particular moment.

MS. LIPTON: But when you talk about shortages, the question comes down to but it's just the ratio on a single day is what you are saying.

DR. WEINSTEIN: Versus the entire month. An interesting question, in fact, is what is the true significance of that ratio? Because, in fact, you could produce, distribute a very large amount of product and have a very large inventory, and that would be a pretty good situation. And so that ratio doesn't necessarily mean that you're in a shortage situation.

MS. LIPTON: So what you find out is you're trending, but you're not sure what you're actually looking at.

DR. WEINSTEIN: Over a period of time I think that you can say that the best or a better way to see the industry is if they had a lot of product in their inventory at the beginning time, that they weren't sort of scraping the bottom of the barrel, essentially. But maybe Jan would like to explain that.

MR. BULT: Thank you, Karen.

In 1998, you have to go back in 1998 when we had the shortage with immunoglobulins. There were a couple of factors. One was, of course, the demand in the marketplace. Patients were very concerned.

In addition to that, we were accused of stockpiling, and this was one of the good proofs to make sure that we were not stockpiling and had all efforts in place to bring products to the market as rapidly as possible. The reason why we developed a ratio was because we had hoped that we could use this as an indicator for future supply. And when we started, we of course didn't know how this was going to work. And as you've seen in the numbers, that is not the best indicator.

DR. GOMPERTS: Other comments? Questions?

MR. WALSH: Just very briefly, I'd like to commend the FDA in their vigilance to improve the collection of data, firstly, and the factor that you consider availability a safety issue. Is there anything that you feel that the FDA can do or that we can help you with, with respect to assisting industry to evaluate different methods of perhaps compliance with GNP observations or issues so that it would decrease the amount of time that the production is effective? Specifically, alpha 1, you know, we're going to be completely out of product, as Nancy Buelow will testify to, for anywhere between six and eight weeks now, and that is because of an extended shutdown after the annual maintenance period to comply with GNP observations.

DR. WEINSTEIN: Well, it's very good for consumer organizations to make public what the situation in the market is, and it sort of highlights the urgency of activities that have to be carried out. Industry decides what they have to do to improve their facilities or manufacturing facilities, and they are working I'm sure as expeditiously as they can, but I do think the that role of the consumer group is to highlight the necessity of having these products available.

We, of course, in the FDA will work as hard as we can to make certain that companies are going to be able to provide a safe product.

DR. GOMPERTS: I will take one more question, and then we do need to move no.

COL. FITZPATRICK: I just wanted to go back to what Dr. Chamberlain said for a minute, and your question about what are the costs and is the outcome worth the effort. Since you've been collecting data since '98 or this data has been available, one question would be what outcome would you attribute to the collection of data versus public pressure or pressure from organizations? And the other would be is there a way to evaluate whether collection of sentinel events, such as spot shortages or inability to fill orders or inability to treat patients wouldn't accomplish the same thing, as opposed to the collection of which data would be best to collect; in other words--

DR. WEINSTEIN: All data. Well, let's look at this carefully, again. One of the very good outcomes of the current situation is, in fact, this idea of helping us to prioritize our efforts there. We now know, as we did not know it in an earlier time, exactly what the, say, monthly distribution is. We can determine that the licensing of, say, a new facility, a new plant is going to have at least the potential of raising this a significant amount or, potentially, not. And when we have multiple things that we have to do, we can perhaps focus our attention on those things that are going to have the most impact on raising the current level of--raising the amount of product in the short term if there's a shortage situation that we're trying to rectify. Perhaps other things could be delayed.

As far as sentinel information, there is this element of being able to help consumers find where a product is available, and some of this sentinel information might be helpful in that sense.

It also would be probably an earlier or give us more real-time information that is, as I said, the stuff is a month old by the time we get it. It's good for historical analysis. But are there certain triggers, in fact, that will be able to give us a sense of what the actual, say, uses of the product? Are people having difficulty in getting it? As I pointed out, there might be a difference in usage, say, in winter months versus summer months here that is not captured in the simple distribution data that we're getting.

DR. GOMPERTS: We do need to move; is that okay?

DR. EPSTEIN: Well, Mark is closer to the data than I am, but my experience is that the monthly data does not help us predict shortages because we always learn after the fact, but that it provides us baselines and that it equips us to think intelligibly about the current situation. So, for example, if we know market share of a particular company, we know they're going off-line, we then understand the impact of what we're dealing with. That would just be one example. So it's the issue of having norms.

DR. GOMPERTS: Thank you. Moving on to Mr. Patrick Schmidt.

MR. SCHMIDT: Good morning. It's going to take me a minute to get set up.

DR. NIGHTINGALE: While Mr. Schmidt is getting set up, I do not wish to say anything that would interfere with the discourse we have here, but I would remind you that the next break in the meeting comes after Ms. Fredrick speaks.

MR. ALLEN: Just for the record, I had a question for John.

John, in your community, do you guys have a better idea of what the demand is on a monthly basis?

MR. WALSH: The bottom-line demand exceeds supply with only one manufacturer in the market and with a projection of who knows somewhere between, you know, let's say anywhere between now and three years we may have another IV product or two, and between now and four or five years, we may have some aerosol products on the line, which would really address shortage issues.

MR. ALLEN: But if a manufacturer were to say to you, in two months, I'll have X amount of units available, would you be able to tell by that whether or not there'd be enough for--

MR. WALSH: Yes.

MR. ALLEN: Okay.

DR. GOMPERTS: Mr. Schmidt, you're on.

MR. SCHMIDT: It's going to take me just one more second. I'm a little nervous because I have a very temperamental laptop, and without my 11-year-old daughter here, I never--

[Laughter.]

MR. SCHMIDT: I never boot it up without her here. So I apologize. A screen saver courtesy of my daughter, so forgive me, please.

Let me also first say I am nervous, and not because it's a U-shaped crowd or U-shaped panel, and everybody is smarter than I am because that happens to me all the time or that I'm turning my back on business partners and community members that I promised never to do. I'm nervous because I'm excited. The enormity of the questions that were asked and then our opportunity to maybe offer some of our perspectives as a plasma derivative distributor to solve some of those problems, that's what I'm excited about.

[Pause while Mr. Schmidt set up his computer.]

MR. SCHMIDT: It's a pleasure to be here. I think it's my second or third time in front of the Advisory Committee. It's always an honor, and thank you, Stephen, for inviting me.

We were talking about the enormity of the questions. This was the first question I think on the agenda that was proposed. And thinking back of our history, every innovation that our company has undeservedly been accredited has come from other sources, from our customers. Every idea about innovation has come from our customers.

I was driving to work the other day, and I noticed on the side of the road an idea, and how to address the availability of blood products throughout the world. And while I was driving, I saw something, and I knew it was a sign, and this was the sign that I saw.

[Laughter.]

MR. SCHMIDT: And it was on the side of the highway. And I couldn't help but notice that if they were called the First Day Adventists, they would be the FDA, but--

[Laughter.]

MR. SCHMIDT: This is what our company lives for. Everything we do, if it doesn't meet this minimum standard, our company does not get involved in it. And I'm the president and CEO of FFF Enterprises. We were founded in 1988, and I'm going to try not to make this a commercial for FFF. I probably will end up doing so. But we're located in Temecula, California, the up and coming wine region of the South.

We have four business organizations, and I'll go through them very quickly: Plasma derivatives, vaccines, specialty distribution of these high-tech products. We have a glove division. We're very excited about addressing the latex allergy problem in the U.S. And something I want to touch more about is a division called Lifetree Technologies. We have an integrated clinical trial manager, and we have innovative lot tracking system to track plasma derivatives to the end users and hospitals. It's in place today, and we're using it today.

Lot track ID, investigational drug accountability system, we first developed at M.D. Anderson. Lot track IM, an inventory management system. All surrounded around lot tracking and managing inventory real time. It's being beta tested at City of Hope in Duarte, California, right now. This is lot track IM. I can take some questions about it later if there are any.

And then NuFactor, which is a direct-to-consumer business that we have distributing antihemophilic clotting factors.

We have a very large share of the albumin market and a very large share also of the intravenous immunoglobulin market, the IVIG market.

We talked about shortages. And I think Mr. Bult talked about unpredictable, is unpredictable. An executive in the room today who asked not to be named, a dynamic, handsome man, said, "The risk of something happening in the plasma derivative business is 100 percent." And it's so true. So you can plan for unpredictability, and that's what our company does.

I want to touch on what causes a shortage: raw material shortages, manufacturing stoppage--we talked about that this morning--team biologics. It might have been risky putting that up there, but we support the efforts of team biologics. We support the efforts of making the products in the U.S. safer. And as far as I know, team biologics at this point are still undefeated. An undefined distribution system causes shortages. And I think in this part of the U.S., we have an undefined distribution system for some products.

What we're talking about today is the scarcity of utilization and demand data. This has a dramatic impact on shortages, the scarcity of data. I'm not from this industry. I was a former professional football player. Statistics are a big thing for us. And I was struck by how little data there is on demand and utilization data in the U.S. surrounding these very important products.

Our company has a legacy of responsible distribution of a variety of different shortages. Today, we're involved in the TD vaccine shortage, the thrombin shortage and the recombinant Factor VIII shortage, things we're helping to manage today. The 2000 influenza shortage was a public health nightmare, and I'm afraid we might have another one coming on our hands.

So, if you have a lack of data, a standard approach to allocating scarce critically short products is something similar to this: We call it the shotgun method. We have a limited supply. Let's ship the same amount, and this is an overcharacterization, an overdramatization, let's ship the same amount to everybody. So here's what happens, and this is what we're setting out to change. You can only change this with information systems. So everybody gets ten. This is just an example. Everybody gets ten. But their real demand, unknown to the distributor, to the manufacturer because of the unavailability of the information is the lower number.

So what's the result? In black, you have an excess of supply, exacerbating shortages. In red, you have the worst-case basis, unmet patient need. In only one instance there, in Texas, is where you hit it right on the number. If you have the shortage of supply, you have to stretch the available product as far as possible. To do that, you have to know what the demand is.

What we do at FFF is we combine the science of distribution with the art of allocation. Allocating critically short products is an art form because the information is dynamic, and it's rapidly changing. How much is in the pipeline? What is the pipeline? Is it from the time it leaves the manufacturer's dock? What happens to it after that? The pipeline is big and large after it leaves the manufacturer's dock. How much is in the pipeline? Today, very few people know that information. To be effective, we have to know it.

We created something that we call interactive allocation, and it's based on a combination of people, and it takes human resources to do this and technology. And here's the difference, the same scenario, same product. Here's what their actual demand is, same number of units distributed as well. Here's what the distribution to them is, and that's what the outcome. And, again, it's a combination of people and technology, not mutually exclusive of one another.

What should we do in the U.S. to monitor the availability of blood products? Another enormous question. I can't tell you if we get the data what to do with it. I can't tell you who should, but I'm here to maybe offer some solutions on how to get the data. We believe we have some technology tools that can help us do that.

I'm going to segue into our Lifetree Division. We have what's called an integrative clinical trial manager. And if you imagine, and I know this group is very aware of the accuracy of the data involved in clinical trial data. It's a web-based system. It complies with all standards of 21 CFR Part 11. It's remote data capture, electronic data entry system. If you have a web browser, you can have access to our system.

Document management, IRB process validations in the future, electronic signatures, data capture. Again, a remote, electronic real-time, not 30 days after the fact, not after we're already having a problem, but real time.

Customer reporting. So we took this system, so you're probably wondering why are we talking about a clinical trial manager system in terms of data capture. It's a very smart system, but it's not smart enough to know what it's capturing. It can capture data, it can capture any data, and it can do it remotely. And on a very short notice, we created just some slides, this is an actual shot from our application, we created some slides where today manually, within weeks, manually, blood centers could be giving us their data. Not giving it to us, but we could be capturing the data. I don't know who is going to get it or what we'll do with it afterwards, but we can do this in a very, very short period of time inexpensively. This is an actual screen shot. It took us about two hours to create this screen, so very, very quickly.

So, if you use your imagination here, it's 5 o'clock, it's at the end of the day, what happens? We start collecting data from around the U.S. It goes into a central location, a central repository, if you will. Again, who is the custodian of that information? That's for you folks to decide, but that's what we could do.

Being a former football player, would you like to see instant replay? We could do that again.

[Laughter.]

MR. SCHMIDT: I'll go on. The next morning we could have real-time data collections, data on collection. What did we collect the night before? And not knowing this business real well, we just made up some numbers. These are not actual numbers, just so you know.

The next morning, units released for distribution. What do we have in inventory? This would be an aggregate number. And we could do this very quickly, very efficiently. And then real-time data on units transfused. What do we have, what's available, and what was used? And is this system in use today? Yes, it is. It's in use in development today in clinical trials around the U.S., international surveillance projects, international patient registries we're in development of, and a DNA sample documentation program is what we're using our ICTM application today.

And I think it'll give us, if we're asking what is the impact, thinking ahead, TSE deferrals expand, we need to know what the impact is. If we're asking what the impact was, I believe it's too late. And I think a tool similar to this we'll be able to answer that question--what is the impact? What's the availability? If we're asking what was, I think it's too late because it's incumbent upon all of us I think to create a system that meets the needs of our patients because they're not aware of this meeting taking place. They're not aware of this scarcity of information. And what they're expecting from us I think is very important, a perfect outcome, and we owe that to them.

So the last thing I'd like to close with is somebody asked me what would you like if you could ask the Advisory Committee what their recommendations would be--nobody actually asked me that. I'm making this up--but here's what my wish list would be: Mandate lot tracking in all settings of health care. The lot tracking can take us to the demand data, it can take us to the ICD 9 code, it can take us to utilization data. In all settings, this is a minimum requirement I think that should be done.

And then I think what we're talking about here today, the last part of the wish list would be implement a data acquisition strategy. We can acquire the data. I think that's the easy part. What to do with it beyond that would be for much brighter minds than I have for this group here, and that's our presentation today. Thank you very much.

[Applause.]

DR. GOMPERTS: Any comments or questions for Mr. Schmidt?

[No response.]

DR. GOMPERTS: Thank you very much.

MR. SCHMIDT: Thank you very much.

DR. GOMPERTS: Thanks for your input.

MR. SCHMIDT: It's our pleasure.

DR. GOMPERTS: We're moving on to a consumer panel. And what we'll do here is to take each presentation, one after the other, and then take comments and questions for the panel as a whole at the end of it.

The consumer panel is to address how can potential shortages of blood products and plasma derivatives best be identified and monitored.

First up is Mr. Bablak.

MR. BABLAK: Good morning. My name is Jason Bablak, and I'm vice president of the Immune Deficiency Foundation. IDF is a national organization dedicated to improving the lives of primary immune-deficient patients through research and education.

I would like to briefly address the issue of monitoring the supply and demand of plasma derivatives from the perspective of a group that has recently experienced a shortage of life-saving medicines.

The primary immune-deficient community experienced a severe shortage of IVIG that began in December of 1997, and although it has somewhat lessened, continues today.

In late 1997 and early 1998, our community was unprepared for this type of event, both in terms of its effect on individuals dependent on IVIG and in ways the foundation could work to mitigate this effect. We had no forewarning of the oncoming shortage, no method for tracking IVIG supplies, and no procedures in place to deal with supply emergencies. Since then, IDF, in cooperation with industry, has identified several key actions that work to reduce the negative impact of the shortage and to better prepare us for future shortages.

From these efforts, which I will briefly describe, we also identified several general themes for dealing with shortages of life-saving therapies. First, identify potential emergency sources of products for patients in need. When the IVIG shortage first surfaced, IDF received hundreds of calls each week from patients, physicians and pharmacists who could not find product.

In order to serve our community better, we developed the IDF Safety Net Program, which includes participation from all IVIG brand donors and provides an emergency supply of IVIG for patients in need. With the help of our distributor partner, FFF Enterprises, IDF has allocated more than 422,000 grams of IVIG through Safety Net, and this equates to approximately 14,000 patient doses.

Number two, encouraged the development of prioritization protocols within the major medical centers and home health care companies so that during periods of severe shortage, the limited supply is reserved for the most medically necessary uses.

Three, survey the population to gain an understanding of the severity of the shortage and its impact on patient health. IDF was able to provide detailed information on the extent of the shortage and its effects on our patient community through a series of patient and physician surveys undertaken during the height of the IVIG shortage. We learned that nearly half of the primary immune-deficient patients were experiencing serious negative health effects as a result of not receiving their product on time.

This data allowed us to quantify the shortage and also to document this negative impact on the public health. This then brought the shortage to the attention of the government officials at the national level and has resulted in swifter actions to resolve the shortage.

Number four, monitor the supply. The plasma fractionation industry, through its trade association, PPTA, developed a method to monitor the supply of plasma derivatives in the U.S. While not perfect, it provides an historical understanding of the volume of product available and also shows trends in the supply line. IDF also developed an informal informational networks with distributors, group purchasing organizations and manufacturers to get real-time product release data and projections for near-term product releases.

Five, identify additional sources of product. We recognize that expanding the current manufacturing capacity for IVIG in the U.S. was a long-term goal and that what we needed was additional supply from foreign manufacturers and new generation products from currently licensed manufacturers. With this in mind, we began to seek out and develop relationships with interested parties in an effort to facilitate the testing and approval of new products for the U.S. market.

With this background in mind, I'd like to now take the next several minutes to answer the question posed by the committee: How can shortages of plasma derivatives be identified and monitored?

The IDF believes that an enhanced supply monitoring system, integrated with a real-time demand model, is necessary to improve our current understanding of the complex IVIG market. First, we need a more robust system of data collection that takes into account anticipated releases of product for each month in advance, as well as near-term projections looking out perhaps one or two additional months.

Secondly, we believe more communication regarding production and distribution issues from industry to the patient community is required.

Finally, we think a system to monitor demand for IVIG must be developed to complement the supply data that's being generated.

We applaud the efforts of industry and PPTA to make available its current distribution data set. We have been able to use this data to monitor the extent of the IVIG shortage, and it has also been very useful to gain an historical context of the volume of product in a given time period in relation to market conditions during that same time period.

However, as can be seen from the data we're going to see from the hemophilia community with regards to the recombinant Factor VIII market, the current data is a trailing indicator only and it's not useful for predicting or even identifying current product shortages.

Our proposal is to enhance the current system in place with the new data point, identifying the projected product releases for each month, along with a longer-term projection of an additional month or two. While this data will not be as reliable as the historical data we already receive, it would be useful in making short-term assumptions, especially when put into context of current market and regulatory situations.

It is clear that the manufacturers have at least some of this data individually and use it in their own calculations. In an industry that is constantly in short supply of its life-saving products, it seems only fair that we develop a method to communicate this information to those that depend on these products so that they may make their own adjustments when necessary.

