Blood Safety Transcripts

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

Fifteenth Meeting

9:11 a.m.

Friday, August 24, 2001

Hyatt Regency Capitol Hill Hotel

400 New Jersey Avenue

Washington, D.C. 20001

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

Arthur Caplan, Ph.D.
Larry Allen
Michael P. Busch, M.D., Ph.D.
Mary E. Chamberland, M.D.
Rajen K. Dalal
Richard J. Davey, M.D.
Jay Epstein, M.D.
Col. Michael G. Fitzpatrick
Ronald Gilcher, M.D.
Edward D. Gomperts, M.D.
Paul F. Haas, Ph.D.
W. Keith Hoots, M.D.
Harvey Klein, M.D.
Dana A. Kuhn, Ph.D.
Karen Shoos Lipton, J.D.
Lola Lopes, M.D.
John Penner, M.D.
Jane A. Piliavin, Ph.D.
Jerry A. Winkelstein, M.D.
John Walsh
Stephen D. Nightingale, M.D.
Capt. Lawrence McMurtry
Virginia Wannamaker

C O N T E N T S

AGENDA ITEM

Call to Order, Conflict of Interest

Current Status of Monitoring Blood Supply and
Demand - Stephen D. Nightingale, M.D., Office
of Public Health and Science

Summary of 8/23/01 Blood Demand Contractor's Conference, Darrell J. Triulzi, M.D.

Break

Public Comment

Committee Discussion and Recommendations

Lunch

Immune Deficiency Foundation Proposal to
Monitor Demand for Plasma Derivatives -
Jason Bablak, J.D. Immune Deficiency
Foundation

Plasma Protein Therapeutics Association
Comment - Christopher Healy, J.D., Plasma
Protein Therapeutics Association

Public Comment

Committee Discussion and Recommendations

Adjournment

P R O C E E D I N G S

CAPT. McMURTRY: Good morning, ladies and gentlemen. Regardless of what this says in front of me, my name is Lawrence McMurtry. I'm the Deputy Secretary--Deputy Executive Secretary of the Advisory Committee for Blood Safety and Availability. I just gave myself a nice promotion there. I wish I had gotten the pay for it.

DR. CAPLAN: When Steve is not here, he is in charge.

CAPT. McMURTRY: I'd like to begin by calling the roll, if I could. Mr. Allen?

MR. ALLEN: Here.

CAPT. McMURTRY: Dr. Busch?

DR. BUSCH: Here.

CAPT. McMURTRY: Dr. Chamberland?

DR. CHAMBERLAND: Here.

CAPT. McMURTRY: Mr. Dalal?

MR. DALAL: Here.

CAPT. McMURTRY: Colonel Fitzpatrick?

COL. FITZPATRICK: Here.

CAPT. McMURTRY: Dr. Gilcher?

DR. GILCHER: Here.

CAPT. McMURTRY: Dr. Gomperts?

DR. GOMPERTS: Here.

CAPT. McMURTRY: Dr. Guerra is in San Antonio today. He had--I think it was grant reviews today.

Dr. Hoots?

DR. HOOTS: Here.

CAPT. McMURTRY: Dr. Klein?

DR. KLEIN: Here.

CAPT. McMURTRY: Dr. Caplan?

DR. CAPLAN: Here.

CAPT. McMURTRY: Ms. Lipton?

MS. LIPTON: Here.

CAPT. McMURTRY: Dr. Lopes?

DR. LOPES: Here.

CAPT. McMURTRY: Dr. Penner?

DR. PENNER: Here.

CAPT. McMURTRY: Dr. Piliavin?

DR. PILIAVIN: Here.

CAPT. McMURTRY: Captain Snyder, unaccounted for this morning.

Dr. Winkelstein?

DR. WINKELSTEIN: Here.

CAPT. McMURTRY: Dr. Davey?

DR. DAVEY: Here.

CAPT. McMURTRY: And Mr. Walsh?

MR. WALSH: Here.

CAPT. McMURTRY: Ms. Pehuja is also not with us this morning. She started law school this week. So she is going to remain active with the Kulis (ph) Anemia Foundation and will be at subsequent meetings but is not here today. And, in fact, I'd like to ask for somebody to propose a resolution commending her on being accepted to law school, if I may.

DR. PENNER: So moved.

CAPT. McMURTRY: Second?

DR. GOMPERTS: Second.

CAPT. McMURTRY: Nobody opposed to that, I assume. Thank you. I'll have that in the notes.

The next thing, this is the part that I have been looking forward to since Steve told me I was going to open the meeting, and that is, getting to read his conflict of interest statement. I'm telling you, this is exciting for me.

The following announcement is made part of the public record to preclude even an appearance of a conflict of interest at this meeting. General applicability has been approved for all committee members. This means that unless a particular matter is brought before this committee that deals with a specific product or firm, it has been determined that all interests reported by the Advisory Committee members present no potential conflict of interest when evaluated against the agenda.

In particular, as specified in Title 18 of the United States Code 208, subsection (b)(2), a special government employee, which all committee members are, may participate in a matter of general applicability, for example, advising the government about its policies related to hepatitis C epidemic, even if they are presently employed or have the prospect of being employed by an entity, including themselves, if they are self-employed, that might be affected by the decision of the committee, provided--and this is the key point--that the matter will not have a special or distinct effect on the employee or the employer other than as a member of a class.

The example given at 5 CFR 2640.203, which implements the U.S. Code, is as follows: A chemist employed by a major pharmaceutical as been appointed to serve on an advisory committee established to develop recommendations for new standards for AIDS vaccine trials involving human subjects. Even though the chemist employer is in the process of developing an experimental AIDS vaccine and, therefore, will be affected by the new standards, the chemist may participate in formulating the Advisory Committee's recommendations. The chemist employer will be affected by the new standards, but only as a part of a class of all pharmaceutical companies and other research entities that are attempting to develop an AIDS vaccine.

In the event the discussions involve a specific product or a specific firm for which a member has a financial interest, that member would exclude him- or herself from the committee discussion, and that exclusion will be noted in the public record.

With respect to other meeting participants, we ask in the interest of fairness that they disclose any current or previous financial arrangements with any specific product or specific firm on which they plan to comment. I will note also for the record that each voting member of the Advisory Committee has in a packet before them or had in a previous meeting in a packet before them a specific waiver for the purpose of participating in the meeting under the terms that I have just described.

When the committee was established, it had been considered that the appointment by the Secretary himself constituted that waiver. Where changes of administration rules undergo review, as do formal policies and action, and the waiver is an additional measure of protection for the committee, it confers no new rights. It does not require your signature. We perhaps have completed the piece of paperwork which we'll be glad to discuss with any member of the committee if they wish to do so, but the bottom line is your rights, your responsibilities, and your protection, which is really the bottom line here, are unchanged by that.

And I think that we have gone through as much of that as we need. So, with that--

DR. KLEIN: Where's our chemist?

CAPT. McMURTRY: Actually, he's out in the hall because there were no chairs.

DR. KLEIN: I see.

CAPT. McMURTRY: So, with that, the agenda, which none of you have--but I do--calls for a welcome by Dr. Arthur Lawrence, the Acting Principal Deputy Assistant Secretary for Health, but he's not here either. So we'll move along.

I'll let you take over.

DR. CAPLAN: Do you want to do the summary?

CAPT. McMURTRY: Yes, let's do this.

x DR. CAPLAN: There was a meeting held yesterday on the contract to try and develop a plan to monitor the blood supply. As you know, with all the discussions of deferrals, with all the discussions of what can be done to ensure safe and adequate supply of blood, it's been difficult to do that without numbers and without having an independent assessment of what the situation is with respect to inventory of blood.

There are lots of ways to compile statistics. I'm always despondent when I find out that I thought we'd get an answer by looking to numbers, and then it turns out that numbers are flexible. So there are a lot of ways to count, and there was a discussion yesterday. Who's going to do the contractor--who's going to recap? That's what I'm not sure, who's doing this meeting.

CAPT. McMURTRY: Dr. Triulzi.

DR. CAPLAN: Okay. And you're going to hear me today say make sure to hit your mikes, the button, when you're talking later. Also, I'm going to ask you to say your names today because we have a new transcriber who's desperately trying to keep tabs on who's who.

CAPT. McMURTRY: Dr. Triulzi, could I get you to come up to the front? We have the press that is wanting to put you all over the television and newspapers.

DR. TRIULZI: Thank you very much. My name is Dr. Darrell Triulzi, and I'm the medical director for the Institute for Transfusion Medicine in Pittsburgh and one of the 29 participating sites in this effort to collect data on blood demand.

I think it would be appropriate to first thank Captain McMurtry and Steve Nightingale, and especially Virginia Wannamaker, in their tremendous successful effort in being able to pull together this group so quickly and so successfully.

Yesterday's meeting was critical because this was really the first opportunity the participants had to get together and really define the parameters for this multi-center study, which is essentially what this is. And it's important to remember that we were really defining how to do this study, that we're not nearly at the point of collecting data in any amount sufficient to provide meaningful information to this committee. We don't anticipate that that data will be available for many months.

We're really at the stages of defining how to report data, which data to report, how it would be analyzed, and that was really the function of yesterday's meeting, a critical part of the meeting. So to that end, I will tell you some of the things we were able to accomplish.

First, we came to agreement on a form for Web-based reporting to make it easy for the 29 centers to report the data being requested. The second, and very important, was that we needed to be sure that we were reporting apples and apples. So we had to come up with a common definition of what is total inventory, what is exported or shipped, should we be including autologous and directed, how do we count neonatal units. So we discussed that and came to agreement on what standard definitions would be so that the data that we are getting from each of the centers would be standardized.

Secondly, there was a recognition that assessing only the inventory in the hospital was probably not the complete answer of what we were looking for in trying to assess the impact of blood supply in the hospital level. So we realized we needed some information that more directly monitored impact on patient care. And we actually came up with quite a long list of potential candidates, including how often a transfusion might be delayed or cancelled, how often surgery might be delayed or cancelled, how often Rh-positive red cells would be used in an Rh-negative recipient, how often physician consultations were needed for shortages. So we came up with a list of parameters or endpoints that we would report that really assess what are we having to do with the transfusion service level to effect an impact of blood shortages.

We also recognized there was a need for a standard operating procedure for this ongoing study, which tells you how early we are in the process, that we really don't have yet a standard operating procedure in which we can codify all the definitions and reporting requirements that we discussed yesterday.

On a very positive note, many centers, I think 25 of 29, have already begun reporting data. The definitions that we came up with and the data reported so far will be useful and still we'll be able to put those in the standardized format. But we don't anticipate having a meaningful amount of data--I want to emphasize that--for many months.

The office of the Department of Health and Human Services has been extremely helpful in beginning the coordination of this, and we in the hospitals are extremely thankful to have the opportunity to participate in what I think is a critical part of assessing the impact of any decisions that might impact the adequacy of the blood supply.

I'd be happy to take questions if there are any questions.

DR. CAPLAN: Questions? Karen?

MS. LIPTON: Can you let us know or do you have an approximate idea of what proportion of blood being transfused is represented by those 29 hospitals?

DR. TRIULZI: Okay. I did a quick calculation of this because this came up yesterday, and the Department of Health and Human Services office, in their wisdom, included the three centralized transfusion services that are in the country--Pittsburgh, Seattle, and Tampa. Just between those sites alone is in excess of a quarter of a million units. Then there's the other 25 hospitals participating, and one of the tools that we realized we needed is for each site to submit to the office a profile of the size of those sites. But using a rough estimate, I would say it's probably going to be about a million units, or about 10 percent of the blood supply, in that range.

DR. CAPLAN: Rick?

DR. DAVEY: Darrell, just following up on that point, the 25 hospitals, do they represent a range of types of facilities--pediatric facilities, small hospitals, academic centers, et cetera?

DR. TRIULZI: They appear to be all represented, although we don't have the specific data on each of those, which is why we want to have a profile of each of the hospitals participating. But we have cities from as small as Bismarck to New York City represented.

There's also geographic representation from the four regions intentionally, so there's Northeast, South, Midwest, and West Coast. And there was an attempt to include both university and private hospitals.

DR. CAPLAN: Colonel Fitzpatrick?

COL. FITZPATRICK: Mike Fitzpatrick. What's the mix of suppliers? About what percentage of blood is going to come from Red Cross and what from non-Red Cross?

DR. TRIULZI: I don't think we know that yet. We'll have to get that from the profile information.

DR. CAPLAN: Harvey?

DR. KLEIN: Yes, Harvey Klein. I have two questions for you, Darrell. One, I'm sure that the committee, in following up on Rick's question, would like to have some kind of analysis as to whether the sample of hospitals selected in any way represents the country? And perhaps by the next meeting, people with survey expertise could tell us whether this is a representative sample for a sentinel-type study.

And the second question is whether or not the suppliers--we didn't hear any supply data, but hospital data, except for the three centralized transfusion services--whether the suppliers will actually be in the same areas as the sentinel hospitals so we can kind of match collection and distribution usage.

DR. TRIULZI: I'll address both points. The first one, regarding the representativeness of these hospitals, I think is why we need to collect the profile data, and I'm sure the office would be happy to present that to this committee, and the committee could give its view on whether that's representative or not.

Regarding the blood center issues, we currently are not structured or don't have a mechanism to collect the data on inventory levels at the blood center at the same time that we're collecting inventory levels at the hospital, although I have talked with my colleagues at Puget Sound and at the institute in Pittsburgh where, because we're centralized, it's easy access to that data. So that we will collect the data at our own two sites, which does represent about 30 percent of the transfusions for the entire group so that there will be some effort to collect that data, but currently not a plan to collect it on a systematic basis.

DR. CAPLAN: Mike?

DR. BUSCH: Darrell, obviously this is a very rapid attempt to bring this program online, I think in great part because the hope was that it could be in place before the expanded CJD deferrals kicked in so you could actually attempt to measure the impact.

You mentioned that there were sites that were accruing some data. Do you think there will be a capacity to look at the impact over the next few months of the expanded deferral?

DR. TRIULZI: If the deferral were to go in in mid-September, we probably would not have more than four to six weeks of baseline data. I can't answer whether that would be a statistically valid sample. My tendency would be to say no. So that is unfortunate. I think that still the office did a tremendous job just to get people to start reporting, even by August 1st. But I think we probably will not even know the first six-week data in a meaningful manner until long after the CJD deferrals change.

DR. CAPLAN: Ed?

DR. GOMPERTS: This is Ed Gomperts. Could you give a brief overview as to how the data is actually going to be managed?

DR. TRIULZI: There's a Web-based reporting form, and the data will be collected by the Department of Health and Human Services office. They will be creating an aggregated spread sheet for the data. We did talk about how to express the data, and we at least came to the following decisions: one, that it needed to be expressed as aggregate data; that participants were comfortable with expressing it as regional data, breaking it down by Northeast, South, Midwest, West; and there was also a consensus that it would be helpful to present the data as the centralized transfusion community's as one data set versus the single hospitals as another data set.

That is as far as we've gotten at this point, and I would say that we really don't have consensus on whether the endpoint data that we want to discuss, for instance, daily inventory over the amount of units transfused, so we need more discussion on how to analyze the data.

DR. CAPLAN: I have two questions. One, how will the data be available to those who want to see it? In other words, who can access it? And, two, has there been a discussion yet of kind of quality control audit to make sure that the reporting stays on target from the different places?

DR. TRIULZI: We didn't discuss the latter point. The first point on how it would be expressed, Dr. Nightingale did say that this would be available on the Web site for public view. Again, the data is not at a stage at which it's ready to be--any of it is ready to be put on a Web site.

DR. CAPLAN: By the way, the Chair is going to take a few questions out of the audience, if there are any. So if you do want to get in after the committee has asked some questions, that will be an opportunity for you.

Ron?

DR. GILCHER: Darrell, perhaps I missed this, but how are the inventory levels established for the hospitals? As an example, in our region this is a joint effort between the blood center and the hospital, which includes medical oversight, to establish what the inventory level for the hospital should be. And so I'm interested in whether there really is medical input into the establishment of the actual inventory levels.

DR. TRIULZI: The study does not intend to define what the inventory level should be. The institutions are defining their own inventory levels, and we're taking basically a daily picture, a daily snapshot of what the inventory levels in the hospital are on each consecutive day. So that it's really at the discretion of that institution to vary their inventory levels as they see fit.

So we're really not trying to affect the endpoint. We're just taking a picture of it.

DR. CAPLAN: Jane?

DR. PILIAVIN: Jane Piliavin. I'm just wondering, in terms of the sample of hospitals, why an actual representative sample wasn't attempted. I mean, now you're saying after the fact that you're going to go and see whether the group of hospitals you've got appears to be representative. I mean, there are in place some rather sophisticated sampling techniques that could have been employed in order to make sure of that in the first place. And I was wondering why this was not done.

DR. TRIULZI: There's a couple factors that Steve Nightingale brought up that go into that. One was the speed at which we were or he was requested to bring this project together.

The second was the financial limitations of the number of sites that could participate with the amount of money available. The sites participating are receiving less than $5,000 per site to participate in this study. So very small sum of funds for each of these sites.

The third is that I think there was an effort to obtain as representative a sample as could be done on first blush. And it may be representative. We don't know yet until we get the profile data back from each of the sites.

And I think Steve also made it clear that if there was a need to change or alter or add sites, that they would be amenable to that possibility.

CAPT. McMURTRY: I'd like to comment on that for just a minute, if I may, also, at the risk of wading in over my head here. We were not really looking for a representative sample of hospitals. We were looking for sentinel sites, and there's a difference. We were unclear and still are unclear what would constitute a representative sample, if you're talking about a scientifically representative sample, of hospitals. That's not something we were looking for.

The information that we're trying to get from our sites now are more descriptive terms, once again, not in an effort to be able to say, well, we represent hospitals, here's what they are, but just so that we can describe who our hospitals--what our hospitals are. That's been part of the problem that we have seen, is that we haven't had any transparency. We don't know where we're getting our data. And so the request to find out who these sites are is just an attempt to be a little more transparent than we've been.

DR. CAPLAN: Karen?

MS. LIPTON: Karen Lipton. I still am concerned, though, even in using the word "sentinel," that I don't think that we have an appreciation that these are truly sentinel sites. And I guess as a committee member I understand there are financial limitations, but I would hate to see us set policy on bad data. And I think that we should make some effort to ensure that we do move towards having either a representative sample or understand what we mean by sentinel, if it's valid, because, I mean, I do feel as if the study is kind of going ahead, and I think it's good to get all this information. I just don't know if we're going to know what we're looking at when we finally get the data.

DR. CAPLAN: Ron?

DR. GILCHER: Ron Gilcher. As a follow-up to the comment that I made earlier, one of my concerns is that if the inventory level that's established for a particular hospital is invalid--and what I mean by that--and let me give you as an example. In terms of shortages or chronic shortages, what hospitals tend to do--you know this and I know this as well--they will increase what they call their inventory level because they begin to hoard. They're worried that the central supplier doesn't have enough reserve.

Now, I've taken a long time in my own system to try to weed that out, and so what we can have is really an invalid inventory system in the hospital which would make it appear that their needs are not being met, when, in fact, they are. So we have to look very carefully at what they're transfusing as well.

Can you comment on that?

DR. TRIULZI: I think that goes to the point that Dr. Klein made, which is to look at the transfusion services in a vacuum may not give you the whole story.

Now, I think there are a lot of communities that, for instance, in centralized one where we would be able to know whether that's happening. But that would be an argument to get the data on the blood center side to assess whether that was happening.

DR. CAPLAN: Could you tell us a little bit about the supervisory structure for the monitoring? In other words, when we're offering some ideas about making sure that this is going to work as a sentinel and exactly how to keep tabs on the overall supply side, who is it that is in charge, or what's the structure of the contracting--of the blood demand contractors? Who's riding herd on this?

DR. TRIULZI: I think that's a Captain McMurtry question.

DR. CAPLAN: What I mean is when you were there yesterday, to put it into slightly more English, did you come up with a steering committee?

DR. TRIULZI: No.

DR. CAPLAN: No. Okay. That would probably be good.

Mike?

DR. BUSCH: Yes, to me, more important than the representativeness, et cetera, I mean, you've got 10 percent of the recipient pool you think representing 30-some hospitals. It's a pretty good sample. To me, much more important than sort of what it is is the continuity and the trend over time piece of this. And in that context, I'm wondering what's the duration of these contracts? What's the anticipated, you know, long-term stability of the study?

And then this whole issue of the interface between the blood centers and the hospitals, one concern--I hate to raise it, but if blood centers know that a hospital in their region is one of these sites, what's to prevent them from oversupplying that blood center? And how can one keep, you know, stable processes in terms of inventory management both at the transfusion service and the hospitals in place or understand any changes so that trends over time can be understood?

DR. TRIULZI: Those are good points, Mike. First, the contracts allow for currently data collection through December. We did discuss yesterday already submitting budgets for continuing to collect data through the fall of 2002, so we'd have at least a full year of data. So it's anticipated that we will be collecting data through October 2002.

Your question about bias is one that we did discuss, that blood centers may treat a hospital differently knowing that they're in the spotlight, so to speak. And we didn't discuss how to either assess it or prevent it, other than to recognize that that may be a potential limitation in interpreting the data.

COL. FITZPATRICK: Mike Fitzpatrick. So as we--if we concur that these are sentinel areas and that you then discover a sentinel event, was there discussion by the group as to what intervention can occur when the data indicates a sentinel event has occurred and there is a critical supply shortage in a region or somewhere else? Or is that just left up to industry to handle?

DR. TRIULZI: I would say that's the next level of sophistication. We're right now just trying to define the parameters for data collection. How to use the data to impact decisionmaking either at the public health level or even at the regional level is something that we have not addressed yet.

DR. CAPLAN: Make sure we identify ourselves.

DR. SANDLER: My name is Jerry Sandler. I'm the Director of Transfusion Service at Georgetown University Hospital, and I'd just like to offer perhaps a slightly different perspective from the hospitals that are participating.

We are very concerned that certain initiatives may impact on the shortage of blood at the hospital level over the next several months. While I think we all participating in this recognize that the quality of data would be very much improved if we could get the community blood centers to report their data, we see this as a hotline to the Office of the Secretary of Health and Human Services at a critical time. If we run short of blood in October, November, and December, this is a way that we can get to someone and say this is a serious problem. The quality of data may not be as terrific, but the ability to communicate that message is very important to us.

Thank you.

DR. CAPLAN: Keith?

DR. HOOTS: Keith Hoots. Your comment raises a question that I already kind of tried to formulate a little bit in terms of the response to the data. One of the things I guess that we've been concerned about as a committee over time is how to, obviously, work nationally to increase donations. And this could be either a very positive impact if it's used very carefully, but it could also be used as a crisis intervention repetitively and actually work to the detriment of the overall continual donation supply for the country.

I think one of the things we probably ought to make sure that, as we monitor not only the data, but the way that response to the data is--how it impacts overall strategies must be part of this schema. Otherwise, I'm a little concerned that in times of shortage, if we whip the horse repeatedly and then it gets to be habitual, sort of up and down in terms of crisis management instead of a more continuous developmental strategy for enhanced donations, that we may actually--it may actually work to everyone's long-term detriment. So I think we need to keep that in mind as well, particularly in light of the issues that were raised about how representative, particularly in the short term--maybe over time we'll be able to refine this sentinel event for the entire organization of data collecting centers and say, well, yes, it is representative so, therefore, we really can respond rapidly to it.

