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Blood Safety Transcripts
DEPARTMENT OF HEALTH AND HUMAN SERVICES
ADVISORY COMMITTEE ON BLOOD SAFETY AND AVAILABILITY
Twelfth Meeting
THE ROLE OF VARIOUS CONSIDERATIONS IN DECISION
MAKING RELATED TO NEW AND EXISTING
BLOOD SAFETY MEASURES
8:10 a.m.
Thursday, August 24, 2000
Hyatt Regency Capitol Hill Hotel
400 New Jersey Avenue, N.W.
Washington, D.C. 20001
P A R T I C I P A N T S
Arthur Caplan, Ph.D., Chairman
Stephen D. Nightingale, M.D., Executive Secretary
Larry Allen
James P. AuBuchon, M.D.
Michael P. Busch, M.D., Ph.D.
Mary E. Chamberland, M.D.
Richard J. Davey, M.D.
Jay Epstein, M.D.
Colonel Fitzpatrick
Ronald Gilcher, M.D.
Fernando Guerra, M.D.
Paul F. Haas, Ph.D.
William Hoots, M.D.
Dana Kuhn, Ph.D.
Karen Shoos Lipton, J.D.
Paul R. McCurdy, M.D.
CAPT McMurtry
Gargi Pahuja
John Penner, M.D.
Jane A. Piliavin, Ph.D.
David Satcher, M.D., Ph.D.
John Walsh
Jerry Winkelstein, M.D.
C O N T E N T S
AGENDA ITEM PAGE
Welcome, Roll Call, Conflict of Interest Announcement 4
Comments by the Assistant Secretary for Health and Surgeon
General David Satcher, M.D., Ph.D., Department of Health and
Human Services 7
Centers for Disease Control and Prevention Hepatitis C
Initiatives, Harold Margolis, M.D., Chief, Hepatitis Branch,
CDC 37
Statement of Issue, Stephen Nightingale, M.D., Executive
Secretary, Advisory Committee on Blood Safety and
Availability 91
Comments by Chairman and Members 99
Comments by Public --
Lunch 191
Committee Discussion and Recommendations --
Adjournment --
P R O C E E D I N G S
DR. NIGHTINGALE: Good morning. My name is Dr.
Stephen Nightingale, and this is the 12th meeting of the
Advisory Committee on Blood Safety and Availability. Could
I begin by calling the roll? Dr. Caplan?
CHAIRMAN CAPLAN: I'm here.
DR. NIGHTINGALE: Mr. Allen is in transit. Dr.
AuBuchon
DR. AuBUCHON: Present.
DR. NIGHTINGALE: Dr. Busch?
DR. BUSCH: Present.
DR. NIGHTINGALE: Dr. Chamberland?
DR. CHAMBERLAND: Present.
DR. NIGHTINGALE: Dr. Davey?
DR. DAVEY: Present.
DR. NIGHTINGALE: Dr. Epstein?
DR. EPSTEIN: Here.
DR. NIGHTINGALE: Colonel Fitzpatrick?
COL. FITZPATRICK: Present.
DR. NIGHTINGALE: Dr. Gilcher?
DR. GILCHER: Here.
DR. NIGHTINGALE: Dr. Gomperts is unable to attend
today. Dr. Goosby I believe is in transit.
Dr. Guerra?
DR. GUERRA: Here.
DR. NIGHTINGALE: Dr. Haas?
DR. HAAS: Here.
DR. NIGHTINGALE: Dr. Hoots?
DR. HOOTS: Here.
DR. NIGHTINGALE: Dr. Kuhn?
DR. KUHN: Present.
DR. NIGHTINGALE: Ms. Lipton?
MS. LIPTON: Present.
DR. NIGHTINGALE: Dr. McCurdy?
DR. McCURDY: Here.
DR. NIGHTINGALE: Ms. Pahuja?
MS. PAHUJA: Here.
DR. NIGHTINGALE: Dr. Penner?
DR. PENNER: Here.
DR. NIGHTINGALE: Dr. Piliavin?
DR. PILIAVIN: Here.
DR. NIGHTINGALE: Dr. Secundy is unable to attend.
CAPT Snyder?
[No response.]