The second tier of our proposal is that the manufacturers provide better communication of current operating conditions with the consumers. While we understand that each company desires to put its best face forward with its ultimate customer, at times it seems that we are the last to know of potential issues that may affect supply. We would encourage each company to develop an early warning system so that when potential issues do arise, a clear line of communication between the consumers and the company exists and can be used to share these developments.

We understand that legal issues may exist that would prevent or discourage the type of predictive system that we've identified here. In fact, it may require a third party outside of industry with a separate funding source or another solution that is not apparent today. We stand ready to help in this process. And as Ann Bult reported earlier, we're working with them to set up some sort of task force where we could get people together to develop some of these additional ideas so that we can develop a plan to provide this type of predictive data.

The final aspect of our plan that we are defining is a demand model for IVIG. We're launching an effort to predict current and future demand for IVIG. Despite what we believe to be a general reduction in demand for the drug during the past two years, the marketplace began to tighten again during the first quarter of 2001. Supporting this belief is our experience with our Safety Net Program, including right now we currently have back orders representing approximately 80 patients. Our current inventory levels are approximately 50 percent of normal, and we have started signing up new hospital customers who are typically served through their larger GPOs.

In addition to this data, several manufacturers are having difficulty releasing IVIG, and one brand has switched distribution channels and brand names which has resulted in significant market confusion. To put it metaphorically, we are skating on thin ice, and we do not yet know if the ice is getting thicker or beginning to break apart. Because of the sense of jeopardy that results from this lack of knowledge, the IDF is undertaking a study to monitor demand for IVIG across all indications. We believe that this data will fill the gap in the current understanding of the IVIG market and will better able us to identify and monitor shortages.

Thank you for this opportunity, and I would be happy to answer any questions.

DR. GOMPERTS: Thank you.

[Applause.]

DR. GOMPERTS: Could we move to Mr. Bias, please.

MR. BIAS: Good morning, ladies and gentlemen. My name is Val Bias. I am the president of the Northern California Hemophilia chapter of the National Hemophilia Foundation. I am also the co-chair of the National Hemophilia Foundation's Blood Safety Working Group. I guess they lucked out that I'm an adult with severe hemophilia, but just so you know.

I am here today to say that the PPTA data alone is not sufficient to monitor the availability of plasma products and their recombinant analogues. As we are currently seeing with regard to recombinant Factor VIII product, there is a severe shortage. The shortage is so bad that in many, many cases, patients have been forced to switch back to plasma-derived Factor VII product. This is contrary to standard medical recommendations, the recommendations of our Medical and Scientific Advisory Council and contrary to the recommendations of this very committee, which endorsed recombinant product as the standard of care and endorsed the proposition that all barriers must be removed for access to recombinant product. The current monitoring mechanisms have ill equipped all of us in the availability, or lack thereof, concerning this product.

Before stating some of the concerns NHF has with regard to the current supply of these products and the current inability to monitor such products, let me give some praise where it is deserved. I would like to compliment the PPTA for compiling this data and would like to commend this committee, the FDA, and the PPTA for working together to make accrued data publicly available. Further, the PPTA deserves the credit for now providing this data on a bi-monthly basis.

The PPTA distribution data is a valuable source to see what the trends have been over the past year with regard to inventories and distribution of product in the United States. This data has also served to give patient organizations, such as NHF, a better picture of what the supply has been like in the United States over the past year. Without this data, patient organizations would be completely in the dark.

With that being said, it is quite apparent that the PPTA data is only one variable in a complex equation. The data itself is only a snapshot of what the supply was like four to six weeks ago in the United States. As we are currently seeing a very critical shortage of recombinant 8, the current data that is provided is not indicative of the current situation. Instead, it's a look back of what was happening four to six weeks ago.

I would propose several recommendations for this committee to consider with regard to monitoring data.

Number one, there should be a mandate that all manufacturers provide their data to an entity, whether that be the PPTA directly, Georgetown Economic Services or the FDA so that a true aggregate data can be compiled. The PPTA has four members in the United States with regard to hemophilia products. Genetics Institutes makes recombinant Factor VIII and Factor IX, yet their supply numbers are not part of the data.

If every manufacturer provided their data to a third party so that the aggregate numbers could be produced, this would give us a better perspective as to the availability in the United States.

Second, the manufacturers of product are all multinational companies. They sell product in many countries, thus, the United States distribution data is only one variable in a large equation. It is impossible to monitor supply without knowing how much product is truly available, how much product is exported overseas and how much is available worldwide.

The PPTA has pledged to provide aggregate data from their European Division and NHF greatly anticipates receiving this data on a monthly or bimonthly basis because it will provide a broader perspective as to the overall supply.

NHF would like to see the total aggregate data worldwide made available so that everyone will know just how much product there actually is and what percentage of it is in the United States. Worldwide aggregate information would be a benefit to monitor the overall availability, inventory, and distribution of blood products and provide data to make much more accurate comparisons and analysis. This worldwide data would also indicate if a shortage, such as we are now facing with regard to recombinant Factor VIII, is indeed a worldwide shortage or just one here in the United States.

If the worldwide numbers are somewhat stable, yet the United States is suffering from an inability to obtain recombinant Factor VIII, then there is a problem. The worldwide aggregate data would help in determining this as well.

My third suggestion is that aggregate distribution data is also needed for the distributors of product. Distributors are very important intermediaries in the supply chain, and they hold large inventories of product for their numerous customers. Just as regulators were created to mandate the compilation of aggregate inventory and distribution data for manufacturers, I would like to request that this committee endorse measures requiring the same information from distribution, distributors such as home care companies, pharmacies, wholesalers and hemophilia treatment centers. This would serve as another missing variable in the overall monitoring of product.

My last suggestion would be that this committee strongly looks at recommending recommendations mandating that manufacturers provide worldwide aggregate data on what is in the pipeline for future distribution. Each company must have a business plan with forecasts as to what will have been available in two months or in advance. It is not possible to compile an aggregate forecast from all of the manufacturers so that we can at least have an idea what is forthcoming in the near future.

I feel that it is most important of my recommendations because this is the one that can allow federal authorities and consumer organizations to participate in situations instead of waiting to react. Perhaps this method will not be able to forecast an emergency shutdown of a facility, but it will be able to anticipate market reaction to forecast slowdowns or shutdowns. The Baxter Thousand Oaks shutdown for maintenance would be indicated in this type of measure.

While this proposal is not able to indicate a suspension in product release, it would be able to provide revised numbers once the suspension has taken place. With regard to the Bayer situation, the last U.S. lot release was in mid-December, yet the announcement of a suspension did not occur until mid-March. I find it hard to believe that Bayer does not have a contingency plan on what may be released in future months. In an aggregate format, these projections would be very valuable to the user community so that patients may be able to advance on how to obtain product.

I wanted to add to that just briefly with some of my own personal comments. When there's a shortage, it has an impact on a patient's life. It is not simply just switching product. It is not soda in the box. You can't just switch product. It does not necessarily, although it may stop your bleeding episode, it may not react in your system the same way, and people should be aware of that.

The idea that here we are in 2001, having worked very hard as a community, as regulators, as government advisers, as manufacturers to achieve recombinant therapy and talking about putting children who have never been on anything but recombinant therapy back on plasma products, I find unacceptable, at the very least. If any of us around this room have, and we've worked long and hard to achieve the products that we have today, this is a professional and personal failure for all of us that this product is not out there and available, that we're actually going to have to go back and talk to families about using plasma-based products, rather than the recombinant products that we've been heralding to them for years.

People who had decided not to have children until the recombinant products were available and now we're speaking to them in terms of using plasma-based products, that we're speaking to patients about going backwards and not going forward. We're talking about delaying surgeries that mean the difference, these are not minor surgeries where you're replacing--cosmetic surgery--this is someone who's been waiting to get a new knee, who's been waiting to get a new hip, who's been waiting to get on with their lives. This is a moral issue, as well as a scientific one, as well as a supply one, and one of the things I wanted to remind the committee today was that's part of the equation. There is a human side to not having these products available, and I think we're missing it.

Nothing I've heard here this morning makes me believe that anyone understands that at the end of all of these supply problems there's a patient waiting to do something with their lives. It has become very common in the hemophilia community for a child to participate in Little League baseball. When I was growing up, that was an impossibility. Today, that's the miracle of the products that we have available. And now we're asking them to put their lives on hold, and personally I feel it's unfair. It's unwarranted. It's a failure that we all need to address.

So I would urge this committee to make whatever strong recommendations are necessary to allow more data to be collected, to allow more information so that we can resolve this supply issue in some kind of immediate terms, but also with some kind of ending terms so that we're not talking about this next year or the year after.

I've been taking factor products for many, many years since their existence, and I almost can't remember when there wasn't some kind of shortage or some kind of threat to supply. Well, we've had enough time now to address many issues along the safety lines. It is now time to think outside of the box and think more about how do we end the supply issues.

Thank you very much.

[Applause.]

DR. GOMPERTS: Thank you, Mr. Bias.

Moving on to Ms. Buelow.

MS. BUELOW: Good morning. My name is Nancye Buelow, and I'm the president of the Alpha-1 Association and a plasma-using patient. I am speaking on behalf of the Alpha-1 Foundation and the patient-based membership of the Alpha-1 Association.

First, I would like to thank the secretary and the committee for your years of effort to see that the blood and plasma supplies are safe and available for patient populations like the Alpha-1 community. We appreciate your commitment and dedication to the issues that are prevalent in every population and also to the issues that are unique in each plasma-using community. As you have been made aware over the least few years, there is only one manufacturer of a plasma-based therapy licensed in the United States to treat Alpha-1. This is unique only to the Alpha-1 community.

A sole manufacturer with a single product continues to make our community very vulnerable. We have experienced numerous shortages in the past due to a variety of reasons on many levels. As of a few weeks ago, the Alpha-1 patient community is facing a total lack of any product at all.

While we are being told that the manufacturer anticipates the distribution to resume in late May, thousands of people in precariously poor health are left without this life-sustaining product to see them through until then. Any lung infection at all can cause valuable loss of lung function. Too many times in the past we have watched our friends lose their battle, as a lung infection causes their death. We have young children losing their parents. We also have young, very sick adults unable to participate in parenting, much less the workplace. Sadly, the last issue of the Alpha-1 News had six pages of memorials.

This situation has reached critical mass. Supply does not meet demand. One manufacturer can no longer produce enough product for our community. With the awareness efforts of the Alpha-1 Association and the Alpha-1 Foundation, fortunately, more alphas are being identified.

We can help those newly identified alphas, begin to monitor their health more aggressively, immediately seeking medical attention with any infection or exacerbation, hoping to stem the tide before too much damage is done to their fragile lung tissue.

We can also help them to understand how important it is for them to practice the best possible health habits. However, they need and deserve a treatment to sustain their lives. Can you imagine being told that you have a life-threatening genetic disorder that has a treatment, but is unavailable for you? That's what happened to me when I was diagnosed, a young mother, with children still in my home. I was diagnosed after being treated for an unremitting case of pneumonia. I was given course after course of treatment and still could not recover.

Once I was given a diagnosis of Alpha-1, I had to wait nine more months for treatment. Sadly, and incredibly, almost nine years later this is still happening. Imagine what it is like to have to wait for someone to die so that you can get enough treatment to sustain your own life.

We understand that there are two more IV products that are in development. We ask the committee to request that the FDA facilitate a rapid approval of this much-needed supply of augmentation therapy for our patients.

We also ask that you support the development of aerosol A1PI therapy and work closely with the manufacturers to provide us with more products and more choices.

Finally, we ask the committee to recommend to the FDA to make A1PI aerosol a priority and support the clinical trials.

While we totally support and understand the diligence of the FDA to assure the public safe products from compliant manufacturing facilities, please consider the impact of increasing complex rules requiring plants to close for substantial periods of time on product availability. We do not wish to have safety compromised. We advocate for ongoing dialogue to maximize the process and minimize the impact on the patient communities.

We, the Alpha-1 Alliance, cannot solve these problems alone. We need the help of the secretary of Health and Human Services and the FDA. We are counting on you. Our lives depend on it. Thank you for your attention.

[Applause.]

DR. GOMPERTS: Thank you, Ms. Buelow.

Moving on to Ms. Hamilton.

MR. WALSH: Excuse me, Mr. Chairman. I'd just like to make one brief comment. I'd like to complement Nancye on her remarks today in representation of the situation of the Alpha-1 community, but I'd also like to thank her for putting herself at risk by getting in an airplane with the poor air quality after not being able to have any access to therapeutics over the past three weeks. I think that's a personal sacrifice that this committee recognizes and appreciates.

I'd also like to commend the National Alpha-1 Association for their direct communication with the patients during this crises in the form of publications in the newsletter, as well as direct mailing to patients representing the "Dear Colleague" letter drafted by the Alpha-1 Foundation and the guidelines for treatment alternatives during shortages, although it doesn't help much when you don't have any product at all, by the Medical and Scientific Advisory Committee of the Foundation.

So thank you very much, Nancye.

DR. GOMPERTS: Thank you.

Ms. Hamilton?

MS. HAMILTON: Good morning. Is it still morning? Yes, it is. Okay. Thank you for allowing us to be here today. My name is Jan Hamilton. I'm executive director of Hemophilia Federation of America, which is an advocacy organization for persons with coagulation disorders.

We've been asked to address today how shortages and potential shortages of plasma derivatives best be identified and monitored, and also how can shortages and potential shortages of blood products best be identified and monitored.

So let's start with what about plasma. Currently, the plasma industry has a mechanism in place for monitoring the supply. Data has been made available to us on a monthly basis from PPTA, and as you've heard already today with current shortage data, it is now made available every two weeks, but is this enough? You've heard repeatedly today that it is not.

The current shortage has been the worst in recent memory, and we really want to know what could have prevented it. It was very unfortunate to have both Baxter and Bayer down at the same time,a need we understand that there is very little window of flexibility for manufacturers to alter that downtime for maintenance, but there comes a question, when inventory is in decline like we have been, is not there some way, and someone else alluded to this earlier today, that companies could make the FDA aware of things that are coming up, and like they would know when the scheduled maintenances are going to be, and possible when some event like the event with the Bayer shutdown recently that if there could have been some way to alleviate that by possibly delaying Baxter's shutdown.

But we also need to know what's in the distribution pipeline. That's been mentioned as well. And what about the supply worldwide versus U.S.? There have been a lot of questions in the minds of the consumers as to whether or not we are receiving our rightful share of the worldwide supply, and I've always advocated that fear of the unknown is the worst fear that there is.

This hiccup in this last report that we had with combining the plasma-derived product with the recombinant product really did us no good at all. It didn't tell us anything except that there's this much factor out there. As Val mentioned, when a person is on recombinant and is forced to go on a plasma-derived product, it may be the only time they've ever been off of recombinant, it may not be, but just like bodies react differently to different brands of product, they also obviously react differently to different purities of product. And it really is of a grave concern to us when that sort of thing has to happen.

We don't seem to be going forward. We seem to be going backward. And surely, at this day and age, we've come far enough to be able to figure out some things that would prevent this from happening. I realize that they are unpredictable things, but if we have some forward trends, as others have mentioned today, perhaps we could begin to anticipate these type of things.

So, in a shortage situation, another question that we have is who makes that decision on what patient gets what product? We've been hearing cases where it's not the physician, where it may be the actual reimbursement mechanism that's making this decision, like Medicaid or whatever. Who are making these decisions? And it doesn't seem to always be at the same level, and it doesn't always seem to involve the physician and the patient which, in our minds, are really the key, the critical decision makers in the treatment.

And at what stage in shortage should that decision be made to have a patient switch product? Again, if we had some sort of projection so that we could know what's going to happen in the short-term and in the long-term regarding product, perhaps there would be a better way to make these decisions.

What about trends? Are we looking at trends in treatment modalities, such as prophylaxis and immune tolerance therapy, to help us make the decisions of what the demands are for these products, and is that information being made available to the manufacturers so that they can project increases to meet those demands so that we don't go into this kind of situation.

One thing that has become very close to me because I've had some people call with just tears in their eyes saying they're afraid. They're afraid if they have to go on monoclonal product in the marketplace that we have today, with reimbursement as much of an issue as it is today, what's going to happen when recombinant becomes available again? Is their insurer going to allow them to return to the more expensive product?

This is a grave concern to us, and I think one that needs to be addressed to the insurance industry. And I don't know who would be the right person to do that. Perhaps it's this committee, perhaps it's a conglomerate of people, but it's definitely something that needs to be explained because we do have a fear that when that happens, we're going to have people that are prevented from going back on the therapy they should have been on all along.

And so you might say how does plasma inventory system compare to the possible system for the blood supply? We've talked today that we heard from the National Data Resource Center that--like some wonderful things, we heard from Mr. Schmidt at FFF of some things that they have in the pipeline. What are some of the red flags that we need to look for and what would sound an alert? We've talked about a lot of these things today, deferral of donors, military exercises, apathy on the part of donor pools, seasonal illnesses and holidays. These are all things that the blood centers address on an everyday, ongoing basis, but some things that we really do need to be aware of.

And then you might say, well, why should Hemophilia Federation of America be concerned about the blood supply versus the plasma supply? Well, guess what, we're real people, too, and we bleed from things that require whole blood or blood components other than the plasma derivatives that we take on an ongoing basis. Therefore, we need to be aware of both the blood and the plasma concerns.

The blood banking industry has made major efforts with publicity and drives, and we've heard today about a lot of the other things that they have in store to plan for better management of the blood after it enters the system and how that could be accomplished. I don't need to tell you, I mean, everybody knows there's 800 numbers and fax machines that can facilitate something and possibly maybe even adopt some variation of the current FDA CBER report or whatever. There's lots of different ideas, and I'm certainly not the one to suggest those things to you.

But another thing that I think we might consider is ongoing education of hospital staff regarding allocation and usage. We have a little bit of referral to that earlier today. Consider ongoing education of physicians regarding the difference, for instance, in type and cross-match or type and screen or type and whole, errors in administration, all kinds of things that are found in those minutes of those Blood Utilization and Review Committees that could be looked at and avoid possible waste.

And then to share the data that is--did I miss one? Sorry about that. And then to remove the near misses from the cloak of secrecy. Hospitals don't want to tell you when they have an error or an accident that could result in a sentinel event. They certainly don't want the lawyers breathing down their necks, but if we can stop the error or accident at the near-miss stage and avoid the sentinel event, thereby removing the fears associated with sentinel events, perhaps it would be progress. It's a little bit like treating at the symptom stage, as opposed to waiting for the autopsy.

How do we know how many near misses there are? The only ones that really know are the people at the hospitals, and the only way we'll really know is if they do report them. So perhaps mandatory reporting of these near misses should be made mandatory. Where that should be reported, whether it's to the FDA, whether it's to this committee or whoever it's to and what to do with that data accumulated is something that would need to be looked at.