On the other hand, in the short term, we don't know that, so if we use it--I think we all want more people to donate, but we've got to be very careful how we do it.

DR. CAPLAN: One comment I would like to ask about, too, is it seems to me, in listening over the years to blood banks, they know a lot about their inventory and what they've got and what's around. Part of the way to power up this sentinel reporting, then, is not so much to make them do something they don't already do. It's to standardize the reporting.

So it seems to me that if we could get agreement from these 29 centers and then perhaps make plans to disseminate this style of reporting standardization and so forth, it wouldn't be too hard to recruit lots of other places very fast to come on line and report in the same way. It's not that no one knows what's going on out there. They all know individually. So this seems to me to be a standardization issue, and I'm sure it hasn't come up yet, given the speed with which we're trying to pull this together. But I would urge that the group that is not running this thing yet, the non-existent steering committee, but soon will deliberate about what they can do to encourage standardized reporting on this.

I understand that each hospital has its own needs and habits and customs, but if this just became the state of the art, I suspect going up from 10 percent to a bigger number would be pretty easy.

Jane?

DR. PILIAVIN: I was quite concerned to hear that this is only solid through December. You don't have to be a blood banker or a hospital administrator to know that there are seasonal variations in blood need--I mean in blood usage and in blood availability, particularly the summer problem, the Christmas problem, and so on.

Again, getting back to sampling issues, which as a social scientist is about the only thing I have to contribute here, sampling isn't just in terms--I mean, you have to think about sampling not just in terms of the reporting units, but also in terms of time periods. And unless you have a good sample of both, you don't know what you've got.

CAPT. McMURTRY: I'd like to talk for a minute about the duration of this. I had a conversation with Dr. Chamberland yesterday about how long we were going to be able to monitor to blood supply, and to say that this is firm only through December is true because of budgetary limitations you can only count the eggs that are actually in your basket right now.

As a young man in Texas messing with the Texas Legislature, I learned that there's nothing more permanent than a temporary tax.

I think that this is going to be similar to that. I would hate to be the bureaucrat that says, no, we're not going to give you money to monitor the nation's blood supply any longer.

So, yes, we are only firm through December, but I'm not uncomfortable with that.

DR. McCARTHY: Dr. McCarthy from Indiana. I agree with most everything that's been said, but I just want to say from a large transfusion service in the Midwest, having been in this now for 30 years, we've all seen the biannual shortages. And I think what perplexes many of the recipients is that why is there a shortage in one area and not another. So I think this is the first step in gathering facts. And the facts may not be enough, but I think the facts should be friendly, should be regarded as friendly data, and not be threatening to some people, and that we may get more facts. But I think it will be good to know if there's a shortage in one area and another, and what can be done with the facts is a different echelon of decisionmaking.

But I think this is a good start, and it's been long needed. This thing really started with the American Blood Commission back in the early '70s when the disparate problems about paid donors and labeling and so forth started out. And I think this is just a last aftermath, if you would, of it.

DR. CAPLAN: I hope that that's partly wrong, that it didn't take 25 years to get the thing rolling. But I suspect you may be right.

The other question I have--and I didn't hear much discussion of this when we were sort of getting ready to launch this effort--that the Chair made clear is I'm very happy that we're going to have this. I think we've got to make sure that it's done right and well, but moving on this at a time when there's a lot of discussion about adequacy of the blood supply, what the impact is--not only of CJD but, you know, we've been in this group to listen to leuko-depletion, NAT testing, and many, many other safety measures. So we're always fishing to try and find out what the impact on supply--it would be wonderful to have--it will be wonderful to have some standardized information on that.

That said, did it come up as to what other countries were doing in terms of monitoring their blood supply? I don't know if Mary's encountered that either, Canada or U.K. Is there any other--do you know, Mike? I'm just curious what the--has anybody standardized out to keep an eye on overall--I mean, in a sentinel-type fashion?

DR. TRIULZI: We didn't discuss it, and I'm not aware of it being done.

DR. CAPLAN: Do you know, Jerry?

DR. SANDLER: I directed a blood bank in the State of Israel for six years. Every morning we made an inventory report, and we sent it to the central one blood collector in the country, and the concept was that the blood was given by the people of the country. It was on consignment to the hospital. And for reasons that we never knew, blood would be moved in and out of our hospital inventory which we had established to other locations. And, obviously, that's a goal for your committee to achieve that in the United States.

DR. BUSCH: There's a number of countries that have introduced hemo-vigilant systems, but to my knowledge, they're mostly focused on risk assessment. But just in the last day, I saw--I think it was distributed by the Web, but Ireland is in the process of reversing their British deferral policy because of unavailable blood supplies for patients right now.

COL. FITZPATRICK: We deal with most of the NATO member countries in Europe, and because most other countries have a national blood system as opposed to a civil blood system, they have some ability to assess their inventory availability depending on primarily the size of the country. Germany is regional, so you had to go to each region to find out an inventory. Belgium is national, and you could go to one point to find out an inventory. France was regional. So it varied from country to country.

But because of the nationalization of the system, there were single points of contact or small numbers of points of contacts we could go to within those countries to determine if they have inventory that could assist us during a war.

DR. CAPLAN: So the steps here may be--I don't know to what extent DOD or Armed Services is free to share its own inventory templates. But either there or in other situations like Ireland or Israel, it would be useful, again, if we're talking standardization and keeping an eye on things, to at least draw upon other experiences and maybe push a little bit toward some international standardization as well.

John?

DR. PENNER: I'd like to just return very quickly to what Mike Busch and Ron Gilcher brought up and, that is, the act of examining the data can change the data. It's the Schrediger-Katt (ph) association.

Is there any attempt to do spot checks with, say, other centers that are collecting blood to assure that the data you're getting is not being manipulated by asking for the data?

DR. NIGHTINGALE: I think it might be easier for me to give a fuller answer after I give my delayed presentation. But in the--immediately, it is clear that sentinels are just that. Sentinels, when they're identified--if you have an outpost somewhere that you're guarding a frontier, you anticipate that the attacking army is not going to come straight at the sentinel.

We've tried to correct for that in two ways. I think the first way is by geographic distribution, and the second way is by making this an evolving process. You've got to start somewhere. We're starting with a six-month contract where we value--we're re-evaluating it right now as we're up and running. But I think that what we're--the basic question we ask is: Is something going wrong in our sentinel sites? Yes or no. Do we have reports that something is going wrong outside of our sentinel sites? Yes and no. Depending on the answer to those two questions, we go in one of four different ways.

If nobody is having problems, we go home. If somebody outside the sentinel sites are having problems, we ask why outside and not inside. There may be a simple answer. There may not be. If both are having--if it's the reverse, well, we've got an early-warning system. And if both are having problems, we ask ourselves why didn't we pick this up earlier?

DR. PENNER: I'm just reflecting on some studies we did on platelet utilization, and our control group, it seemed, once they realized they were a control group, suddenly started improving on their use of platelet in the hospitals. So that sort of game playing is obviously part of what we do. So sometimes what we were able to do is just spot check a few other institutions and find maybe the data we were getting was not really true.

DR. NIGHTINGALE: Well, I believe that--well, not believe. I recall Dr. Linden saying yesterday that New York State planned some activities of its own. That would be an obvious check against us. I haven't talked in any detail with Dr. Linden, but that statement was made yesterday, and we've had conversations with New York State about intensifying surveillance in that state at this time.

DR. CAPLAN: All right. Let me thank you for that presentation.

Do you need a break in order to get to your talk, or are you set up to go?

DR. NIGHTINGALE: Not at all.

DR. CAPLAN: All right. Then we'll heard from--

DR. NIGHTINGALE: I guess the question is: Does anybody else need a break?

DR. CAPLAN: Not yet. I know.

[Laughter.]

DR. CAPLAN: I can tell.

x The next speaker is Stephen Nightingale, who is going to talk to us about the current state of monitoring and perhaps fill in a few of the details from yesterday's meeting. You know, I was up on the Web today, and I saw this article in the Baltimore Sun. And some of you may have seen some other press coverage on this move toward monitoring the blood supply. I just want to, again, make clear we're going to probably spend some time haggling and niggling a little bit about how to do this.

The general push to do it is something that I think the committee is absolutely supportive of and wants to see all organizations cooperate with. It's just vital if we're going to be in situations where we might have steps taken that impact the blood supply, that we have some ability to know what's going on. So at some point, it may be wise for us to simply pass a resolution or motion to that effect. I'm just suggesting that, but that may be something you want to say this morning.

DR. NIGHTINGALE: Thank you very much, and I apologize for the lateness of my arrival.

Dr. Penner?

DR. PENNER: Is that on?

DR. NIGHTINGALE: Is the microphone on? Can everyone hear me in this small, and not uncrowded, room? Yes, I think so.

Where I would like to start is with a brief review of how we got to where we are today. And how we did so really started out in April of 1998 and, before that, in December of 1997. In December of 1997, we became aware, a couple of months after the fact, I might add, that there was a very acute shortage of plasma derivatives in the country.

The Advisory Committee held its third meeting in April of 1998 on this topic and on what the government might do to help alleviate it and, as importantly, to prevent its recurrence in the future. One of the recommendations that came out of that meeting, and one of the responses that came from it as well, was a monitoring of the supply of plasma derivatives in the United States. It was instituted by the industry group that was then known as the IPPIA and now known as the Plasma Proteins Therapeutic Association. I hope that I got that correct.

The monitoring of the supply began in October of that year and was really fully functional in January of 1999, and it had several benefits, but I think as great a benefit as any was the degree of confidence that it provided to the user community. There are many representatives in the community, and they could acknowledge or question that, but I think most would acknowledge that.

It also provided us with some useful information. What we got from that, and continue to get on a monthly, and in fact now every fortnightly basis, is both a measure of the inventory that is on the manufacturers' shelves at a specified time and the distribution of the product over that month. So we get a ratio of inventory on top to distribution on the bottom. The higher the ratio, particularly if it's greater than one, the better, at least subjectively, we feel things are because that means you have, at least, a 1-month supply of product. For products in short supply, that ratio has been well below one, particularly for recombinant factor 8 in recent months.

The second event that got us to where we are today concerns monitoring of the blood supply itself, as opposed to the plasma derivatives, and that came to our attention in February of 1999, when the National Data Blood Resource Center published a summary of its biannual survey that included a projection the supply would fall below demand for blood by the year 2000. We held a meeting on that, had made a variety of recommendations.

And after that meeting, the surgeon general, who is then also the assistant secretary of Health, Dr. Satcher, convened an intergovernment panel that Dr. Epstein led to see how we could go beyond the recommendation of the Advisory Committee. One of the recommendations that came out of that was the monitoring of the blood supply. And, in fact, in October of that year, a contract was let to the National Blood Data Resource Center to monitor the supply from a representative sample of blood establishments throughout the United States, and that contract, in fact, continues to this day as well. We get supply, we get inventory and we get distribution from that.

The review of the monitoring efforts had picked up steam really in January of 2001. Responsibility for that activity was transferred to the Office of Public Health and Science, which is the Office of the Assistant Secretary of Health, for whom I work. And in April, we had a full review of that, on April 20th, here at the Advisory Committee.

The basic outcome of the review was that we found both provided very useful information, but there are issues for both about the timeliness of the report. We get the information somewhere between 4 to 8 weeks after the events that are being counted occurs. And there is a question, if you get monthly or even bimonthly data, what constitutes a trend. So timeliness was an issue.

I think the second, and perhaps most important issue, was that both for the monitoring of the blood and plasma supplies, the question has repeatedly arisen of the relationship between supply of blood or plasma products and demand for those products.

And, finally, there is also an issue that has been raised about the transparency of the review. To the extent that these are designed to provide information to the public, the more transparent, the more visible the process, the more rapid the distribution of the results, the more effective they will serve that purpose. And also for the people who are using the information, the more easily they can verify the sources on which the information is based, the more confidence the government, for example, or any other interested party will have in using the data

A reason why we started with supply, rather than demand, was very simple. Supply is easier to measure than demand. We've had many conferences about that, both public and private. I might add that Dr. Haas, a member of our committee, has been extremely helpful, certainly to me and I think to many others, because of his expertise in this area. But the original way that we measured demand was the ratio of inventory to release at the producer level. And for the reasons that I just mentioned, we felt that that was suboptimal. We didn't necessarily have a better idea.

The second way of measuring demand is the classical economics way of measuring the price of the product. For a variety of reasons that Dr. Haas reviewed at the April Advisory Committee meeting, price is not the best way to do that. You can have a product that is in demand, but still be cheap, depending on market factors, and you can have the converse as well.

The second is inventory--the inventory release at the distributor level. This is not a simple market. The distributors play an important role, and one of the pivotal thoughts that we've had is that the transfusion services in the large hospitals, in the sentinels, if you will, might, in fact, be functioning as distributors on behalf of individual patients, and that's pretty much where we are.

The inventory release at the end-user level, of course, is the optimal one, but there are a lot of problems with measuring that, not the least of which is the privacy of the individual. Others I have a question here. We, by no means, exhausted the topic, but at the moment this is where we are intellectually at the distributor level.

And how should inventory be measured is another question that is very much on the table right now. In the very preliminary data that I'm going to show you, we're just measuring inventory over daily use. For example, if one blood centers that supplies, for example, all of greater Seattle has--let's give a number--500 units, and one hospital supplies, say, half of Bismarck, North Dakota, has 50 units, you've got to factor the difference in the size of the institutions.

There is, for those of you who follow or perhaps lead the business section of the newspapers, you'd be familiar with trailing averages. This is a way of smoothing out daily fluctuations, but there's also a way of filtering out information that you may not want filtered out. So we are still in the process of trying to figure out, as we go along, whether or not it's appropriate to use trailing averages.

Very briefly, what it would appear use of a trailing average does in the data that we've collected so far, is to push the curve a couple of days or 3 days to the right, depending on how long the trail is. If there's a blip, the trailing average filters the blip out, so it takes a couple of days before the curve shifts. So, to some extent, the use of a trailing average negates or at least counteracts the daily collection of data. This is an ongoing topic that we'll be working on.

The third is a definition of a trend. In this business, we're also going to have to learn this as we go on. Looking at data over a year, it's clear, for example, from the NBDRC data, that in the past couple of years, both transfusions and blood collected have gone up. There are trends, if the curve is significant, if the confidence interval at Point A and Point B do not overlap you have a trend.

Of course, when you're looking at data every day, you're sampling an infinite number of times, and conventional statistics are not particularly helpful. Bayesian statistics are what you make of them. So we will need a definition of a trend, which we don't currently have. Certainly, there are weekly, as well as seasonal cycles, in the data that we need to factor up, but since we haven't looked at the data yet, we're going to have to find that out as we go along.

What happens when you have a transient effect, for example, in the plasma business, when a manufacturer shuts a line down for preventive maintenance, and what happens when you have a nonlinear event, for example, when there's a change in the number of manufacturers or a major change in policy, are things that we also need to learn as we refine our usage--plus, an adequate inventory.

At the moment, then, we're looking at a variety of ways of measuring the inventory at the distributor level. And for our benchmarks, initially, we are asking the question, what is the inventory at a time when there's consensus that there is an adequate supply at least nationally. Much of what went on yesterday, and, gosh, I hope I'm not going to repeat more than a couple of words Dr. Triulzi gave to you, is we got a number of very, very valuable suggestions for how to define an event that would make us think that the inventory was inadequate to meet demand, and we're going to pursue that very aggressively over the next month.

There are questions, of course, whether or not an event is a random or a sentinel event. If we had guidelines, well, hey, the stock would be shorter. What's an adequate sample? This was something that was raised several times by several speakers yesterday. When you get started with a sentinel system, I believe Dr. Chamberland said you, when you start to use the example of monitoring the AIDS epidemic in the early days in New York City, I think there's two things that you do is, one, you start out with the best information available, and number two, with experience and with as much of the scientific method as you possibly can, you try to make it as much better as you can, as quickly as you can. I think our current intent is to bootstrap, rather than to restart the calendar every 6 months. I don't think we really have time to do that.

To the extent that a sample is representative of an entire population, well, that's very difficult, unless you know exactly what the population that you want to sample is, and this is the long answer to Dr. Penner's question. I mean, clearly, what we want to do is to put our sensors at the most sensitive parts of the system. By putting the sensor there, at least in the part of the government that I work for, you attract attention to the sensor immediately. In other parts of the government, apparently you attract attention to it sooner or later.

This is a problem that we're going to have to work with. I think that the way to deal with it is to maximize public information rather than try to put it under a blanket and hope nobody finds it for 90 days. I do not think that's the right way to deal with a sentinel system.

There being 5,000 acute hospitals in the United States, can an N of 30, which is our budgetary limit right now, truly be reflective of the population? And the answer is probably not. The next question is, if it isn't perfect, should you do it anyway? And the answer is, yes, at least that's our answer right now.

I mentioned about bias from disclosure of participation. We're going to learn some things from this, at least if we conduct the study correctly.

John, would you like to make any further comments?

DR. PENNER: I think you have to start someplace.

DR. NIGHTINGALE: I'll tell you where we're going to start.

DR. PENNER: And then you can modify it.

DR. NIGHTINGALE: That segues me into someplace. Where did we start? These are the contracts--let me say that while I'm not sure that all of the contracts have been signed, we certainly have handshakes, and we have data coming in from 25 of the 29 sites so far.

In the Northeast, we have two hospitals in Boston, St. Elizabeth's and Brigham and Women's--hey, we're grateful for everybody--the Institute of Transfusion Medicine in Pittsburgh, in--oh, my God, I did these slides this morning, and there's only two of the four here, and I apologize--Mt. Sinai and Columbian Presbyterian are in Manhattan. There is more to New York than Manhattan, and Maimonides Hospital in Brooklyn and Jamaica Hospital are also participants, and my apologies to them. I knew there was something wrong with this slide. In Washington, there are two, the Georgetown University Hospital and the Washington Hospital Center.

One of the initial thoughts, as we started this process, was to have two hospitals in a particular geographic region, and it was not particularly important to us which they were, but we didn't want carbon copies of each other. If we didn't want sort of the University of North Manhattan and the University of Midtown Manhattan were not what we were looking for, and we do have Sinai and Columbian Presbyterian there, but I don't think they would fit that description too precisely.

In the South, we have Emory and Grady. I think Emory and Grady pretty much mirror what the idea was initially. And as you can see as the distribution changes, you'll see how our distribution matured, as we started making phone calls and actually listening to the people on the other end of the phone.

We have Mt. Sinai Hospital in Miami, and we have the Florida Blood Services from Tampa-St. Pete. You can't sample every small town, and Mobile is, by no means, a small town, but one of the concerns was are you just going for the big cities, and the answer is, yes, predominantly, because that, to paraphrase Willie Sutton, is where the money is, but we tried not to go just there. The Ochsner Clinic, Baylor, and Parkland in Dallas are the two other contractors that we have in the South.

In the Midwest, Indiana. Leo, is that the correct hospital?

DR. McCARTHY: It is.

DR. NIGHTINGALE: Thank you.

Northwestern, University of Illinois, Chicago Circle, the University of Iowa, the University Medical Center in Minneapolis, and St. Alexias Hospital in Bismarck. We will make allowances for the weather in North Dakota in the winter when we analyze the data, but it is not without interest, by the way. There was a reason for Lackington, North Dakota, to at least identify, albeit as a sentinel site, what happens in the part of the country when it gets rough in the winter.

And in the West, we have the Puget Blood Center in Seattle. Puget, Pittsburgh, and Tampa-St. Pete, of course, are three communitywide networks for blood banks. And Denver General is now Denver Medical Center and the University of Colorado. We have Cedar-Sinai, and Harbor, and University of Arizona, Tucson. Those complete the list of our 29 contractors.

Preliminary data. Presented this yesterday in what really is a public session. There are caveats here. Number one, we only have, so far, in the data bank, 25 of the 29 sites. This is not final data. We only have 537 total days of observation. Yes, that's a lot. This is a big system. The database has been unscrubbed. A technical term is have we gone back and verified every entry for accuracy? Have we done our best? Yes. Have we scanned for obvious errors? Yes. Would this be ready to submit to FDA for a new drug application? No.

And for those reasons, the only slant that I have on these slides, if you look at the placements of the Bermuda Triangle, and they say, "Not for use in navigation," this is not for use in navigation, but it is an indication of where we are. And I am also bringing this out so that we can get feedback from you. If you don't know where we are right now, you can't help us get better, and that's the reason for going public at this point.

This is total inventory of daily distribution of red cells. What this is is the average inventory over the amount that went out, that was either transfused or it was exported to another site. For example, if you're in Bismarck, and you have blood, and Fargo doesn't because it snowed in Fargo, and you send some blood to Fargo, that's blood that went out the door. And it also includes any blood that might have outdated, although there's not a lot of blood being outdated these days, and I think that that is an observation that we need to follow up on.

If we're flush with blood, you'd expect a percentage of it to be outdated. We really have to think about what that percentage might be, but right now there is not a lot of blood that is being outdated. And there's a modest amount, it would appear, that the exporting is in line with traditional numbers, maybe about 1 out of 7, 1 out of 6 is being exported, for the median. This is not an average, this is a median. Half are below and half are above. I'm giving this number because this data is unscrubbed.

Yes, Jane?

DR. PILIAVIN: What are the units here? I mean, 7.4 what?

DR. NIGHTINGALE: Actually, these are pure numbers, but what it is is this is the number of bags on the shelf divided by the number of bags that were released by the system in 24 hours. So what you are looking at could be construed as day's worth of inventory. Just like, if you are selling cars, and you have 900 cars on the lot, and you sell 100 cars a day, you have 9 days' worth of inventory. That is a way of looking at these numbers, and I think--

DR. PILIAVIN: So it's basically days.

DR. NIGHTINGALE: Yes.

DR. PILIAVIN: Thanks.

DR. NIGHTINGALE: That's right, it's basically days.

I'm not going to show you a complete analysis because I don't have comfort in the data sufficient for a complete analysis. I'm showing you where we are right now. If you look at two of the segments, the Northeast is a little bit above the median, the South is a little bit below the median, but we're tracking in the same area. And if you just look at the data that was collected for really the first week where we have 25 out of 29 reports in the computer right now, the median is very close to the overall median for the data. So I think we're up and running.

This is a number that if you just look at the total inventory over the amount transfused, you'll see you actually have a 9-day inventory because one of your questions is don't let any blood outdate, and the second is don't export blood. This will give you a measure of the slack in the system, but it remains for further observation to see how good this measure of slack will be. But, certainly, if anybody has any comments about this measure, I would be more than happy to receive them.

DR. DAVEY: Steve?

DR. NIGHTINGALE: Rick?

DR. DAVEY: One thing, obviously, I think you are aware of that the inventories for different blood groups will vary considerably.