DR. NIGHTINGALE: CAPT Snyder would be in transit.
Mr. Walsh?
MR. WALSH: Here.
DR. NIGHTINGALE: And Dr. Winkelstein?
DR. WINKELSTEIN: Here.
DR. NIGHTINGALE: The public service notice
regarding conflict of interest that has been read at the
beginning of all prior meetings of this Committee is
included here by reference and will be read in full after
Dr. Satcher's and Dr. Margolis' comments.
Dr. Caplan will introduce Dr. Satcher.
CHAIRMAN CAPLAN: I'm very pleased that the
Surgeon General is here with us again. We have been
wrestling for some time with a variety of issues, and as I
think he knows, today's meeting has more of an open agenda
than we've had in the past in terms of trying to respond to
requests by him and the Department. We're going to touch
upon a number of topics that this group has wrestled with in
the past, and I suspect members of the Committee may bring
up a few others maybe that are brewing as we go along.
But I know he's going to comment to us about a few
of the issues that we have been wrestling with and bring us
up to date. So let me turn the floor over to the doctor.
DR. SATCHER: Thank you very much. I'm again
delighted to be able to join you at least briefly--I'm
sorry. Thank you. I'm delighted again to be able to join
you, if only briefly, to again express our appreciation for
the outstanding work that you continue to do and also to
help try to put in perspective how we see the challenges and
certainly the significance of today's meeting.
This summer, in response to your request, our
office prepared summaries of both the origin of this
Advisory Committee and your accomplishments to date, and I
think all of you have now received the formal summary as of
June 12th, probably, and the latter summary you should have
received by now. I think it was sent out on August 4th.
There are six broad areas which you have addressed
in a very productive fashion: hepatitis C lookback, the
issue of the transmissible spongiform encephalopathies, the
shortage of plasma derivatives, the shortage of blood
products, reimbursement for blood products and plasma
derivatives, and the management of error in transfusion
medicine.
In each of these areas, I think we've made
substantial progress, and we want to thank you for your
contributions. But as you know, there are many challenges
which remain, and now is certainly not a time to relax our
vigilance or lessen our efforts when it comes to assuring
the safety and availability of the blood supply.
Let me take a minute to review our progress with
the hepatitis C lookback, which was the first major issue.
A guidance to the industry that incorporated your January
1999 recommendation was published by FDA on June 17, 1999.
A proposed rule based on this guidance has been prepared by
the Department, and it is presently awaiting final review by
the Office of Management and Budget. And I think you're
probably familiar with that process. The target date for
completion of direct notification is September 30, 2001.
We've received assurances from the blood industry
that direct notification will be completed within that time
frame, and we have reviewed assurances from the
Commissioner--we have received and reviewed assurances from
the Commissioner of FDA that her agency is prepared to
monitor compliance with this process.
Our general notification program for hepatitis C
is also underway, and Dr. Alter provided you with an update
on this program last January, and today I understand Dr.
Margolis will provide you with an additional program update,
in fact, in just a few minutes, and this is very important.
But I do want to mention something else. In
January of 1998, when the Secretary announced the initiation
of the hepatitis C lookback efforts, she acknowledged your
first set of recommendations on this subject, but she also
said something else which I want to remind you of. She
said, "I intend to go even beyond your recommendations. I
consider these steps to be only the first phase of a
comprehensive plan to address this significant public health
problem. So it is my intention to reach effectively as many
people at risk as we can. Today's decision," she said,
"will allow us to move immediately to address concerns among
transfusion recipients at greatest risk. At the same time
we will educate the public at large, evaluate our efforts,
and take even more steps to address unmet needs as we
identify them."
And this is what we have tried to do with the
hepatitis C lookback. This is the same approach we've taken
to the other challenges, I think, to blood safety and
availability that we've encountered, and we hope to continue
to do so.
But I do want to mention the latest effort in this
regard because we have had discussions about sending a
letter from the Surgeon General to every household in
America about hepatitis C because of the magnitude of this
silent epidemic affecting four million people. And the
struggle, of course, has been that there's only one model
for doing that, I guess, in the past and that was when
Surgeon General C. Everett Koop sent a letter about the
HIV/AIDS epidemic.