But certainly no information gathered is worth the paper it's printed on unless it's shared with those people who can do something about it: the participating hospitals, the institutions, the blood banks, the physicians, this committee itself. And, therefore, if we do share this information, then we can avoid some of these pitfalls in the future, reduce the errors and accidents, increase the usability of existing blood supply, severely reduce or eliminate the sentinel events, thereby helping to avoid shortages.

And I'm saying right now about the blood, but this really does pertain also to our own plasma-derived or recombinant products because those are some of the things that we need to take into consideration in the whole picture, looking at the whole picture, not just one little segment of it. And I really think that these usage trends are something that need to be looked at. Are the manufacturers looking at these trends in taking into consideration planned expansion? When are they planning to make plan expansions? Only when we get into a shortage like this? Then it's going to take one, three, five years to get up to speed again.

So what we're asking is for cooperation from all of those involved, and we need the enthusiastic cooperation from all of those involved in order to keep these shortages from continually appearing and impacting on the lives of the patients. As Val said to you a few minutes ago, the patient is what we need to remember. That's the bottom line. Their lives are in jeopardy. And to hear somebody like Nancye stand up here and talk about people having to wait nine months for treatment or to be without treatment for six months or to hear a person with hemophilia having to go for three to five months with minimal treatment is just not something that we should accept in 2001.

Thank you for your time.

[Applause.]

DR. GOMPERTS: Could the panel members please approach the microphone for questions and answers from the committee. Thanks.

Are there any questions or comments?

DR. KUHN: I guess I have one question to address to Val Bias and one to address to Nancye Buelow. And the one that I'd like to address to you, Val, is I know you were saying basically what we have done as a committee and as industry is we've taken like three steps forward, but we have taken two steps backward now as its recombinant and having to then pose the problem of people going back on a plasma-derivative product.

The question I have for you is when people do have to go back, because it's inevitable they're going to have to go back on this product, even though it is not what we would like to see as the best standard of care, is there going to be a shortage in that plasma-derivative product? I guess, to me, it seems like there's going to be a great pull upon that product when there is no more recombinant product and how long would that last?

MR. BIAS: Well, two things. I would expect that eventually, of course, plasma supplies are in great supply at the moment. As a matter of fact, a number of wholesalers approached individuals that we monitor immediately when there was a recombinant shortage, almost before it was announced. "Well, there's plenty of plasma here." Sometimes you wonder how those things happen.

So I would imagine, at some point, there will be a reduction, but what I'm more concerned about is, after the shortage is done, and I haven't experienced one of these shortages, where I haven't experienced even if it's moderate or minimal, a price increase once we get back online.

What I'm worried about is the patient who switches to the plasma-based product which is cheaper, and then goes back to their HMO when recombinant is in good supply, and their HMO says, "Forget it. Isn't that working for you? Are you bleeding?" And we've lost what we started with, the whole reason for moving these patients to these products, we may have lost the game at the insurance level.

And NHF polled some insurers, and they said, no, that shouldn't be a problem, but they didn't factor in a potential price increase, and I've never had one of these shortage things happen where we didn't get it on the back end somehow.

And then, secondly, it's not the big insurers I'm worried about, it's the HMOs who do some very individual cutting.

DR. KUHN: And then my second question to Nancye is, you are talking about the Alpha-1 shortage right now, and I'm sure--I guess the question I have is the manufacturer who produces the product you have is the product line or the manufacturing line the same manufacturing line that produces the plasma derivatives, as well as the recombinant products or are they different lines?

MS. BUELOW: I'm not sure about that. John probably has that information.

MR. WALSH: Well, they have two facilities. One produces some recombinant, but the line that we're involved with in North Carolina does not produce any recombinant product that I'm aware of out of North Carolina. Obviously, there's no recombinant product available for Alpha-1, and we would not anticipate there'd be any available from three to ten years.

DR. GOMPERTS: Steve, you had a comment?

DR. NIGHTINGALE: Yes, I have a question for all four panelists. I heard each of you say a great deal, and at the same time I heard each of you say the same thing. Is there any, within the four of you, I'm by no means trying to split you up, I'm trying to identify if there is unanimity, are there any meaningful differences between the concerns that each of you expressed? Is there anyone in the room who self-identifies as a representative of the consumer community who has a perspective other than the one that was expressed by the four discussants?

[No response.]

DR. NIGHTINGALE: That is a substantial help to the executive secretary.

DR. GOMPERTS: Thank you, Steve.

Other comments, questions?

[No response.]

DR. GOMPERTS: That was excellent. We do have a comment. Okay. Beg your pardon.

Thank you very much for that perspective, very important. Thank you.

We are moving on to the next group, and we are going to change in order here. Ms. Jacqueline Fredrick, American Red Cross, does have some pressure from the point of view of air travel. So Europe first.

MS. FREDRICK: Thank you. As someone who commutes between two cities, I appreciate that.

Mr. Chairman and members of the Advisory Committee, the American Red Cross is pleased to join in today's deliberations concerning the important public health issue of blood availability. Blood is a precious and national resource made available only through volunteers who generously give part of themselves to help patients. The American Red Cross is uniquely positioned to address the human element of blood availability as we interact with the people at either end of the process, our volunteer donors and patients in need of transfusions.

The safety of the blood supply is our number one priority at the Red Cross. And through our national system, we continue to strengthen our infrastructure to ensure safety. A similar investment is now needed to ensure availability. We face an increased demand for blood, coupled with challenging new threats to the safety of the blood supply, such as variant CJD. It will take a major investment of time, money, expertise, and resources to make the American public aware of the need to recruit the next generation of blood donors.

The Advisory Committee has raised the important issue of where responsibility resides for ensuring an adequate blood supply and the role of the federal government. The government's role is vital in the area of blood safety. The FDA serves as the independent representative of the American public to ensure blood safety by means of blood centers' compliance with statutes and regulations. Through FDA, the federal government has a place in extensive regulatory and auditing structure that serves the nation well, as it tempers the impact of regulatory actions on availability.

The federal government has responsibilities for blood safety in two other very important areas. First, research funding to study blood-borne pathogens and other safety issues must be made available to NIH and CDC.

Second, Health Care Financing Administration, as the largest third-party payer of patient needs for blood, must make certain that Medicare patients have access to state-of-the-art blood products by appropriately reimbursing hospitals.

These important responsibilities, regulatory oversight, federal funding of research, and adequate reimbursement, rest with the federal government. We do not believe that it is the role of the federal government to routinely monitor the availability of the transfusible blood product supply, but rather that that role rests with the private sector.

Responsibility and accountability for providing an adequate and safe blood supply rests with this blood banking community. Blood availability starts with understanding and meeting the needs of the hospitals and the patients we serve. As a result, it is the obligation of the Red Cross and our blood banking colleagues to forecast, plan for, monitor and act to meet the demand for these life-saving products.

Information on availability is needed on a daily, monthly, and long-term basis if the blood banking community is to successfully project and meet patient needs. These information management and projection tools are the underpinnings of an available blood supply.

Historically, availability has been based on a supply-driven model, which emphasized an individual blood center's ability to bring in blood donors and collect blood. The result is, and was, cyclical and seasonal patterns of shortages throughout this country which have become worse as the population ages, and the need for blood continues to grow. Local demand is setting national patient needs, as opposed to patients needs across the nation setting our local blood supplies.

By changing to a need-driven model, one based upon projected patient needs, the Red Cross can expand and stabilize collections to ensure an adequate supply of blood products whenever and wherever they are needed now and into the future. The Red Cross is now instituting a nationwide system that enables us to have real-time view of blood collection and inventory data for half the nation's blood supply by blood type, by product, by location, and by customer every day.

We are coupling this collection and inventory system with information on individual hospital needs. Combining these data will enable the Red Cross to plan for and pinpoint where specific blood products are needed and where potential shortages may occur. We will then take immediate action to ensure that products are available wherever needed.

This new system will also include data on blood usage and nationwide access to collection scheduling information. Red Cross collects at 500 different sites every single day. A critical component of this model is the collection of individual hospital blood needs and fulfillment of those needs. Today, we monitor the fulfillment of blood requests from every hospital, all 3,000 of our hospitals, on a daily basis, and I get that information on a monthly basis.

The innovative model will enable the Red Cross to anticipate and prevent potential blood shortages. We will be able to utilize our resources to meet the needs of all patients, reduce unneeded inventory and minimize waste of this precious resource, thereby fostering greater stewardship of the nation's blood supply. We don't only do this on a daily, and weekly, and monthly basis, but essentially every day I can look at a 3-week, 3-month and 18-month plan on what the blood supply is going to be, what I'm collecting by blood type and product.

The American public recognizes that blood is a national treasure that should be available to all people at all times so that patient needs will be met. When asked to give, the American public has never failed to be generous. A national public opinion poll conducted last month on behalf of the Red Cross found that the majority of blood donors understand that their blood donation is used to help people most in need, no matter where they live in the United States.

Among blood donors who were not previously aware that blood donations may be used to help people outside their local area, almost all respondents reported that the location of the patient receiving their donation had no impact on their willingness to donate blood.

Clearly, the American public supports the use of blood for patients in need. People who voluntarily donate blood are among the most generous philanthropists who give a piece of themselves to save another. At present, the Red Cross is blessed with seven million blood donors, people who have donated at least once in the last four years.

In the coming months, Red Cross will reach out to all of those, sending letters to current and lapsed donors. The effort is being undertaken by Red Cross to assist in the establishment of the next generation of blood donors who will help ensure the adequacy of the blood supply. This effort will be backed by multiple and sustained communication with our donors. The goal is that blood will be available every day, no matter what day of the year it is.

Red Cross will make the commitment to invest resources in availability, as they have had in safety. The tools and the capability are out there. The money is available. It's just a matter of the blood community delivering on its promise and executing its plans.

We appreciate the opportunity to share this vision for the blood supply in availability, and I would be happy to answer any questions.

DR. GOMPERTS: Thank you.

[Applause.]

DR. GOMPERTS: Any comments or questions?

Karen?

MS. LIPTON: Jackie, we heard a lot this morning on the plasma-using community about, first of all, public disclosure of this information, how important it was, and then we also heard Jay talk a little bit about the significance of this type of information for making public policy decisions. Would that be part of your intent to use the information this way?

MS. FREDRICK: Absolutely. And we believe availability is a public policy issue and, hence, developing the systems.

But, yes, I think it's our view that if you are looking at public policy, let's say a new deferral criteria, that the best place to collect that data and understand its impact are those of us who interface with the donor and have access to that because we're the ones that have to plan for it. Retrospective data doesn't do any of us any good, in terms of action. So, yes, absolutely. And we have done that in the past. When we went from ear sampling to finger sampling, I knew exactly, on April 14th, that the blood supply would drop 6 percent if we didn't take action. So, yes.

DR. GOMPERTS: Jay?

DR. EPSTEIN: Jackie, could you make a little bit clearer, at least for me, how the redesigned system of needs to driven collection differs from the system now in place?

MS. FREDRICK: Absolutely.

DR. EPSTEIN: What will really be done differently at the operational level?

MS. FREDRICK: Well, Jay, right now, generally, in this country in almost every blood center, blood collections and our ability to collect blood from donors, our perceived inability to collect blood from donors, is what sets how much blood we're going to collect every year. So it's a collection-driven model, as opposed to understanding what the demand is out there. Right now I have information that says, potentially, we are under-resourcing red cells about 6 percent. That's just our 3,000 hospitals.

So the idea here will be to evaluate demand by each hospital, build the model up based on need, and then collections will have to meet what the need is. Right now I would dare say most blood centers start by how much blood we can collect or think we can collect. So it's actually a reversal of how we do things.

DR. GOMPERTS: John?

DR. PENNER: It's still a little bit easier to do the ear stick than the finger stick and a lot less painful.

MS. FREDRICK: I know. I'm a blood donor.

DR. PENNER: Going back, Jackie, the Red Cross is handling about 50 percent of the blood.

MS. FREDRICK: Uh-huh.

DR. PENNER: What is the mechanism then that one could address the entire population of the United States with respect to inventory, and outdating, and things of this sort, a way of triaging blood that is in various areas of the country if you're just looking at part of it? How are you going to get together with the rest, the other 50 percent, so that one could say that AABB has some blood here that's going to outdate in about a week, and you've got a Red Cross center just a couple of blocks away that needs this? How are you going to operate there?

MS. FREDRICK: We've actually given some thought to that because now we have the ability to have transparency across our system. One would be our feeling that every blood center and blood organization needs to build this capacity. I mean, these are tools that are used routinely in every industry that I know of, for-profit industry. So I think the idea would be, if we had the structure in place, then one could move the blood just by working together to do so.

But there has to be a concept that we're going to collect for patient need, not based on my community needs 10,000 units so that's all I'm going to collect from my community, right? It's the responsibility that we have to collect for patients nationwide.

DR. PENNER: So you would certainly consider then an interaction--

MS. FREDRICK: Absolutely.

DR. PENNER: --of the entire group of the blood industry programs.

MS. FREDRICK: Absolutely. Absolutely.

DR. PENNER: And that should not be under governmental approach, though.

MS. FREDRICK: No, I do not believe so, because--

DR. PENNER: Have you done independently with all of the group here--

MS. FREDRICK: Yes.

DR. PENNER: --getting together and deciding where blood might be transferred from one area to another without any concerns about additional pricing and other concepts?

MS. FREDRICK: Yes. I mean, pricing really isn't an issue. When a patient is need, you have to have the blood there. But, yes, I think we would all work together. I don't think it is effective for government to try to move blood around. That's not the role of government, in our opinion.

DR. GOMPERTS: Thank you.

Ron?

DR. GILCHER: Jackie, I have two comments. I am sure that the results of polls are dependent on how the questions are asked. And, in fact, in our area, which is, of course, a much smaller area, it's very clear to our donors, since we are an exporting center, that we take care of home first. That's clearly understood. And, in fact, that has been reflected back to us in surveys that we've done within our own area, and that's sort of contrary to the statement that you've made with your poll.

The second point that I want to make is that the supply of blood is really dependent upon the number of donors that, in fact, show up and then how many donors we defer. And so what I'm saying is that a deferral policy clearly has to be I think very clear and that it also has to be based upon what I'm going to call "correct," and put quotes around the word "correct," medical and scientific data, when, in fact, we have two organizations that have a difference of opinion, we send two different messages to the public.

We're in that situation right now, and I think this is a potentially very serious problem that we have to address because we can end up with clear-cut shortages, and I agree with you because of the VCJD issue, and we have to I think address this and work this out in a manner that's equitable.

MS. FREDRICK: I absolutely agree with you that each blood organization ought to be accountable for meeting patient needs and needs to take into effect all of the parameters that affect blood donation, be it variant CJD deferral, our processes, our location, et cetera. So I think to just focus on variant CJD, obviously that's the issue at hand, but there are going to be more emerging threats to the blood supply. And if we keep focusing on these one-time events, so to speak, we miss the opportunity to build the robust supply for the future.

In the area of deferrals, I think we all have our policies. This one happens to be one that is more evident, but it's our opinion that there's tremendous scientific uncertainty and there are not concrete medical and scientific information. If there were, we'd all have an easier decision. And so prudent, cautious, informed judgment is at hand here, and I don't think it will confuse donors. I think we confuse donors.

DR. GOMPERTS: Thank you.

A question?

COL. FITZPATRICK: Jackie, I want to go back to Karen's question for a minute. I think part of that question was the desire of the panel and the committee to have that public information. So is Red Cross willing to make your projections and the data you have available public?

MS. FREDRICK: I guess all data, I would ask Mike to what end do you need the data, and what will be done with it? I mean, we can all collect data, and gosh knows we collect a lot of data in our business, but to what end?

COL. FITZPATRICK: Well, we've had discussion that we have the National Blood Data Resource Center collecting data, but we don't have a tool to make a prediction based on that data. And what I heard you say is that you have predictions for 3, 6, and 18 months of needs within 50 percent of the country, which is a tool that we have just said we lack.

MS. FREDRICK: Right.

COL. FITZPATRICK: So the need would be to share that tool so that it could be used to predict the needs of the nation and not just 50 percent of the nation.

MS. FREDRICK: Sure, we could share that tool, yes. It's a computer-driven tool, and an aggregate of data, but, yes, we can clearly share the model, and how the model works, and how the data is aggregated, and how we make decisions. But the whole point around is this is not to collect data. The point in our model is to know what the information is so you can take action so the outcome is every patient every time the right unit of blood. That's the end point of the system.

But, yes, we can share the model and the computer process.

COL. FITZPATRICK: To meet that need, the data needs to be available to meet the need.

MS. FREDRICK: And we have that data, and our commitment is to meet the need, just as I believe it is every blood organization's responsibility to have that data prospectively and not to have retrospective data. It does me no good to look back at data collected in '99. I knew in '99 what I collected, right? Unless we're going to use that to somehow change practices and behaviors going forward, it's just data.

COL. FITZPATRICK: Right. But what I heard you say is you're willing to share the model with them.

MS. FREDRICK: Yes.

COL. FITZPATRICK: And one other question, not to beat a dead horse, but when you mentioned the role of the federal government in your statement, you attributed to the FDA the role of statutes, and regulations and an auditing structure that has served the nation well, and to represent the American public in ensuring blood safety.

MS. FREDRICK: Correct.

COL. FITZPATRICK: Yet we are faced with a dilemma being created by the Red Cross concerning safety. What will it take to come to some kind of closure between the Red Cross and the FDA so that this statement would, in fact, be true; that you're acknowledging their role?

MS. FREDRICK: We absolutely acknowledge the FDA's role in setting the safety standards in this country and have always done our utmost best to ensure we are in compliance with those standards, and we believe they do represent the American public in setting safety goals.

Having said that, all of our organizations use those as our starting point and go above and beyond in the systems we built. For example, the American Red Cross has over 20 surveillance systems that monitor every process, every product as we go along. Now, nothing in the regulation says to me I should be monitoring every filter I use out there in every blood region, but we do because we think it is a better, more robust system.

So we absolutely agree that the FDA sets the safety standard. The Red Cross is making absolutely not one claim about safety, as it relates to any expanded deferral criteria, but we think, in our judgment, given the information we all have, that it is prudent and cautious to be more conservative today.

DR. GOMPERTS: Thank you.

DR. NIGHTINGALE: Before Dr. Busch, if I might interject, Jackie, I'd like to suggest a couple of clarifications and generalizations you have that might clarify the issue the colonel addressed.

First of all, when you state that it is not the role of the federal government to monitor the availability of the blood supply, I might suggest, as an alternative, that it is the role of the federal government, and I can't remember if it's under Chapter 41 or 42 of the U.S. Code, but it is the Public Health Service Act, to preserve the public health of the United States. And that is very different from the part of the code that deals with the FDA. And I think if we were to have the discussion about the monitoring, it would probably come under 42, though it need not.