DR. NIGHTINGALE: Dr. Davey has anticipated my next slide. We had dinner together, and I guess I discussed that because here we have for A-positive--

Don't worry. I'm not going to go over all of them, I'm going to go over most of them, and I'm sure the blood bankers were interested in this.

Again, while these probably correlate with expectations, this is still preliminary data. For all observations, as opposed to 7.4, there's a little bit more A-positive in the system than there is for O-positive. Is that a meaningful difference? I do not believe so on the basis of the data that we have right now. But, again, this is for discussion and for input from the public about how we should anticipate it.

And, again, I've compared the other areas that we're tracking pretty much the same. The median for the past week is a little bit higher. Is this statistically significant? My expectation is no, but my warning is that this is not data on which you want to put statistical test. This is an exploratory data analysis that I'm presenting to you.

So a little bit over for A-positive. B-positive, there's a lot of B-positive in the system. Is it statistically significant? I don't think so, but it tracks about as well. AB-positive is the next one. There's not a lot of AB-positive in the system relative to the others, but what is "not a lot of"? There's reason: AB-positive is blood that outdates. And overall you can see for the last weeks it pretty much tracks this.

The two items that you want to look at--and, again, I'm cautioning you not to look at it too deeply--are O-positive, where the amount of O-pos in the system would appear to be right about the same level as the mean, and there's not a whole lot of variation in there. And of such interest at the moment is O-negative inventory, which is actually a little bit high.

Now, one more time, this is a snapshot of preliminary data. This is being presented so that we can gain input from you, the committee, from you, the public, and from those who will have access to this presentation later. We want suggestions for how we can do this better. We do not feel that we have a template which we can simply plug in and monitor the blood supply in a manner that would suit everybody's purposes.

The platelets are a separate issue. Platelets, for those of us who are not blood bankers by training, which includes myself, outdate a whole lot faster than red cells, so there'd be a lot more turnover. For the random platelets for all else, it looks like we have about a 1.4-days' supply of platelets. Again, what that means depends on the experience of the user. The platelet market is much more active than the blood market. For the apheresis single-donor platelets, we have a number that is pretty much in the same line.

What we asked on our samples is please provide any comments on any actions that you took in response to finding that supply was inadequate to meet demand. Most of the comments so far about platelets, and I think the preliminary analysis would appear that this would be perhaps more closer-to-normal business practice than it is for blood because of the short half-life of the platelet.

I'm up to the last slide now, and this is the summary of the preliminary data that we have. Where we are with the monitoring system is we have a single-point estimate. We are just about to get daily averages. We will consult with our consultants, we will consult with the committee about how to analyze and to improve the collection of this data. We have a second round of contracts for the data that will be coming up for the first year and that I anticipate, after the second round, after we found out how much we've learned from a 1-year analysis of the data, we will be prepared to put out a formal contract pending approval of the Secretary, but I think that the support for continuing this process is pretty strong right now, and I don't expect that to change.

I am done. Ms. Lipton?

MS. LIPTON: Just a quick question. When you're doing inventory, and I think you mentioned this, but I couldn't quite recall, you are including directed autologous, and then what about special inventories, you know, for a red-cell antigen specific. Does this include everything? Are you tracking those separately?

DR. NIGHTINGALE: We had an extensive discussion of that yesterday afternoon. Unfortunately, I had to be out of the room when that discussion took place.

Ms. Wannamaker? Ginny? Ginny, would you like to comment on that question?

MS. WANNAMAKER: These were all issues that we knew that we had to discuss, and we wanted to discuss them together, and we did that yesterday. This preliminary data that Steve has has--it's not distinct as to whether or not there is--there is autologous and there is directed in these numbers. We did discuss yesterday how we're going to handle that so that our numbers may not reflect those later on. But this particular data has everything that people were collecting.

We had initially asked people to include those in. Some people, I believe, had done it. I'm not sure that all were. But we have had discussions about that, and we will be looking at this a little bit differently. But for those numbers, yes.

DR. CAPLAN: Rick?

DR. DAVEY: Steve, just to revisit a topic that the committee has already discussed, to some extent, this was the first time I think we had a listing of the hospitals that are participating, and I understand the necessity to have big hospitals. As you say, that's where the money is, and they need to be very clearly represented. But I was struck that there were no, for instance, military hospitals. There weren't very many, maybe except one, kind of community hospitals, like in our area, Sibley or Suburban, included. I know it's going to be tweaked, and you're thinking about it, but could you comment on that a little bit?

DR. NIGHTINGALE: Yes. I think I'd reiterate the comment that I made before I got up to the podium, is that it is not practical for us to put a monitoring in each of the 5,000 hospitals. I think that we started with a plan that was really based on geographic distribution, duplication within a metropolitan area, and a budget that was $113,000 for sites, and we busted that. We went up to--I had to stop when I hit $155,000. That was how it was taken.

So the plan is to see how this group performs. I think that the best way we'll see how the group performs is we will either receive or not receive reports, comments or, indeed, criticisms, just the point of having this talk, that you're not shining your light where the problem is. If we find that our group of sentinel hospitals is flush, say, over Thanksgiving, over Christmas, during a traditionally low period, and we're receiving reports from other institutions that, say, we're not flush, then we will revise our plans accordingly.

Short answer to your question is we started where we thought the money was most likely to be, and like any good investigator of money, we are prepared to react and respond to events as they reach our attention.

DR. PENNER: Steve, I wonder, as this data emerges and becomes public, if some hospitals may not use it as a standard, looking at the data and saying, "Well, we only have 5 days' worth. We better go out 7 days' worth. And those hospitals that have 10 or 12 might even decline in their amounts. But looking at this kind of sentinel group, they think maybe they should be in line with it. And I'm wondering whether that impact will really be good or bad.

DR. NIGHTINGALE: Once we have the data scrubbed with an N of 29, I think that we can give confidence intervals. And the question is I don't know whether the distributions will be normally or plus on--or uniformly distributed across an area, and I don't know what the variance will be.

What I do know it will have is a measure of what inventories were at a time when shortage events were reported. Remember, a shortage event at the moment is any action taken in response to discovery that supply was inadequate to meet demand.

We talked yesterday about making a typology, about classifying those different responses, and several of the members presented specific events that should be used, delaying elective surgery, for example. We will have a great deal of data, I think, within 90 days, I would hope, by which we could improve the reporting system. Honestly, two ways of doing this are to go into a dark room and figure it all out by yourselves or come in the bright lights and request comments like that, and we have chosen Plan B.

DR. CAPLAN: I've got Karen, Harvey, Jay.

MS. LIPTON: Steve, we've talked a number of times about some of the hospitals that aren't in the reporting and using them to see if there are some other problems. How are you going to get that set up. Will that be through the website, encouraging people to, if you're not within this range, and you know, if it's showing that these hospitals aren't having a problem, but you are, how are you going to--how is that going to work?

DR. NIGHTINGALE: Yet another hanging curve. I am, using forums such as this to publicize the existence of this system and to encourage individuals who wish to report, to do so in whatever format they like. For the record, I don't have it up here, but I could type it in, my address is 200 Independence Avenue, Southwest, Room 736, Washington, D.C. 20201. My e-mail is posted on our website, which is www.dhhs.gov/Blood Safety. And the direct line to my office is 202-690-5558. Thank you.

And I mean that very seriously. I think one of the ways we're going to find out about the shortage is simply going to be the number of events that are reported. If nobody is calling up, we're going to make the assumption that the problem is less than it would be if we were getting a lot of telephone calls.

One other thing that I have not mentioned here, of course, is what happens if you find that things are tight in North Bismarck, but they are not tight, say, in South Bismarck. We have had discussions with America's blood centers. And I think Ms. Fredrick and I raised this topic the last time we met, is that there is data on supply that the blood establishments do collect. And if we found an imbalance, we would certainly want to go to the suppliers to see if there was a technical fix. We anticipate a great amount of cooperation from those, as the need for it arises. So we haven't given up on measuring supply. What we're trying to do is complement the measure of supply by a measure of demand.

DR. KLEIN: Yes, Steve, I'd like to make a comment. I noticed a number of my colleagues writing down numbers. These really are numbers. These are not data. There was no common definition of even what an inventory is, and I know that some inventories, that people call inventories, weren't included in the so-called inventory collections. Now that's going to be straightened out. It's going to be tweaked, but I think it's important not to try to think that you know something about the O-negative supply in the Northeast.

The second point I think is related to that, and that is that let's take the Northeast, where one of the sentinel hospitals is Brigham and Women's. It's one of the few hospitals, one of the 8 percent or so, that collects its own blood. And knowing something about their supply is important, but really tells you nothing about the regional Northeast and the New England Regional Red Cross Center, which supplies virtually all of the rest of the blood north of New York City.

So I think we can be misled by these numbers now. I'm sure they'll get a lot better. But if you've written them down, you may not go out of this room knowing any more about the status of the blood supply than you did when you came in.

DR. NIGHTINGALE: Yes, and let me make one additional comment. The existence of a contract between the government and an individual is a matter of public record or at least FOIA-able, perhaps not all, but certainly all Health and Human Services' contracts are. But we do not plan to report data on an individual center. We never did. There's a variety of descriptions for it, one of them is trade secret, the other is plain old-fashioned privacy, and the other is that's not what we're interested in. The government does not perceive it to be the business of the government to know, on a daily basis, what B and W's blood inventory is.

What we have done is chosen 10 hospitals, and we've oversampled New York City, to get a first-pass estimate of what's going on in the Northeast. We have alternate sources for what's going on in the Northeast from the blood establishments, the producers of blood. And we will have a third source from our contacts, formal and informal, the telephone, about what's going on in the parts that we haven't sampled.

As I said earlier, if the State of New York does its own sampling, we will have a fourth. I wouldn't be surprised if other regions might do something of the same. Again, Harvey's point bears reemphasis. These are preliminary data. I think that my point also bears reemphasis, and that point is that there are two ways to get off the ground, and one is to do it by yourself, and the other is to go public and ask for help, and we have chosen B.

DR. CAPLAN: Jay, and then we'll go to Jane.

DR. EPSTEIN: Steve, I apologize I came a little late.

Have you attempted also to capture percent of inventory outdating on each day? I understand that outdating is a phenomenon that's multiply determined, but on the other hand, in the end, it might prove to be a useful indicator. If having a larger daily inventory turns out to correlate with a larger outdate, it would be one of way surmising you're collecting too much, for example.

DR. NIGHTINGALE: That information is being collected, I think. Until I have a scrubbed set, I'm not prepared to do any analysis of that as an indicator, but I am very hopeful that that will prove to be a useful one.

DR. CAPLAN: Jane?

DR. PILIAVIN: I want to second and third the comment about this not being data that people should take away and quote as gospel. There is a saying among those of us who work in the data business is that the plural of anecdote is not data. But what I originally wanted to ask was whether you have, indeed, observed in this set of anecdotes any of these shortage events?

DR. NIGHTINGALE: Yes, there are comments. I am not in a position to go further than that. But I think the truth is this is not anecdote, this is not data, this is a progress report. This is a seminar writ large. For people who were starting a new investigation, the ones who survive usually go not only to the people right around the corner and ask for advice, but the people who might have some information that they would not otherwise have access to unless they went public with their concerns.

I think one of the things that we have observed that I think is a preliminary observation, and I think it's an observation rather than an anecdote, is that the platelet business is different from the blood business. What we may end up observing is that the AB-positive business is different from the A business. And if anyone has comments about that, particularly if anybody has suggestions for an exploratory data analysis, we would be most grateful for them.

DR. KUHN: Dana Kuhn. Dr. Nightingale, prior to your presentation, this committee had some concerns, and I just wanted to follow up on some of those concerns and maybe you can address them. They were concerned that these 29 sentinel entities, there was a need for a standardized reporting, and also there was a need for a sampling throughout the seasonal time periods. Also, there was a concern of conducting spot checks to avoid manipulation of data or to avoid, as one person put it, hording or probably speculative obtaining of product.

Will this entity be addressing this or has it already addressed this?

DR. NIGHTINGALE: Yes. I think we've addressed it. You usually do questions in reverse order and hope that you remember number one. I do remember number three, which is the validity of the data.

We have asked each of our contractors to prepare a standard operating procedure for this, and include it in their standard operating procedures. And I think that the blood banks, as just about everybody in the room, I believe, will agree, is a very heavily-regulated business, and you regulate adherence to standard operating procedures pretty carefully. So I believe that that was the most efficient way to meet our fiduciary obligation to ascertain the accuracy of the data. I don't know of a better way, honestly.

Now, having done number three, I've got to ask you what one and two were again.

DR. KUHN: The need for standardized reporting and then the sampling--

DR. NIGHTINGALE: Okay. Stop there. It's been a wild week, and it's almost over.

The need for standardized--the reason we had a contractor's conference yesterday, so we could get everybody together, after they'd done it for a while, and said, "Okay. How are we going to standardize this?" If you have one of these conferences before you get the thing going, you're likely to need another one, and we scheduled a teleconference for September 25th, and I anticipate we'll be doing them on a regular basis.

That was number two, and I'm sorry.

DR. KUHN: The sampling throughout the seasonal time periods.

DR. NIGHTINGALE: I think, originally, we cut contracts for 6 months so we could get off the ground, but not be constrained if we found that we were going the wrong way or, for that matter, if a lot of other people found we were going the wrong way. The expectation right now is that we will continue pretty much the way we're going, depending on budget, honestly, among other things, for a 12-month period.

The thing I'm not certain of yet is whether or not we'll have full confidence that we have a full data set from October 1, '01, through September 30, '02. I would like to have one full month of lead-in before I started really collecting data for the record. That's pretty much been the experience with the plasma and with the blood supplies. They started in October, and looking back on them a year later, they were really fine by January of the following year, but a 90-day roll-in is what I need.

COL. FITZPATRICK: Steve, Mike Fitzpatrick. Just to kind of respond to Dr. Davey's, part of his question, I think we discussed on either teleconferences or previously at this meeting that since DOD collects as much as it transfuses, that we wouldn't be representative of what's happening nationally, and so we weren't included in the model.

Sort of going along with what Karen asked, though, if the committee felt it desirable for us to mirror and report similar data, as you are reporting, I have the means to do that, but it would help if I were asked to.

[Laughter.]

DR. NIGHTINGALE: I understand the comment. I think I had similar reticence. I will just say that the conversations I've had with the other suppliers, and with Red Cross and Celso was particularly helpful yesterday at the contractors' conference. I hope to develop those.

I don't want to scare anybody off.

COL. FITZPATRICK: Well, I wasn't implying that we felt left out. What I was meaning was that in order to obtain the resources to mirror what you are doing and provide the committee that data, if the committee asked, I could probably get the resources to do it.

DR. NIGHTINGALE: Understood. Of all of the branches of government, yours is certainly the one most likely to get the resources it needs.

DR. McCARTHY: Leo McCarthy. Just a couple comments. Again, to reinforce what Dr. Klein said. I think the contractors, many of us, we hadn't seen this data either and we haven't manipulated it, so this was new to us. This data was handled by the office, Steve's office.

One of the things that I think one needs to be very cautious when we leave this room and return to our areas, is not to think that there's a week's supply of blood on the shelves, because, as we might find out--and I'm not trying to be Cassandra-like--that may not be proven to be true over this next group of holidays.

Lastly, is just a question, which I guess we should have dealt with yesterday, that just follows up on Karen's comments and I believe the Colonel's. Steve, how would one respond or your office respond to a hospital who feels left out, who wants to participate n a program, provide the data on an every-day basis, and would not expect to be paid for that? I don't know that we discussed that contingency, but I can imagine, as we just heard, some of the people, even outside the government, may want to do that in some part of fly-over America, in Omaha or something like that. I don't know that we discussed that.

DR. NIGHTINGALE: Actually, your next-door, St. Vincent's, called up a couple of days ago and asked that question. And I believe that here is--I'm going to throw the question back out. One of the reasons why we looked at Indiana University as well, it's a large bank. It also is a major transplant center, and one of the inclusion issues is--that we would like to look at--and we'd go back to Dr. McCarthy to look at--is what's inventory right in the middle of transplant season, which is usually Christmas, which is usually when things go tight.

We really don't know the answer. We have a couple to that. I think that St. Vincent's--and correct me if I'm wrong, Leo--does a lot of heart transplants?

DR. McCARTHY: Open-heart surgery.

DR. NIGHTINGALE: They're a heart center. And this is not to say that that's all one hospital does. This is information that I would like to obtain. I think it's something that we could--we should all think about--is: data that comes voluntarily, the same as data that comes on contract? I haven't got there yet. I think perhaps by September 25th we will get there. Where I have gotten is that there's a finite amount of money in the pot, and some of that money we do anticipate, at least keeping it available for monitoring the plasma supply, which is a very, very different business, the demand for plasma.

Dr. Chamberland.

DR. CHAMBERLAND: While I understand people's concerns about trying to get additional data, more sites enrolled, trying to address this elusive goal of representativeness, I think having participated in yesterday's meeting, we have a big job ahead of us just to put in place a standard operating procedure, protocol, case definitions, criteria, data collection form, have that disseminated, and as uniformly as possible be followed by the 29 participating sites, and people shouldn't be discouraged by that or think that that's unusual because when you get a multi-center study up and running off the ground, it takes a lot, a lot of hard work and time to do that in a careful way so that you have confidence in the data that you are collecting. And so as Steve and others have emphasized, and Dr. Truelzi, as a participant, this is at a very preliminary stage, and if data collection were to be expanded to other sites, particularly, as was brought up by Dr. McCarthy's sites that might have an interest in volunteering their data, I think that would raise potential concerns that unless other sites were willing to follow a uniform protocol using the same definitions, et cetera, it really would make it very difficult to aggregate these data and analyze them in a way that you have confidence.

So I think there's a lot to do just to get the 29 sites up and running, view it as a pilot that's something that we need to, at the end of the 6 months, take a step back and look at, see what worked, what didn't work, how can we improve, do we need to change the participation by individual sites, you know, add, subtract, get more, et cetera. But I think, you know, caution is the word here. This is very much needed and very just laudatory that Steve and his office--as someone who works in government, I know how difficult it is to get contracts let, and they did this in an extremely short period of time.

DR. NIGHTINGALE: Quick and dirty?

[Laughter.]

DR. CHAMBERLAND: But I think we have to take a step back and sort of do some of the things that were outlined in yesterday's meeting, that participants very much wanted to do.

The other thing that was of interest to me is that many--the hospital transfusion services all have--operate using computerized data collection systems. There apparently must be a finite number of these software packages that they operate on, and some are more user friendly than others, that allow you to manipulate the data and to extract the data to be reported to the system. So again that's another caution for hospitals outside the 29 that might be interested. There was a lot that I certainly didn't have an appreciation for going into this meeting.

DR. NIGHTINGALE: If I could just thank Dr. Chamberland for expressing what I had hoped to express myself. Dr. Chamberland's words are the guidelines under which we are proceeding, and they are the guidelines under which I hope you have received the information that I've presented.

DR. WINKELSTEIN: Jerry Winkelstein. Steve, I understand, or I hope I understand that the reason you've made the presentation is to get feedback.

DR. NIGHTINGALE: Absolutely.

DR. WINKELSTEIN: That it's a work in progress. I'd like to suggest that committee members spend some time thinking about this today and actually produce some written suggestions for you as a housekeeping suggestion.

DR. NIGHTINGALE: Thank you.

DR. WINKELSTEIN: Because I think that would be useful. I certainly have a few, and I'll just, rather than publicly give you the advice, I think probably most of us should spend some time writing out a few suggestions. If this is truly a pilot program we should assume that it will be changed in January and our role is to make that a better program.

DR. NIGHTINGALE: That is the reason for having this meeting today.

DR. CAPLAN: Mike?

DR. BUSCH: Steve, I commend you definitely for bringing this on board, and I think it's an excellent pilot activity, but I think a lot of the comments and criticisms are that the transition to it, a sustainable program, is not clear. The commitment, and, you know, having worked in a number of multi-center studies, these are typically 5-year programs with the well-funded coordinating center that manages a variety of these issues and has policies about publications, forms a steering committee that really oversees the program. In most studies, presentation of data like this in such a preliminary context simply wouldn't be allowed. It's so preliminary. Not that it's--I think it would be useful now in the context of a very pilot-oriented program.

But the question is: do you intend to continue to manage this program through your office on an annual allocation program, or is there some mechanism to set up a real sustainable study group?

DR. NIGHTINGALE: I think the first answer is, obviously, given the position that I am in, if this did not reflect a commitment of my superiors, including the Secretary, to continue this program for as long as it was beneficial to the public health, I wouldn't be up here giving it. I think the details of where this program will--before that, this is also a component of the Department's Blood Action Plan that was signed in on November 23rd of 1999, and my presence at the podium reflects a commitment of the new administration to maintain that policy.

Where we go with this in the long run is something that we have discussed extensively within the Public Health Service and among the Advisory Committee members. It is not clear that this has a convenient home in a particular spot with the Public Health Service. It's come to the Office of the Assistant Secretary for Health because since October of 1995 the Assistant Secretary for Health has served as the Blood Safety Director for the Department, and that continues in this administration. So we clearly plan to keep this going as long as it is useful. That is my understanding from my superiors, and I have no expectation that that would change.

Where do we do it? Do we keep it in this office? I hope not. This has been a long summer. There are several other things that I have to do at the same time. There's three of us and we're hiring a secretary. It's tough. I hope that the computer will make it a whole lot easier.

Where does it site? Does it site within the Public Health Service? Does it site within the private sector? That's a very complex question. But let me add the one reason that is implicit in what I'm saying, but explicit. Is when an entity, whether the government develops a program, whether AABB, ABC, Red Cross, Immune Deficiency Foundation, develops a program, that entity will face a very legitimate challenge, and that challenge is: did you develop this system for the public good or did you develop this system to spin your own version of the public good? And that above all is the reason why this is being presented to the public for this time, for their input, with the tape running.

The final version of this program, when it comes out--and the January version may be it, that's what I'm shooting for, and that's why I'm holding this program right now--should at least address the concerns that are raised at this meeting, raised at the meeting yesterday, and that I'll hear about later. If we had a lot of time to put this in progress, we might not be pushing it so fast, and that's another reason for bringing it to the public early rather than late.

It is my own view that we don't have a couple years to fool around with this program. We need this information right now. There is major policy change in donor deferrals that's going to be coming fairly soon. We need to know the impact of that policy change on the capacity of the blood supply to react. And when we put the UK donor deferral in, we missed an opportunity to get data, and I don't want to miss that opportunity this time.

DR. WINKELSTEIN: So I have another housekeeping question. Forgive me. Have our previous resolutions in any way addressed this problem? Do you need something more specific? Is that an agenda item for today? Should we be thinking in those terms?

DR. NIGHTINGALE: I think we got a pretty strong boost in April. This is a different meeting from the one in April. This is much more a nuts and bolts meeting. It's nuts and bolts this morning. It's nuts and bolts this afternoon. There may be a big picture into it, but I think we've dealt with the big picture before, we'll deal with the big picture again. This is--if you have a thought, a constructive, or just a plain old-fashioned suggestion or criticism of this, this is a real good time to give it because this is a time when those criticisms are going to be addressed.