There have been some major changes since that
time. At that time the Surgeon General's office had what we
call franking privileges, and there was no problem in
sending mail to all of the families in America. Congress
has long since withdrawn that, and now only Congress has
such privileges, so it would cost us between $30 and $40
million to send such a letter.
However, in discussions with leaders in Congress,
especially Congressmen Bliley and Coburn and Brown and some
others, we agreed that we would take advantage of the
franking privilege of the Congress to send the letter. So
on July 27th, we held a press conference with members of
Congress and announced that, in fact, a letter from the
Surgeon General would be sent to all the constituents
through congressional mail. And so we have moved forward
with that in terms of putting together a letter, and the
Congressmen have sent letters to all of their colleagues
urging them to transmit this letter.
There are a couple of things that we have to deal
with. One has to do with the separation of the different
branches of government and to make sure that the letter is
written in such a way that it doesn't imply any breach of
that separation. So we'll be changing a sentence here or
there from what you received, especially the one that says,
"We have joined with the Congress." We're going to say that
differently.
[Laughter.]
DR. SATCHER: So nobody will misunderstand what
we're saying.
The other issue, which I think is a very important
one, is we are sending letters to all of the households,
hopefully, and we hope that it will get the desired
response. Now, if you ask me if we're prepared to deal with
the desired response, that raises another issue that we
should be aware of, and that is that there are not adequate
federal funds to support the state and local level programs
to respond if people will, in fact, as we would like for
them to do, if everybody would come seeking testing and
treatment as indicated. But I think that's a good problem
to have in the sense that--the good problem, of course, is
that we have put the problem in perspective by sending this
letter, and hopefully it will lead us to move forward to
make the resources available to deal with this issue. We
want to deal with it. So that's sort of where we are there.
Today I understand that one of the things you're
going to be discussing is the role of various considerations
in decision making related to new and existing blood safety
measures, or I guess to answer the question: What are the
principles on which a policy to assure a safe, available,
and affordable blood supply should be based?
So we certainly look forward to the outcomes of
this meeting, but I also want, before in conclude, to
express our appreciation to those members of the Committee
whose terms are expiring. I want to thank Mr. Allen, Dr.
AuBuchon, Dr. Gomperts, Dr. Penner, and Dr. Secundy for your
contributions to the Advisory Committee during your
three-year term, which ends, I guess, on September 30th of
this year. But we certainly want to assure you and to
assure those who are not here that they have not seen the
last request for public service, and we hope that they will
be as generous with their talents in the future as certainly
they have been as members of this Committee.
Now, one final comment. In addition to advising
us on blood safety, I want you to know where all of this
fits into the bigger picture of Healthy People 2010, the
nation's health plan for the next ten years. And I think we
released that plan on the day when the government was closed
down, and we were having this national meeting, and you met
and I remember coming here to meet with you. But we did
something for Healthy People 2010 that we've never done
before in Healthy People. We have now over 400 objectives.
We felt that there needed to be a national strategy based on
easily definable and a set of indicators that we could
easily communicate to the American people. So we came up,
with the help of the Institute of Medicine, with leading
health indicators, and that's what you have at your desk.
You have a bookmark of the leading health indicators.
It's interesting. Five of them we say are
lifestyle and five are health system indicators. And what
we hope to do over the next ten years is use them as we use
leading economic indicators. We want to monitor the
progress, and we have measurable objectives on each one of
these indicators. And we hope to monitor the progress of
the nation toward reaching these measurable objectives.
Now, I won't go any further than that except to
say you might want to take any problem that you're concerned
with and ask yourself how these indicators would impact upon
that. I did that last week with cancer, and you might want
to just go through and say if your concern is reducing
mortality from cancer in this country, how would each one of
these indicators relate to that? I think you'll be
surprised to find that virtually every one of them has an
impact on the risk for cancer.
I want you to know that because I want you to help
us also with the whole issue of Healthy People 2010 and
moving this nation forward to meet those goals and
objectives.
Thank you very much.
CHAIRMAN CAPLAN: Do you have a minute for some
questions?
DR. SATCHER: Sure.
CHAIRMAN CAPLAN: Okay. Why don't I open the
floor for questions, comments. Dana?