And the other clarification that I would make I think is, supportive of your statement, is that much of the information that you or, say, the New York Blood Center collects is your own business until decided otherwise by a compelling national interest. And I think it is worthwhile stating that it is the view of the government that it is your own interest, absent a compelling reason to the contrary.

What I think Mike was saying is that there is concern that compelling interests may arise. So, to get to my clarifying question that might help you resolve the issue right here is, has the Red Cross considered what, if any, of the data that it will be collecting under its new plan would be made available on a confidential basis to an appropriate government agency?

MS. FREDRICK: Well, I don't know because I don't know what the request is and for what it would be used for, but clearly we have been a leader in surveillance research around transfusion medicine. So, of course, to the good of the American public, we would. But, again, I think we want--I think everybody wants to understand, not just the Red Cross, to what purpose would we collect the data.

It is not a surprise to any of us in this room that we have had blood shortages for as long as we've collected blood. So collecting more data, I don't know of what benefit that is. I would hope that this group would say how is it that we're going to grow the next generation of blood and ensure the blood supply in this country. And I don't have any doubt that government, certainly, in a public health policy, can oversee the availability of the blood supply.

I just don't think it's the role of the government to collect that data on a daily basis and a monthly basis because it's really the people who must act that need that data.

DR. NIGHTINGALE: But your statement didn't, in any way that I heard anyway, deny the need of the government to have access to that data under certain circumstances, and that that need might be a perception of the public that a blood shortage might be imminent.

MS. FREDRICK: Sure. I mean, we've always shared our information and published it.

DR. NIGHTINGALE: And I know that, and that's why I'm taking valuable time to get this on the public record because it was my impression that your written statement was perhaps overinterpretted.

MS. FREDRICK: That could be that people overinterpret it. I don't know.

DR. NIGHTINGALE: Yes. And I might finally state for the record a reminder that there is information that is collected by the private plasma sector that is shared selectively with the government on a need-to-know basis.

MS. FREDRICK: Sure.

DR. NIGHTINGALE: For example, Dr. Weinstein and my colleagues in the FDA get information that I do not because I don't need it.

MS. FREDRICK: Right. And I understand that. But, again, I would challenge this group to say we can't just collect the data. We need to do something about the data we see. And so I would encourage this committee, if you feel there is a public health issue collecting the data, then you must accept also the responsibility that we have to do something about the data we collect. And that's I think my point is not just to have data collected.

DR. NIGHTINGALE: You are saying what I hoped you would say.

DR. GOMPERTS: Mike?

DR. BUSCH: Jackie, a very impressive and ambitious statement. And actually to correct Steve, this very sentence he quoted, you didn't state it quite that way. You stated that the role of the federal government to routinely monitor the availability.

MS. FREDRICK: Right.

DR. BUSCH: You added the word "routinely," which is an important word because I think that we all support that the process of monitoring historical usage patterns has been very important, and we all think that's appropriate and probably done best on a national level.

I actually agree with you that the strategy to best monitor usage on an ongoing basis is the blood center interfacing with the hospital. One of the three or four new things that you referred to in this document was the development of a new system to interface between the hospital and the regional collection facility, and in your program at a national level, through that process, to be able to actually obtain from the hospitals anticipated utilization data.

Historically, there's been a fire wall between hospitals and blood centers, for legal and other reasons, that I thin is historical in terms of a lot of exchange of information about which units which go into which patients, et cetera, and I think a lot of that should be revisited. And I think developing mechanisms to, you know, using computer mechanisms to exchange data much more effectively between hospitals and blood centers is a very good approach, and I very strongly support that. But it's an approach that's going to take work, and time, and experimental design of methods to develop an optimal strategy and breakdown of historical barriers in terms of the willingness of hospitals to share detailed information.

So I think it's a great idea, but I think it's ambitious to expect that it will happen overnight. And I think that anything that the Red Cross does to accomplish that goal should be something that becomes generalized industrywide. I appreciated your comment that you'd be willing to share the tools, as you develop them. But I think likewise there should be an aggressive effort of non-Red Cross blood centers to develop new strategies to get that kind of information, and that should be a national endeavor to develop a better mechanism to communicate that kind of stuff.

The second thing you talked about, which I think was also great, is a new strategy to try to get new donors in, letters to donors, and you are anticipating that worked. We just did a letter campaign, in collaboration with Reds, monitoring the effectiveness of mailing letters to large numbers of donors versus e-mail and quantify the relative effectiveness, et cetera. And, clearly, letters and e-mails were more effective in doing nothing, but the net effect was increasing donations by 1 percent.

MS. FREDRICK: When we did it, it was 25 percent.

DR. BUSCH: Twenty-five percent of your donors you sent letters to came back several months later?

MS. FREDRICK: Last January--and it's paired with some telemarketing stuff. You never just drop a letter. Last January--I'm looking for some of my folks--I think we sent out mailings to something like 260,000 Group O donors and I believe 60,000 came in to donate, and we're going to do that again, as I mentioned.

DR. BUSCH: Again, a very good thing and excellent to monitor these programs, but I think again it should be a national effort to try to identify strategies. The communications should be national programs, and the concern is that it not become a Red Cross-isolated activity. But, in general, I think a very good proposal and one that we all endorse.

The third thing, again, that I think is very important is the concept of nationalizing the donor base, of moving away from the regional blood center and the idea that you're promoting of blood collected anywhere can be distributed anywhere, and the donors support that. They don't have any problems with that.

Again, I agree with that principle, and I think we should be moving that direction. So, all in all, I think lots of wonderful components to this, but again very ambitious. And the expectation that Red Cross is going to succeed at this ambitious plan over the next months to offset this impending loss of donors I think is too ambitious. And I support the whole program, but I think that the idea that Red Cross can take this on by themselves and accomplish this by themselves creates a bad image in the system, that there's not a partnership, that there's not a collaboration.

And I think the sharing of the procedures, the sharing of the data, the sharing of the strategies, the recruitment strategies, is the best way for a national blood program.

MS. FREDRICK: Okay. Can I respond to that? Let me be clear on a couple of things. I am not suggesting we nationalize the donor base. I'm not sure what you meant by "nationalize the donor base." But I do believe that we have to, all of us, all of blood providers, have to take the responsibility that our donors have to donate for patients everywhere and that it's just not a matter of helping someone in St. Cloud, Minnesota, if their sister is getting a bone marrow transplant in St. Paul.

So by that I meant that we have to I think start looking at meeting the needs of patients everywhere. You and I agree with the nationalization.

I also want to be clear on what we can, today, do with our hospitals. I can monitor blood usage by hospital, by blood type, by product, what they get, what they return, et cetera. We are not yet crossing that barrier, which I think you were alluding to, is once that unit gets inside the hospital, is it going to Cardiac or is it going to Onc or where?

But, Mike, we're doing most of this today. This isn't a new program at all. And I will tell you, last year, we were faced with the U.K. deferral, which was about 2.2 percent. We actually only realized .13. We were faced with finger sampling, which was a true and real 6-percent loss. So we recovered 8 percent of the blood supply and still grew our collections.

So I think we have shown we can do it--9 percent, last year, recovery. And I think we have to. I think to set our goals low and to think that all we can grow every year is 3 percent or 2 percent is absolutely wrong, and we'll never get there.

DR. GOMPERTS: Thank you. One last question.

Dr. Lopes?

DR. LOPES: Given that your donors are understanding that their blood itself may go to people elsewhere, don't you suppose that they are already assuming that data, at least, would go to the Public Health Service freely and without any barriers?

MS. FREDRICK: I know of no information that our donors have any opinion about data and information because we certainly never asked them.

DR. LOPES: But you haven't asked them.

MS. FREDRICK: No.

DR. LOPES: I mean, I should think that donors would simply assume that information about a critical resource like this would be available to the Public Health Service.

MS. FREDRICK: It could be. I don't think we'd not do this because I don't think donors have an issue with this. I mean, I don't think donors would be preventing this. Now, clearly, you have confidentiality issues.

DR. LOPES: Oh, no, I think donors would be thinking that this data should be going to the Public Health Service and probably are assuming that it is already.

MS. FREDRICK: I have no information. We just have never asked our donors about data.

DR. GOMPERTS: Thank you.

One comment?

MS. SULLIVAN: Jackie, one thing that I have always wondered about, at least for the last three and a half years, is, and since you brought it up today, in your daily monitoring of 3,000 hospitals, what percent of those hospitals or what percentage of their total need is, for which percent is the Red Cross the sole supplier?

MS. FREDRICK: Oh, we have that data, but I have no--it's well over, I think, 90 percent/92 percent, something like that. I mean, I just don't know the exact number, but someone in the Red Cross does.

DR. GOMPERTS: We do need to move on; is that okay?

Thanks.

MS. FREDRICK: Thank you.

DR. GOMPERTS: Thank you very much. It's important.

Dr. Hillyer?

DR. HILLYER: Thank you, Mr. Chairman and members of the committee. Good afternoon. My name is Chris Hillyer, and I speak to you today in my capacity as a member of the Board of Directors at American Association of Blood Banks, but I'd also like to wear my patient advocacy hat, as the AABB does, and tell you I'm a professor in the Department of Pathology at Emory University and manage the transfusion medicine program for five hospitals there with over 100-, almost 150,000 blood components given per year.

Our hospitals across the board feel that there's an apparent lack of blood, but I have to use the word "apparent" because I believe that there's not an adequate amount of data. I believe collection data is very helpful, and I might even disagree or appear to disagree with some of you. I believe retrospective data are helpful, not as good as sentinel alerts, not as good as micro or macroeconomic-type data and utilization data. But as one authority said to me recently, collection data, without utilization data, is essentially worthless.

I'd like to take the utilization idea one step further. I'd like to take that to the shortage area and to the patient impact area, and I don't believe we have that data. So, when I say an apparent lack of data, I believe the data are limited. If you go to the literature, in the last 10 years, you'll find two peer-reviewed publications right from here at a time when he was in Boston. There is more data, of course, from the National Blood Data Resource Center. And the IOM, the Institute of Medicine, had in 1995 a council looking at blood availability, and there is commentary and some summary data available, but not in peer-reviewed form.

So, if one goes on a search for data since 1995, one comes to the NBDRC. And what you find is that their data are available to their members, but not necessarily widely distributed. So it may be a contradistinction to Jackie. I believe that we should be data driven, that we should count on our data being very usable, as real time as possible. And I believe if we are not real time yet, that we should get and can get to a more real-time and usable situation.

The plasma industry showed us this morning that things happen that retrospective data don't predict prospectively. That makes sense just from those words. But I don't think that that should stop us. And so I'm going to return, actually, to some of the written verbiage and try in the next two minutes to say that there's an urgent need for money, and that while this committee may not be in a position right at the moment to directly recommend or may be to recommend money, I believe that--and I'm speaking on behalf the AABB--the AABB believes there is an urgent need for data, utilization, use, not just utilization, not units transfused globally, use data regionally.

And let me ask two things, respectfully. First, that you make collection of usable, accessible and unfettered data a top priority for the committee, and, second, that you lend your strongest support and recommendation to Congress, the Bush administration, the secretary of Health and Human Services, to designate an appropriate office within the Public Health Service to be responsible for this and to fund this important issue of blood data collection for the future.

Across our country, it is expected that blood will be available. It is an expectation of our public, and it is an expectation that it will be safe. When blood is not available, I would suggest to you that these potential recipients are not safe. I make this broad statement wearing my transfusion medicine hat. Many times each year i must consider closing our emergency rooms, operating theaters, and trauma centers, and that's no joke. Sometimes I have to do it, and that is a tremendous fiscal and human cost. I can't quantitate that. There aren't data.

During the past 10 years, my perception is that blood shortages have dramatically increased in frequency, but I can't say this because no shortage data are available, and I don't believe they're available nationally, and they are certainly not available regionally.

If you go to the folks from the NBDRC, in their publication, dated March 2001, they'll show data from 1999 that 6.6 percent of hospitals reported that "elective surgery was postponed during the year due to a blood shortage." That's a fantastically high number, in my opinion, because I know of the pressure on hospitals not to say that they'll do that, and I would be surprised if that was underreporting. Indeed, in writing, and I quote again, it was written, "These data are difficult to interpret."

I believe, the AABB believes, that we need more data, and we believe that the NBDRC needs to be at a fiscally viable position to widely disseminate, not just disseminate, but widely disseminate and continue to collect its data.

This morning, presentations were made about collections, including the number of units. These data, again, need to be widely available, but also ongoing and long term. Donor deferrals have increased dramatically--ear lobe and finger stick, changes in malaria risk location, and now the idea of a potential change in the variant CJD deferral standard.

But the impact of these policy changes on blood donation and collections is not known. The impact on how much of that blood will be available to our patients and in what time frame is not known. I submit to you we need to know.

I also believe we need a substantial array of data regarding utilization. We, in transfusion medicine, do not really understand how utilization changes as the practice and science of medicine evolve. Is the majority of blood used in operative settings for obstetrics emergency, for trauma care? Are the special products needed for cancer support available in adequate supply in a timely fashion? Again, I submit to you we don't know, and I submit to you that we need to know.

To quote Dr. Wallace in 1995, the blood supply is adequate, but we do not know if the amount we collect drives the amount we transfuse or if utilization would actually be higher if more blood were available.

I further believe that uniform data are needed regarding hospital ordering practices and how these ordering practices are filled by blood centers. We need to anticipate trends, we need to use the data, as Jackie said, to do something, to anticipate and to understand how hospitals fill their orders, their relationship to the inventory, and how hospitals manipulate their orders, as they are forced to do from vendor to vendor, sometimes putting contractual and pricing arrangements in jeopardy.

All of this, as I have laid out, is a lot of data, and collecting it is both difficult and expensive, which brings me to my second request, a need to designate a responsible central body and the need for federal funding.

The committee heard the reports of Drs. Wallace and Greer, providing data by the NBDRC. The NBDRC is an independent affiliate of the AABB. And for more than 50 years, the AABB's highest priority has been to maintain and enhance the nation's blood supply.

Thus, in 1996, recognizing the significant need for data, the AABB conceived of and founded the NBDRC. The AABB is proud of the significant work the NBDRC has done, but the NBDRC is just that, independent. The AABB does not and cannot financially support the NBDRC in its entirety, and the NBDRC cannot financially support itself in its entirety, either through sales or through its membership. Both the NHLBI and the PHS have stepped forward to provide stop-gap measure funding, but that funding ends FY '01.

I believe the AABB, we both believe that this effort is imperative; that is, collection, utilization data, its analysis and its widespread distribution, to ensure more than a "adequate" blood supply for our citizens. I and the AABB are very concerned that as long as no specific agency or office within the Executive Branch is charged with the responsibility for supporting these critical data initiatives, that patients will suffer. And if the government decides not to support data collection right now, we risk losing the progress we and the NBDRC have made.

And perhaps most importantly, the data are needed to fully understand the relationship between collection and utilization to accurately anticipate when or even if supply will not meet demand. With the more recent increases in donor deferrals and impending policy on variant CJD, that time may, in fact, be now.

In summary, national and regional statistics regarding blood collection and utilization are direly needed. As the committee knows, policy makers can't make informed decisions without adequate data. Blood safety and availability are integral and inseparable requirements for ensuring optimal patient care. Therefore, I and the AABB strongly urge the committee to recommend appropriation of federal dollars towards the collection, interpretation and distribution of a national blood data as a central resource.

Thank you very much.

[Applause.]

DR. GOMPERTS: Thank you, Dr. Hillyer.

Any questions or comments?

MR. WALSH: Real quick, if I may.

Do you think the AABB are willing to make it mandatory, as a membership requirement, to submit data to a program such as that from your membership? Currently, I understand that 33 percent do now.

DR. HILLYER: If I could, I would prefer to let the AABB speak for that.

MS. LIPTON: I think the answer is yes. I mean, I think we would consider that, and in fact in some other areas we've been talking about making data monitoring. I think the issue is that, for many of these places, it would be tremendously costly. You heard about what it would cost in a hospital system, not so much the blood centers, but in the hospitals to, first of all, define what you're trying to collect. This is not an insignificant issue. When you define what is your inventory in a hospital, we don't even have standard definitions. So we don't even know what we're asking them for.

But I think the other thing is that these information systems do not talk to each other in hospitals. We heard this, and it is absolutely true. It requires the dedication of an individual to go in there and do it or you have to develop the software and give it to them. And it's just we just don't have the resources to do this. So we could, yes, could we consider doing this? Absolutely. But would we need help to implement this? Yes, we would.

DR. GOMPERTS: Any other comments?

DR. GUERRA: Yes. How sensitive is the system that currently is in place when you anticipate a shortage, say, in a hospital setting, an acute care type of setting, that says within a matter of hours or is it cumulative over days or how do you arrive at that?

DR. HILLYER: I'd like to offer my own experience rather than a broad statement.

It often is our hospital that picks up that there's a regional blood shortage. We feel the shortage by looking on our shelves, not looking at a wall of red blood. We see a wall of white refrigerators, and we go back to the blood center and say, "What is your inventory on your shelf?"

I believe that Karen is completely right, that "inventory" is a tricky word. We have three different inventories; one is available, one is reserved, one is out of the blood bank coming back. People keep their reserved for transfusion inventory anywhere from 24 hours to the entire hospital stay or 72 hours, depending on the type of hospital.

So there are these inventory issues being played. How much do you have? And as Dr. Gilcher said, there are people who are hording blood, and we know that, and they do that because they have faced these shortages.

So the answer is I can't tell you. It develops over a period of days. There's a sneaking feeling. And somewhere around Friday at 3 o'clock I start to call the head of surgery, the head of anesthesiology and the head of all of the hospitals. And then the question becomes what do I want to do? And the answer is I don't know.

DR. GOMPERTS: Okay. Thank you.

Moving on to Dr. Bianco.

DR. BIANCO: Before I start my statement, I want to just say that we agree entirely with Dr. Hillyer. We need data.

But America's Blood Centers or ABC is an association of 75 not-for-profit, community-based blood centers that collect nearly half of the U.S. blood supply from volunteer donors. We thank the committee for the opportunity to comment on the monitoring of availability of blood products in the United States. Actually, we like the name of the committee; that is, Blood Safety and Availability.

We are all aware that blood shortages exist and have worsened in recent time. The National Blood Data Resource Center from ABB reported a week ago or so that an additional 1.1 million allogeneic units would have to be collected to meet patients' needs this year. This estimate does not include any additional referrals being considered because of the theoretical risk of variant CJD. These additional referrals may add one million to the NBDRC estimates, for a total of over two million donations or a 14-percent increase needed at a time when blood centers are struggling to maintain the status quo. And we saw the flat curves presented to us by NBDRC this morning.