DR. CAPLAN: One issue that I think has come up time and again is, as we sort of build this sentinel system, want to make sure that there is an adequate steering committee, oversight and accountability for the system. So one issue is where is it house? Another is sort of who's steering the ship and ultimately making decisions?

So what thought's been given, Steve, to the sort of structure, executive structure on the sentinel system?

DR. NIGHTINGALE: I think the toughest critic is the public, and that's the critic we're eliciting at this time. We are using the Advisory Committee, once again, as the surrogate for the public, but we're doing that in a public rather than a private session.

At the time same time, I should say that there have been extensive private discussions with people in the community, both those who are participating in the study, and those who are not. I would mention in particular, Dr. Brendan Moore, of the Mayo Clinic, who's been here before, declined to participate in the program. He felt his center was not representative, but he sent us a 5-page very detailed critique which was extremely helpful.

At the moment, supervision comes from my superior, who's the Assistant Secretary for Health. The immediate supervision are my colleagues in the Public Health Service who are on the Advisory Committee as well, and many members of Dr. Epstein's office. There is a working group on blood safety where I think that the--the supervision of this with Dr. Williams--there's a light in my eyes and I can't see Alan if he's in the room, okay, I see his hand--Dr. Williams, who has a very extensive, very valuable background in this area--

DR. CAPLAN: Here's my point: I think if we're going to be in a situation where there are major concerns about inadequate blood supply in the face of the pursuit of increased safety and how that debate is going to sort out, it's going to be very clear--it has to be made very clear who is going to be at the helm of the sentinel system. So I didn't really mean to put you on the spot--

DR. NIGHTINGALE: No, no, no.

DR. CAPLAN: --for running the names out, but I think that should be there. We can sort of suggest, perhaps, that as you--part of the standardization and reports and so forth, should include some attention to what's the best way to make sure that the public, which donates the blood, understands who's keeping an eye on the supply of it in terms of really administering the program, making the decisions. That's, to me, taking a public perspective, I want to know names and zero numbers to speak.

DR. NIGHTINGALE: That's easy.

DR. CAPLAN: And they may all be within the Departments. That's okay. I just want to know where they are, so if Jerry's sending suggestions in, he knows sort of who's reading them at the other end, and then who's accountable.

DR. NIGHTINGALE: Okay. On January 8th of 2001, Dr. Satcher signed a directive that established the responsibility for monitoring the blood supply in his office at the time he was the assistant secretary for health, as well as the Surgeon General. So to where does the buck stop, that stops here. It stops in his office. And the person who is implementing it is myself.

DR. CAPLAN: All right. Well, if we've exhausted the subject here, maybe we can take our 15-minute break. Thanks, Steve, for the update on the details. We'll get back here in 15 minutes.

[Recess.]

DR. CAPLAN: We're going to go into the public testimony part of the meeting. Our first 5-minute set of comments, let's hope 5 minutes, Jackie Fredrick from the American Red Cross. So if it could get the room to quiet down so we could hear her.

Jackie.

MS. FREDRICK: I'm getting older. I need glasses.

Mr. Chairman and members of the Advisory Committee, thank you for allowing the Red Cross to make some public statements here.

As you know, the American Red Cross is committed to developing a stable and sustained blood supply to meet increasing patient needs and hospital demand for these life-saving products. We believe it is incumbent upon the blood banking and transfusion medicine community to commit to a new way of doing business by accurately forecasting the demand for blood and ensuring that blood collections are geared to meet specific patient needs. The Red Cross has instituted a series of new initiatives that are enhancing our ability to monitor the amount of blood collected, distributed, and in inventory at each blood center nationally and nationwide. With this information, along with the results of market research that enhances our understanding of how to effectively reach our generous blood donors, we are working to make chronic cyclical shortages a thing of the past.

We have been asked today to comment on HHS' plan to monitor the demand for blood products. We believe in order to ensure blood availability, it is critical to have an effective means for monitoring hospital and patient needs as well as available inventory. The recent announcement by HHS regarding a sentinel system with real time information from 29 hospitals on the supply and the demand for blood and blood products will be useful for all of us in the blood services enterprise.

At the April meeting of this Committee and at the June meeting of the TSEAC Committee, the Red Cross shared with the Department of Health and Human Services our short- and long-term plans to increase blood collections. Today we are pleased to share with the Committee and our blood services colleagues, the initial results of our efforts to monitor and increase the blood supply, as well as our comments on the present HHS data collection effort.

Before I outline our recent activities and results, we would like to update the Committee on research to determine the further impact of our expanded donor deferral criteria related to variant CJD. This information is directly relevant to the discussions today on blood availability.

The Red Cross commissioned Wirthlin Worldwide to perform a telephone survey of a nationally representative sample of Red Cross donors to determine the number of individuals that would be deferred because of our expanded variant CJD criteria. The findings of this survey indicate a total of 3 percent of our current Red Cross donors will no longer be eligible to donate under our expanded guidelines with a margin of error of 0.6 percent. In addition, approximately 1 percent of eligible donors will erroneously self-defer even though they are actually eligible to donate. Take together, the results of the survey indicate about a 4 percent loss of our donors in the American Red Cross, equivalent to approximately 235,000 units from the expanded deferral.

Based upon our collections experience this July and forecasted collections through mid September, the Red Cross believes that donations will cover this anticipated loss. Although the Wirthlin survey shows the Red Cross national system experiencing a loss of between 3 to 5 percent, our collection goals are based on prior modeling that indicated an 8 percent loss in donors. The Red Cross is working hard to ensure an adequate blood supply to our hospital customers. Our recent summer initiatives have already shown positive results towards increasing blood collections.

There are those who have speculated that donations are actually decreasing in this country. We have found exactly the opposite. In fact, presenting donors to the American Red Cross surged to 7-1/2 million in our last fiscal year or a 6 percent increase compared to the prior year. This corresponds to a potential 8-1/2 percent gross increase in productive units if one uses fiscal year 2000 deferral rates that did not include our loss of donors due to finger sampling. So an apples to apples comparison would have actually had us growing 8-1/2 percent in presenting donors. This would have corresponded to over a half a million units of blood collected.

The Red Cross has seen an increase in our blood collections for this summer compared to past. We embarked on a targeted advertising campaign, personal contact with our donors, and new programs to monitor and forecast blood collections and distributions. Our July 2001 collections of 551,949 reflect an almost 8 percent increase over July of last year. That's over 39,000 more units collected this July than the past July, or an equivalent increase in two days of transfusion need for our 3,000 hospitals. In addition, 31 of our 36 blood regions also increased their supply from July to July.

These increased collections have had a direct impact on our inventory. Our total red cell inventory is 33 percent higher this August than last August. Type O inventory has increased 83 percent over last August. This shows that our targeted efforts to contact Group O donors, over 2 million of them, paid off. Distributions of red cells are up 3-1/2 percent in July compared to last July, as well as increases in Type O distributions.

Our recent campaign highlights our ability to increase blood collections by using the right strategy and resources. Our goal now is to make this sustainable.

We are moving forward with long-term initiative to build upon our positive experiences this summer, the lessons learned and what we are hearing from our donors. We are increasing our collection staff, our collection sites, and our collection goals. We are developing specific appointment scheduling methods that will expedite the donor experience, and are planning to expand our telemarketing and call management systems as well as our blood donation record process. All of these initiatives are geared to making the donation experience quicker and more enjoyable for our volunteer donors.

The Red Cross is also working on its projection and demand models so we can forecast where blood is needed before there are shortages. We have been forecasting collections for over a year and we believe out models will continue to accurately predict what has been collected.

I heard the prior conversation about what happens if you find out there is blood needed in Bismarck and it's in Madison, Wisconsin. In fact, the Red Cross has been moving blood all summer because of our inventory model and our demand model. And that will allow, in the Red Cross, inventories to be balanced absolutely across the country.

All of us in blood services are challenged by meeting growing patient needs for life-saving blood. Chronic, cyclical blood shortages have long plagued this enterprise. HHS' announcement about the sentinel system for monitoring the blood supply is commendable. This is a good step towards better understanding issues of the blood supply and the need for blood in the 29 participating hospitals. This information will be useful to everyone in blood services and will provide a snapshot of certain variables that impact the blood supply.

The Red Cross would like to thank the Committee for the opportunity to provide our views on this important public health issue. The safety of the blood supply and its availability are top priorities of the American Red Cross. We believe it is a shared responsibility of blood collection organizations and hospitals to collect data on supply and demand, and therefore, institute programs to ensure blood availability.

I'd be happy to answer any questions.

DR. CAPLAN: A couple of questions we have time for. Karen?

MS. LIPTON: Karen Lipton. Jackie, we were talking earlier about the HHS monitoring effort, and people were talking about it would be important to have some data if we could from the blood suppliers. Have you been asked to participate in that?

MS. FREDRICK: No, we haven't been asked. I think Steve came about end of June and we discussed the agenda here, but as I said, I think it was at the April meeting when we talked about the same thing. We would be glad to provide data. We'd like to be asked to provide the data. And we'd like to participate in how that data's going to be used also.

DR. NIGHTINGALE: Jackie, you can anticipate all of the above. Thank you very much for the offer.

MS. FREDRICK: Thank you.

DR. HOOTS: Of your new donors, do you have a sense of how many are either first-time or nonchronic donors? Are you harvesting a new pool, or are you moving pools of donors around?

MS. FREDRICK: I can't tell where that question's coming from.

DR. HOOTS: Right here.

MS. FREDRICK: Oh, I'm sorry. I can't see the red light.

We haven't analyzed the July data yet to know what percent are new donors and whether we've increased the frequency. We're going to actually monitor donor behavior over time because one of the theories we all have is do you actually cause donors to donate more frequently or do you just move them more current in their donation process?

So right now I actually can't tell you whether the first-time donors or the frequency have changed.

DR. CAPLAN: Let's do two more. We'll do Ed, Dr. McCarthy, then we'll get done. Ed first.

DR. GOMPERTS: Mr. Fredrick, the estimate from the point of view of deferrals, the percentages and estimates that you've given, on what--was this an estimate or did you actually have some hard data? Did you do some surveys? How was that done?

MS. FREDRICK: I'm sorry. We did a representative survey of our blood donors who have donated in the past 12 months, and it was done to determine the national impact, though I do have geographic data, but it's large geographic data. It was done by telephone surveys, statistically significant sampling, detailed questionnaire. So, yes, we have very detailed data, and I would say that that has been shared in its detail with FDA, with Alan Williams and Jay Epstein.

DR. GOMPERTS: Thank you.

DR. McCARTHY: Leo McCarthy. Jackie, in your ramped up efforts to increase donations, could you comment on what singular efforts are being made to recruit the minorities, which have been under recruited for a long time. Is there a focus on minority recruitment?

MS. FREDRICK: Yes, there is a focus on minority recruitment, but it tends to be very focused locally, where I believe it should be. We can provide guidance from national. But, for instance, in Detroit, which I think two weeks ago came out as the country's largest African-American city, we have a three-year initiative there that will grow collections by 58,000 donations and 24,000 donors. We have a large hispanic initiative going on in the Los Angeles area.

But clearly, going forward, those have to be a focus for us to be successful, particularly in urban areas.

DR. CAPLAN: I've got Rick, then I've got Harvey, then I think we'll go.

DR. DAVEY: Rick Davey. Jackie, in your survey of the deferred donors, I wonder if you got information on the number of deferrals or the percentage of deferrals that may include repeat donors and platelet pheresis donors? There's some indication that the deferral criteria may more heavily impact those particular groups.

MS. FREDRICK: Yes. We did. We specifically asked a question about apheresis, and apheresis donors or platelet donors will be impacted by a higher rate, and I want to say I think it's about 4 or 5 percent of platelet donors. We also did look at first-time donors and regular donors and I believe first-time donors had a higher incidence of deferral than repeat donors, which would make sense, because we've kind of done the UK deferral already to those. But I have that detailed information. I can get you those numbers.

DR. KLEIN: Jackie, first, if no one else is going to say it, I'm going to say how encouraged I am that the data on collection looks so good. I think this is wonderful news, and the organization really needs to be commended.

Having said that, this is the other half of the data. I mean, this is the collection side. We heard a little bit about the hospital side, and I certainly would hope that at least some of the regional center, if not the whole system, would be able to supply that kind of information so we can put the two together. Do you have the kind of information that might look at need, such things as orders that were partially filled or orders that weren't filled. Even though the collections are going up, that may not be supplying the hospitals, and perhaps you have some information that might be of some help.

MS. FREDRICK: I do. And we are monitoring that. We call it fill rate. And it's not perfect, because just like the discussion about what's an order and what's filled and if people are over-ordering, but, yes, I have that by blood region and by blood type, I believe. I will tell you, I'm a little leery about the data, but what we have done is we've already set our goals for the next five years for blood collection, specifically in detail for the next three years, and we have taken the fill rate loss, that I call it, and we've actually built it into our collection number such that within 24 months, we would meet 100 percent of the orders placed today. Now, I don't personally believe that we are under transfusing the country by huge percentage points, but--so, yes, I have that information.

DR. NIGHTINGALE: This is Steve Nightingale. I would just like to state for the record that I fully concur with Ms. Fredrick's comment about the fill rate. That is one of the many things under "other" that we have considered, and frankly, we considered it a much hard measurement than how many bags of blood are actually on the shelf, how many bloods were actually transfused, how many were actually sold to another center and how many were actually outdated. I would be very concerned about basing either projections or estimates of current availability of blood or demand for blood on estimates, such data as that.

MS. FREDRICK: Jay?

DR. EPSTEIN: Thanks, Jackie.

MS. FREDRICK: Sorry.

DR. EPSTEIN: Could you comment on how readily blood moves around the country because one of the issues with deferrals is that they can have disproportionate regional impacts, and so one of the challenges to the system is moving blood around from where it may be in surplus to where it may be in shortage.

MS. FREDRICK: Yeah. Within the Red Cross it moves very readily. We have, every morning, a call of 5 people, 1 at headquarters, 4 out in the different business units. We look at our inventory, we look at it by region, and then we move the blood. I think--I don't have recent numbers, but at least two years ago, between platelets and red cells, I want to say we moved almost a million units a year, but it can be moved within, you know, 5 minutes notice.

DR. CAPLAN: That's within Red Cross or everywhere?

MS. FREDRICK: Well, when we get a call from outside the Red Cross, which we did in July, a city in the midwest who was going to have to cancel surgeries. I think we were an hour and a half down the freeway from that blood center, and I think we had blood to them within 4 hours or so.

DR. CAPLAN: Okay. Dr. Bianco is next, from the ABC.

DR. BIANCO: Hi. I'm Celso Bianco from America's Blood Centers. ABC's an association of 75 not-for-profit locally-controlled blood centers that collect nearly half of the US blood supply from volunteer donors.

ABC thanks the HHS plus Safety and Availability Committee for the opportunity to comment on the monitoring of availability of blood products in the US.

ABC wants to congratulate HHS, particularly Dr. Steve Nightingale and his staff, for the creation and implementation of a system to monitor the daily status of US blood supply at the hospital level. To our knowledge, this is the first time that this happens in this country. It is incredible that every Monday we know the gross revenues of new movies around the US, but we do not know how much blood we have on hospital shelves.

This program will generate critical information for the health care system. We thank you and each of the sentinel hospitals for your efforts.

I want to make a couple comments that are not in the sheet. The way I see sentinel programs of this type, there are two that I want to mention. One is a program that has been going on for many years through the Centers for Disease Control that is the hepatitis surveillance program. There are four centers in the whole country. Yes, they are representative of their communities, but they are not necessarily representative. But they have been extremely important. And I know that Dr. Chamberland knows much more than I do about that program, but a wealth of information has come out from this program over the years and has helped us manage hepatitis and the blood supply for many, many years, particularly more recently since we knew about non-A/non-B hepatitis and introduced hepatitis C.

The other one, that I think is a little bit more similar to what we are talking about here, that is was scrambled a little bit just to get it very fast, is West Nile virus. As we heard of it, 9 people die in New York City, and then you create a very fast surveillance program. What you want it to do is to be very sensitive, and there are big cities, I think that that was very smart, because those the are the ones that are going to feel the impact first. You want to have a first signal so that you know what is happening. And the same way with the birds. We're going to test the birds that are dead, not the ones that are alive, because those are the ones that will have a higher prevalence of infection.

And ultimately, that does not mean that this is representative of the bird population in the United States or the danger of West Nile virus, but this is telling us where each one of our cities will go, spray, take precautions in terms of mosquito containment, and protect the population. And, actually, if you will look at West Nile virus, we were scared to death when it first came up, but it has been a very controlled, serious, has been spreading epidemic, that has not been a real threat to the blood supply.

Changing to another item, America's Blood Centers will contribute to the HHS effort by generating complimentary data to monitor the blood supply at the blood center level to help us understand a complete picture. The system that we are creating is simple in order to ensure timeliness and compliance. Essentially, ABC member centers will indicate every morning, through an Internet based system, how many days supply they have in their shelves. If they have three or more day supply, they will click a green button, for a two-day supply, they'll click a yellow button, and for a one-day supply, they will click a red button.

The software will compile the data according to regions, and since what we know from the HHS system, these regions, we will make sure that they correspond to the regions chosen for the HHS sentinel hospital monitoring program. The regional data will be posted at the America's Blood Centers website on the afternoon of the same day. The posting will indicate how many blood centers in each region have designated their status as green, yellow or red. Individual centers will not be identified. We expect to have the system operational within one month. This will provide HHS, our blood centers, and the public, with daily information about the status of the blood supply. Certainly this is not a sophisticated statistically based system, but as all of us think about that, ABC member centers are certainly ready to provide more data that is appropriate to HHS and to investigators studying the problem.

ABC members are extremely concerned about blood shortages, because they threaten patients' lives. Implementation of deferrals because of the travel to countries with BSC [?] will have a serious impact on the blood supply. We commend FDA for all the thought and balance brought to this issue, and are aware that a guidance will be issued in the next few weeks.

We hope that the guidance will propose a gradual implementation that provides time for us to recruit new donors without major disruptions. We also hope that FDA will assure our member centers that the integrity of the blood supply must be balanced against the theoretical risk of variant CJD, and that they should follow the timeline proposed by FDA. We are extremely concerned about premature implementation of the full donor ban before the system is allowed to adjust. We are also very concerned about the potential reaction of the public, leading donors to self defer. We believe that the HHS data, together with the ABC data, will help guide us through the several phases of implementation and help preserve the supply of life-saving products that our patients need. It will also assure that additional blood is available to support the areas of the country that will be hard hit by the deferrals.

Finally, I want to announce to the Committee that one of the most difficult problems created by the donor deferrals has been addressed. A [?] deferral will eliminate Euroblood, a 30-year old program that provides 140,000 red blood cells a year to the New York Metropolitan area. This represents about 25 percent of New York's supply. Two days ago members of America's Blood Centers pledged 75,000 units of red blood cells to the New York blood centers. One of our members, the American Red Cross, made a similar pledge. We are proud of our members for making such a difficult commitment in face of the uncertainty associated with the impact of deferrals in their own areas.

ABC members thank again this Committee, HHS and FDA, for the opportunity to participate in this effort. It will benefit every patient that needs blood. It's about life. Thank you.

DR. CAPLAN: Committee questions? John?

MR. WALSH: Thank you for your presentation, and also ARC. Is there an effort to standardize a protocol or data collection device between you and ARC at this time?

DR. BIANCO: Not at this time, but certainly this can be worked out, and particularly if the groups that are doing those studies within HHS, Dr. Nightingale's, create a standard, we certainly will make within the limitations of computer systems and definitions and all that, adjust it in such a way that we could comply with that standard.

DR. CAPLAN: Jane?

DR. PILIAVIN: Your green, yellow, red system sounds certainly very easy for the centers to use. I was just wondering to what extent it's going to give information on the different kinds of blood.

DR. BIANCO: We can break it down by group, but there is a certain--even if it is not an exact correlation, Jane, there is a certain correlation between what is available at the blood center and the blood type distribution. The correlation gets destroyed when you get down to one-day supply. You look at your shelf and you have only A's and AB's. But as you have a normal blood supply, the blood type distribution is more or less. So we feel that this will give us at least a preliminary thing, can be started fast, and will be easy for our centers to comply. The definitions of a day's supply are also different from center to center. Some will include what we call in-process inventory, that are the units that were collected are sitting in the refrigerators, waiting for the test results, waiting for the MAT [?] results until they could be labeled and ready for shipment. Other's don't. And so we have to harmonize a lot of those definitions, and to be able to do exactly like the movie producers and movie houses do.

DR. CAPLAN: Jay, then Dr. McCarthy.

DR. EPSTEIN: Celso, in the same vein, are you going to lump the platelet inventory with the red cell inventory, because it's certainly going to confound the number.

DR. BIANCO: No, we are not. This is at this point was designed for red cells, but hearing all the things that I heard today, and our initial thought was to make it very simple, but probably, Jay, we should include a platelet set of buttons for both random and single donor. I don't think that this will add much to the programming, and again, it should not be a major task for the inventory management in each center to be able to respond to that question.

So thanks for the suggestion, and we'll do that.

MR. : [Off mike, inaudible.]

DR. BIANCO: No, it's not. It's because the question is hard.

[Laughter.]

DR. McCARTHY: This kind of cooperation that we just heard from both of you is very heartening, and I just want to know if this is really going to continue beyond the New York blood center, i.e., we jump months ahead. This data, we have refined data. We can predict there's going to be a shortage in an area either U or X or U or Y. Can we then expect that if one--if a transfusion service such as mine has needs that aren't being met by organization X, that organization Y will backfill, if you would, the orders in a--to a transfusion service that's not primarily one of their primary customers, and I'll use your word. Because I think that's one of the questions that I want answered, and I guess that's one of the goals why some of us have voted with our feet to come here.

DR. BIANCO: Well, I believe that that exists, and as we are able to improve collections, that it will get better. And I do not want necessarily to comment myself, but for instance, the AABB exchange has existed for many years exactly with the purpose of providing blood to moments of need. I think that what is going to happen here is as we start learning more about the process, as we start learning more about shortages, certainly these mechanisms of cooperation can occur, the mechanism of cooperation here between Red Cross and ABC is really New York Blood Center, that is, New York Blood Center is going to manage their needs and their requests to both organizations to supplement that immediate loss of 140,000 units.

I understand your question, and I seriously trust that we can get--I will be able a year from now to give you a better answer because I have a sense that we all are acutely aware of the potential for shortages and acutely aware of the impact that blood has in the health care system. So I think this is the beginning, at least I'm being very optimistic.

DR. CAPLAN: I'll do one more from Keith.

DR. HOOTS: Yes. I also would like to congratulate both of you for this humanitarian approach in New York. I was actually kind of surprised. Is there any other example that you know of, of the US not being self-sufficient in blood?