DR. KUHN: Dr. Satcher, I want to just thank you
and the CDC for the efforts you put through in putting--in
this "Dear Citizen" letter. I think it is much timely and
much needed, given the fact of how long we've been dealing
with the issue of hepatitis C.
But two questions I wanted to ask you, because I
think what I'm hearing you say is there's a little concern
about how Congress has withdrawn the franking privileges of
the Surgeon General. Is anything being done to reinstate
the franking privileges? And then, second, is anything
being done to request funding perhaps in this next budget so
that this letter can go out to the citizenship of the United
States?
DR. SATCHER: Well, the letter is going out. It's
going out through the good graces or the support of
Congress. So that part is taken care of. They have made a
commitment to send this letter to their constituents. So it
does raise the next question that you raise. What will be
the impact of this letter and will we be prepared to deal
with it?
If we receive the maximum impact, it would stress,
severely stress the system as it now exists and really bring
into sort of bold perspective the fact that perhaps there's
not adequate support for the state programs in this area.
We know that already. However, we don't know how many
people will need--how many people will have insurance
coverage and things like that as they seek to respond, how
many people will go to the public system.
We could envision a situation where the "public
system" was, if not overwhelmed, severely stressed. And
we're aware of that, and we hope that Congress will continue
to increase funding in this area.
But certainly I can tell you that the Association
of State and Territorial Health Offices are concerned about
being able to respond properly.
CHAIRMAN CAPLAN: Jane?
DR. PILIAVIN: Your talking about how the letter
is actually going out to all the households makes me think
in terms of our unhoused people. Is there going to be any
way of trying to reach them? Clearly, many of those people
have substance abuse problems, and almost none of them have
access to medical care.
DR. SATCHER: That's a general problem that we
have with all of our programs, and I can only say there are
different levels of programs in different communities
throughout the country now dealing with that.
You know, one of the best examples of that has
been, I have to recall, dealing with tuberculosis, when we
had the dramatic rise in tuberculosis from '85 to '92,
almost a 7 to 10 percent increase a year. What we
discovered was that much of that problem was in the
population of homeless people in this country, so we had to
develop strategies, including directives of therapy, to
reach them.
So there are strategies that can deal with this
issue, and CDC certainly is always looking for new
strategies, and maybe Dr. Margolis will comment on that.
But, yes, we are concerned about that population, and jails
and prisons, homeless, those are major challenges for our
public health system. And it really raises the bigger
issue: To what extent is our public health system now
relevant to a population in which so many people are in our
jails and prisons? For example, I believe the rate of HIV
infection in jails and prisons is like eight times what it
is in the rest of the population. So any public health
system that does not respond to that and which people
are--the jails and prisons are part of the community.
People go in and out, in and out. So whatever goes on there
is actually a part of the general community, whether we like
it or not. And, therefore, increasingly our public health
system must include how we deal with the jails and prisons.
We're trying to deal with that. As you know, my
Deputy Surgeon General, Dr. Ken Moritsugu, before he became
Deputy Surgeon General, was Medical Director of the Bureau
of Prisons. So we have improved our working relationship.
CDC has some targeted programs both for the homeless and for
people in jails and prisons, working very closely with the
correctional institutions. So we are going to be sending a
letter.
I didn't respond to your point about franking, and
I can't. You know, it's a tough issue. Congress made this
decision, and I guess there has even been a ban on using the
franking privileges now for a period of time.
DR. NIGHTINGALE: We discovered that you cannot
send franked mail within 90 days of an election. When we
had the press conference on the 27th, it was the impression
of both the executive and the legislative staffs that that
ban was 60 days. A lot of work has gone into this so far,
and obviously a lot of work remains to go into it. The
commitment is to do the work.
DR. SATCHER: But there are a lot of things--to
get back to your basic question, there were a lot of things
that happened to the Office of the Surgeon General during
the last four years, between '94 and '98, in terms of
reduction in budget and things like that that have been a
problem for us. We have found ways to compensate for them
in many cases to get reports done and to get them out. But
it's not because of the resources in that office. It's
because we have managed to use the agency resources, and
they have been great in providing them and responding to
requests to get things like the mental health report and the
oral health report and the tobacco report done, not because
of money in our office but because we have been able to work
with other people, and now working with Congress directly in
getting this letter out.