Shortages are worsening because demand is growing and the donor base is shrinking. Demand continues to increase because of the aging of the patient population and increased use of blood-intensive procedures like solid organ transplants, hematopoietic progenitor cell transplants, aggressive chemotherapy, and so on.

Our volunteer donor base is aging, as the population ages. We must now resort to emergency blood appeals during times of the year that used to be periods of abundance. In the opinion of ABC members, the fragility of the blood supply is a public health issue, and I'm very pleased to have heard this said by both Dr. Lewis and Dr. Epstein from FDA, and that fragility must be addressed, and measures that affect the available donor base must be managed carefully to prevent shortages that injure patients.

We thank the committee for recognizing that we need meaningful data. But, again, we must recognize that the available data already suggests that U.S. blood supply is on the verge of a crisis. Before we suggest responses to the questions of the committee, we would like to make a few important observations.

We need to define blood shortage. For instance, we suggest that we define a blood shortage as a supply inadequate to meet the needs during a specified period of time. We must take into account the inherent characteristics of the blood supply, short shelf life. RBC's expire in 42 days. Platelets expire in five days. So shortages of platelets, for instance, are a very interesting set of data. Collections vary seasonably and are affected by external factors: seasons, storms, holidays, disasters.

There is an association between blood types of donors and recipients in this country. Even in times of plenty, there are type-specific shortages. Type A predominates among the blood donors and Type O among blood recipients. Only 15 percent of the donors are rH negative. Universal donor is rH negative.

According to NBDRC data, in 1999, we collected 1.27 million units more than in '97. Nevertheless, we experienced many shortages, as evidenced by the documented urgent appeals all over the country, demonstrating that this impressive increase in collections was not sufficient to meet demand.

The objectives of the data collection efforts need to be, one, to produce reports that accurately convey the status of the blood supply to the public, to the media, and to government; and, two, to provide indicators for the management of shortages, and also in the same tone of excess collections.

Data collection efforts must be funded. These efforts require substantial investments on the part of the organizations collecting and providing data and on the part of the organizations analyzing and publishing the data. We suggest one measurement that we feel would be easy for the public to understand. We suggest that blood availability reports show the average number of day's supply of each product in a month.

The public will understand our predicament when we say that we had an average one-day supply of O positive blood and less than a day's supply of O negative blood during the last month. The public will learn that blood centers are very concerned when they have less than three days' supply of life-saving blood on their shelves. The number of days' supply could become the report card on the health of the blood supply.

There are many parameters that should be monitored according to the questions before the committee: Collections by type and by type of product and by blood type, distribution, type of product and blood type, deferrals, discards because of collection failures, discards because of test results, frequency of donations by first-time and by repeat donors, because that measures the effectiveness with which we are maintaining a solid donor base that is dedicated and continues to donate, outdates, blood appeals, and resource sharing, that is, imports and exports, and data on what we were hearing a few minutes ago from Dr. Hillyer, data on inventory management practices.

All blood centers, actually, no blood center that I know, it may be with the new system that Jackie Fredrick reported maybe they have it, has a back-order system. We just tell the hospital we'll supply the blood in a few hours, if we get it.

We do ration. We say that starting on Monday, you are going to have only 50 percent of your standing order of O. And we do, at certain points, triage; that is, distribution restricted to emergencies approved by the collecting facility.

We need also data on demand, and this was extensively discussed today. Unmet needs could be estimated, for instance, for requests to AABB clearinghouse when they are not filled. Retrospective data can be obtained from companies that mine health care data, certain billing tapes, for instance, from hospitals from around the country, and we urge today that that the--that this committee may have access to some of this HCFA data, but, for instance, blood use by hospitals, DRGs and all of that.

We, actually, ABC has had several discussions with one of those companies, Data Advantage, based in Knoxville, Tennessee. This company has access, for instance, like several other companies, to data from all U.S. hospitals.

We need to analyze those reports on supply at regular intervals, by month, as has been done, and by quarterly and yearly. We need to know the average number of days' supply in inventory by product and blood type. We need reports on utilization, as, again, extensively discussed today.

We, also, would like to know, and since this committee can consider issues of cost, as we consider other measures that, for instance, as the committee discussed leukoreduction a few months ago, what is the financial impact of blood costs on hospital budgets by measurement of a percent of hospital budgets spent on blood and blood products, and percent of hospital billings that depend on blood over a period of time? And, actually, I'd love to know the answer to one question: What percent of the hospital billing would disappear if blood products were not available?

In conclusion, the third and final question posed by the committee asks where the responsibility should reside. ABC members believe that U.S. blood system is in a crisis. There is urgent need for data. ABC members also believe and agree with FDA that this is a public health issue and that funding data for collection activities by blood centers, by contracting organizations, should be provided by the public health system.

There are professional organizations like AABB and NBDRC that can generate a good part of this data. There are also private corporations that can provide essential data on utilization. What is lacking is a concerted effort, organized by the public health system, and adequate funding. We hope that the public health system can dedicate as much attention to blood data as they currently do in monitoring the West Nile virus spread, transmission of spongiform encephalopathies, and variant CJD. We sincerely hope that this committee will urge HHS to support these data collection efforts through coordination and funding.

Finally, we also believe that there are sufficient indicators of blood shortage to warrant a national recruitment effort supported by the public health systems.

Thank you.

[Applause.]

DR. GOMPERTS: Thank you, Dr. Bianco.

Any comments or questions for Dr. Bianco?

[No response.]

DR. GOMPERTS: Okay. We're now--Steve?

DR. NIGHTINGALE: Very quickly, we have not had a formal period of public comment. Every meeting of the Public Advisory Committee needs to allow time for such public comment. The one constraint that we do have, however, is that the public comment be addressed specifically to the topic at hand.

We have announced previously that public comment is limited to an arbitrary period, something that we have ignored in the past, but there is some urgency to get on with the business. So I would ask is there any member of the public who wishes to comment at this time on the matter before the committee?

MS. HALPERN: Good morning, ladies and gentlemen of the committee and ladies and gentlemen of the public. My name is Lisa Halpern, and I am the vice president of the Red Cell Business Unit at Haemonetics Corporation.

As most of you are aware, Haemonetics has been an innovator in technology for blood collection processing for 30 years. As a member of the blood transfusion community, we have a strong interest and commitment to blood safety and availability. In fact, our corporate mission statement is not that different from this committee's. It is to provide and create innovative products to advance the safety, quality, and availability of the world's blood supply.

As we introduce our technology to blood centers around the country, it is apparent that the greatest threat to a safe and available blood supply is our ability as a nation to recruit and retain blood donors, not that different from what Dr. Bianco was saying.

Although I was not in the audience yesterday, I understand that numerous speakers have identified the continuing and compelling need for donor education and motivation int U.S., as well as in other nations. Our purpose in making a public statement today is to encourage HHS to promote, indeed, lead the effort the recruit the blood donors of the Internet age.

Although individual blood centers and associations all have individual programs to educate donors, top-quality programs, using state-of-the-art communication and education tools are expensive to develop and implement, and individual efforts consume too large a portion of scarce resources.

Furthermore, it is often difficult for these directly or indirectly competing organizations to work together to combine resources for donor education. With the exception of times of national emergency, there has never been a coordinated national campaign to promote blood donation.

HHS is in the unique position to make a difference in donor education and recruitment. The Department has none of the competitive conflicts that make it difficult for blood centers and manufacturers to work together to promote blood donation. It has successfully spearheaded many public health campaigns in areas such as smoking cessation or highway safety. It also has the resources and focus to bring to bear on this problem.

Secretary Tommy Thompson announced this week the beginning of a new public health initiative to increase awareness of organ and tissue transplantation. Our hope is that this initiative will provide significant resources to promote the awareness of the need for the most commonly transplanted organ--human blood.

Traditionally, HHS public service campaigns have included public service announcements, educational programs for schools and other outreach methods. All of these are important techniques and tools that generate public awareness that would help donor recruitment in all blood agencies.

Haemonetics would certainly be willing to support the effort to develop these tools, both financially and with contributions of our knowledge, experience and skill set. I would expect that our customers, partners, and competitors in the blood community would also be willing to support a collaborative effort to generate public awareness about the continuing and constant need for blood.

As a company, haemonetics has a long history as a supplier to blood banks, where we work closely with our blood center customers to help recruit donors to apheresis technology. As a company, we also have a long history of supporting blood donation by our employees. Through these experiences, we have gained insight into donor recruitment, particularly in the area of the interface between new technology and blood donor recruitment.

One observation is the unique appeal that donating by apheresis appears to have for younger, so-called Gen X, Gen Y donors who are drawn to anything high tech. We are very successful at recruiting young new donors to red cell apheresis.

Promoting the technical advances and sophistication of blood donation and component preparation in the 21st century may make blood donation more appealing to this critical demographic group. However, if apheresis donation appeals to some donors, it also creates a donor education challenge.

We would like to share our internal experience with one means to meet this challenge. Haemonetics uses an Intranet-based website as a recruiting tool for blood drives held at our factory. Web-based communication, with all of its vitality, its power for interactive personal learning brings new life to communication and education efforts and turns awareness into motivation.

By posting personal donor and patient stories from our own haemonetics community, procedure information and automated real-time self-scheduling, we were able to dramatically increase the number of donations and decrease the burdens of recruitment and registration at our blood drives.

We believe this is a transferrable and scalable approach towards recruiting blood donors. An HHS-sponsored general public website on blood donation and public announcement advertisements on popular browsers or websites could reach an entire new population of blood donors who use the Internet as the media of first choice for communication and information.

Recruiting blood donors and developing public awareness of the importance of blood donation is not a new idea. Many of the people in this room probably have donor recruitment posters framed in their offices. Some of them dating back to World War II. So why do we see this as an important issue for today? What has changed is how critical it is today to supplement our continuously shrinking and aging donor pool with, if you pardon the pun, the new blood of the next generation.

We believe it is time for a coordinated, well-planned campaign to increase public education and motivation, and we believe no one is as well positioned to lead this charge as HHS. Thank you for your time and attention. We appreciate the opportunity to speak today on the vital issue of protecting our nation's blood supply.

[Applause.]

DR. GOMPERTS: Thank you.

MR. ROBERTSON: Good afternoon. My name is Derrick Robertson, and I represent 19 hemophilia treatment centers who either have factor delivery programs under the 340(b) Act or are considering developing these programs.

I just want to talk very briefly on the whole issue of the data that's available through the PPTA and other sources. I think one of the crucial things is, as I agree wholeheartedly with Val, this is a very valuable tool and has been very useful for us to be able to look at it and understand not only the inventory, but by using the data to get an understanding of what the production levels are from month-to-month.

I think it's very important to define, and maybe this committee could define exactly what a shortage is. I think that that's critical because in the community, the hemophilia community, now there has developed over several years what I would term the psyche of shortage. It has never been able to get to a situation where you can feel comfortable that you can get the medicine that you need.

And I think in trying to understand what is causing this situation would be absolutely critical. For example, in my view as having done economics, as an undergrad, that essentially you have to look at demand versus supply. That's the basics. And if you don't understand the demand and you don't understand the supply, you really can't determine if there is an actual shortage. I think we have not, we have touched on the supply data. We're beginning to collect that. We probably need to do a better job of collecting the demand data. But in doing that, it's going to be important to look at demand not just for what product has been ordered by the treatment centers or the home care companies, but what product would likely be prescribed by the physicians because those ordering patterns are usually limited by what allocations there are at a given point in time.

I think in looking at the supply data, it's also important to understand whether there is an issue of worldwide supply or just supply in the United States and how that compares to the demand. Because what I have been looking at, in terms of the data, seeing where one manufacturer, and these are all guesstimates, having 50- to 55-percent of their production coming into the U.S. and the rest going worldwide. Another manufacturer may have as little as 30 percent, and like I said just guesstimate.

But if these numbers turn out to be true, then we, as a community, need to realize that probably part of the situation comes down to corporate decisions that will take, well, this amount is going to be in the U.S., and this amount will be in Europe, and this amount will be in Asia, and we need to then have that information to be able to better advocate with the companies that, well, can't you put more here, not trying to pass any judgment on whether those corporate decisions are right or wrong, but simply an ability to be able to address them.

I think, in interpreting the data, somebody needs to ask the questions not just to have the data there, but to look at this and ask the questions why do production fall by X percent in this month? Why is it down? There is no FDA action going on, there's nothing. Why was production down? And getting those answers I think will allow us as a community, to allow you as a committee to better understand what the situation is. Is it simply a lack of availability in the United States or is it truly a shortage that exists for recombinant product?

Because if you are able to interpret the data, then you can address the issues. Is the issue one of demand that is spiraling through prophylactic treatment, through inhibitors? Is that reason? If that's the case, then the community needs to understand that, well, if we're going to choose these type of treatment regimens, there's a consequence to it or is that the manufacturers are not putting enough into the U.S. market to meet all demand? And, again, not whether that's right or wrong, but whether we need to be talking to the manufacturers and say, "Is there any way you can bring more here?"

Is it that the worldwide demand is growing so much that the manufacturers think we can't keep up? Is it a capacity issue? And if that's the case, well, again, we probably need to be advocating for more capacity, which I know the manufacturers are addressing.

Historically, it just seems as though each time, especially in the recombinant marketplace, there is an opportunity for increased supply, it doesn't translate into the market equalizing. When one manufacturer brings on a new manufacturing suite, then you think that a 30-percent increase in production will help stabilize the situation, it doesn't realize. And I think as we move forward, as Val so eloquently said, we don't want to be back here a year from now or two years from now looking at the same issue. We need to understand what the issue is and really get to the bottom of why we have the situation we have today.

So getting the data is excellent. I think more importantly we need to interpret that data and get some questions answered from the manufacturers and also from those that are providing the demand.

I thank you for your time.

[Applause.]

DR. GOMPERTS: Thank you.

MR. WILLIAMS: I'm Alan Williams from the FDA Office of Blood, and as some of you know, I also have a continuing research interest in the characterization of donors and the screening of blood donors. And I'd like to make three succinct comments which I think are relevant to today's topic. I'll also mention I will add my congratulations to the data collection already in place. I think that the NBDRC has done a terrific job in the face of many hurdles to allow us to have data where we had none several years ago.

First of all, I'd like the committee to consider not only monitoring blood safety numbers or blood availability numbers on an ongoing basis, but also take a little broader academic approach to the whole recruitment issue.

As shown in the donor recruitment workshop held last year, there are many approaches taken to increasing the number of blood donors who come in, and some of them, like the Canadian Blood Services approach and several approaches in the country, have involved letter-writing campaigns and telerecruiting, which certainly are very effective, although they do take a lot of resources.

We also have seen an increase in the use of incentives to bring donors in to donate, and there are some data from the Red studies which show that over the years this has increased over time, and not to say whether or not there are safety issues related to those, clearly there are other mechanisms in place which are attracting donors that need to be considered.

Also there's increased use of the media and other mechanisms, such as telerecruitment, and it's really an unknown how far these mechanisms can be taken before they break, and you simply cannot bring in additional donors. Or, alternatively, you start to bring in a lot of first-time donors, which do, in fact, replenish the donor base, but in most cases it's for a single time and ultimately is much less efficient than using repeat donors.

Secondly, I'd like to make a please for consideration of federal funding for some psychosocial research related to the knowledge, attitudes, and behaviors of potential blood donors in the general population to get at the root cause of what motivates the people to come in, what are the hurdles, and where can educational strategies best be brought forward to assist the recruiting process that's done at individual blood centers.

Additionally, I'd like to make a plea for some rapid survey data capability available on a regular basis. You're all familiar with the travel survey which was used to predict the loss of donors due to U.K. travel. That was, in fact, done in short order with no budget, and the data have been used extensively at many public meetings now. And this sort of capability needs to be in place to address issues like this into the future. And the better mechanisms and systems we have, the better the data will be.

Briefly, the screening of donors was mentioned earlier and the efforts of the Uniform Donor History Task Force. I'm a member of this task force, now currently a liaison. There is a questionnaire product that has been developed and is ready for further study. We would very much like to see this questionnaire validated for comprehension by an external force, and this, unfortunately, will take some funding to do. So I'd like to urge committee support for the modest funding that it will take to do some validation studies of this questionnaire for comprehension. And I think ultimately it will produce a better questionnaire for screening blood donors and reduce unnecessarily loss.

Number three, I'd just like to say beware of oversimplification of donor loss issues. The travel survey predicted a donor loss of 2.2 percent based on our random sampling of donors. On-site deferral by some blood centers was as low as the 0.13 percent mentioned earlier today, but keep in mind that donor loss on site is what is recorded. Clearly, there was national media which brought this deferral into place. When donors called in to make appointments, they were often screened by the blood center as to whether or not they met this deferral criteria. They received educational materials in the mail and on site at the blood center. And what you're seeing is the near final result of those who, based on the interview, self-deferred because they saw this question for the first time. So just understand that difference.

We see a similar magnitude of difference in screening for AIDS risk factors between deferral of the first-time donor population and risk factors in the general population. So that's not unprecedented at all.

I'll end there. Thank you very much.

[Applause.]

DR. NIGHTINGALE: Thank you.

Are there any other comments from the floor?

MR. GOLDFINGER: Yes, I'm Dennis Goldfinger from Los Angeles and thank you for the opportunity to say a couple of words.

Dr. Hillyer gave his perspective as a hospital-based physician on the situation of blood shortage in this country. Clearly, one difference between me and him is he said that he had a ten-year experience with this. One of the few advantages of being old is that you have more experience. And, clearly, the magnitude of the shortages that I'm seeing in my own career are beyond anything that I had ever seen earlier, certainly in the last several years.

I can't say that I've ever seen a patient die from lack of blood, but I'm actually amazed that that's not happened.

Tuesday, my last day in Los Angeles before coming here, we actually had no O negative blood on our shelves and had to delay transfusion while we were waiting for some blood to come from the Red Cross. It's just amazing that we've not seen situations where patients actually have suffered--either died or suffered serious morbidity as a result of not having blood due to these shortages.

The Red Cross cannot meet the need that they're projecting if they lose an additional several percent of their donors. I'm amazed at how well they've done after having lost various percentages, most recently with this ear stick to finger stick thing. They've come through very well. But they can't come through any further.

Right now, the Red Cross in Los Angeles fails to meet the blood needs of the city. There are many hospitals that collect blood, and we obtain blood from outside of the city in order not to be without blood.

In my mind, the only way that we can meet this need is by pumping more money into the system. Blood banking, the blood banking industry really survives on a shoestring budget. They're always just at the brink of disaster. Virtually every for-profit endeavor that has come along to collect and deliver blood has gone out of business. There's just no money to be made in blood banking.

But now, not only can't we make money, we can't even deliver the blood that we need. The only way is by increasing the money that is pumped in.