DR. BIANCO: The Euroblood program was not created out of self-sufficiency or doesn't exist because of a question of self-sufficiency. The Euroblood program was created over 30 years ago, and Dr. James Louie, I think, is in the list of speakers, from New York Blood Center; he can tell more. But it was created when Dr. Aaron Kellner, the founder of New York Blood Center was creating the New York Blood Center in the mid '60s, and was trying to replace paid donors with volunteer donors. In traveling to Europe he realized that several European centers were collecting whole blood with a single purpose of using the plasma for the manufacture of derivatives, Factor 8, for instance. And so he worked through the process for several years, with Europeans, with FDA, and ultimately they created a system in which there are three centers, one in Switzerland, one in Holland, and one in Germany, that are FDA licensed collection centers of the New York Blood Center. They're inspected every year, and they work as if they were a center in Queens. And the units of blood are separated. The plasma is retained by them for manufacture. The red cells, with the test tubes for the testing, are shipped to New York for the appropriate testing and release.

So the program is not--there was a question--in the early '70s, yes, it was self-sufficiency because we wanted to get out of a paid donor system. It remained because the program worked beautifully, and actually helped New York a lot in the early '80s because the AIDS crisis hit Europe many years later, so if you compare actually the incidents of AIDS by transfusion in New York in the early '80s, it's not very different from other places in the country simply because the blood supply was diluted with units that were essentially negative. Now we are seeing the reverse of the thing.

DR. CAPLAN: Thank you, Celso. I'm not sure who's presenting for the New York Blood Center.

DR. BIANCO: It's Dr. James Louis.

DR. CAPLAN: Okay.

MR. LOUIE: I'm James Louie from New York Blood Center. I'm the executive director of our Long Island Region Operating Unit, and I want to thank the Committee for allowing me to speak today, and I was to applaud the efforts of the Committee, and for the new initiative on the sentinel study, so that we can better understand the blood supply and how to assure adequate availability throughout the country.

I also want to express our thanks and express how grateful we are to the American Red Cross and to ABC Centers on their pledge to assist the New York area in the coming months and year to get through this anticipated stressful situation with the blood supply in the New York Metropolitan area.

And the New York Blood Center really would like to very much participate and work with the Committee and with the study group to better understand the supply, especially in the New York area of course, and to assure good availability of blood.

I want to just convey our concern, the Blood Center's concern regarding the impending reduction of the nation's blood supply that will result from the actions of the Red Cross with the implementation of the recommendation of the new deferrals, and also the pending recommendations from the FDA TSEAC Advisory Committee. These actions will precipitate a tremendous loss of donors, donations throughout the country, and which hundreds of thousands of donors will be deferred because of this. And these losses will be justified by the theoretical risk of transmission of variant CJD via blood transfusion.

There has never been a documented case of transfusion-related vCJD, and there's no solid scientific evidence for the support of restriction of donations based on residency in continental Europe.

The impact of these restrictions will be most severe in the New York Metropolitan area. Our population is very cosmopolitan. It has large numbers of immigrants, citizens with dual citizenship, business and leisure travelers who will be deferred because of the new donor restrictions. The New York area surveys indicate that we will lose approximately 25,000 donors who donate approximately 35,000 units of blood per year. We have a historical reliance on blood from Europe from three European countries, Holland, Switzerland and Germany, and these are FDA-licensed, and regulated, and monitored blood centers that we have worked with for almost 30 years now. And we have been making great strides in decreasing the number of units that we get from these centers over the past three years, but still we import about 180,000 units per year.

The loss of the Euroblood and the implementation of deferral criteria, combined will cause a loss of about 200,000 units--of available units in the New York Metropolitan area. The magnitude of the loss will have a significant medical impact on the area, and concerns of the medical impact has been expressed by the Greater New York Hospital Association, Senator Charles Schumer of New York, the American Hospital Association, the ABB and the AMA. So these organizations and the New York Blood Center have argued for a measured approach that would balance the impact of the severe shortages that we anticipate with the theoretical risk of transfusion transmitted vCJD.

We urge that the Department then create an orderly transition for implementation for new policies aimed at vCJD, and careful management of this transition is very important, so that the public is not confused about the implementation of the criteria so as not to overly defer people who might be confused by a new criteria, and also take into consideration the impact over the short term on the medical care situation in the New York area.

We are totally committed to the safety of blood supply, but we ask that the Department consider blood adequacy and availability in its deliberation.

I want to thank the Committee for, again, the opportunity to speak, and I'll be open to any questions now.

DR. NIGHTINGALE: I think we can take one or two questions, but it is approaching noon. Are there any from the Committee members? Dr. Epstein.

DR. EPSTEIN: Well, Dr. Louis, I certainly appreciate your fair description of the condition in New York, and I also understand your statement that there's, as you said, no solid scientific evidence on CJD risk or variant CJD risk. But I think it is very important to emphasize in a public forum that although the risk is considered theoretical because there has been to date no demonstrated or proven transmission, research in animals has suggested that this is possible, and no one is able to exclude that possibility. Unfortunately, there are a lot of unknowns. We don't know if it's actually transmitted. We don't know how many people are harboring the infection now and we don't know how big the epidemic might get.

So it is the view of our expert scientific advisors, and also government officials, that it is only prudent to take feasible precautionary steps to reduce that risk, and of course, we recognize that those steps must be tempered to avoid blood shortages that would themselves be safety risks. So we certainly agree.

And therefore, we have tried to approach the problem, as you say, with balance, and tremendous efforts have gone into trying to avert any acute blood shortages that could result from deferrals that are implemented over time. And I am prepared to say that it is FDA's current thinking, that as it proposes deferral standards, that they should be implemented gradually over time in a phased approach to allow the system the necessary time to offset potential losses, both through additional donor recruitments, and also through supply arrangements that can move blood from where it may be available in surplus to where it may be deficient.

So, really, my point here is to emphasize that while there is certainly a risk in diminishing the donor base, there's a very good reason for it, which is the need for prudent safety measures.

MR. LOUIE: Yes, and I agree that--and I appreciate all the effort that Committee and the Advisory Committee has put into this in consideration of the situation of New York. And we're just approach this, asking for a balanced approach because of the nature of the severe shortage that would occur with the sudden cutoff of our supply from Europe, and with the--and the impact of the deferrals I think having a greater impact in the New York Metropolitan area than other parts of the country.

Certainly we are working very hard to increase our donation rates. For example, this past year we're tracking now at a 15 percent increase year over year in the New York area in terms of total blood collections, red cell collections, and even more than that, for platelet collections. So we're looking forward to great strides in beating this challenge as we look to putting new deferrals in place for improving the safety of the blood supply.

So we're very strong advocates of having a very safe blood supply, but we want to just state our case, that we need time, and we need a balanced approach to this transition.

DR. NIGHTINGALE: We have comments that I know from American Association of Blood Banks, American Society of Clinical Pathology, the Committee of 10,000, Hemophilia Federation of America, and the National Hemophilia Foundation.

Is there anybody else in the room who wishes to make a public comment at this time?

[No response.]

DR. NIGHTINGALE: It's about 3 minutes after 12, I would like to conclude the comment period about 12:30, but if necessary, we will go beyond that time.

The American Association of Blood Banks. I think Ms. Wiegmann.

MS. WIEGMANN: Good morning. I'm Theresa Wiegmann and I'm General Counsel and Director of Government Affairs for the American Association of Blood Banks.

We have provided a written statement for the Committee, so I'm not going to go all the way through that. I'm just going to try to summarize some of our positions in light of this morning's conversations.

I'd like to start by saying that the AABB commends both the Committee and the Department for working to obtain blood supply and utilization data. The agency has taken important first steps on this critical public health issue. However, we do share many of the concerns raised this morning.

We agree that timeliness is important, especially in critical states of flux like we face today. But in order to get meaningful data, we believe that we need to think in a more long-term picture. We need to step back and answer a number of questions before moving forward with data collection and analysis.

We need to assure that the data that we obtain is representative on a national scope. We need to determine how the data will be analyzed. How are we to match the new data that we obtain with the existing blood supply and utilization data we already have? And we will need to figure out how the supply side and the utilization side of the data are matched.

Quantitative, in addition to qualitative data are also needed. We need to measure trends over time and across geographic regions. And as was stated on numerous occasions this morning, it is critical that we have uniform definitions before moving forward with data collection.

As soon as possible, we urge the Committee and the agency to establish an expert panel to address these important issues. In order to serve the public health and the patients' needs, critical questions need to be answered before we obtain data.

In forming an expert panel, we must recognize that neither this Committee nor government representatives alone have the necessary expertise to answer all of the questions about blood supply and utilization data that we have. We would encourage an expert panel to include representatives from the transfusion services, from hospitals, blood centers, and data experts. And we believe that the National Blood Data Resource Center should continue to play a valuable role in collecting and analyzing blood supply data.

Now, we heard this morning, and we appreciate the fact that currently there's only a limited amount of federal funding available this year for data collection efforts. However, we're not satisfied with that response.

Patient access to a safe and available blood supply is an absolute public health priority. Therefore, we believe that we all, this Committee, the agency, the AABB and others in the blood banking and transfusion service community, must work together to do all we can to assure that there are adequate federal dollars supplied both this year and in coming years to support long-term blood supply and utilization data. And AABB welcomes the opportunity to work with the entire community to obtain, analyze and distribute this data on a long-term basis.

DR. CAPLAN: Questions, comments from the Committee?

[No response.]

DR. CAPLAN: Okay, thank you.

DR. CAPLAN: Who is left?

DR. NIGHTINGALE: The ASCP.

DR. CAPLAN: Who is speaking for the ASCP today, or did they just give us a statement?

DR. NIGHTINGALE: The representatives for the ASCP--the document is unsigned.

Ms. Wannamaker?

MS. WANNAMAKER: I'm not going to speak for ASCP, but I believe they only wanted to present the information and not stay.

DR. NIGHTINGALE: It will be entered into the record and we appreciate their comments.

Next, I believe, is the Committee of 10,000?

DR. CAPLAN: The Committee of 10,000?

DR. NIGHTINGALE: Mr. Cavanaugh is here.

DR. CAPLAN: Yes.

MR. CAVANAUGH: Good morning. Good morning. I'm David Cavanaugh, government relations staff for the Committee of 10,000.

While the acknowledgement of the need for whole blood supply and demand data via HHS's support of the new system is laudable, paralleled by PPTA's reporting on blood products, we are concerned that today's discussions of the new system in the context of recent and likely increasing donor deferral policies do not take into consideration, first and foremost, the often stated national commitment that there be a safe and adequate supply.

Though the timing seems prudent, pitting output and demand against deferral losses overlooks the large issue, which for now I will call the arm-to-arm tracking. In fact, it seems merely intended to accentuate the impact of CJD deferrals on supply, de facto supporting efforts to remove the deferrals rather than increase the supply.

As we have said repeatedly, when supplies are dangerously low, as certainly ours are at present, a wonderful opportunity exists for triggering a national blood donor campaign using the most persuasive official in government, the President of the United States. Whether as part of a talk to the nation on some breaking news event or in the form of short PSAs, there is a crying need to return the nation to seeing blood donation as a civic responsibility.

Even the Red Cross knows that, if asked, many of those in the best of health will come forward. We must not hide behind the stigma over paid donors or the empty logic that any new donor is less safe than an existing, repeat donor. The former readily converts to the latter in a matter of weeks.

The debate regarding America's blood supply must be conducted in the larger context of real costs and real benefits. All too frequently, the debate is focused on narrow constructions of cost and benefit which fail to illuminate the national costs of blood-borne epidemics such as HIV and HCV.

Whenever this nation faces serious blood shortages, reentry of potentially risky donors is always offered as the possible solutions. We as the stakeholders in a safe and adequate national blood supply seem to repeatedly shy away from the larger issue of blood and plasma donations as a national priority. We have yet to undertake a national initiative with the will and commitment to succeed in greatly expanding the national donor pool.

We consistently discuss ways to squeeze more out of existing donors or to allow the reentry of donors whom we now believe to present less of a risk due to new testing, screening and inactivation technologies. Where is the national will? Why are we not tapping the resources of the Congress, the White House, leading entertainment personalities, and other leaders such as Secretary Thompson, who approved this process? These could be brought to bear as part of a serious and far-reaching national initiative.

We as consumers of blood and blood products continue to be ready to participate at any and all levels in the development and implementation of such an initiative. Yet, for the last ten years, even in the wake of the HIV and HCV epidemics, no such initiative has been proposed or developed. Is it a lack of will or are there issues that prevent such an initiative? This is a question we have yet to hear a sufficient or satisfying answer to.

In our current debate, only the consumer advocacy organizations appear to be looking at this component of the overall landscape. However, from our perspective, to ignore these significant economic impacts on the health care system hides some of the most significant costs associated with the blood supply over the last 20 years.

It results in conclusions that are not fully rooted in the reality of the blood safety landscape. It also distorts how we may view a given safety improvement's relative benefit. A good example of this was our failure to adopt hepatitis B core antibody testing as a surrogate test for HIV during the early years of the AIDS and blood epidemic. That option was deemed too expensive at the time; i.e., the costs outweighed the benefits.

However, how many HIV transmissions could have been prevented if all blood was surrogate tested at that time? We would all agree in hindsight that thousands of individual lives could have been saved, and the costs of treating blood and blood product-associated HIV and AIDS would also have been greatly reduced.

Conservatively speaking, the money saved certainly would have exceeded $100 million. One clearly sees the potential for history to repeat itself when our conclusions are based on narrow constructions of cost and benefit, constructions that fail to include the potential costs of another blood-borne epidemic.

From our perspective, what is sorely lacking in this discussion is any attempt to promulgate and implement a national blood policy that is inclusive of all aspects of how blood and plasma are collected, processed or manufactured, as in the case of fractionated products, distributed and used.

Essentially, what is needed is a broad national policy tracking the nation's blood supply from the donor's arm to the recipient's arm. This is not currently in place and we are left with decisions taken on a case-by-case basis, each assessed essentially in a vacuum, compartmentalized from the broader policy questions of the blood supply as a national system.

It is important to remind ourselves that the provision of blood and blood products is a business in our society. It is a marketplace where blood and blood products are treated as a commodity. There are some differences between the so-called volunteer blood banking sector and the for-profit blood banks that collect plasma from manufactured protein derivatives. There is also a difference between the blood banking side of the equation and the manufacturers of blood derivatives, such as the group of drug companies producing factor concentrates for the treatment of hemophilia.

Even with these differences noted, blood is still a commodity moving within what is seen as a marketplace. The view is that this is a free marketplace. However, this is not reflective of reality. It is a marketplace where profits from the sale of blood and blood products are underwritten to a large degree by the American taxpayer, the public sector through programs such as Medicare and Medicaid.

This is because consumers of blood and blood products have literally been priced right out of the marketplace. Essentially, this is a marketplace where profits are artificially supported by public sector dollars. Yet, the public sector does not exercise a commensurate degree of oversight regarding the operations of the marketplace.

It is also the public sector, taxpayer dollars, that bears the brunt of the fiscal responsibility for disasters such as the HIV/AIDS and hepatitis C crises. As a society, a safe and available national blood supply is a goal we all share. However, in order to attain that goal, we must begin to change the ways in which we view the reality and associated issues of the blood supply and its protection and enhancement.

We must abandon the worn-out adversarial relationships between the producers of blood and blood products, those tasked to regulate them, and the end users whose health depends on a safe and available blood supply.

We must consider all the issues in the context of a national policy that addresses all of the components necessary to sustain a safe and available blood supply. We cannot continue to confront the various questions such as nucleic acid testing, leuko-reduction, and the costs of safety margin increases as individual and compartmentalized issues. They are all necessary parts of a national blood policy that continues to be conspicuously absent from the discussion of our nation's blood supply.

For the last four years, the Committee of 10,000 has been proposing that we undertake a process involving all the relevant stakeholders, with the goal of promulgating and implementing such a blood policy. From our perspective, this is the only effective vehicle for the development of sound public policy regarding the blood supply.

Thanks very much.

DR. CAPLAN: Questions, comment?

[No response.]

MR. CAVANAUGH: Thank you.

DR. CAPLAN: Jan?

MS. HAMILTON: Good afternoon. Thank you for once again giving us the opportunity to address this esteemed panel regarding issues of interest to the hemophilia community.

Today, we are concerned about the availability of an adequate product supply and the possibilities of creating a monitoring system that would avert the situation we find ourselves in at this time.

The last time we met, we asked to have more warning about impending shortage so that the advocacy organizations can alert their constituents to take care and not hoard. What has become exceedingly clear is that there must be a system to keep tabs on what product is available and where it is.

We are delighted to hear that Dr. Nightingale has been working with various suppliers of whole blood and components since the spring to have an idea of what is available on that level. It is understanding that this was to be a pilot project that could possibly expanded upon on other levels of the blood and plasma supply.

There have been several suggestions of methods to accomplish monitoring the supply of blood and plasma needed to treat the patients of many patient communities. Because there are many peculiarities of each of the communities that utilize blood, plasma, and coagulation products, there must be a design that would address the specific needs of each of these communities.

We do not presume to offer a design for this product. However, we do have some suggestions that would affect the design of the system. The system needs to be designed with input from the manufacturers, hospitals, physicians, HTCs, home care companies, and consumers. The system should be developed and administered by at outside or impartial organization, perhaps a university research department or similar agency.

The components should be a way to take a snapshot of the level of supply in all of the above-mentioned areas, and should start with a product as it is released from the manufacturers and end with consumer inventory. Reporting and participation should be voluntary, but encouraged.

The snapshot should be taken at regular intervals for the information to be useful. The easiest system would be one that is computer-based, and the various parts of the system could e-mail their data at a specific time, in a specific day, on a regular basis, such as a particular day of the month and hour of that day.

There needs to be an outside, impartial agency, again we stress, to review the data and make it public. We appreciate the data that has been coming from PPTA for the last couple of years, but it's just not enough; it only tells part of the story.

Additionally, these factors are just a part of the larger picture; other factors such as which manufacturer is scheduled to be down for maintenance and which one needs to be out of distribution due to FDA findings. While we understand the importance of not violating Sherman antitrust issues, there has to be a way that some agency could hold the appropriate data and act on it.

In other words, if a company is scheduled to be down for maintenance and a problem develops in another company, causing them to have to be offline for cause, the company's scheduling maintenance should be able to be asked to postpone their down time for a period of time, if at all possible, without harm to the product, to allow for a leveling of product availability rather than dire shortage. We realize there are restraints on who could have this information, but if we put on our thinking caps, we can make it happen in an unbiased manner that does not violate antitrust regulations.

In the matter of coagulation products, there's another hurdle that must be mastered, and that is of allowing the product to follow the patient. There are many reasons for a patient to leave one home care company and go to another. Just a few of them are some of the smaller companies that can no longer serve Medicaid patients and those patients must seek product elsewhere. Some companies go out of business. Some patients move to an area not covered by their current home care company.

In times of severe shortage as we are facing now, these scenarios are a bit difficult to maneuver. However, if the manufacturers and the home care companies would agree to work on a release system, then the patient could rightfully expect his or her product to follow them wherever they go.

The Hemophilia Federation of America is thankful that this Committee has heard our voice, along with the voices of other consumers in the hemophilia community and others that use blood and blood products such as sickle cell, immune deficiency and alpha 1, to name a few, in the past, and we are confident that you will hear today and take the appropriate action, if not for your own Committee, then refer the situation to the appropriate agency.

In closing, we would like to offer to work with whoever designs a possible program to accomplish this much-needed task in whatever capacity we are needed.

Thank you.

DR. CAPLAN: Thank you.

Steve?

DR. NIGHTINGALE: Just one very quick comment. We do recognize that portability is a specific issue for some communities, and in a time of shortage it's an issue that has to be addressed. It's like several other issues that have been discussed here, not only by me. We need to figure out how to monitor this process along the way, and we look forward to an active dialogue with all stakeholders on the issue of portability. It's very high on my radar.

DR. CAPLAN: Okay. Is there anyone who wants to testify who has not done so?

DR. NIGHTINGALE: NHF.

DR. CAPLAN: Oh, NHF, okay. I think we have Mark Skinner coming up.

MR. SKINNER: Good afternoon. Thank you for the opportunity to present today. My name is Mark Skinner. I'm President of the National Hemophilia Foundation. The Advisory Committee's consideration today of the monitoring of the demand and the supply of the blood supply is crucial.

Regrettably, people with hemophilia have been suffering from an acute shortage of recombinant products for a long period of time. These shortages have been unpredictable and they have been unexplainable, and the solutions to this problem have been even harder to find. A monitoring system to provide real-time information on demand and supply would be of great assistance to our community.

What NHF and the bleeding disorders community has been doing on our own has been inadequate. It has not provided us all the information that we need. PPTA has been working with the community and they have provided us monthly production data, but ultimately it is only retrospective and it doesn't provide us the prospective analysis that we need.

We also have been doing quarterly surveys of our hemophilia treatment centers, and through that information we are seeing now a very acute change in treatment patterns. Medical care is currently being compromised and we are seeing the trend grow. The shortage is here, it is getting worse, and we don't the data to manage or to predict or to anticipate the kinds of things that will be coming forward.

For these very troubling reasons, NHF is enthusiastic about the blood monitoring system announced today, insofar as it goes. Access to real-time information is critical for both short- and long-term product use, and a similar system for these products, for recombinant drugs, would be extremely helpful in better managing the current shortage situation.

We strongly suggest the inclusion of the hemophilia treatment centers in any monitoring program. In addition, we would suggest that this include the manufacturers, the home care companies, distributors, and hospitals, as we've already discussed.

The National Hemophilia Foundation has been exhausting its options to manage the current supply. Through our medical and scientific advisory committee, we have adopted new guidelines, recommending in many instances what is sub-optimal care. We are committed to recombinant product as the standard, state-of-the-art care for those with bleeding disorders, and we have been unfortunately left with the situation of recommending something less than that as an interim measure. Conservation alone on our part is not going to solve the problems.

We have initiated a series of meetings with manufacturers. We have met with some this week; we'll be meeting with others next week to explore each and every option we can to enhance the production and the supply, and to develop an additional base of information on which we can make decisions and recommendations.

We have requested a meeting with the Acting Commissioner of the FDA, and unfortunately to this point we have not been granted that meeting to express our concerns at the highest level and to see what role the FDA can play in a solution to this problem. We are hopeful that we will be able to garner such a meeting and we'll be able to raise and elevate the dialogue to the appropriate point.

We have been recently discussing with the manufacturers, and these discussions will continue about the management of an emergency supply system. Most of the manufacturers have adopted an emergency supply. We think this is a critical part of going forward, and understanding what the current supply is and what the projections are would be an important part of the development and the management of that program.

There are a number of public health consequences to what is occurring today, and the goal should be to manage those and to avoid them going forward. Data alone won't solve all the problems. There clearly is a need for greater production capacity, and there clearly is a need to provide greater availability to the products going forward.

As important as the system that has been announced today is for monitoring blood, it is important that we address yet another and probably yet perhaps more critical shortage, that of recombinant products. So we would encourage the expansion of the program that was announced today as quickly as possible to include the monitoring and reporting on the recombinant products.

Thank you.

DR. CAPLAN: Thank you.

Comments?

[No response.]

DR. CAPLAN: Thanks, Mark.

All right, what I propose is this: we take an hour break for lunch. We will reassemble to talk a little bit more about monitoring plasma products when we get back. I think we've got a short recombinant DNA presentation.

DR. NIGHTINGALE: We do have a presentation by Genetics Institute, Wyeth, or whatever you are these days.

AUDIENCE PARTICIPANT: We can't hear.

