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.]
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