I took a deep breath on that one.
DR. GUERRA: Dr. Satcher, again, thank you very
much for making the time to join this Committee at our
meetings.
You commented about the public health
responsibility and certainly the increasing demands on a
system that has not always had in place the kind of
structure and support that is very much needed. And
certainly one of the very important considerations relates
to the identification of individuals that have infection
with hepatitis C virus and that are progressing with their
illness and that obviously need a variety of interventions
for more clearly defining the status of their disease and
hopefully connecting them to some treatment.
But on the public health side of that, we
certainly need to make sure that they are protected against
hepatitis A and hepatitis B in the instance where they're at
risk for those diseases, and also that they have access to
detox programs when they're substance users or
alcohol-dependent, which obviously is a comorbidity that
puts them at even greater risk.
The resources are not in place to do that. In the
instance of my own community, where in a relatively short
period of time we presently have over 5,000 individuals in
our registry for hepatitis C that we have identified just
with some minimal efforts that we have been able to support
through our own department for screening and counseling,
doing the testing. But we don't always have enough vaccine
for the adult population because they're not eligible for
the Vaccines for Children Program to protect them against
hepatitis A or B, and then also to try to get them into some
system of care.
Unfortunately, many of these, as you well know,
are within some of the minority communities, for instance,
within the Mexican-American or Hispanic and Latino community
that has many who are uninsured and/or marginally employed
and not eligible for any benefits.
In the instance where they do have some coverage,
you know, managed care organizations will not often cover
the preventive measures and/or the work-up that needs to be
done.
Is there something taking place at the federal
level to try to at least put some of that responsibility on
those systems that are in place for serving uninsured or in
the instance of managed care organizations that they do have
an obligation to their patient populations? What is your
sense also of the availability of additional vaccine program
support for these populations?
DR. SATCHER: Well, let me just say I think we
continue to review our relationship certainly with managed
care organizations who contract for the care of Medicaid
populations, and I think that is going to continue. It is a
very serious problem, because we have had cutbacks in
several areas in terms of the budget, and as we look at our
budget situation today, it is really critical that we look
at the areas of need in public health.
Those have been documented very well by, again,
the Institute of Medicine's report in 1988 talked about a
public health system that is in disarray, and documented the
loss of infrastructure, especially at the State and local
level. We have been trying very hard to rebuild that
infrastructure. We have made some progress, in terms of the
State public health laboratories, for example. Most of this
progress has come about in response to emerging infectious
diseases and the threat of bioterrorism.
We have a long ways to go, as you point out, so we
are looking for strategies of public-private partnerships to
reach the populations in greatest need. You know,
ultimately, obviously, we keep dealing with a system that
is, in some ways, well-funded, but not well-organized, in
terms of the need for universal access and dealing with the
lack of universal access in this country, which is one of
the things that led the World Health Organization to rank us
so low among the nations of the world. I think we're ranked
number 37 in terms of health systems efficiency.
So, part of what we're trying to do is figure out
a way to adequately fund the system and then organize it in
such a way that it operates most efficiently for everybody.
So, the gulf between here and there is still pretty wide.
You know that better than I do, because you have been on the
front line a long time, and, obviously, our relationship,
federal, state, and local, putting together a public health
system that is well-coordinated, is one of the real
challenges that we face.
CHAIRMAN CAPLAN: Mike?
DR. BUSCH: Yes, Dr. Satcher, I would also like to
commend both you and the Public Health Service for the
enormous focus and contribution in the area of blood safety.
I think today the risk of blood is extraordinarily low, and
the progress is really a tribute to the system and to the
incredible accomplishments of technology, in applying it to
blood safety. I think the discussion over the next day will
be how much additional resource can we afford to put into
closing that last bit of the window period or adding safety
measures, such as leukodepletion, which have marginal safety
benefit.
So, we're struggling with the enormous resources
that are required to further reduce risk in blood safety. I
think there are two related areas, though, where a modest
amount of resources could have a high impact. One of the
other charges of this committee is the availability of
blood, and there remains a serious problem with blood
donations.