DR. NIGHTINGALE: There will be a presentation by the entity formerly known as Genetics Institute immediately after the lunch break and we will conclude the agenda after that presentation. It is our hope that the meeting will conclude on or about 3:00 p.m. this afternoon.

MR. WALSH: Mr. Chairman, my understanding is that the Immune Deficiency Foundation has a presentation as well. Is that correct?

DR. CAPLAN: Right. We'll do the recombinant first and then go into those.

[Whereupon, at 12:30 p.m., a luncheon recess was taken.]

AFTERNOON SESSION

DR. CAPLAN: We have a presentation coming from Patrick Gage, who actually is someone I know and have known for some time from Wyeth. Some people have asked could we be updated on recombinant factor product work, and it turns out that we can.

So without further ado, Dr. Gage.

DR. GAGE: Thank you, Arthur. It's a great pleasure to be here. I appreciate the opportunity to talk to the Committee, Dr. Caplan, Dr. Nightingale, and all the distinguished members of the Committee.

I'm Pat Gage. I'm President of something called Wyeth-Ayerst Research, and since Stephen brought up the question earlier, who are we, Wyeth-Ayerst Research, the organization that I'm responsible for, is the R&D division for Ethical Pharmaceuticals for American Home Products, Wyeth-Ayerst. It incorporates Genetics Institute, which was acquired by American Home Products in 1997, and includes all of the R&D activities of Genetics Institute. And, of course, Genetic Institute, one of its principal programs has been in the area of hemophilia, recombinant factor production.

I'm also responsible for process science and manufacturing of protein pharmaceuticals, which includes all of our hemophilia products. And so it was great to have an opportunity to come and talk to you a little bit about our program and our commitment in the area of hemophilia.

This is just a quick summary of my ten minutes' worth of remarks. I'm going to talk a little bit about our mission, our heritage in the field of recombinant factors for hemophilia. In some sense, we're focused today on issues of supply. I'll talk a little bit about supply of recombinant hemophilia factors both for hemophilia A and also I'll mention our work in hemophilia B.

Our vision from the beginning has been to dedicate ourselves to the development of safer treatments for hemophilia, both in the area of, first, hemophilia A and then hemophilia B, and as we got into this to work with patients, treaters and families to assure that we provide the best service to the community that we can. We pledge ourselves to listen to the community and work with patients, treaters and families to make things better for those patients. And it's one of the reasons why we're here today, to try and listen and learn from this activity.

We feature ourselves as a leader in developing advanced treatments for hemophilia, and we also are proud of our outstanding record of manufacturing consistent supplies of factors for both hemophilia A and hemophilia B.

Leadership. We were the first to develop a recombinant Factor VIII product. This is a partnership that we formed with Baxter. We had the technology to clone, express and develop a manufacturing process for recombinant Factor VIII. Baxter, our partner, worked to do the clinical development and register the product, and currently markets this on a global basis.

This was the first approved recombinant Factor VIII product in 1992. It's Recombinate. It's a very important product to the hemophilia community, and we're proud to continue to manufacture a portion of the bulk drug substance for that product.

The Genetics Institute was also the first and the only company to develop a recombinant Factor IX product, BeneFIX, which was introduced in 1997. And, thirdly, we were the first to introduce a recombinant Factor VIII product formulated without the use of any albumin. So it's albumin-free formulated recombinant Factor VIII. That's ReFacto, which was introduced here in the United States in January 2001.

I want to talk to you a little bit about our supply situation. This slide indicates where we make these recombinant proteins. It's a very complicated picture because we have established manufacturing capacity where we can to try and get product to the market as quickly as we can.

Our principal manufacturing facility for protein products is in Andover, Massachusetts. We make bulk drug substance there for Recombinate Factor VIII and BeneFIX; that is, recombinant Factor IX. In fact, 75 percent or more of our manufacturing capacity in our Andover facility is dedicated to hemophilia products.

We make ReFacto in Stockholm which is supplied to us, and this is finished and supplied to the European and U.S. market. That's the recombinant Factor VIII albumin-free formulation product.

We are developing new facilities in St. Louis for the production, for enhancing our supply capacity for ReFacto, and we will be working there further to bring on a follow-on product which I'll discuss a little bit later, ReFacto AF, which we perceive to be the ideal recombinant Factor VIII product of the future.

And in bringing those products on, we will bring on additional fill/finish facilities in Greensboro, North Carolina, for ReFacto and ReFacto AF, as well as fill/finish facilities in Europe. And we've also brought on additional capacity for BeneFIX, because of the rapidly growing market for that product, through licensing a facility in Upper Merion, Pennsylvania, for the production of additional capacity.

Let me talk for a minute about BeneFIX. BeneFIX, as I said before, is the only recombinant product for hemophilia B. It supplies the majority of the global need for Factor IX. We have supplied this in an uninterrupted fashion since 1997. We're very proud of this. It's not only the science before what we do, but we're very proud of our performance in manufacturing.

And we've added this additional capacity which will come online at the end of 2002 or early 2003 to assure that we can have up to double our current capacity going forward. So we want to assure the community we're doing everything we can to supply this product according to the needs.

First-generation recombinant Factor VIII, again a partnership with Baxter. We were the first to do this, first product approved in Europe and in the United States. Manufacturing technology was transferred to Baxter. We continue to supply bulk drug to Baxter for that product, and that product in our facilities is manufactured in Andover.

In order to provide more product to the community, we elected to bring on a new product, ReFacto. This is the first FDA-approved albumin-free final formulated recombinant Factor VIII product. It's manufactured in Stockholm for us, where drug substance and drug product is produced for the world, but it's in very limited capacity and we are working really hard to enhance that capacity by bringing on the St. Louis facility.

We acquired the St. Louis facility, have been installing the technology, have been making product now, and we're actually in the process of filing for approval of this facility and auxiliary facilities to produce finished product for the United States.

Additional supply of this product is dependent upon the approval of these facilities, and we think actually that approval of these new facilities is our best way as a company to help address the severe shortage of recombinant Factor VIII products in the world.

The ultimate best product, though, in our view, for hemophilia A is one that is manufactured without the use of any animal- or human-derived substance throughout the manufacture. This is a standard we set with the introduction of BeneFIX recombinant Factor IX, which is produced using serum-free technology and culture, purified without the use of monoclonal antibodies and formulated without any albumin.

And we've developed the same technology for ReFacto and we are operating this process now. We will be undertaking clinical trials and hope to have this product approved early in 2003. We think this not only will provide the safest possible recombinant Factor VIII product for hemophilia A, but it will also allow us to significantly enhance supply of the product, hopefully to eliminate supply shortages in the future.

In summary, Wyeth Genetics Institute is committed to the hemophilia community, to meeting the needs for recombinant factors and for new treatments of the future, for example, gene therapy. We're doing everything we can to produce as much recombinant Factor VIII and recombinant Factor IX, and to bring on new capacity as quickly as we can.

We are working diligently with regulatory agencies both here in the United States and also in Europe to get these new facilities approved as quickly as we can. And we think we've planned never fast enough because we can never anticipate problems with other manufacturers in terms of issues that create shortages, but we think we have planned to bring on additional capacity that in the longer term will meet the needs of the hemophilia community.

With that, I'd like to stop and if there are any questions, I'd be glad to answer them.

DR. CAPLAN: Steve?

DR. NIGHTINGALE: Dr. Gage, thank you for the presentation. I would comment to you and to the Committee and the public at large that it has not been our practice in the past to have presentations that might be characterized as infomercials, and it will not become such.

Dr. Gage's presentation touched on an issue that is of concern to the Advisory Committee on Blood Safety and Availability, and it is simply this: It is the general practice of the pharmaceutical and biotechnology industries that as soon as they have received approval for a product that sells at a non-trivial price, particularly in a market where there is extraordinary demand, to have anticipated that demand and to be ready to go from the get-go, and I believe the get-go was licensure of March of 2001.

It is of concern to the Committee, to the government and to the public that as much safe product can be made available as soon as possible. And could you amplify for the public really the reasons, if you can, why we're six months after launch and we're still waiting?

DR. GAGE: You're talking about ReFacto?

DR. NIGHTINGALE: Yes.

DR. GAGE: Yes, of course, Dr. Nightingale. ReFacto was developed and filings were made essentially simultaneously in the United States and into the European Community. The European Community approved the product several months, many months before the U.S., and so we began to introduce the product into the European Community prior to the approval of the United States.

The Stockholm facility that we have, that we have access to product from, has very limited capacity. It's about 200 million activity units and the bulk of the product, because of the earlier launch in Europe, ends up in the European Community. About 40 million activity units of that comes into the United States.

Our goal for supplying the United States with higher levels is to bring on our St. Louis facility. But because of the timing of the regulatory approvals, rather than hold material back and not put it out for use by patients at all, we've put it where we were approved, and that was Europe.

DR. PENNER: This Committee has a stake in this inasmuch as we recommended the hemophilia population transfer its use of product from a plasma-based to a recombinant form, and that was with the supporting information from the manufacturers that that would not be a problem. However, it is a big problem.

As noted, just about all the centers--and we've got a number of representatives around here; Keith and others, I think, can attest to this that we just can't get recombinant Factor VIII in any way, shape or form. So a lot of patients now are going to have to be shifted, or are being shifted to the plasma-derived product which some have not been exposed to before. So I think there's a big problem that we're not understanding here from our earlier discussions over two years ago on this matter.

DR. GAGE: Maybe I could follow up to your question, Dr. Nightingale. In terms of timing for additional capacity, if things go well we would expect European approval of our St. Louis facility at the beginning of next year, late this year, beginning of next year. And at that time we will divert our entire European supply from our Stockholm facility; that vast majority of that material will come into the United States, significantly enhancing the supply here.

And then, of course, when the U.S. approval comes, which we expect to come somewhat later, that will significantly enhance the availability of ReFacto here in the United States. Our St. Louis facility has a capacity which is about three times greater than our Stockholm capacity. So when this capacity becomes available and becomes licensed, it's going to significantly enhance our ability to supply recombinant Factor VIII.

DR. PENNER: The time, then, you're saying is like February or March or April or November of next year?

DR. GAGE: It's the first or second quarter. Of course, we don't want to over-promise. We're trying to move it as early as possible in the first half. We have our own internal company goals, but we're saying in the first half of 2002 for European approval. And because of some difference in the application of the regulations here in the United States, it's more likely to be the third or the fourth quarter in terms of the U.S. approval.

So as I said, the best we can do is we supply St. Louis material to Europe and move the Stockholm material to the United States until the U.S. approves--actually, it's the fill/finish facility, Catalytica, in North Carolina that is the bottleneck right now in getting approval, not the actual--we don't anticipate the approval of St. Louis to be the bottleneck. It's actually putting the material in the vials and doing stability testing and getting the approval, but we are working with the FDA to try and expedite that process.

DR. CAPLAN: Larry?

MR. ALLEN: Doctor, do you have any idea of the demand, the needs in this country for this country?

DR. GAGE: We think we have--I mean, our staff working in the hemophilia business and R&D, I think, have a good awareness. We're always open to learning more. We met this week with officials from the National Hemophilia Foundation at our St. Louis facility to have a dialogue about this, to better understand the needs of the community, and to educate the community through the NHF about what we're trying to do to meet those needs.

I know it's a severe need. As I said, it's something that was unexpected and it came out of nowhere in terms of the problems with one of the major manufacturers. It takes quite a while actually to increase capacity for producing these kinds of products because of the need for new facilities and then regulatory approval of those facilities. So it's impossible just to turn on the spigot and get an enhanced supply, but we're doing everything we can.

MR. WALSH: Do you have a ball-park on whether your enhanced supply--what percentage of the market here in the U.S. it will satisfy?

DR. GAGE: We think with our ReFacto AF process we will be on the order of 1.5 billion units' worth of material capacity.

MR. WALSH: And what's the total demand?

DR. GAGE: That's a good question. It continually grows. I'm not in the best position to answer that question.

DR. CAPLAN: Larry?

MR. ALLEN: Just sort of a follow-up to what John was saying. Do we have any estimates on the demand the past few years? Is that something you're using as a build-to?

DR. GAGE: Maybe I would ask one of my colleagues, Neil.

MR. FITZPATRICK: My name is Neil Fitzpatrick. I'm the Director of Marketing for YFGI. The U.S. demand for recombinant products is about 1.2 billion units in terms of what we see. If you look at worldwide demand, certainly the European market is about equal to that, as well. So we're looking at about 3 billion units in terms of worldwide demand for recombinant products; with ReFacto AF, as Dr. Gage said, being able to supply about 1.5 billion of that.

DR. GAGE: And we actually hope that we would produce more than that, but that's our current--what we're projecting.

DR. CAPLAN: Keith, and then --

DR. HOOTS: Just a comment about that. I think those numbers are as good as any that I've been able to come up with. And as Dr. Gage said, the big thing is--the hardest part is estimating the degree of increase in demand, particularly worldwide, because as the quality of care rises and enhancing the developed world toward a more developed economy, if they can afford to purchase this, then their demand is going to go up tremendously.

So a thought that this will be half the world supply when it actually reaches this is probably not true, if that last five years has been any indication, because the worldwide use for Factor VIII has gone up substantially; that is, before the present shortage became so critical, in the last 5 years, probably as much as 50 percent.

So whether that will be sustainable over the next five years is anybody's guess, but if all impediments to usage were removed, the answer would be unequivocally it would be at least that or greater.

DR. GAGE: I could add something to that comment from Dr. Hoots. When we started the BeneFIX recombinant Factor IX product, we made a market estimate in terms of how many units we would have to make, and today we sell twice as many units as we thought the entire market was, not just the share we were to get. So these estimates are actually very hard to make, and by the time you start a project like we did with BeneFIX which was in the early '90s to final approval in the late '90s, of course, that market changes dramatically.

DR. CAPLAN: Okay, thank you, Dr. Gage.

DR. GAGE: Thank you.

DR. CAPLAN: Let's go right to Jason Bablak. We're going to spend a little bit of time here looking at other blood product supply issues and he's from the IDF.

MR. BABLAK: Good afternoon. My name is Jason Bablak. I'm Vice President of Public Policy for the Immune Deficiency Foundation, and today we're going to talk to you about a proposal that we've worked with some of the other stakeholders in putting together a system to monitor availability and potential demand for plasma derivatives.

So today we're going to talk about our proposal, and I want to keep this in mind as we go through all of this that the ultimate goal here is to effect a positive outcome in patient health care. And we get into the presentation, I'll explain how we think that might happen, but that is what we're looking to accomplish here.

What we're going to talk about today is a conceptual framework for how a system might be put in place and how it could be accomplished and run. We think that the implementation of this system needs to really be managed through this Committee and through the Department at HHS, and obviously it needs consumer and stakeholder input from this proposal, as well as when we move forward in creating the actual system.

In some initial conversations we've had with other stakeholder groups, we've come up with some ideas that would focus us as we created this system. The requirements would be it would need to be aggregate data. We wouldn't want to report data on any individual company or product. It has to be voluntary both from the statistics that we create out of this as well as participation by the consumers, physicians, et cetera.

The potential benefits to the consumer would be--these are just examples and obviously there will be more as we develop this system. But perhaps if there is a shortage, consumer groups and physician groups that represent those consumers and treat them might develop a standard for allocation in times of short supply. It could help potentially with reimbursement issues. Are the products being reimbursed? For what indications are they being reimbursed, and are they being reimbursed for adequately? It might be able to help us encourage participation in the patient notification system, and also safety issues. First and foremost, for our community availability is a safety issue.

So here what we're talking about, just so that we're all clear, IVIG, the different factor products, and as we go forward we can determine whether it's useful to break out, for instance, the plasma-derived into high-purity and different levels of purity or how we would want to collect that data, and then also the alpha 1 protease inhibitor.

I apologize. The plasma and recombinant factor units are in thousand units, not in units. But this is really what the market is for plasma products in the year 2000. We have data for some through PPTA. Others we don't have any data, and then for the alpha 1 protease inhibitor we have some data from the Alpha One Foundation.

Basically, our rationale for creating this system is that shortages of plasma derivatives result in adverse health consequences for consumers. IDF documented this, as we had a severe shortage of IVIG in 1997 and 1998, and we basically surveyed our physicians and consumers and determined that greater than 50 percent of immune deficient patients were suffering adverse health consequences in relation to the shortage.

Monitoring the supply of these therapies could provide advance warning of shortages, and this would allow consumers and physicians to make informed health care decisions, or at least more informed than what is currently out there. I think all the interested parties, including the consumer groups, physicians, government officials and this Committee, have agreed that this system would be useful and should be created.

I'm going to briefly just go over what I'm going to talk about in more detail, but just so that everyone is on the same page as to what we are proposing for a plasma derivative supply monitoring system.

Basically, we would survey hospital pharmacies, physician practices, home health care companies, hemophilia treatment centers and consumers on some regular basis and then take that information and report it to HHS and then make that publicly available. What we would do with it from there, I think, still needs to have discussion as to how we might interpret that and what groups might do once that data is made publicly available.

Starting from the beginning, we think there are probably four different areas, major areas, that you would have in a complete data set to monitor product availability. And I'll go through each of these in a little more detail, but the first would be information from manufacturers regarding projected releases. Obviously, the manufacturers are in a position to know what they're going to be releasing, when they're going to be releasing it to some extent.

Now, sometimes things come up that change things, but at least there's some level of ability of the manufacturers to have an understanding of what they anticipate releasing into the market over a certain period of time.

The data from manufacturers regarding the actual product distribution in inventory, and that's data that we are currently receiving now through the industry association; the amount of product in the pipeline, meaning the amount of product that actually has been released, and then where it is in the system of distribution; and then also we think that a demand model to project the future product requirements going out 1, 3, maybe even 5 years so that people could start making informed decisions based on what the market looks like, because it takes that amount of time if new facilities or new production is needed.

Regarding the information from manufacturers regarding projected releases, this could obviously provide an early warning for release difficulties. If a manufacturer is having trouble getting things out the door, they're the first to know about that and therefore they are in the best position to inform the community.

The problem with this is it's difficult to define, quantify and aggregate throughout a manufacturing industry, and so therefore our best hope here is to continue to work with the individual manufacturers so that they work with the communities that are affected if there are difficulties and to give us the early warning if that's possible.

The second area would be data from manufacturers regarding actual product distribution and inventory. And as I stated just a second ago, we're currently getting this through PPTA, and I think everyone has agreed that this provides valuable information. Unfortunately, it's only one piece of the puzzle and we need to supplement this with further distribution and use information, and that's where we come to the main part of our presentation, which is the amount of product that's in the pipeline.

So once it has been released from the manufacturers and before it gets to the consumers, where has it gone, where is it? And that's going to give us a more detailed look at whether or not there's a shortage.

So here we think this can supplement the current information that is already available from the manufacturers. This can provide a system to monitor supply and alert of potential product shortages. And I think this is important as well, is this sets the platform for designing and implementing a demand model for plasma derivatives, because once we start to collect some of the data, we'll begin to understand where some of this product is being used, how it's being used, and that sets us up so that we can then go to the next step of creating a viable demand model.

In the plasma derivative distribution chain, there are several areas where we should look to determine where the product is, and I have broken down into four levels and that certainly is not indicative of the level of importance, but just where they appear on the chart that is going to follow this slide.

The first is the major distributors, group purchasing organizations, national home health care companies. There's a finite set at this level and it would be akin to getting data from, we think, the manufacturers, and I'll talk about some difficulties that we expect to encounter at that level.

Level 2 would be the hospital pharmacies, hemophilia treatment centers, regional and local home health care. Level 3 would be treating physicians, and Level 4 would be consumers. And because the Immune Deficiency Foundation has the most expertise with IVIG, I've given just a sample here of the distribution of IVIG as it gets down to the patients, with some estimated numbers of participants at those levels.

So as you can see, at the beginning there is the manufacturer. There are approximately six U.S.-licensed manufacturers or distributors, brand donors, of IVIG. There are probably about ten major distributors, another ten group purchasing organizations, national home health care companies. And as you can see, the solid-color lines represent what would be a typical distribution, but there are also lots of dotted lines that make this a little bit more difficult to track on a 100-percent basis, if you will, like we are getting now from the manufacturers.

So what we're proposing to do is more of a statistical sample survey, and so we would have three different phases, basically, starting with the hospital pharmacies. And then we would have basically objectives of what we were trying to find would include what indications are being treated, does the supply meet or exceed demand, what products are facing shortages, which indications are facing shortages, what types of hospitals. Do hospitals limit products or indications?

And then we would do this basically by a monthly sample of a phone survey of 100 hospital pharmacies which would be stratified by hospital size and type. And this just shows you how many hospitals there are, I guess, in the U.S.

The next slide. This is a sample of one survey that was done. It shows you that there was an 83-percent success rate of actually getting through to the hospital pharmacists. So the take-home of this is that we think that it would be a short implementation time to get something like this up and running. It wouldn't cost all that much and we have likely participation from the hospital pharmacists.

Phase II would be prescribing physicians in the non-hospital setting, hemophilia treatment centers, regional and local home health care companies. Here, what we would be looking for are what indications are being treated, the same sorts of questions. And then the last one would be would the physicians curb demand for certain products or applications? So that is getting us a little bit more information on the use of the product. And this also would be a monthly sample, phone survey, of about 100 physicians, HTCs and home health care companies.

This is just--you probably can't see it from there, but this was a survey that the IDF sent out to our physician community when we had our shortage, and it's just to show you that we have experience doing this. We had a very high success rate in this endeavor and it was not very costly, and we had, like I said, lots of participation from our sample.

The next level would be Phase III, consumers. Here, we would ask basically is the product available in the amount and frequency prescribed because in times of a shortage things can change and so we would want to keep tabs on that. If there are particular products facing shortages, which consumers might be facing shortages? Are they in certain regions of the country? Are they in certain areas, rural versus urban? Has the frequency or dose changed? Has patient health been adversely affected? And have reimbursement or insurance problems been encountered?

And this we would do probably as a semi-annual sample survey of 400 consumers per indication, and we might either do that on a rolling monthly number or do it at two different times of the year. We haven't completely decided what might be the best way to do that. Once again, we did a survey of our patients and had pretty good success with that as well.

So this was the slide I started out the detailed presentation. So it just goes through and summarizes the details that I just talked about.

The next steps: we think we need to get consumers and stakeholders together on a working committee or advisory panel to basically take this and put it into an implementable form, identify the general contractor who would oversee this type of project, and then the subcontracts of who would be doing different parts of either the data-crunching or sending out and developing the surveys, that kind of thing. We need to develop the data collection reporting system and identify funding sources.

Long term, this is a list of the different parts that I have talked about. The first one is the manufacturers' estimates of projected releases, and I think there we will encourage the manufacturers to continue to inform us when problems arise, but there's not much beyond that I think we can do.

Manufacturers' distribution and inventory data we are currently receiving, and I understand that there's always a process there where they might either do it more frequently or do additional types of data collection.

The two that are white, the distributors' estimates of product in inventory and the information platform for estimating product demand, are things that we'll probably need to work on in the future. But the three in the middle are what we kind of talked about during this presentation as the things we think we can put into place pretty quickly and start to get some real data on the supply that is in the pipeline.