In my role as a member of the board of directors of the Southern California Region of the Red Cross, I have jokingly--well, not so jokingly said that we need to double the price of a unit of blood in order to have sufficient money in the system so that we could hire sufficient recruiters, so that we could hire more nurses. And this is not some kind of an ingenious method, although I like some of these other methods, for example, using the Internet. But for now, the simple method is to pump more money in so that we can hire the people who can recruit and collect the blood that we need.

The Red Cross has kind of taken me up on it, I think, because they're going to come through with a very major price increase. And I do think that this will help.

The problem is going to be now wearing the other hat as a blood bank director that the hospitals are going to get killed. And so it's very--this same problem came along with NAT testing, and most recently with universal leukocyte reduction, and I'm very happy, Dr. Nightingale, that you've gotten a member from HCFA to sit on this committee, at least for--or to be present at these meetings at least for a period of time. It's essential that HCFA recognize their responsibility to pay for the increased costs associated with the need to deliver a safe and adequate blood supply.

Thank you.

[Applause.]

DR. GOMPERTS: Are there any further comments?

[No response.]

DR. GOMPERTS: Okay. Steve, do you want to comment?

DR. NIGHTINGALE: The comment I want to make is that we, the government, are aware that we have not had a lunch break.

[Laughter.]

DR. NIGHTINGALE: And it is the intention of the government to see if we can conclude our business without one, and the reason for that has to do with the point I made at the beginning of this meeting, that there are many conflicting interests today, and we are trying to accommodate them all.

If, however, we need to break in order to conclude our business here, we will, but I would ask the members of the committee to begin their discussion, and I am aware of some motions that members wish to offer. Should we perhaps go around in alphabetical order, as we often do, and ask for comments before we introduce motions?

DR. CHAMBERLAND: Can we at least have a little bit of a break, just ten minutes or something? I think it would help the discussion if we could all kind of get up, move around. Some of us have to check out of hotels and things like that, whatever. But I think it would be appreciated if we could have a little bit of a break.

DR. NIGHTINGALE: I find myself overruled by Dr. Chamberland.

DR. CHAMBERLAND: I may be in the minority.

DR. NIGHTINGALE: Ten minutes.

[Recess.]

DR. NIGHTINGALE: Could the members please take their seats?  Members? We are under a new "No more Mr. Nice Guy" policy here.

DR. GOMPERTS: Prior to that plea for a break, we had started going around the table--that was great. Get that on the record.

Mary, do you have any comments?

DR. CHAMBERLAND: Yes, I actually do. Okay. A couple things.

One is I want to say that I'm more optimistic at the end of today--near the end of today's session than I was perhaps when I arrived in that I think I--I know I arrived thinking that it would be an overwhelming, incredibly daunting task to think that it would be possible to monitor supply and availability.

And while I think it's a very complex task, I was energized by a number of the presentations. There's a lot of work that's been done, is in the process of being implemented, some exciting, innovative approaches.

However, no one presenter, no one organization has the whole picture. Everybody's got a little bit of it. Okay? But we don't have the complete picture.

I also think I'm being somewhat pragmatic in that I think it's going to take a lot of work to put the whole thing together, to have a comprehensive system of surveillance in place that has long-term monitoring, forecasting, and early warning components to it, if you will. And I don't think that--while this has been a really good venue to learn about what's ongoing or planned, this is not the place to build the nuts and bolts of a surveillance system. That, as has been alluded to by many speakers, takes a lot of work, starting with standardized definitions, methodologies, resources, et cetera. So I don't think we should leave here thinking we're going to build and know exactly what this system of surveillance and monitoring should be.

Where I don't want to get pessimistic is to think that it's going to stop here, because I think personally what's needed is a bringing together of different bodies, organizations, with different types of experience and expertise. And these may be perhaps, you know, groups that don't usually kind of sit down under an umbrella and meet and thrash out details, because I think there's real contributions from industry, I think there's some real contributions from the private sector, I think there can be some lessons from the years of experience that we've had in public health surveillance for various diseases and whatever.

And I think what's needed is identification of an entity to lead that effort. Someone has to bite the bullet and pull all of you out here around the table who have the contributions to build such a system together. So I think if we could leave with some notion of who or how that might happen, it would be a real significant step forward.

DR. GOMPERTS: Thank you.

We'll move up the table here. Raj?

MR. DALAL: First of all, my personal compliments to all the speakers, including comments from the public. I thought they were very articulate and thoughtful contributions to today's deliberations.

I just want to comment on the importance of having macroeconomic supply-demand data in order to guide public policy and strategy. And let me use an analogy from a totally different part of the economy.

If we were discussing crude oil production, which is a national security matter, we would want to know the supply-demand equation quite clearly. We'd want to know how many oil fields, how much oil there was, what the production rates were. We'd want to know a lot about the utilization in terms of what kinds of automobiles and how power was generated. And we would establish national policy drawing upon some of that information in terms of mileage of automobiles and whether we wanted an electric car at some time in the future. Basic macroeconomic data of this nature is essential in order to guide rational and thoughtful public policy.

In that regard, I think we need to think how broadly or how narrowly we draw the boundaries of this supply-demand. Particularly should we include the European community or not? For example, if BSE and TSE issues have a real bearing on the supply of European plasma, what impact will that have on production of plasma in the United States? And how does that supply-demand equation work? So can this be isolated as a U.S. problem or, in fact, is this a broader global--and how do we define that box?

Then, third, I said macroeconomic because I wanted to distinguish that from the data and the information that individual organizations collect and use in order to run their businesses and their enterprises competitively and better than the next enterprise next door. And I think as we think about all this data that we want to collect in order to get a macro view, I think we also need to be very respectful of the proprietary or the data that is considered proprietary within organizations so that they can use it to compete effectively.

DR. GOMPERTS: Thank you.

DR. DAVEY: Mr. Chairman, I'd like to also thank all the speakers. I thought it was an excellent discussion this morning. And it seems to me just in summary that, while we do have data out there, it is not very coordinated. And I would certainly support a greater effort to both collect additional data and meaningful data, especially on the utilization demand side. Therefore, I would certainly support some of the comments about having a central focus, an office or a focus at the PHS level with appropriate funding to move this forward.

I think just another comment is that I was impressed that if we do collect data and more useful data, how that data is used is really going to be a challenge. I think we heard from so many different groups today, patient advocacy groups, certainly, hospitals, blood centers, the AABB, the Red Cross, ABC, the FDA, this committee, everyone is going to want to use this data and use it in different ways. So I think one of the real challenges to any group or focus or funding target will be how to gather the data and then analyze it in a way that makes sense to all the competing groups that need these data, because the data are going to be used in many different ways.

I think Dr. Wallace actually mentioned earlier in his discussion that any analysis, any summaries, have to be done by very experienced people. And I think that Marian and her group certainly fulfill that bill, and there are others also that are key. We need experienced people. We need to target the data so that it's most meaningful to the people that need it. But I think we do need a central focus, a national focus, and appropriate funding.

DR. GOMPERTS: Larry, did you have a comment?

MR. ALLEN: Can you come back to me?

DR. GOMPERTS: Okay. Jay?

DR. EPSTEIN: Thanks. Well, I guess I'm struck by the difference between the supply monitoring problem in the area of plasma and derivatives and that of whole blood. I think that in the area of whole blood, the problem in a nutshell is that the system is not currently operating in the mode of needs-based collection. In other words, there's a sense that you could get more blood and there are various ways to get more blood, but we don't routinely get the blood because we know we need the blood.

And in that direction, I thought I heard a very big disconnect. I heard Dr. Wallace tell us that the data you need is really hospital-based, that ideally it ought to be based on utilization related to DRGs, that the data are hard to gather, and that in a survey of 30 hospitals, only six can provide it; and if you want to get more of that data, it would require a huge influx of resources and human effort.

And then I heard the Red Cross say later in the day, oh, we've done it, we have that system, we can model this, you know, we are already at the point where we can collect according to demand.

So those statements can't both be true. I mean, if Red Cross is talking about 3,000 hospitals and, you know, if Dr. Wallace is talking about a reasonable national sample, then there's a disconnect. And so I would just say I don't understand that disconnect unless people are talking about different things. But they didn't seem to be. On the one hand, they were saying, you know, this is hard to get. On the other hand, they were saying, well, we've got it. So I don't get it.

[Laughter.]

DR. EPSTEIN: The situation with respect to plasma derivatives I think we heard is quite different, which is that the supply instabilities relate to the fact of a small number of manufacturers and that the instabilities that we see appear to relate both to anticipated and unanticipated events related to production. And I'm not discounting the fact that there have been changes in demand.  But I have the sense that those changes have been gradual over time, although there may have been, you know, ramping up, for example, of off-label use of IVIG over a decade. But, still, those tend to be gradual, whereas the crises are coming because of, you know, punctuated disruptions that mainly have to do with the oligopoly structure of the industry. And I'm not sure what the remedy is, but I think the challenge is to improve the predictive potential based on what we do know about reserves and events as they unfold, and that there's been a communication gap. Where the information exists, it's not being optimally utilized, at least it's not available to the consumer.

On the central question, which is what role should government play, I would like to respond to the charge that government has no role to play in the monitoring of supply. I think in the ideal scenario that's the right answer. In the ideal scenario, you know, our health system is privatized, and we generally allow market forces to sort out the balances between supply and demand. However, government has gotten involved because the system isn't working optimally. That's what the consumers are telling us. And I think that it is both within our legal mandate and our need for government to play a role precisely because there are these supply instabilities and they are recognized as a safety problem.

So that said, my instinct would be to agree with Dr. Davey that the proper role of government ought to be as a facilitator and a coordinator. Personally, I would like to see some focus within the PHS. However, I don't actually think government can be the vehicle for the data gathering. I think that it's been pointed out by Mike Busch and others that, you know, all the data exists widely distributed in the blood system, in the industry, and the more you can get at the local level, the better it will work. And really what we're talking about is feedback loops between the data you might gather and the actions you might take today and tomorrow.

So I tend to think that government should play a facilitating role, but that there is a large role to be played in the private sector with regard to the actual data gathering and analysis. Government needs to have the products of that data gathering available so that it can do what's necessary. And I do agree with Rick that a coordinating and facilitating role, as well as, you know, a major funding role, is necessary and appropriate.

DR. GOMPERTS: Colonel Fitzpatrick?

COLONEL FITZPATRICK: Thank you, Mr. Chairman.

First, Mary has said pretty much what I was going to say, succinctly and better than I would have said it. So I can go to the punchline, I guess. And Jay followed up with that, so that's really good.

The key points are that we need to monitor supply and demand. We're not doing very well with demand, and we really have a tough time finding out what demand is. Timing is everything, and I couldn't be here yesterday because the Department of Defense is involved in a worldwide war game, and part of my role in that war game is to respond to an immediate demand for blood.

This is probably the first time in about nine years that we have played it actually down to the donor center level within the Department of Defense. Part of the rules that they were to follow this year was to strip all the liquid blood they had on their shelves and immediately send it to two places that we have, one on each coast, to distribute from overseas.

Then, in turn, they were to call their civilian counterparts and ask what could you supply me immediately so that I know how much I need to leave on my shelf to continue to treat the patients that I have in my hospital.

I'm still getting reports back, but so far I know of two of our 23 donor centers who were told I can give you nothing. And that's not to say that three days from now they wouldn't be able to give us a lot, because I know that if we went to war, the population of the United States would respond, and we would have donors at the doors. But the immediate need is of a great concern.

And this is the first time in my experience in 26 years that a civilian donor center has told one of our donor centers I can give you nothing. So that tells me that we are facing a critical problem.

As far as the comments about a national campaign, I'd like to go back to that for a minute. I think I agree with Mike. What we do need is a national donor campaign. We need to mobilize the population of the United States to understand that blood donation is a responsibility. Fragmenting the population by enacting different donor deferral criteria between major collection organizations within the United States is not going to help that.

Most of the predictions of the success of donor campaigns have been based on the Canadian experience. In Canada, there are two collection agencies, Heme Quebec and Canadian Blood Services, and they did enact different donor deferral criteria initially. But they had a national campaign based on a national need.

If we don't have a national campaign and if we fragment the donor population and confuse them, I don't believe we will see the impact that the Red Cross is predicting.

To show that DOD is in support of a national campaign, just last week I got the Chief of Staff of the Army, a four-star general who is in charge of all the Army, to sign out a letter encouraging blood donations to DOD donor centers and civilian collection agencies that is being distributed to the commanders of all Army installations as we speak.

We don't have the resources within DOD to collect at all our facilities. We have to collect at the facilities. We have donor centers. We have to make the donor base available to civilian collection agencies at the other sites. And I intend to do that, even in the light of losing up to 25 percent of my eligible donors.

So the bottom line to me is that we need to be able to flatten those peaks and valleys in that supply and demand graph that the NBRDC showed us. How can we do that with a 42-day dated product? One is through research. The Army has developed an anticoagulant solution that will allow 66 days of storage. However, we have yet to be able to find a manufacturer willing to invest the capital to take it to licensure because there is, quote, no need for longer shelf life of red cells.

We do have a frozen blood program which we use to offset critical shortages overseas. It would be possible with advances being made technologically to make that useful within the United States, and there are license applications before the FDA that will make that more practical, allowing storage of thawed, deglycerized red blood cells for possibly 14 days after thawing.

So, to sum up, I believe that--I support the Red Cross and the need for a national campaign for blood donors and that we do need to expand and grow the donor base. But I feel that we need to have confidence in the FDA, in their ability to lead us in safety regulation, that we should all nationally follow the same safety regulations when it is a major issue, such as variant CJD; and that if we can figure out a way to monitor supply and meet supply--I'm sorry, monitor demand and meet demand throughout the nation, we can even out the supply.

And so as far as the role of the government, we should be able to monitor the supply. We should be able to develop a way of intervening when demand outstrips supply, and that I think is the challenge that faces us.

Thanks.

DR. GOMPERTS: Thank you.

Ron?

DR. GILCHER: Essentially I agree with everything that has been said, so my comments can be short.

I believe that Health and Human Services does need to support the endeavor of data collection and analysis with respect to supply and demand of our blood supply. But there is an infrastructure in a sense that's already in place, and that is the National Blood Data Resource Center, and I think that should be looked at very carefully as an organization that can, in fact, do much of this.

However, there's one piece that is missing and that's been brought up repeatedly, and that is the utilization data that is needed to complete the picture.

On the other side is the issue with respect to plasma, and what I heard this morning is that when there is only one supplier, then it really is a critical situation. And that has occurred specifically with the Alpha 1, and that is something that I still do not completely understand how these shortages can occur. But I think where there is only one supplier, this really puts that particular group of individuals in very, very serious jeopardy whenever the supply is curtailed for whatever reason.

DR. GOMPERTS: Thank you.

Fernando?

DR. GUERRA: Let me try to look at this a little differently. First of all, let me offer the following suggestion. You know, about maybe 14, 15 years ago, when it was very clear that our country's public health system was in disarray, the assignment was given to the Institute of Medicine and the National Academy of Sciences to see how that could be addressed. And so I would suggest that because we're talking about a system that is tremendously important for the public health and well-being of this country, that from what we've heard over the course of the last several years, it is in varying stages of disarray that perhaps it could be presented as an important question and issue to the Institute of Medicine, perhaps as an assignment to look how we can try to make things better in terms of assuring the availability, dealing with issues of distribution and inventory, monitoring, surveillance of the nation's blood supply and blood products, and see how a group like that that is totally nonpartisan and certainly has access to some of the best minds and creativity that could take on what I think is a tremendously compelling and very important question.

The other has to do with the suggestion that I think we've heard several times, and I think it would lend itself for some near-term opportunities to really take this on as an important public health campaign to promote, one, the concern related to the need that exists, related to shortage and at times some distribution issues, and try to increase awareness for developing the next generation of donors that will really buy into it as an important public health responsibility that has to be shared. And I think it would certainly fit in well with any number of other public health campaigns that are going on, and one that probably with some support could certainly be launched in the near term.

The other is the one that has to do with trying to centralize the effort for gathering data, and I would certainly support the notion that this is something that does have important public health implications and opportunities. I think that there already is in place, at least within parts of the public health system, ways to monitor some of this if there were some additional support for expanding that capacity.

But I think that, you know, especially in large urban areas, public health departments that have many different responsibilities, one of those is for having that close working relationship with the hospital care community that deals with anything from reporting of individuals with certain conditions or clustering of those conditions or issues related to diversion, whenever there's a shortage of beds or nursing shortages, et cetera, just to try to keep the community apprised of some of those critical needs that exist. And I think that, you know, it certainly could perhaps, with a well-designed and supported system, perhaps even as a public and private and voluntary type of effort could certainly be organized and would have the capacity for doing the kind of monitoring and the tracking that I think is essential.

Then just my final point--and I think we learned this very clearly in the recent shortage of the influence vaccine during this past season--is that we didn't anticipate it early enough to try to put into place some contingency plans without really trying to engage the private and proprietary interests in a way that would have allowed us to better handle the distribution of the vaccine. And I think that so it is within the blood industry that there are those proprietary interests that at times make it very difficult, and that if, in fact, there is an opportunity to try to more clearly define the roles and responsibilities, and if some of that rests within Health and Human Services, and particularly the public health system, that it be linked to some authority to be able to deal with issues of distribution, because when one can certainly anticipate or recognize that there is a shortage and a maldistribution of some of these critical products, there has to be a way to quickly move in and say, look, let's redistribute this instead of hording and stockpiling.

DR. GOMPERTS: Paul?

DR. HAAS: A couple weeks ago, Steve asked me to make some comments on today's issues from an academic economist's perspective, and I in a silly way actually wrote some ideas out. And all morning long, I've been re-editing those comments. And once I thought I had them pretty well down, now I listen to the comments around the table, and I should be re-editing again.

So I guess the good part about all that is what I'm going to say is a lot shorter and I'll spare you an economic lesson, although I think there's some issues there.

The second part, though, is that I'm not at all convinced that what's going to flow is going to flow as smoothly as I would have liked. But some of it's repetitive, and I think I have a few points that are not repetitive.

The market we're working in, it's hard to call it a market. When we think in terms of an academic way of looking at things, markets are when private sellers are interacting in a way that they compete, knowledge flows very easily, we don't have to talk about how we gather this data because the market mechanism does that. And this is not that type of market. It's a market in which government and private sector are inextricably intertwined. And the bouncing back and forth of bureaucracy, in effect, becomes a necessity because of that intertwined nature. It's not something that is necessarily something that we're ready to throw away, because we're saying the market won't give us the type of blood products that we'd want, so we do want government to play a role, but we can't figure out what that role is and a way to allow the market system to work the way we'd like.

And, again, I come back to my fundamental point, well, we're living in a dream world if we think the market's going to do it. So our real task is what I've heard come down the line, that we've got to sit together and think about how we get these two entities working very closely together.