About half the blood centers in the country now
are struggling to collect enough blood, and I know that
about a year ago, there was discussion about a public health
sort of campaign where yourself and others were going to do
television commercials, et cetera. I have not heard much
about that. We have seen in Canada an enormous impact
through an orchestrated public health campaign to increase
blood donations, and I think that is an area where I could
imagine Give Blood being added to this list, because I think
that giving blood is a process that encourages safe health.
The process of going through that interview, understanding
risk behaviors, et cetera, could ramify out in terms of
broader public health safety implications for individuals as
a whole, as well as, obviously, it is a good, you know,
general sort of ethical behavior.
So, that is one area I would like you to comment
on; the other is the issue of blood safety around the world.
We heard, at our last meeting, the reality of developing
countries, where I think it is estimated that, in developing
countries, perhaps 20-to-30 percent of the blood that is
collected is not even screened for basic antibody tests, and
that translates into more units of blood than are collected
in this country altogether every year, are being transfused
without even basic screening.
I will show later that basic screening with
simple, inexpensive tests would interdict probably 98
percent of the infectious units. So, I am wondering, I know
there was the major focus by yourself and the world on
Africa recently. I am wondering if there's any potential
that significant U.S. research or resources safety could be
put towards bringing forward basic blood measures in
developing countries.
DR. SATCHER: Well, let me start at the end, and
forgive me if I forget some of the things you have raised,
but I think the World Health Organization meeting in
November is certainly an opportunity for us to engage in a
discussion about the global implications of blood safety,
and to also put into perspective how we and the United
Nations World Health Organization can best contribute to
blood safety worldwide.
So, I think that is what this is all about. That
discussion is underway. I believe we had--at the World
Health Day, I believe we had a discussion about blood
safety. So, we are trying to put that into perspective.
Let me just say to you that is a very difficult issue. I
have visited the hospitals in Africa. I remember going to a
hospital in Kissimu, western Kenya, where the choice between
transfusing blood that is not, as we would define it, safe,
and saving the life of a child with cerebral malaria is such
that, are you going to take the time to go through the
process when you know that you have got to give the child
the blood? You have got to choose between safety right now
and a child that will die within the next week or so, but a
child that might well be infected with HIV because of that
unit of blood that you have to give anyway because of the
situation.
So, we have got to deal with all of the things at
the same time, but I believe we can get to the point where
we can make the technology available to developing
countries. When I led the team to Kenya and Tanzania after
the bombing of our embassies, and the whole issue of blood
safety was another issue we dealt with there, where again
the systems were just not in place. So, hopefully through
the United Nations, through the World Health Organization
and our participation, that we can begin to bring to bear
our technology on blood safety in developing countries.
As far as--Damon, do you want to comment?
MR. THOMPSON: On the PSAs.
DR. SATCHER: Yes. The next issue is what are we
doing about getting people to contribute more blood in this
country. It is a very important point, because we have had
an evolving strategy in which we have made several
recommendations, some of them we have implemented, but,
also, we have had a public campaign. Damon, who is the
Director of Public Affairs, do you want to comment?
MR. THOMPSON: Sure. Hi, I'm Damon Thompson. I
am Dr. Satcher's communications director. I'm pleased to
announce that just a couple of months ago, we teamed up with
the AABB, America's Blood Centers and the Red Cross. We
provided the HHS studios at their disposal, as well as the
personnel. They brought some people in who had benefited
from blood products from around the nation, and we spent the
morning cutting several public service announcements, and
the AABB and the ABC and the Red Cross have all committed to
working on the distribution of those PSAs.
If you like, I can get you some information. They
sent us a storyboard with some of the photos involved. If
you like, I can see that Steve transmits to you the
storyboards for those, so you can see just what we have
done.
DR. SATCHER: When do we expect those? I remember
spending the whole morning doing the taping of those PSAs.
MR. THOMPSON: Right. You know, I'll have to
check. I'm under the impression that they have already gone
out. Sometimes it takes awhile to get into the pipeline.
DR. SATCHER: But we agree with you. It's
something we need to really do. It's too late to add it to
the indicators, but--
[Laughter.]
CHAIRMAN CAPLAN: Karen?