So for the future we would talk about how we would get the major distributors, group purchasing organizations and national home health care companies to participate in this kind of system. We would talk about making a demand model for plasma derivatives, and perhaps incorporating other relevant data sets.

I know that in the hemophilia community there is data that is collected at the hemophilia treatment centers in the CDC and there might be a way to start incorporating some of that data into some of this usage data to develop a demand model and other types of issues.

So one issue I just want to talk about quickly is getting the distributors in. Basically, the issue here is does the manufactured product equal what is available in the system. What we would want to do is real-time monitoring of the amount of product available at the top of the distribution system, and this could be done in a couple of different ways.

One way would be a monthly survey of key distributors. Another way would be to create a reporting system similar to what PPA does at that next level. So there are a couple of different things that could be done.

There are some requirements for that I won't go into, but I think the important one is on the bottom, is that we need consistent participation by all major distributors to make this data meaningful, given that it's a very small set and to do a statistically significant sample of that would be, I think, difficult.

These are the strengths and limitations of distributor monitoring, and I'm going to have, I think, two slides on doing some demand modeling. And basically there are some things we need to get some further information on, which is basically indications for which prescribed, number of users per indication, average amount prescribed per use, and then the average frequency of use per user. Some of these in different communities are more well-known than others.

And then these are some dynamic factors: Are there new prescribed uses coming online, are there off-label uses that we're not aware of, what's the percent diagnosed within the community, what's the age at diagnosis, the life expectancy of a chronic user and is that changing? The same thing with the weight of the chronic user, recommended levels of dosage--as we know, sometimes that can change as well--the price, and insurance coverage. So these are all things that, if we're going to create an accurate demand model, we need to start getting some answers for.

That is the end of my presentation. I'd be happy to answer any questions.

DR. CAPLAN: Let's do a question or two.

Keith?

DR. HOOTS: First of all, I'd like to applaud your efforts here. It's a very good first pass, I think, at what needs to be done. I think that getting back to just kind of the parting words, I think, that were in the morning session talking about portability, we're spending some time cogitating about the difficulties of that.

I think that in order to assure portability, you have to have this kind of data, particularly about the supply line, the pipeline, because only when you identify where the problems arise can you create strategies to transfer the specific product in a way that would allow the patient to have it.

An example would be--and we're all, you know, unfortunately way too familiar with this scenario--your company provides you with insurer A. They change your coverage to insurer B. Insurer A has coverage with a given home care company for the product you need. Insurer B doesn't recognize that contract and doesn't have a similar contract. They have somebody else who, in a time of shortage, may not even have the product you need. So how do you get your product now because you now have a provider who doesn't have a pathway into the system?

If we knew exactly what inventory issues were involved, then we could probably suggest, without even having any governmental participation, some real reasons why maybe a slightly above cost transfer could occur between suppliers. That might be one way to circumvent the portability issue without having to get into a lot of legal and governmental mandates about it.

MR. WALSH: I'd like to commend the IDF as a member of the Committee, as Keith has done, as well as on behalf of the Alpha 1 community. And I appreciate the inclusion of the other consumer communities in the development of some of these concepts and the commitment from IDF to collaborate very closely with the plasma user communities in the further design of the system and ultimate implementation and oversight.

Our Alpha 1 experience--you know, since 1993 we argued for direct consumer allocation because of the inequities in distribution and the portability issue. And I think we're a simple case in one sense and a complex case in the other. With only one manufacturer on the table, direct consumer allocation ended up equaling direct distribution, and that certainly doesn't work for all communities and that is certainly understandable.

But we were able to make certain that 300 consumers that didn't previously have access to product now have access to whatever product we can make available to them by cutting out some of the distribution inequities. So I applaud Jan Hamilton coming forward with the Hemophilia Federation and her statement to move toward that in the hemophilia community, and I know that immune deficiency community has similar problems in many circumstances.

The Alpha 1 community did not make a statement today because we embrace the situation with the hemophilia community and their critical shortage of recombinant product, and want to commit ourselves to do whatever we can to help them out. We have one manufacturer, and I won't beat a dead horse here. The Committee well understands our situation. Demand exceeds supply, and it's going to for however many months moving forward.

We don't have any immediate relief on the horizon. There are two companies submitting PLAs for additional I.V. products, and there are four-plus companies developing aerosol products. The aerosol we won't see for 4, 5, 6 years, if we ever see it. The I.V. products, we don't have any specific projections or comfort level that there will be a market any time in the near future, but we are committed to work closely with the FDA and the industry to get fast-track approval and licensure as soon as possible. Both of those trials are completed.

Finally, you have before you and the Committee has copies of a letter that we distributed from our medical director in the Alpha One Foundation to all consumers on augmentation therapy in the Alpha community, and this was our way of dealing with the shortage.

Whereas we should be getting normal allocation of 28-day supplies shipped to us on a regular basis every 28 days, it is extended well beyond 32 and 34 days now, and increasing every week. So we have a critical shortage. We've got people missing windows for transplants, we've got people having many more exacerbations than they had 12 months prior.

We just went through a 60- to 90-day period in which we didn't have any product at all. So asking our community to try to stockpile a little bit for when we get sick or during flu season or when you travel or have a family event is a little but unrealistic right now, but that is what we've asked our community to do.

We've also reached out to the physician community, the treater community, and asked them to make certain that they have written the prescription properly, and actually given them the formula so it's 60 milligrams per kilogram per week and not 90 and not 120, and make sure you don't have a 400-pound gorilla that you're writing a prescription for. It's really a 200-pound male gorilla.

[Laughter.]

MR. WALSH: So, you know, we're doing whatever we can to stretch the limited supply of product that we need, and we further appeal to this Committee to assist in whatever way and extend our appreciation to this Committee for its support in the past and moving forward into the future and embrace this effort to do data monitoring that is ultimately going to have an effect on health care.

We strongly suggest that as distribution data is designed to change behavior and positive effects on patient health care, we really need to clearly articulate what effects that may be. In our community, we'd like to take the leadership role that the NHS MASAC and IDS MASAC has taken and actually look at an algorithm or treatment protocol for dealing with prioritization of product during allocations.

And if somehow there could be a facilitation to provide that through each individual community--it's different for each indication--I think that would be very helpful. And our community is going to try to work through that process, as the hemophilia and immune deficiency community has.

Larry Allen expressed at lunch that there are real specific problems related to the sickle cell community that I know he wants to talk about as well. But again I thank the Immune Deficiency Foundation for the leadership role here, and we pledge our support to work with you and the other consumer organizations and stakeholders to design a program that will work.

DR. CAPLAN: Larry?

MR. ALLEN: Well, first of all, we in the sickle cell community are looking forward to collaborating with the other foundations and all the members here in regard to these issues. And one of the things that we did discuss over lunch was the standards of protocol for transfusion. It's different around the country and that's something we want to bring to the table at some point here so we can resolve that issue.

Thank you.

DR. CAPLAN: I think what I'm going to ask for is the--I want to thank Jason for his remarks. I'm getting a sense that there might be three matters of business we do after we hear from PPTA. One is we probably should say something about the IDF proposal, and we could extend it out to keeping tabs on blood products more generally just using that as a paradigm, if you want to think of it that way.

It seems to me we may want to say something also about where we are with the sentinel system, if we want to express some desire to see it solidly founded, grow, permanent, expanded. And then perhaps Ron had something that he wanted to put on the table, a possible resolution that he talked to me about which he may or may not want to do.

Those are three items that I've got for the Committee action. There may be other things that you've got, but those are three that I've got.

So let's go to the last presentation. It looks like Chris Healy.

MR. HEALY: Good afternoon. My name is Chris Healy and I'm the Executive Director for PPTA North America. As you may know, PPTA is the trade association and standards-setting organization for the world's major producers of plasma therapeutics. Our members include Alpha Therapeutic Corporation, Aventis Baring, Baxter Bioscience, Bayer Corporation, Grupo Griffels, Octopharma, and ZOB Bioplasma.

PPTA supports the efforts aimed at providing consumers, regulators, treaters and other interested parties with information and data that will assist in rational decisionmaking. To this end, PPTA has made industry-aggregated U.S. inventory and distribution data publicly available for the last three years. We believe that these data have served as a surrogate early-warning system during times of critical shortage.

During the more recent Factor VIII shortage, PPTA began publishing the inventory and distribution data on a bimonthly basis. And as I'm sure you're all aware, our member companies are working diligently to rectify this shortage and to provide consumers with their preferred product choice. However, it's unlikely that there will be a positive, major change in the current supply situation before the end of the year.

Turning to the data issues addressed today, we're pleased to lend our support to the concept of data collection aimed at the total future usage of plasma therapeutics. This is a key data set that will help industry, consumers and regulators alike in terms of planning to meet future need.

In short, industry's current data collection efforts tell us where we are today and the proposal for total future shortage will tell us where we want to be tomorrow. With this information in hand, companies can begin taking steps to reach these future goals.

We acknowledge the desire of IDF and other organizations to obtain better information about plasma therapeutics held in the distribution pipeline and agree that this warrants further discussion and consideration. However, in terms of accommodating supply shortfalls and increased project demand, we believe that the current data collection and data collection regarding total future usage will better serve consumers, regulators and the industry.

In closing, I'd like to thank the consumer representatives and the members of this Committee for the appreciation you've expressed with respect to the industry's current data collection efforts. We've been working diligently to provide this information in as timely a manner as possible and we recognize that the more timely the data, the more valuable it is. As a result, we're examining ways to provide the data even more timely and provide it to you on an even more timely basis.

Thank you again for your support of the initiative and thank you for the opportunity to address the Committee.

DR. CAPLAN: Questions?

[No response.]

DR. CAPLAN: Okay, thank you.

MR. HEALY: Thanks.

DR. CAPLAN: Well, let me not put a motion forward, but explain what I was thinking about with the IDF and blood product modeling and tracking. It seems to me that we might want to say something to add blood products to the sentinel system that has already been developed and perhaps go further along the IDF lines both in terms of scope and in terms of detail.

In some ways, they must have had a longer meeting than they did yesterday about dimensions to cover. We've got a more developed proposal there in some ways. But be that as it may, it seems to me since we have a group that is vulnerable to shortage in a particular way, counting becomes very important and then managing scarcity becomes very important in terms of what might happen were things to become scarce on the donation side. So the Chair is very interested in hearing perhaps a motion or expression of support along those lines.

Ron, did you want to say something about the thing we talked about?

MR. GILCHER: My comments really address the issue of availability. On February 28th, I believe it was, 1999, there was a meeting at the NIH, convened by NHLBI, where approximately 20 individuals from the Red Cross and 20--these are CEOs from the independent blood centers, were invited to a one-day discussion on the issues of blood availability and recruitment of blood donors.

The conclusions of that meeting were very interesting, although not a lot has come out of them. The conclusions were three-fold, first that there was inadequate reimbursement to the blood centers, and we certainly know that issue, and that if there were better reimbursement, blood centers could spend more dollars on recruitment.

The second issue was that there needed to be support from the top down--I'll say that and explain it in a second--and that there needed to be support from the bottom up. Support from the top down was specifically that the highest members of government, specifically the President, the Vice President, Cabinet members and critical legislators, really needed to support the importance of the blood supply in this country, and specifically to address leaders within the country, specifically at the industrial level, about the importance of allowing the employees of their organizations to donate blood, and to do it with time off while they're at work.

And, of course, the third issue, then, was educating from the ground up meant addressing children really at the grade school level. And, for example, ABC has put in a "Your Blood, My Blood" program really to do that.

My objective today is to propose what Dr. Penner and I discussed, and I loved his term, a no-lose situation, as opposed to a win-win, where we would propose to, in fact, address the very highest levels of our government, specifically the President, to address the issue of the importance of the blood supply to this country and to encourage industry to support the donation of blood. That's a quick summary.

Thank you.

DR. CAPLAN: And we did hear some testimony this afternoon and this morning from groups asking for more attention, if you will, to the donor supply side. So that seems in the spirit of some of the things we've been trying to look at.

Well, let me move back, then, to my third possible issue and we could see what bubbles forward. I don't want to get in the way of this nascent effort at HHS which has come forward about the sentinel system. If it's not useful to have us jump up and down and say go fast or spend more and keep it around a long time, then I guess we shouldn't. But if it is useful, then I think we should.

So I don't know politically whether it matters or not. If it gets in the way, then maybe that's not such a prudent thing to say right now, but it becomes very clear, you know, from all that we've heard that if we're going to get into any issue of safety versus availability and we don't have any numbers and we can't detect changes relatively fast by some standardized mode of counting, we fly blind. We've flown blind for a long time. It's probably time to start looking out the window.

So I don't know. What do you think? Is that useful to have?

DR. NIGHTINGALE: This is the time in the meeting when, if I want Art to go in a different direction, I usually have my elbow in his ribs. My elbow is plainly visible.

[Laughter.]

MR. WALSH: Mr. Chairman, what if we try to craft a motion that includes the three areas that I think we've talked about today that at least are clear to me? One is to support the continued implementation of the sentinel program with regard to blood supply, data monitoring for blood supply, and take into consideration recommendations made by this Committee and the public.

Two would be to coordinate with the American Red Cross and the American Blood Banks to establish a standardized protocol to capture collection data that relates to the sentinel program, a comprehensive view; and, third, to support the design and implementation of a plasma derivative data monitoring system, as presented by the IDF, and encourage all stakeholders to participate in that process. And I'm open to any change in language.

DR. HOOTS: Just a friendly amendment to say something to the effect that at least for the first and the third that there be appropriate resources considered, if not allocated, at least projected about this so that we can get it done not only in a timely way but in a way that, like we discussed this morning, gives data that we can all be quite comfortable in using to make projections about the future.

MR. WALSH: I think that's a good point.

DR. CAPLAN: Paul?

DR. HAAS: In the same sense, not only plasma-derived products, but recombinant products.

DR. KLEIN: I'd like to also make a friendly amendment. I think one of the things we felt was lacking this morning, as I see it, was the suggestion of a steering committee of some kind so that we have some expertise directing not only where the pilot is going and how it functions, but kind of the long-term stability of the entire enterprise. So I do think we do need to include in your wording somehow the idea of an infrastructure that would be able to include people with expertise and experience in this area.

MR. WALSH: I probably should have waited and crafted a more carefully articulated resolution, but I would concur with all of the friendly recommendations.

Do we want to try to redraft that, Mr. Chairman?

DR. CAPLAN: These recommendations are so friendly and the text is probably clear enough on the record that I'm going to suggest, instead of trying to write it out, that we ask Steve to write it up and on this one we go for approval if you want to do it by mail or we can make minor changes there. I'm not hearing anything that people are saying they have to see us sort of go to the mat on language on this one, unless you want to do it.

So that might open the door to a motion.

MR. WALSH: So move.

DR. HAAS: Art, may I just --

DR. CAPLAN: Yes.

DR. HAAS: Maybe I just don't understand close enough, but it seems to me we want to be very careful about monitoring blood supply and distinguishing that from plasma recombinant products.

As I understand the plasma recombinant process, it's much more complex, and if we lump them together I'm afraid we'll get results that are not going to be helpful. So I think if we're going to have steering committees, maybe we need two and then maybe we need oversight over that. I hate bureaucracy and I just created one, but I think we have to be careful about the different sets of data.

DR. CAPLAN: Well, let me say as an ethicist I love bureaucracy and I think that it keeps people off the street.

[Laughter.]

DR. CAPLAN: But why don't we go with the understanding that we're calling for the monitoring of these three areas in the amendment and we can, if you will, break out how to structure that?

I mean, I understand exactly what you're saying, Paul, but I think we don't want to confuse what it takes to monitor A, B and C. So on the record, we'll ask Steve and Mac to kind of give us that proposal that says we want to monitor A, we want to monitor B, we want to monitor C, requisite infrastructure, long-term commitment, use the IDF proposal to get some idea on the plasma products, maybe form a committee to think about how to do it on the recombinant thing.

But, again, my hunch is on this one we don't really need--we'll be here forever if we try to craft it and we'd do better to look at a draft is what I think.

All right, so there's a motion rattling around there.

MR. WALSH: Yes, so moved.

DR. KLEIN: Second, second.

DR. CAPLAN: All right. Any further discussion?

[No response.]

DR. CAPLAN: All in favor?

[A chorus of ayes.]

DR. CAPLAN: Opposed?

[No response.]

DR. CAPLAN: It looks like it's unanimous. Okay.

If we were going to summarize Ron's motion, it might be--I don't mean to take the three parts you presented, but a key part you presented was that we try to direct the Secretary to ask the President and the administration to make blood donation at this critical time a top priority for the American people, and industry to speak out on it and to try and make it easier for people to donate, and time off and this sort of thing.

That seems to me--I'm trying to summarize here where we're going, but it seems like a very timely moment to do this. People are paying attention to what's going on on the supply end. It might be good to ask the Secretary to do that. I don't know whether he can do it or not, but we could certainly ask that that be done.

DR. NIGHTINGALE: If I may make a comment--this is Steve Nightingale--the Secretary has on numerous occasions made clear his interest in promoting blood, as well as organ donation. And I believe when you go into his offices, which is where Jay and I were this morning, on the table is not candy, not a small piece of candy; it is a large button, too large to swallow but also large enough to see. And I think even from the back of the room you can see "Donate Life." I think you will be preaching to the choir, and people who join choirs are often very receptive to preaching.

DR. CAPLAN: Are people amenable to this notion, then, that we ask the Secretary to take his well-known enthusiasm for this subject and transfer it throughout the administration, trying to mount an organized effort to push for blood donation at this important time and to encourage steps that will make it easier for Americans to do that? Do you like that language?

Motion?

MR. GILCHER: So move.

DR. KLEIN: Second.

DR. PILIAVIN: I would like some more specific language along the lines of what was originally proposed to speak to the captains of industry because that really is where the problem is, where the roadblock is.

DR. CAPLAN: Okay.

DR. PILIAVIN: They don't want to spend the money that it will take to give people time off.

DR. CAPLAN: So we can add specifically in a sort of friendly amendment mode something about making sure that people have the ability to take time off from work, and that industry, universities and other institutions support their ability to do that without penalty, is what we're talking about.

MR. DALAL: Art, as a member of industry, what evidence do we have that industry is the roadblock to stimulating donor recruitment?

DR. PENNER: I've got information.

DR. CAPLAN: John, do you want to go there?

DR. PENNER: Just experience with the auto companies that at one time there was no problem in getting a set-aside time for the workers to come down, donate and go from there. That quickly tightened up as things got a little tougher in selling cars and the monies were a little less, and then suddenly that was turned off. So the union was not able to just mobilize workers to come down for a 100- or 200-unit donation. You probably have the same experience.

DR. HAAS: It's very similar. I sit on the Blood Services Committee of the Western Lake Erie Region of the Red Cross, and every time we have a discussion about donations that very point is coming up that industry that used to be very receptive now is less likely to even have a donor drive. And when they do have a donor drive, they don't get as many participants.

MR. DALAL: "Industry" is a very broad term, as we all recognize, and each one of us may have a story where there was a hurdle, either temporary or something more than that, that got in the way of a group donating blood. I would argue that there are numerous anecdotes and stories where corporations have gone out of their way.

When I lived in New York City, I was aware that the New York City corporations were particularly supportive of blood donations and the captains of industry in New York City were supportive. And I can say that my corporation and biotechnology companies support blood donation.

So I think the way Art described the resolution, which was more broad, was something that I could support. But I don't think singling out industry without more evidence that if you focused the President and other senior individuals in government on industry that you would solve the problem.

DR. HOOTS: Would you accept or would Jane accept a friendly amendment that might address--instead of using the word "industry," what if we said "employers?"

DR. PILIAVIN: That sounds fine.

DR. PENNER: You just really want to encourage them to do this because it's really giving company time for the donation. It's not just saying, well, you can do it here after hours. It's during working hours is what we're after. Is that what happened in New York? Did they give time off for them to donate, because that was what happened in Detroit for a while and then it was just shut down very quickly and the union couldn't get through?

MR. DALAL: I would say most blood drives in corporations that I am aware of--and I'll acknowledge that that's a small sub-set--occurs on the company premises, on company time, during work hours.

DR. DAVEY: Actually, if we could, either in a subtle or not so subtle way, also encourage these higher government officials to not only support donation but to donate themselves, I think it would be a great idea. Actually, the last President who donated was Carter and that's quite a while ago, and some people have speculated that if Gore had donated, he might have got 500 more votes in Florida, but we don't know.

[Laughter.]

DR. KLEIN: The Secretary donated on Tuesday at NIH.

DR. CAPLAN: Ron?

MR. GILCHER: Yes. I just want to say again that what I'm talking about is really generic, and I can tell you that in our own area very clearly as we see more foreign ownership occurring of large organizations, and I'm not going to single those out, we have seen decreased donations; that is, they clearly do not want to give the employees time to donate. That I can actually give you data on.

So I'm really saying that if the top members of government would support the importance of the blood supply--that's my number one point--and the importance of donating, those two points need to be in the resolution, the importance of the blood supply to our great nation and the importance of being able to donate. We would go a long way toward opening doors because it would become the way.

DR. CAPLAN: So on the table here are whether we get a little more specific about trying to remove obstacles when they exist in employment or university--I'm saying university, too, because this has come up at our place about donation. Are they in, are they out, can they take the time off? It has happened at Penn.

Do you want to get a little more specific or not? And then we could say, Steve, just when this resolution moves, call attention by PSAs, maybe a national day devoted to, that kind of thing. I mean, that's what we're really pushing here, is the--I also think it's important to work with religious groups on this area. If you can really get your thing in sync and have everybody speak out at the same time, I think that would be very powerful.

However, those are subsidiary ideas. The main question is do we want to follow Jane's suggestion and maybe it a little bit or just let it go and come with more general language about removing obstacles, which I'm open to ideas about?

MR. DALAL: I might just add another observation. I think at the previous Advisory Committee meeting a young lady went on record and spoke about her company's efforts to use the Internet to appeal to the younger generation of donors.

I bring that up simply as an example of different approaches to appeal directly to individuals to overcome behavioral, psychological, other kinds of hurdles which may be as important in recruiting a new generation of donors in the United States as, say, getting employer support and the support of corporations.

MR. GILCHER: Raj, that is the education from the ground up. Very clearly, if our high school students do not understand social responsibility and community responsibility, we're all done. That's why we have to start at the grade school, so we need both. But specifically at this point in time, we really need to get support from the top down. If we don't have support from the top down, we won't get the support from the bottom up. Support from the top down will lead to support from the bottom up.

DR. HAAS: It's always a problem of trying to decide what type of message we want to send. There's a huge message out there that includes everything that we were talking about--the teenagers, the college students, the workers, the non-workers, everybody--and that clearly has to happen.

I'm aware of some social science that is talking about the social capital, people's willingness to be involved in activities has gone down rather significantly over the last generation. So there's a huge problem society-wise that relates to this very issue that we're talking about. And the more I think about potential ways of doing that, the more I'm afraid that we cloud the message.

And maybe I tend to agree more with Ron right now that if we can get really sharp message coming out of the leaders of this society and really pound on that, that may be the best way to go.