One comment that I did write before that I would like to make here is that anyone who's been through a basic principles of economics course knows that this market doesn't fit that textbook model. Theoretical economists dismiss that. In a sense, they say markets will eventually clear, but it takes time.

If you've had those principles of economics courses, you probably heard of a guy named John Maynard Keynes, a relatively well-known individual who was reported to have said: In the long run, we're all dead.

Well, he was referring to the Great Depression. It seems eminently clear that that comment is fairly apropos to what we're doing here, and I say it only half in jest.

The issue in front of us--well, Val Bias I think made a point so eminently clear when he spoke from his heart. Not that his other comments weren't important, but certainly from an academic economist's point of view, the person often gets lost in the whole process of what this economics is all about. And, boy, Val made that eminently clear, that if we're not thinking of that person or persons who are getting blood products, we're missing a very, very real part, I think, of what our responsibilities are as a committee.

I said earlier that effective markets coordinate that demand and supply, but what I've heard today is that we have a good sense of the data that's needed, but we're really not sure how to coordinate or how to pay for it. We're hearing that government has a role. Again, that's that bureaucratic part of the process which is an absolutely essential part of the process.

In the plasma area, just to repeat another point, it was made clear by the industry, it's been made clear here, that that market is going to remain a market of very few sellers in the marketplace. The textbooks call that an oligopoly, as Jay remembered his economics well enough to get that term right.

You have a situation there where when we're thinking about getting the data, as the organizations told us, it's hard to report it out because of the proprietary nature of the data. Well, we need the data.

We run into a very interesting situation in that PPTA very correctly pointed out their restrictions because of antitrust. And as I was listening to that--I actually had some of that written in my comments--I started to think, well, you know, when I was taught this stuff, and I go back to 1890 when the Sherman Act was passed, and then all the amendments up through then, the Antitrust Act was put into place to protect the consumers.

Think about what's going on around the table today. The Antitrust Act is, in fact, making it harder for the consumers to get what they need or what the producers need to do to coordinate their production.

One of the things I think this committee ought to be doing is making an explicit move to try to see if there's some way we can work with that. If we don't address that particular part, one arm of government is going to keep another arm of government from doing its job very effectively.

I want to repeat another point that came up. In trying to figure out this whole demand and supply thing, I think the emphasis on getting demand data is critical. You can't have two parts of the puzzle--or one part of the puzzle and say we're going to solve the problem. But several speakers made the point that we've got to be very careful with the demand data.

We have a hard time figuring out who needs the product but just aren't presenting themselves because they know the product's not available. If we lose that side of the picture, we again are going to put ourselves into a position where we're going to have at least--we're going to still have a shortage. It just might not be as clear with the numbers.

This is almost painful for me to day, but an economist by his very training is supposed to talk about the importance of efficiency. That's the way more resources become available to do things.

In the markets that we're talking about--I guess I'll be even a little more specific in the sense that none of us want products to be left on the shelf and not get used. It has been made clear throughout today and at other meetings that we want to keep outdating at a minimum. Well, if I try to meet that goal, that element of efficiency, we create the other potential of an error of saying because we're keeping the inventory so tight that when the demand has a spike, we're not ready to go.

The inventory structure, as we've heard a lot today, is not the just-in-time inventories that we have out in the private sector. We can get it kicked up in emergency situations, as we heard. We heard just the Army response in the sense of saying, well, if we can get it there in two or three days. Well, in that military situation, if it were real, that's a disaster. In some of the communities that we're talking about, we have another disaster.

So we've got to also rethink what we ought to be doing with these inventories. Maybe it makes sense to absorb some cost, some throwaway, if, in fact, we can get enough donors in at the front end, of course, to try and help the situation.

I think as kind of a general comment as I try to pull this stuff together, I, too, was encouraged by all the interesting ideas of how different agencies are gathering data. I'm discouraged in the sense of in this world of trying to bring private and public together, that at least right now--and I agree--I forgot even who has said it right now--this is not the place to work out--it was Mary, I think, who made the comments--trying to work out the details today. I'm discouraged in the sense of saying how do we really coordinate this, and I guess I want to go back to the point I made a few moments ago in the sense that if we're afraid really to talk because the other arm of government called antitrust says that we're not allowed to talk about certain things, we've got to get that clear so this information flows. We have to get out of our heads that the marketplace is going to be able to bring this information together because of the very complexity of this marketplace. We've got to get people that are competing in the private sector, meeting with individuals in the public sector, and have a true sharing of knowledge. That's a complex task. That's an incredibly complex task. But it seems to me that we have to keep that focused there, or we're not going to get to where we want to go.

Thank you.

DR. GOMPERTS: Thank you, Paul.

Larry?

MR. ALLEN: Well, if everyone would kind of bear with me here, I'm going to kind of jump around a bit, but I'll do my best here.

First of all, I again would like to thank all the speakers for all their presentations over the last couple of days. I do agree with some of the comments by Mary and Jay. I definitely feel that the system as it is now has failed, and definitely there's some room for improvement.

I also think that we could do a better job of not only collecting the data but defining and utilizing this data, and I think I agree here with Mary that it's going to take some time to find the proper model in order to do this.

I'm reminded of a couple of things from the last few meetings. We talked about the leukocyte reduction. We went through all of that. My son, unfortunately, was one of the people who got leukoreduced bloods and the filters failed, and then we're talking about shortages again today, and I realize I'm thinking back to January when he had to have transfusions, and we waited two and a half days for blood. And at the time, I didn't think anything about a shortage. I actually thought it was because they were trying to match the blood, the antigens the way we've asked them to do.

But I guess what I'm getting to now is the fact that we're here because of the Nancye Buelows, the Jan Hamiltons, the John Walshes, the Dana Kuhns, the Michael Allens of the world. That's why we're here. And their quality of life is most important to me especially.

So I just want to emphasize the fact that even though we're nowhere near the finish line here, I just want all of us to keep that in mind because when you're in situation such as my family has been in, you don't have a lot of options. And this committee for a lot of people is the final step. And if we can't get it done here, it's not going to get done.

So I just think we need to just keep that in mind, not that anyone here has forgotten that, but it's just something that I needed to say. And I just want us to work along those lines of trying to find the avenues to help everyone as best we can and work with the government and work with the private sector to get this accomplished. I just think that that's where we have to go at this point, because without that one day all of us are going to be on the other side of the boat here.

DR. GOMPERTS: Thank you, Larry.

Dana?

DR. KUHN: I, like Paul, was asked by Steve to pontificate upon this subject today, but instead I'm going to give the Reader's Digest condensed version of the insights that I believe that I have into this.

As I see it, there are two issues before us, and one is data collection for blood, as I would put it, and then the second would be data collection for plasma derivatives and recombinant. And since I'm not an expert in the area of blood but I feel like a pretty good expert in the area of plasma derivatives and recombinant, I'd like to share some insights that I have into this subject.

It has come to my attention that, as manufacturers manufacture the product, either the plasma derivatives or the recombinant, it is sent out from their jurisdiction and it goes out to what I would call resellers, and even 340(b) programs. And then it goes to the end user.

Somehow, some way, there is a lack of data from the time it goes out from the manufacturers until it gets to the end users. And that could be because end users may store it up and put it in their refrigerators. It may be that some of the resellers, because of good business practices--I'm not saying bad business practices, but good business practices--are concerned about their livelihoods and the continued ability to sell it to their customers, these products.

But I think the point that I am seeing is that it is very important to try to glean information from those end users, because in a business, that is really where you're going to get your information about how much product is really being used.

I would like to suggest that in maybe some of the recommendations that we make or whatever entity is going to hopefully collect this data, that we would also include another element, which would be, as some of you know, the hemophilia treatment centers are required to collect data. CDC has asked them to collect minimal data. And that includes the age of patients, it includes the severity, and it also includes those who are on prophylactic treatment and immune tolerance treatment.

I think you will gain a lot of information of usage once you can get that data also. Plus, if manufacturers were able to have that kind of information, they would probably be able to adequately plan for production. And then I would think that, as I heard before, if you had that information, at least in my business sense of understanding, if you had that data, you would be able to predict the unpredictable in case there may have been a bacteria or microorganism that came along in one of the product lines and it's being held or decided what to do with it, you'd be able to kind of plan for that rainy day, because there are other production lines. Hopefully it's not going to affect all the production lines.

So I think that these need to be taken into consideration as one collects the data and as one provides that data to those who are responsible for manufacturing and making sure there is enough supply for the end users.

DR. GOMPERTS: Dana, thank you.

Yes?

MS. LIPTON: Just a few comments because I think everybody said everything I was going to say.

I do want to say that I came here today prepared to advocate that we really need to collect demand data, and nothing I've heard today has changed my mind. I have been talking to hospitals. As you know, they're the greatest number of our members. They feel an acute shortage. They firmly believe that something is going on and that they don't even have adequate data upon which to try to plan their own practices in the hospital. And I think that's really where the rubber meets the road, is the people who have to treat the patients.

I do believe this is a public health issue. There is no doubt in my mind that there is a role for HHS in this, as Jay said, and maybe not necessarily collecting the data, but certainly in feeding some of it back so that we as a committee or HHS, or whoever's going to be in charge of this, has some idea what's going on for this critical public health issue.

I know that it sounds like a daunting task, but I also believe that there are people out there and that they could be brought together. I mean, I'll make no bones about it. I believe the Data Center has the capacity to at least start this, could have a very broad umbrella group in it. The Data Center not only has a board who are all expert in this area, it has advisory committees, an there's nothing to say that you couldn't expand them to include government experts and other people.

The national awareness campaign, which is sort of what I like to call it, I think it's a great idea. I think we need to do it. They've done it in Canada successfully. I think it is a role for the Federal Government. Our problem isn't in trying to develop the materials. Our problem, frankly, is in buying the air and time that we need to put it out.

We have public service announcements. They get played at 3 o'clock in the morning. If you want the message out there, you buy the time in the media, and that's what gets the attention of the people that we need to reach.

I guess, last, I was so moved today by a number of the comments. All of these shortages ultimately do affect patients, and we had a number of examples that, actually, Chris isn't here anymore and he didn't want me to repeat. But we know that shortages are affecting the way people are practicing transfusion medicine. They are changing dosing levels of platelets. They are doing things, and they are doing it because of supply. It has nothing to do with whether we believe it positively affects the outcome for patients. And then to have Val stand up there and talk about the issue of replacing therapies and then potentially facing, you know, the future that they go back to insurance companies and find that everything that we've worked for in this committee is suddenly gone because they will be on plasma-derived products. I think it does--it starts with the patient. That's our obligation, and I think we need to do something to move this forward.

We intend as the AABB to go to Congress and to ask for funding. So I don't have any hopes, frankly, that it will necessarily come through HHS without that. What I need from this committee to do that, though, is a recommendation so that we have something to carry to Congress to say this committee said fund this initiative.

DR. GOMPERTS: Thank you.

Lola?

DR. LOPES: When I first received Steve's announcement about this meeting, I thought that we'd be dealing with fairly ethereal issues of what the data are that we need to collect. It has come as a surprise to me that we are really in a state of some serious shortages, both for people who have chronic needs for blood factors and for those who might need to go to an emergency room or have an important surgery scheduled. I think the issues of data are very central to this.

In terms of the data about supply, I am surprised that there have been greater efforts among the for-profit organizations to share data and make it available than is the case among the non-profits. I don't know all the reasons for this, but I believe that that information is a national resource that we would be remiss not to see to it that someone be allowed to have access to all the data about supply.

On the demand side, it was interesting to me from Dr. Wallace's comments how little information seems to be available within hospitals about usage patterns. And I don't know how much of this is because the data is not captured in the first place and how much of it comes from the fact that the data is lodged in different databases and different departments.

Now, in my corner of the world, we have seen huge steps in the last five years in the use of data warehouses as a repository for data for multiple databases, making it trivially easy for users to get access to data that five years ago would have been expensive and almost useless by the time the programming was done to get them.

I think that one of the things that could come out of this group is the recommendation that someone, a federal source, fund model user projects with hospitals to develop ways that hospitals themselves can build the databases that they need to use themselves and that we in turn can call on for the data we need on demand.

DR. GOMPERTS: Thank you.

Do you have a comment?

MS. PAHUJA: Finally a time where I feel like my age can be of advantage. I would just like to speak to the issue that was brought up earlier about attracting--or the aging donor population. I speculate that I am closer in age to the Gen X/Gen Y population than some of the people in this room, so I feel I can speak to that.

While attracting them and recruiting this new population presents challenges because of their perceived attention span, I don't think their return to repeat donation is any different than why their parents give or why older members of the population give, which is a perceived trust in a system that works.

At present, Dr. Gilcher said it's a system that seems fragmented and sending mixed messages. What is a shortage? Is there a blood shortage if there's blood available in St. Louis but not in New York?

There's no clear-cut answer, and I'm, I think, as confused now as I was before, as I am when people ask me that question as a recipient of blood products. So I feel that it's very important that we send some kind of cohesive message, whether it is to HHS that this is something that they should undertake as a national program to encourage donation or to develop some kind of coordination of all of the data that is being collected and that should be collected that's not.

DR. GOMPERTS: Thank you.

John?

DR. PENNER: As one in the retarded level, I must admit that I've been impressed so far with a good deal of the information that's been coming through. Particularly, the Red Cross presentation was impressive, the fact to me that they've been able to get their system up and working and pretty much identify some of the needs throughout their system.

What bothers me, it's their system and we still have 50 percent of the population served by others, and I don't see where one is going to be able to transfer things very easily unless there's some pushing together in this area. And I'd like to be able to know that when Los Angeles is down that Oklahoma City can send 500 units or 1,000 units, and that you can triage that blood very quickly.

We've got FedEx out there that can label everything and tell you where it is and how far it's going to be before it's going to show up. There's no need why we can't do the same thing with the units of blood throughout the country. So we do need something as an oversight.

Then, lastly, I'd just like to make a remark about the demand situation. We did hear that there is considerable variation in physicians' approach to blood usage. I think we need to attend to that as well, and that may be an area that requires a little bit more attention.

DR. GOMPERTS: Thanks.

John?

MR. WALSH: Yes, thank you, Mr. Chairman. I think everybody's articulated, certainly better than I, the issues and identified them, and I would just like to reaffirm that I have the utmost confidence that this committee will be able to come up with some specific recommendations that address the issues identified and that will make certain that the appropriate follow-up is undertaken.

I definitely feel that I'd just like to highlight there are two issues, as Dr. Epstein indicated. One is the blood supply and the other is the plasma derivative and recombinant supply. And I think that although both of these require additional comprehensive data collection on both the supply and demand side, they are a little different, if not a lot different, and I think need to be addressed appropriately.

I'd like to thank this committee for their focus on all plasma derivatives, and our community has asked for help and has done everything we can that we're aware of to pursue this. And I think that as Linda Lipton indicated, I think that we should as plasma users mobilize our constituencies and get up there on the hill and support follow-up on the funding request, if that's what it's going to take. And I would pledge that from the Alpha 1 community.

DR. GOMPERTS: Thank.

Jerry?

DR. WINKELSTEIN: Most everyone has spoken, made comments that I agree with. The only thing I'd say is that what concerns me the most is that you can monitor and you can study all you want about how material is entered into the system and how it's utilized, and I certainly endorse that. But what worries me is that it seems that the number of individuals that are eligible to give blood is going to decrease, because there are going to be emerging infections, contraindications to donations. Those are only going to grow rather than reduce.

And so a large part of my concern is not just monitoring availability but the other part of that, which is to increase availability. So all of the people that have spoken about increasing donor populations or the populations from which donors can be obtained seem to me to be very important. And I think that if we're going to make a recommendation, I'd be enthusiastic about having the recommendation be twofold and certainly include some recommendation for public education so that there can be more donors rather than fewer, because I think that would solve a lot of your problems.

DR. GOMPERTS: Thank you, Jerry.

Steve will have the last word, but I do personally want to make a couple of comments. I think during the tenure that I've had on the committee--and we've addressed many issues, but I think this particular one is the most important that we've been asked to talk about, address, and make recommendations on.

It's complex. It's heterogeneous. There are many issues. But there is no doubt in my mind that there is data being generated. Responses are taking place to various issues, and the data from the National Center presented earlier this morning indicates responses to shortages. But clearly this isn't adequate, and we need to do a better job from the point of view of getting that data, targeting that data, making sure it's meaningful, and then interpreting it accordingly.

So, Steve, you have the last word here.

DR. NIGHTINGALE: Actually, I have the next to the last word, and what I wanted to use my time for was to tell you what will happen to the last word, which is yours.

We have discussed bringing this issue to HHS. We have looked for the need to identify a locus within HHS to coordinate this activity. We've talked about looking for leadership within HHS. If you are looking for that, look no further except around you, because it happens to be the way HHS is structured now, and, surprise, has been structured for quite some time, has been the leadership for the issues that cross-cut agencies, as opposed to those that are specifically within it, is here because it's really not anywhere else.

The actual office that deals with the cross-cutting blood issues has been until recently Mac and myself. And that has not been a bad thing because the real structure has been this committee, where we are in the process of dealing with the issue of the blood supply, as I'm talking to my colleagues, and in this respect, you are my bosses here.

My proposal that I made to Dr. Satcher to have this meeting was to use this as an introductory, an exploratory operation to collect as much information as I could from the public. We had initially started out with the idea that we could subcontract the data collection of the blood supply. That was, as you've heard, successful to a far greater extent than I think most people in or out of the government realized, but to the extent that it was unsuccessful--and I think that was the extent that it hit the rocks, which it did--was because of an underestimate of the complexity of the task.

I think we've made huge progress in elucidating the complexity of the task and identifying potential solutions to some of those complexities.

We have not, as Mary pointed out and I think everybody appreciates, gotten to the bottom of it yet. I think that the process that I see coming out of this meeting is, first of all, I look forward to your specific recommendations, and we will, as is always the case, communicate those expeditiously to the Secretary.

I am hoping for an expeditious response from the Secretary to those recommendations. However, at the same time that I am hoping for that response, there will be a great deal of staff work done. The first part of the staff work will be to incorporate not only the suggestions and the recommendations, but at the same time, the members of the blood community have been very generous, and I mean that with their suggestions. I did not mean that in the way that somebody--the suggestions have been very constructive. I want to get as many of the--as complete an expression of the views of the community on how to monitor the blood supply onto a piece of paper as soon as I can, and the minimum amount of time I think that's going to take is 30 days, because that's going to take some floating back in draft with the members of the committee.

I think that I have to have a distillation, I need the essence of those views, before I can take those views to circulate them within my colleagues in the different agencies and try to achieve a consensus within the agencies about what the best path to take is. But having gotten that consensus, I need--and this is my challenge right now, is to get the attention of the new administration on this issue.