MS. LIPTON: I can just comment on the
distribution. We do try to get them out. I think one of
the things that we find is that we compete with so many
other--when you are going into a public service
announcement. And we have talked a lot about different ways
to get around that. Some suggestion is that if you plan
earlier and ask them to place them in December, you get a
better result than asking them for next week.
But I do think there is also a contrary view that
you get what you pay for. And if we are not willing to put
money into purchasing advertising time that will get us at
Super Bowl or something like that, that we will be always
placed in the slots that the stations have allocated for
public service announcements.
I also want to mention that there is an NHLBI, a
committee to try to increase blood donation, and they have
been looking at the Canadian campaign and trying to look at
different ways, and I think they will come up with some very
productive recommendations for all of us, in terms of trying
to put together both the national campaign, which I would
call an awareness campaign, and then coordinating that at a
local level with recruitment, which really has to occur in
the communities.
DR. SATCHER: I know I have gone over my time.
Damon knows a lot about the franking issue, because I think
he was working in Congress during the time when the
situation was different, and the decision about not allowing
franking privileges for the Surgeon General was not limited
to the Surgeon General. Basically, except for Congress,
they were done away with. But, will you say something about
this, Damon?
MR. THOMPSON: Yes. It was part of the budget
agreement, and it had to do with the Postal Service budget
and getting more efficiency out of the Postal Service. The
Postal Service agreed to be able to achieve a certain amount
of savings, but, in return, they wanted to be relieved of
the responsibility of providing free postage and expense for
all of the agencies of government.
So, no agency was particularly singled out or
anything. It was part of an overall budget agreement. I
should also note that as far as the 90-day ban on
mass-mailing for Congress, that does not mean that Congress
has to sit on its hands on this message until after the
election. There are many things that are available to them,
many resources available. There are radio and TV shows,
which they do, newspaper columns, town hall meetings. There
are still many avenues available to them to be able to
disseminate this message, and it will simply be any
mass-mailings, and that is just to duplicate the verbatim
letter and send it out to each doorstep. That will probably
have to wait until after the election, but that doesn't stop
them from communicating the message from now to the election
in many other ways.
CHAIRMAN CAPLAN: Well, I want to thank Dr.
Satcher for coming, and you should know that the committee
is more than willing to try and push forward some of our
recommendations in tandem with you when the letter goes.
You should feel free to tie into our interest in making sure
that the system is available to respond, as well as to
notify.
DR. SATCHER: Thank you very much.
CHAIRMAN CAPLAN: Thank you.
We're going to hear next from Hal Margolis about
basically where we are with the notification and hepatitis C
information programs that we've been trying to push forward
and track.
At the last meeting there were members of the
committee who expressed interest in having an update on this
important area, so--in terms of CDC activity, so Hal has
agreed to present to us on the latest situation.
While I've got the floor here, by the way, I
always was interested in doing an empirical study to see if
there was a correlation between insomnia and organ and
tissue donation, looking at PSA targeting. Blood could be
added to that.
DR. MARGOLIS: Thank you.
What I'm going to do this morning, was asked to
do, is to try and focus on--is this thing on? Can you hear
me? Is to focus on some of the issues in terms of
look-back, and I kind of phrase it in--realizing that most
of the effort and most of the concerns have been around the
issues of targeted look-back, but I think as we recognize,
it's not the total answer to the issue and the problems.
And then I'll also touch on some of the things
that Dr. Satcher mentioned in terms of CDC's efforts in
terms of hepatitis C education and identification of
infected individuals. I think one of the things we've all
learned, is that while we have initially focused on
identifying persons who are infected by transfusion, that as
soon as you start talking about hepatitis C, you actually
start talking about all this group, and you really can't
wall these things off.
I have given everybody a copy of these overheads,
so you shouldn't have to take too many notes in terms of the
direct data. This kind of gives the summary. We've used
the denominator of an estimated 300,000 individuals who have
been infected among the 4 million estimated by blood
transfusion. The data that Miriam Alter presented the last
time, which until the next survey, but based on the power of
that last survey, gives us an estimate that about 1,600
individuals have been identified through the targeted
look-back, which, unfortunately, only represents about a
half of one percent of all the infected individuals.