DR. NIGHTINGALE: May I ask if the following conveys both the sense of the entire Committee and the concerns that Mr. Dalal expressed?

Would it be the sense of the Committee to encourage the most senior managers of the government to support efforts by both the private sector and community-based organizations to remove obstacles to donation of blood?

I think that what the Secretary would do with that is say not only blood, but also organ donation as well. I think that would be very congruent with his current stated policy.

DR. CAPLAN: Ron?

MR. GILCHER: Steve, as part of that I think it's so important to have in this resolution that the President address the importance of the blood supply to the nation as part of this.

DR. KLEIN: I was going to say precisely that. We need to make this a national priority, and a national priority starts at the top of the nation and I think that's number one.

COL. FITZPATRICK: We have an effort which has not come to fruition yet to get Secretary Rumsfeld to sign a letter encouraging DoD participation. A quote from here certainly wouldn't hurt that letter probably getting signed.

The other thing is rather than removing obstacles, it might be better to put it in a positive note and extol the virtues of successful organizations like the Saturn Group and that sort of thing. I'm sure Steve can do that.

DR. CAPLAN: So we might try some language that goes more speak out on, set a national priority for it at this--I want to say at this important time because we are facing an interesting, important time just now about supply, and then encourage Americans to follow the lead of those organizations that have shown success in terms of encouraging donation and make than a priority of the administration, is what we're talking about.

COL. FITZPATRICK: Even to the point of I think Congressmen who recognize constituents on the floor or in a record --

DR. CAPLAN: Right, right.

COL. FITZPATRICK: --that that goes a long way toward something. So even if there was just some recognition of those successful organizations in a national forum, that goes a long way to getting other groups wanting to get that same recognition.

DR. CAPLAN: Jane?

DR. PENNER: I think Ron had it right.

DR. CAPLAN: Jane?

DR. PENNER: No, I'm sorry.

DR. CAPLAN: Jane, then John. Let's do it that way.

DR. PENNER: All right, go ahead.

DR. CAPLAN: Jane first. He's so excited, he can't --

DR. PENNER: I thought we were equal these days.

[Laughter.]

DR. PILIAVIN: But he had already said me.

DR. PENNER: Then it's yours.

DR. PILIAVIN: I don't want to be the person who goes against this sort of "let's all be upbeat and happy and emphasize the positive." But I would like to point out that the current administration is sort of in tight with business and sort of shares their values and is unlikely to come up with this idea on its own of specifically speaking to business organizations regarding bottom-line issues. I mean, it's just not going to occur to them.

This is why I think it may need to be said specifically, perhaps not in the resolution but perhaps when it is presented to whomever it is presented to. I would be very unhappy if--and these may be anecdotes that should not be pluralized into data, but I have heard for years from blood donor professionals that this is an increasing problem.

And we all know that the way to get an organization, a specific one organization to cooperate in blood drives is to start at the top and work down, and that usually involves the executives of those organizations both being an example and providing space and time for their employees to do it. I have been associating with blood bankers for over 20 years now and I have heard this story consistently and more and more urgently.

And, finally, as a social scientist the effectiveness of moral appeals has not been demonstrated very well. It sounds good, and people nod their heads and then they go about their business. And what does work is providing structural support for people to do the things that they know they ought to do, and structural support--in the past, I know that many blood donor organizations have built satellite centers to make it easier for people to go to fixed donation centers.

Obviously, the long practice of taking bloodmobiles to places of work is an example of trying to remove barriers and obstacles in the way of time and effort and thought for getting people to donate. And all of a sudden, if you make it a national priority that this is something you ought to do, those social science--I don't want to say laws because I don't think we have any laws--those social science principles are not going to go out the window.

And we do need to emphasize trying to do all of those things that will make it easier for people to do the right thing, and I don't think just saying, oh, let's all be happy together is going to help this.

DR. CAPLAN: So you reject the "Kumbaya" principle?

[Laughter.]

DR. CAPLAN: John?

DR. PENNER: I agree with Jane, also, but Ron's, I think, approach going directly to the President level is, I think, the way to get impact. And as was stated, from his aspect it's really a no-lose situation. You can't really come out any better than apple pie and mom and blood.

DR. CAPLAN: Well, let me try a suggestion here and then take a few more comments if they're out there. One way to handle specificity so that people have something concrete to work with is to simply list in an IE form we want this to be a priority. It's vital at this time that the administration draw attention to it. Situations such as when there are obstacles in gaining access to the opportunity to donate are when situations exist where people can't get time off from work to do that, that sort of thing.

We could do some of those in a list, you know, an IE kind of thing. It doesn't quite then get into blanket statements about all of industry. We're just sort of saying when it happens or if it happens, these kinds of situations. And I do think it's true that in a tighter economy it's going to become harder to take that day off, get that time off to do this.

So would that work to handle some of that concern, Jane?

DR. PILIAVIN: Yes.

DR. CAPLAN: Yes, all right.

Other comments, and then maybe I'll hunt for a motion here.

DR. KLEIN: Art, if I could make a comment, I think perhaps one way we might want to word it if this is going to be a national priority by the highest levels of the administration, then what those members could do, they could enable government employees to donate. They could encourage leaders of industry to allow their employees to donate and they could appeal to all eligible Americans to donate.

MR. WALSH: Mr. Chairman, as we're referencing examples, if you do that in an IE or EG formula, just a reminder that the Five Points of Light was an excellent example of a lot of corporations getting together for multiple awareness for donations.

DR. CAPLAN: Steve?

DR. NIGHTINGALE: I'd like to introduce a procedural point. The way that the proceedings of this body are communicated to the public are two ways. We post the transcript on our Web site as soon as it becomes available and we post a summary of the meeting, not a short by any means--I think the mean is about 16 pages; the median is slightly less, but in the same range. And both will reflect the sense of the Committee.

But to make something specific--and here I'm speaking to the various reporters. I don't see ABC Newsletter, but I'm sure you're here somewhere. I will make an effort to craft these to suit the sense of the Committee and I will submit them to Art for approval as part of the minutes.

Would that procedure be acceptable to the Committee?

DR. CAPLAN: The Chair would take a motion on that and then we'll go into the discussion mode off of that.

MR. WALSH: So move.

DR. CAPLAN: Okay. Second?

MR. GILCHER: Second.

DR. CAPLAN: Discussion?

DR. KLEIN: I would frankly like to see the wording of the resolutions before they are made public. Maybe other members of the Committee would as well.

DR. CAPLAN: You can get them on the Web site.

DR. NIGHTINGALE: No, you wouldn't put them on the Web site.

DR. CAPLAN: I don't mean the Web site. I mean e-mail.

DR. NIGHTINGALE: Yes.

MS. LIPTON: E-mail.

DR. NIGHTINGALE: Yes, yes.

DR. CAPLAN: So we should be able to e-mail these around.

Okay, with that proviso, all in favor?

[Several hands were raised.]

DR. CAPLAN: Opposed?

[No response.]

DR. CAPLAN: Okay.

DR. NIGHTINGALE: Actually, before we go to the third, there is probably--I need to make a comment, I think, in regard to what Dr. Klein may have been referring to.

Before I was able to come to the room today and after I was able to come, there have been discussions about forming executive committees and sort of management and stewardship of the blood monitoring effort. This particular function, as I indicated in my presentation, was transferred to the Office of the Assistant Secretary of Health on January 8th of this year, and that is where responsibility for it is and remains.

To the extent that there is an executive committee for it, that executive committee is the immediate Office of the Secretary, and I expect that to remain there for the indefinite future. I certainly hope that it will. And I believe that it is the sense not only of the Advisory Committee but the people who have attended meetings the last two days that it stay up there and not be delegated.

And I think, finally, it is my intent to continue to seek input from the widest possible spectrum of views, and it is my own view that appointing an executive committee to funnel that information would be premature at this time.

DR. CAPLAN: Okay, so the last area, which was do we want to say something about sustained funding, supporting what has been done, moving it ahead rapidly and keeping it where it is.

DR. KLEIN: I'm not sure that I quite understood the last comment, Steve, but I think the terms that were used earlier had to do with a steering committee with expertise. And if you feel that that is not a necessary function, well, I think you probably ought to say so and that ought to be open for discussion.

I frankly believe that in most large studies expertise is something that is desirable, as well as participation by those individuals who are involved both in the public sector and in the federal sector.

DR. NIGHTINGALE: I seek the widest participation from those who have such expertise.

DR. CAPLAN: Well, if you had a steering committee that had some money, it doesn't necessarily have to usurp the location or administrative control. It's just there for steering.

Al, sitting in for Jay.

DR. WILLIAMS: Alan Williams, sitting in for Dr. Epstein. One other consideration. Folks with expertise might not have the time to put into steering committee activities, but there are a lot of folks with expertise that could contribute to a workshop. And I think a very targeted workshop to address some of these key issues that we need to flesh out would probably help a lot in the process.

DR. NIGHTINGALE: Yes. We had a workshop yesterday and I would like to integrate the suggestions of that workshop before proceeding. I think that we plan, among other things, to have a conference call of the contractors on the 25th and I would like to get the input of all the contractors before proceeding further.

Where I'm coming from is that I'm not prepared to limit my options for input to this process at this time. That will come, but the more input that we can get, I think the quicker we will reach public consensus. And I think that limitation of input at this time would reduce the chances in the long run of public consensus for the end product, regardless of its merit.

DR. CAPLAN: Mary?

DR. CHAMBERLAND: I think perhaps some of the unease that has been expressed by members of the Committee and members of the audience about the effort to get this pilot project underway and then its long-term life, if you will, might be assuaged if there was some sense that this effort was structured in a more--at least using language that is more traditionally applied for multi-center research endeavors which usually function around an organizational structure where there is a principal investigator. There is epidemiologic and statistical support, as needed.

And while I think it's very good and laudable that Steve and now Alan's suggestion about getting input and advice, either actively seeking it or in a workshop format, is very good, when push comes to shove you really need a small number of people to develop a protocol--definitions, procedures, monitor the data, and then have the ability to statistically manipulate it.

And I think that I in no way want this to be construed as anything negative about the efforts that Steve and Ginny and others in his office have put forward in a very short period of time, but I think that Steve would be the first to admit that, you know, his office is not organized to conduct research, at least not at this point. I mean, it just doesn't have that kind of personnel in place, at least that I'm aware of.

And perhaps if there were, you know, efforts to see if some of these gaps could be filled, maybe that would assist in alleviating some of the concerns that have been expressed.

DR. NIGHTINGALE: Yes. The way that I would hope that those concerns would be alleviated is that there are three bodies that are actively involved in the review of the process at this time.

The first is the group of 29 prime contractors. They were chosen partially on the basis of their geographic distribution, but I believe both the list and the folks who appeared today would indicate that they were also chosen in part on the basis of their expertise, and the expertise in that group is very substantial.

The second group that has been asked to comment is this Advisory Committee, and I would respectfully but very firmly disagree with the statement of the American Association of Blood Banks that this Committee doesn't have the expertise to contribute to it.

I'm looking around the room and I see a lot of it, including the President of the American Association of Blood Banks sitting next to me. There's very substantial expertise, and I believe the CEO--and I have the President of the American Association of Blood Banks, neither of whom have been bashful about giving me advice.

The third group is the--I'm not sure what we call it, but, Alan and Richard, we have an internal Public Health Service blood availability committee that met by teleconference last week and will continue to be actively involved in it. I do plan, as soon as I have a halfway scrubbed database, to provide it on a regular basis to them. Alan is the only person outside my office who has got the raw data right now, but that will continue.

If you want to get to the bottom line of why do I have my feet in the ground, it is that the Department has an imperative, which is to get an effective monitoring system up as quickly as possible. And an advantage of having it in the Office of the Secretary, as I indicated earlier this morning, is that sometimes you can move faster in that office than you move elsewhere. Speed is a distinct concern here. It's not the only concern, but it is a distinct one.

DR. CAPLAN: Ed?

DR. GOMPERTS: Steve, I first of all want to acknowledge the really outstanding contribution that you and your group have made. Just witness what took place earlier today, for example, hearing from the American Red Cross the positive notes that they sounded, and ABC, et cetera.

So there really are two issues. There is the political one, which you are clearing driving and are very successful at. The second issue is the data that is going to be generated, and the validity of that data has to be unquestioned. That's really the bottom line, and I think this is probably what Harvey is driving at.

DR. NIGHTINGALE: Yes, and my response to that is that there are two issues for validity. One is the accuracy of the data that is collected. And as I indicated earlier, by requiring in the contract to each of the 29 participating sites that they incorporate the data collection as a standard operating procedure, this makes the data subject to verification. So I believe that that issue is addressed.

The second is the issue of analysis of the data, and when you have unscrubbed data there are limitations to how far you want it to go. I put the data out yesterday in what was a public forum with a great deal of thought about the uses, and for that matter the abuses of the data. I was not prepared to present that at one public meeting and not another public meeting. It is in the domain.

And the decision about how much of that is mine, and you never expect that any decision that you make publicly will be endorsed unanimously, but I did explain the reasons. And the reasons for putting that data out were to encourage as much comment as I could from as broad a variety of sources that I could in the future design of the project.

I also said that the project is initially designed over a six-month period so that there is reevaluation at the time. I'm not prepared for closure at this point on what the second phase of this data will be. There are a lot of folks with interest in the data. There is also a lot of concern that any entity, and particularly the United States Government, might spin this data for its own purpose.

I am very sensitive to that concern and I am taking very substantial steps to minimize that concern. I can't make it go away completely, but I've done the very best I could and will continue to do so. Given the volatility of the data right now, I think that the current course is the best one.

DR. CAPLAN: Karen?

MS. LIPTON: One suggestion, then, Steve, and I do understand that you're trying to get this up as fast as possible. But I still think that there is a sense around this table that people would be more comfortable with a more traditional structure, and perhaps if you could be persuaded to say you'd come back at the end of the pilot with a recommendation on how it could be structured on an ongoing basis which would you allow you freedom to do what you need to do now, but I think allow all of us some comfort around the table that in the end it is going to have, as I think Mary said, more of a feel of a traditional research project.

DR. NIGHTINGALE: I'm a little short on the answer because I think when you bring a project at this stage of development to this public a forum, most would think that that commitment was already implicit in the action.

DR. CAPLAN: Well, how about a sense, then, listening to this discussion, that the Secretary be encouraged to move this sentinel system forward as quickly as possible, that funding be appropriate to what is required?

It didn't seem to me that you were carrying around too much money in your 29-and-stopping budget. Didn't you tell us you went to 155 and got shut down? So this is not a lot of money to try and monitor the system. So we could go with a resolution that says move it forward quickly, fund it adequately to get it up and running, and then move to set up an appropriate supervisory infrastructure.

I mean, I think we're going to want something there once it exists. And you aren't a biostat person anyway, so what are you going to do?

DR. NIGHTINGALE: I'm going to bust my budget to hire one.

DR. CAPLAN: That may be, but it's the appropriate infrastructure whether you give it away or contract it out. I don't know. You could fight that with the Secretary.

So does that seem like a reasonable--not that I make resolutions.

CAPT. McMURTRY: It's already seconded.

DR. CAPLAN: All right.

All in favor?

[Several hands were raised.]

DR. CAPLAN: Opposed?

[No response.]

DR. CAPLAN: I think that may conclude our business, unless there are some other motions that people want to bring forward.

John?

MR. WALSH: I have another no-lose one here, and it's that time again. HCFA has been kind enough to send representatives to our last two meetings. I don't know if there's a HCFA rep here today. Is there? No.

At any rate, the Committee was very helpful, and Dr. Nightingale specifically, with the Secretary helping with the outpatient prospective payment crisis that we had last year. And that is still looming out there and it was a tentative solution, a band-aid, if you will. So I would propose the following resolution to see if we could rally support from Secretary Thompson as follows.

For purposes of the Medicare outpatient prospective payment system, the Department of Health and Human Services Advisory Committee on Blood Safety and Availability recommends that all plasma-derived and recombinant analog therapies be placed in permanent separate payment categories, similar to blood and blood products. Maintaining unique reimbursement will ensure that hospitals' current and future costs are recognized and safeguard patient access to these critical therapies.

DR. CAPLAN: Discussion?

MS. LIPTON: Well, I don't think you want to be treated like blood is treated under reimbursement.

[Laughter.]

MS. LIPTON: Oh, outpatient, okay, okay.

MR. WALSH: Yes, this is for outpatient.

MS. LIPTON: Right.

MR. WALSH: This is not inconsistent with the Committee's position last year.

DR. CAPLAN: Does someone want to move that? Oh, I'm sorry, we did. Excuse me.

All in favor?

[Several hands were raised.]

DR. CAPLAN: Opposed?

[No response.]

DR. CAPLAN: All right.

Rick?

DR. DAVEY: I don't have a resolution, but just a quick question perhaps, Art, for you or Steve about the CJD deferrals. There's been a lot of discussion, as we know, in the past few months about these impending deferrals and the discussions involved a number of organizations--the FDA, ABB, Red Cross, ABC, New York Blood Center. And those discussions have been very useful and progressed the issue along quite nicely.

And the TSEAC committee has also been very involved in this, appropriately. Those folks are experts on preon [ph] diseases, but they're not experts on blood. So it puzzles me a little bit why, during the course of these important deliberations on this issue, this Committee, the Committee that is charged to advise the government at the highest level on blood safety and blood availability, really hasn't been involved in this process.

I've been puzzled by that. I just wonder if there are any comments on that, please.

DR. NIGHTINGALE: My comment is that there is a time for everything, including committees' participation. At one point in the classic CJD deferral, we considered that issue here. I think the question at the time from my own perspective was that if the issue--the interest of the government is in airing an issue publicly and fairly and openly. It is less pecking order of one committee or another, and to some extent one picks and chooses one's committees not so much on the basis of their appropriateness for a task but whether or not the task is getting done. I think that there may be a role for this Committee to play in that discussion in the future. I very much hope that it becomes unnecessary for us to play that role.

I hope I wasn't speaking too cryptically there. If you'd like me to unwind that a little bit, challenge me.

DR. CAPLAN: John?

DR. PENNER: Just one quick item. For the next meeting, I'd like to request a report on the status of the hepatitis C lookback.

DR. CAPLAN: Steve, who is on and off any further departures, aside from the Chair?

DR. NIGHTINGALE: Well, I was saving this for the last word. Is this the last one?

DR. CAPLAN: I think so.

DR. NIGHTINGALE: Okay. There are six people whose contributions I would like to acknowledge. They are the members whose terms expire on September 30th of this year, and this really comes from the heart. If there was irritation in my voice before, there's no irritation in my voice right now. These are good people who have done the public a real service and it is an honor to be the spokesperson for the government to thank them.

Dr. Fernando Guerra is not here today. He is at work at the Baird County Health Department and could not attend this meeting. His expertise in public health and his overall wisdom, his personality, have been invaluable to the Committee.

It's easier, I suppose, to speak about somebody who's not here, but the sentiments that I have for Dr. Guerra are shared for every other member of the Committee who is here, so I'll try not to embarrass both of us. This is no tears, no hugs at this point, but a very sincere thanks.

Dr. Piliavin. Let me say one thing about Dr. Piliavin. In the early stages of this Committee when it was much less cohesive than it now is, we had a very difficult discussion over classic CJD in January of 1998. Dr. Piliavin was the person who set us on the right track. And the subsequent successes that the Committee had have many parents, but I think that her contribution just on that day alone was comparable to the contribution of any other member.

Jane, I'm obviously speaking on behalf of all of us when I say thank you, and not only for that.

It's going to be a challenge to embarrass Dr. Haas and Dr. Kuhn to the same degree. I'm probably not really up to it at this point, but that is not because I don't want to be. Their contributions to the broad functioning of this Committee, the perspectives that they have brought to the Committee, have represented not only the communities that they perhaps obviously represent, but the community that we're all trying to represent, and that is the American public.

I think I can embarrass Dr. Busch a little bit. The original challenge of the Committee was to find consensus on hepatitis C, and the real challenge, however, was to find consensus based on science rather than on emotion. I believe that the validity of the Committee's action, and perhaps why I was as supportive, perhaps even defensive of the Committee's expertise a few moments ago relates to Dr. Busch's contribution to the resolution that we achieved with the hepatitis C issue.

That resolution was based on fact. The fact that that resolution was possible was based on Dr. Busch's untiring scientific, honorable efforts.

Michael, again, I speak for the Committee, I speak for the government and I speak for all affected by that in saying thank you and congratulations.

Dr. Caplan, I don't know how to say this. Your leadership is beyond my capacity to say thanks. I believe there are many people in the room who could say it better than I, but I've said it as best I could.

Having said that, now let's come back to reality for a minute, which is what is going to happen next. I do not know. There is a new administration in place. We have solicited nominations. The nominations remain open until the 31st of August. The procedure that we have followed in each year is to receive nominations until that date, and I would encourage anyone who is interested to call (202) 690-1351. That is Captain McMurtry's number. He is the Deputy Executive Secretary of the Committee and he will be at his phone next week to answer those questions.

We will prepare a nomination packet--there are very strict laws on this--that goes through about five departmental filters before it goes to the Secretary. The final decisions on these matters are the Secretary's and I do not know what the Secretary's thinking is on this because I haven't asked him and he hasn't told me. But I will and he will, and I will let you know as soon as I do.

One last time to you 6 and to you 18 and to the additional 6, thank you very much, all.

[Applause.]

DR. CAPLAN: Dana?

DR. KUHN: I know that they usually give a dying person their last request, and I know in some States they often give retiring judges their last wishes. In this case, I just have three wishes, or I guess requests that I would like to have as I am terminating my time with this pleasurable Committee which I have enjoyed.

And I would be remiss if I didn't follow up on some of the consumer organizations and the requests that they have had in the past which I would hope that this Committee would visit in the future, and that was the comments of revisiting, I think it's a 30-year-old national blood policy, and discuss the need for the development of a new national blood policy, a better one, if there can be a better one.

And then the second is to discuss the need and the development of a national no-fault compensation program, as I have seen the history that we have. In the past, there has been a lot of litigation, needless litigation, and I think there can be some lessons learned from the past and applied to the future, and I would like to see that discussed, and then also to reiterate what Dr. Penner said. I think it's time for a follow-up report and discussion on the progress of our hep C recommendations in the past.

DR. CAPLAN: Okay. Well, I think that may be all of the Committee's business today. When is the next meeting, for those staggering back on?

DR. NIGHTINGALE: Without my Palm Pilot in front of me, I can only guess that I believe it is the 2nd and 3rd of--if the 2nd and 3rd of February are a Thursday and Friday --

DR. CAPLAN: And while we're looking that up --

DR. NIGHTINGALE: That's when it is, and it's here.

DR. CAPLAN: I want to thank the members of the Committee who are leaving for their service. The Committee has always struck me as a fascinating group of people who came to these issues with a lot of goodwill. I was talking to Jane the other night, trying to decide if anybody had ever listened to us over the first four years, and I sort of think if we were batting about .400, that was about where I would put us relative to things we started. But, of course, that would put us in the all-star hall of fame by some lights.

All right, so the meeting is adjourned.

[Whereupon, at 3:29 p.m., the meeting of the Advisory Committee was adjourned.]