My goal for that task is June 30th right now. I'd like to have it earlier. I don't think, I really don't think I will. We do have limited resources within my own budget, first of all, to fund the continuation of the National Blood Data Resource Center effort through December 2001. That contract is somewhere between me and Mary, and there's a lot in between it. But that contract is out of our office right now.

There is roughly $100,000 plus some change for a contract that I hope we can develop to make some--at least to do an exploratory analysis of how to measure demand. A hundred thousand bucks isn't going to do it, but what I'm giving you are performance measures that I've got to hold myself to. I very much hope that that contract can be developed by June 30th and put out for bid on July 30th so that on August 30th we can have the contract in and a mechanism for awarding that before the end of the fiscal year.

Obviously, there's some urgency to determining what will be the next year's--what will be the budget in 2002 for these activities, and that's the other thing that has to happen right now.

At the conclusion of this, there will be the need for an additional meeting of the Advisory Committee on this. I would not convene the committee until I was ready, but obviously I need to be ready sooner rather than later, and with my colleagues and with your support and input, we'll do it as soon as we can.

DR. GOMPERTS: Thanks, Steve.

I think moving ahead now to recommendations, a number of the committee members have been focused on some recommendation activities. Who's coordinating that? Ron?

DR. PENNER: It's in Mac's hands.

[Laughter.]

DR. PENNER: A condensation of a number of things that Ron and Dana and I had tried to put down on paper so that there is some place to start. And I guess we can read it off as we go.

Whereas--Ron, why don't you read it off?

DR. GILCHER: It's easier for you.

DR. PENNER: All right. Whereas, patient access to a safe and available blood supply is a public health priority--I don't think anyone will argue with that one--the Advisory Committee for Blood Safety and Availability recommends that the Secretary of the Department of Health and Human Services and Congress work to ensure that an appropriate office within the department has the responsibility to support national blood collection and utilization data.

Moreover, adequate federal dollars should be provided to support collection, analysis, and distribution of these critical public health data.

Specifically, the following should be addressed, and the first bullet is identify an agency responsible as a repository for such data; second bullet, to support programs to develop the data; and, third, to encourage organization of blood collection centers as well as manufacturers of blood products for the purpose of identifying and addressing areas of short supply of blood and blood products.

So that's kind of a consummation of some of the ideas, and I don't know that it's particularly specific or particularly arguable at this point, but maybe we could get--

DR. GOMPERTS: John, is there a second recommendation?

DR. PENNER: There is a second recommendation, if we'd put it up. It's just with demand, and this was my--I don't know if we've got that up. That was with respect to the demand area, and that is, support a program of physician education designed to improve blood product utilization throughout the United States and to encourage support for such programs through the Department of Health and Human Services, the idea there being to try to improve utilization of the blood products on the demand side so that the products are being used appropriately, which I believe is not the case now from personal experience, as well as comments that have already been mentioned.

DR. GOMPERTS: Okay. Let's return to the first recommendation. Jerry?

DR. WINKELSTEIN: I was going to suggest the second recommendation should be turned into--could be modified to become a third recommendation, so you'd say support a program of public education designed to improve blood and blood production donation throughout the U.S. and encourage support for--et cetera, et cetera. Because I still think public education is a big part of what we've been discussing today.

DR. GOMPERTS: Okay.

DR. WINKELSTEIN: So I would suggest we add that as a third recommendation.

DR. NIGHTINGALE: We are two recommendations right now. In the interest of conciseness, could we put physician and public education in there to avoid the repetition--

DR. WINKELSTEIN: Then you'd have to say improve blood and blood product utilization and donation, or donation utilization for parallel construction.

DR. NIGHTINGALE: Okay.

DR. WINKELSTEIN: But I'd start that by saying, for parallel construction, I'd say support a program of public and physician education designed to improve blood and blood product donation and utilization, and I'm happy to have it that way. Just put it in parallel construction.

DR. GOMPERTS: Let's just put in those corrections. I just have one question, John. When you say support, who should be supporting?

DR. PENNER: Presumably this should be HHS support.

DR. GOMPERTS: Okay.

MS. LIPTON: Can we just go back to the first one?

DR. GOMPERTS: Let's just finish--allow Mac to complete--you're done? Okay.

MS. LIPTON: I'm trying to understand the third point. Go back to the other one, recommendation one, Mac, third bullet.

DR. PENNER: The attempt there was to try to bring the blood collection centers and those involved in distribution of blood products in some--getting them together in some way to address these areas of short supply, namely, when there is an absence or a deficiency of blood in, say, Detroit and we have full knowledge that there's plenty of blood in St. Louis, we should be able to have some means of addressing that.

I'm harking back to the old days when I was responsible for a blood center in our area, and that whenever we got in short supply, my associate knew exactly who to call. He's try about five or six centers that he knew had a surplus, and he's extract some, and then vice versa. So they had their own little built-in system, but it was not a nationwide thing. It was kind of catch-as-catch-can. And if you had somebody who was really very good at it, you never ran into supply problems. And if need be, you had an excess, you could always dump it in Los Angeles, because they paid more. And that way we'd end up charging our institutions much less for the blood because we were making more from L.A.

MS. LIPTON: So is the word organization or something else? That's what I--

DR. PENNER: Maybe it could be rephrased. You'd need some help there.

DR. NIGHTINGALE: Coordination?

MS. LIPTON: That's what I--

DR. PENNER: Collaboration.

MS. LIPTON: Collaboration.

DR. PENNER: Collaboration would be a better word.

DR. GOMPERTS: Paul?

DR. HAAS: I guess I'm looking at that third bullet. Was the intent to include cellular and plasma products there? And I think if it is, I'd almost suggest we should separate out the plasma as a separate bullet because there's sufficiently distinct problems in the two areas, that to lump them together I think potentially confuses the problem. Someone else can work with the wording.

DR. GOMPERTS: I think what is missing here, in the first two bullet points, you're focusing on the data, collection of that data. There should be another bullet point indicating that that data should be made available so that blood centers, collection centers, can respond to the information. In other words, making and ensuring that the data collected is available so that there is a national response or a regional response or a local response to specific shortage issues.

DR. PENNER: So there would be another, which would be to, I'd say, support programs to develop data and then ensure availability of data...

DR. NIGHTINGALE: And ensure its availability to the public?

DR. PENNER: Yes, that would be good.

DR. NIGHTINGALE: Mac, do you think you could just take that--continue in part two. You don't need a separate bullet.

CAPTAIN McMURTRY: Okay. Don't do a separate bullet.

Give me the words again, please.

DR. NIGHTINGALE: And ensure its availability to the public.

DR. GOMPERTS: I think if you could qualify that, rather than ensure its availability, ensure that the data collected is available, rather than "its."

MS. LIPTON: Is data singular?

DR. NIGHTINGALE: That is a very complex question. There are parts of FDA that consider data to be singular, and there are other parts that consider it to be plural.

CAPTAIN McMURTRY: I'm easy. I'll go whichever way you want.

DR. PENNER: No split infinitives.

Maybe a question is: Are there other items out there that ought to be included? Because it would seem to me that if we had something to work with that we could add to or subtract and think about, that would provide us with the means of getting back to you and deciding whether this is something that you wanted to pass on. I don't know if you wanted to get this determined today or whether you want to just let this simmer for a bit.

DR. NIGHTINGALE: I want everything that you--I want every idea that you want emphasized, emphasized in a recommendation. The purpose of my penultimate comments were that don't worry if you don't get it in the--if it's in the transcript, it won't be forgotten. This is for emphasis.

DR. GOMPERTS: Mary?

DR. CHAMBERLAND: Just a couple thoughts in terms of how the current verbiage might be interpreted, particularly the first bullet: identify an agency responsible as a repository for such data.

I think to follow up on the comments that Jay and Rick Davey made, that not necessarily does the Federal Government have to be the collector of these data, and, in fact, there are a lot of hurdles and burdens imposed on the government when it seeks to collect the data that would not fall on industry. And I think the words that Jay and Rick used were coordinate and whatever.

So I guess I wanted to get a sense from the committee what their reaction to Jay and Rick's comments was about should the government actually be collecting the data. And repository of data to me implies that we might actually be collecting it as opposed to somehow coordinating or facilitating an effort to see that the data are indeed collected and shared and acted upon, et cetera.

DR. EPSTEIN: I think the word that needs to be changed in draft recommendation one is where it currently says support national blood collection and utilization data, it should instead say facilitate the availability of national blood collection and utilization data. Third from last line, third word, strike support and say facilitate the availability or facilitate the gathering and dissemination.

DR. CHAMBERLAND: Jay?

DR. EPSTEIN: Facilitate the gathering and dissemination of national blood collection and utilization data.

DR. CHAMBERLAND: Just to follow up, Jay, and, again, I guess this speaks to why such a recommendation needs to be very clear. I took the term support to mean kind of that the Federal Government would somehow, through whatever efforts, have resources that could be brought to bear on the issue, that they wouldn't themselves be doing the collecting, but could provide resources either through a contractual method or some other mechanism for others to collect the data. And I guess if you insert facilitate to that, it would somehow change what the intended meaning was.

DR. EPSTEIN: Well, you know, money is the ultimate facilitator. I don't think it precludes it, but it doesn't lock us in if we can come up with clever alternatives.

DR. DAVEY: Mary, maybe just on your first bullet, the comment might also be--it might also be modified to say, to identify your concerns, identify an agency responsible for facilitating the collection and analysis of data, facilitating, collecting, and coordinating data to appropriately monitor the national blood supply, something like that.

DR. NIGHTINGALE: Or even better, because it might be shorter, just to say identify responsibility for this activity. That addresses your concern, Rick. It also addresses Mary's concern. Because if we say agency here, among other things, remember the 60-day hold before you can ask anybody how old they are. That's for any federal question. There's a 60-day hold. Identify responsibility for this activity. Or you might even say assign responsibility for this activity.

DR. HAAS: Does activity need to be more specific since the first bullet...

DR. NIGHTINGALE: You could, or you could assume that it referred to the previous paragraph.

DR. HAAS: I wouldn't assume it.

DR. GUERRA: Steve, don't you think that's too permissive, that rather it should almost be a requirement, a mandate or something?

DR. NIGHTINGALE: These are supposed to be your thoughts and not mine.

DR. GUERRA: Well, I think, you know, if, in fact, this is an issue for public health, I think it almost has to be more--

DR. NIGHTINGALE: Do it.

DR. GUERRA: Yeah, to do it.

CAPTAIN McMURTRY: What would you like?

DR. GUERRA: I guess where we say responsibility to facilitate, I would just say responsibility to gather and disseminate national blood collection and utilization data.

MS. : We don't want to collect it.

MR. WALSH: Yeah, I don't think we want the government to do that.

DR. PENNER: Are you going to use assign? Which I thought wasn't a bad idea.

DR. NIGHTINGALE: Was or was not a bad idea?

DR. PENNER: Was a good idea.

DR. NIGHTINGALE: Assign responsibility here, the first two words in the first bullet.

COLONEL FITZPATRICK: To keep with what Jay and Mary have been saying, though, wouldn't you want to drop--bring back the facilitate into that bullet, or coordinate? Because what they were saying was that you could contract that repository.

DR. NIGHTINGALE: I tell you, what I suggest is if Mac types a bridging phrase, assign responsibility for this activity, then we can work on the bridging phrase and see where we go from there. If it's okay, let's just type in assign responsibility for this activity as the first bullet and let the committee decide how they want to--assign responsibility for this activity. Type that there right now. Okay. Get rid of the rest of it. Okay. Get rid of the A, and then add to it as you will.

DR. DAVEY: Steve, I think that's fine as written, the first bullet. Just leave it brief.

DR. NIGHTINGALE: Get rid of two. I can work with this one real well because it doesn't tie my hands.

DR. WINKELSTEIN: Well, as long as you do what we want, go ahead.

[Laughter.]

DR. NIGHTINGALE: And if I don't, I've got to come back.

DR. GOMPERTS: Yes, Dana?

DR. KUHN: The question I just have about this recommendation, does it address the need for obtaining the data that may be missing between the time that a manufacturer puts the product out the door to the usage by the end user? That's the question I would have in here.

DR. NIGHTINGALE: Then I would suggest in the first paragraph where you have collection and utilization, you insert the word collection, distribution, and utilization. Would that address your concern, Dana?

DR. KUHN: Yes.

DR. NIGHTINGALE: Is there stylistic concern about whether or not there's a comma after distribution?

DR. PENNER: Have we got enough whereas's in there?

DR. GOMPERTS: Paul?

DR. HAAS: If we're through with that, I want to come back to my concern about plasma. But I can wait.

DR. WINKELSTEIN: I have a small point, which is the whereas really applies to number one and number two. And at the risk of sounding like I really care about wording, I would say whereas, and go all the way through to work to, and make number one work to ensure, and number two would stand as it is. The whereas applies to both suggestion one and suggestion two. That's the only point I'm making. So I'd cut it out--I mean, I'd have it as an introduction to both. A small point.

DR. GOMPERTS: Okay.

DR. PENNER: Actually, you can leave the whereas out and it would probably handle it just as well.

DR. GOMPERTS: Paul?

DR. HAAS: If I caught what we said earlier and we do want to keep plasma and whole blood separate, I think we can just create two versions of the third bullet. And the third bullet, with minor modification in the third bullet, the fourth bullet would be encourage collaboration of plasma manufacturers for the purpose of identifying and addresses areas of short supply of plasma products. Something to that effect. Then I think it would take out--probably take out the manufacture of blood products in number three.

DR. GOMPERTS: Let's get that one down.

MR. WALSH: I also think we need to include recombinant.

DR. HAAS: Yes, yes.

MR. WALSH: Which wouldn't be covered in the--

DR. NIGHTINGALE: Paul, would you dictate for Mac--

DR. HAAS: I'll just give it to Mac.

MR. WALSH: Mr. Chairman, just for the record, while we're working on this language, not related to this resolution or a proposal for another resolution, knowing that this is not the platform nor the meeting content to address this, but I would ask that the committee make certain that at another point in time, i.e., another meeting, we follow up on the issues related to the interruption in production and its effect on the access to product from different consumer groups.

DR. NIGHTINGALE: Noted. I don't need a specific resolution for that.

DR. EPSTEIN: Steve, could I raise another point while we're wordsmithing? In the first bullet, a little trouble that we haven't captured, the notice of predictive models. It's buried that we want in the third bullet of item one to identify and address areas of short supply. But I think that in the first bullet, we're interested not just to gather and disseminate collection, distribution, and utilization data, but also to develop useful predictive models.

DR. NIGHTINGALE: Hold that thought. I think both of us can--

DR. EPSTEIN: I've been holding it for the better part of three years.

[Laughter.]

DR. NIGHTINGALE: Surely another 30 seconds is possible.

Mac, are you caught up?

CAPTAIN McMURTRY: I think.

DR. NIGHTINGALE: Okay. If you could move the cursor up to distribution and utilization data, put a comma, and, Jay, would you do the honors?

DR. EPSTEIN: Well, just say predictive analytic models.

DR. NIGHTINGALE: And development--

DR. EPSTEIN: Or analytical models--and development--

DR. NIGHTINGALE: Development of predictive analytic models.

DR. EPSTEIN: Well, yeah. We want to get in the word shortage, though, and development of analytic models to predict shortage.

CAPTAIN McMURTRY: To predict shortage?

DR. EPSTEIN: Yes, and development of analytic or analytical models to predict shortages.

[Pause.]

DR. GOMPERTS: Mac, could we go back to the beginning and we can read through this again?

DR. DAVEY: Leaving out the whereas makes it not a sentence, unfortunately.

DR. NIGHTINGALE: Could I see a show of hands for those who would prefer whereas as the first word?

DR. WINKELSTEIN: I love whereas.

[A show of hands.]

DR. NIGHTINGALE: One, two, three, four, five, six, seven--I see eight hands. That would be a majority.

DR. PENNER: It sounds congressional, doesn't it?

MS. LIPTON: But you also want to capture the thought that it modifies one and two, so one doesn't start until work, with the word work.

DR. EPSTEIN: Yeah. I think what we need to do is after DHHS and Congress, it should be colon, and then strike the words work to, just put number one, ensure.

DR. NIGHTINGALE: If that is the case, can we delete the phrase specifically the following should be addressed, and just make those two, three, and four?

DR. GOMPERTS: Yeah.

DR. NIGHTINGALE: Does anyone disagree with that?

DR. WINKELSTEIN: Those are subsets of number one, though, aren't they? They're specific to number one.

DR. NIGHTINGALE: Okay.

DR. WINKELSTEIN: I think it's better this way.

DR. PENNER: So you don't need a one if there's no--

DR. NIGHTINGALE: No, you'll need a one because you've got a two coming.

DR. PENNER: Oh, I see. Yeah, that's right.

DR. WINKELSTEIN: Two and three were combined.

DR. PENNER: Combined, yeah. So there will be a two.

DR. GOMPERTS: Could you move it up? Just move it up so we can--I think we're okay on the whereas section.

It looks like we're just about done and ready to vote on this. We're ready?

DR. DAVEY: So moved.

DR. GILCHER: Second.

DR. NIGHTINGALE: Dr. Davey moved, Dr. Gilcher seconded the recommendation.

For the record, I would like to read the recommendation so that everyone knows exactly what they are voting for and that the transcript will show what they voted for.

The motion that Dr. Davey moved and Dr. Gilcher seconded reads: Whereas, patient access to a safe and available blood supply is a public health priority, the Advisory Committee on Blood Safety and Availability recommends that the Secretary of the Department of Health and Human Services and Congress: one, ensure that an appropriate office within the department has the responsibility to facilitate the gathering and dissemination of national blood collection, distribution, and utilization data and development of analytic models to predict shortages; moreover, adequate federal dollars should be provided to support collection, analysis, and distribution of these critical public health data.

Specifically, the following should be addressed: assign responsibility for this activity, support programs to develop the data, and ensure that the data collected are available to the public, encourage collaboration of blood collection centers for the purpose of identifying and addressing areas of short supply of blood and blood products, encourage collaboration of plasma manufacturers for the purpose of identifying and addressing areas of short supply of plasma products and recombinant material.

Two, support a program of public and physician education designed to improve blood and blood product donation and utilization throughout the U.S., and to encourage support for such programs through the Department of Health and Human Services.

DR. GOMPERTS: Okay. All those in favor of the recommendation?

[A show of hands.]

DR. GOMPERTS: Any against? Abstentions?

DR. NIGHTINGALE: The vote was unanimous. The only abstention was the Chairman, who votes only to break a tie. There were no objections to the motion.

DR. GOMPERTS: Well, that brings us to the end of the deliberations. I would like to thank and congratulate the committee for their staying power, dealing with the hunger, as well as the public representatives. Thank you.

DR. GUERRA: And thanks to our Chairman for doing such a good job.

[Whereupon, at 3:03 p.m., the meeting was adjourned.]

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