What you all want to know is this last part, is
that, okay, and so how well has our general look-back effort
done in terms of identifying everybody else? I think you
got some glimpse of that in the targeted look-back data,
which indicated that about one-third of these 1,600
individuals already knew their hepatitis C status. And so,
again, I think we have to at least presume that, as defined,
general look-back has been having some effect.
I put this up here just to remind everybody that
there are a number of reasons why targeted look-back is not
going to identify everyone. The single donor, even the
donor with multiple donations who didn't come back after HCV
testing came in place. And then a number of issues about
the recipient, including, you know, the obvious, you can't
find them, they changed their name. And the last one, which
I think we never discussed here, but which is a very real
issue, is that many people in the United States in fact have
been transfused outside of the United States and they're
among our American citizens, and in fact, as we look at this
from a public health perspective, that's also an issue, and
again, targeted look-back was not going to identify those
people.
This one's the sound bite. You know what the
recommendation is. And now what our challenge has been is
how do you put this into the context of what we at CDC have
been calling now our national strategy for prevention of
hepatitis C, which has four components, very simple:
prevent new infections; identify those individuals who are
infected, and make sure that they are evaluated for liver
disease and treatment; surveillance and research in terms of
the issues that we don't know about.
Now, what we have done from a CDC perspective is,
okay, how do you get this out there? How do you deal with
both the public and private sector in terms of
implementation of any strategy? And we've really focused in
four areas, and you know, you've heard from me and others on
my staff and other in the PHS that in fact communication of
information about hepatitis C has been our number one
priority, and I'll show you some data that says, "Boy, we're
sure not there, but we've made a lot of strides."
The other big issue, which Dr. Satcher brought up
and which we're just beginning to do some things on are what
we call state-based prevention activity, and when I talk
about state-based, it's not all just public sector, it is in
fact public and private, and I'll give you some glimpses of
where we are at this point.
Obviously, surveillance to figure out how well
we're doing is key to this, and again, I think everybody on
this committee knows the issues about hepatitis C and the
difficulty of surveillance, but I'll show you again some
glimpses of things that may help us.
And then, lastly, you know, we don't know
everything, and so there is a research agenda to this, which
I will not discuss at all today.
In terms of the communication issues, as I've
said, we've primarily focused on education of health care
professionals. The other big issue has been public service
advertising or general public education, and then education
of persons at risk, in the risk groups, and as I said, we
have started at CDC. All of our effort over the last two
years has really essentially been focused on the transfusion
recipient. But as we have seen, as we put out just general
information about hepatitis C, other risk group issues come
up, and very quickly you're dealing with the whole universe
of risk of HCV infection. We've only now begun to target
some activities at injection drug users, and most recently,
there was a joint agency symposium on hepatitis C or
hepatitis and HIV in the injecting drug use community. It
was held in Baltimore and co-sponsored by CDC, NIDA, CSAT,
which is the Center for Substance Abuse and Treatment, and
really realizing that the issue now in this last population
is one that everybody said, "Oh, gee, it's HIV that's the
problem." In fact, now drug users or people who had
previously injected, are now dying from their chronic liver
disease because their HIV disease can in fact be managed in
terms of long-term treatment.
Put at the bottom here are the array of things
that we are doing which have gone--and which we are funding
at CDC, which include cooperative agreements with a number
of NGOs, and in fact, there's a new round of funding that
has just begun that is going to expand out. I can't tell
you who everybody is because the awards aren't officially
out yet.
Starting with the recommendations for hepatitis C
control in 1998, which really gave the blueprint and the
framework for how we're going to PSAs, to distance learning
issues on the Web--and I'll give you some of that data
later--to the STD and an HCV hotline that is in fact
available to a group that you probably aren't aware of,
called the STD Prevention Centers, which exist--there are 10
of them around the country, and in fact, train both
practitioners on the medical direct physicians as well as
others dealing with STDs and prevention of these diseases,
with counseling messages. You know, again, one of the
strenghts of HIV prevention is in fact formal counseling
scripts. How do you do it? How do you do client center
counseling? Well, that's not there for hepatitis, and in
fact, we're developing those now, and those will be in the
PT centers within the next six months or so, and again, this
available to both, and heavily used by both the public and
the private sector.
DR. EPSTEIN: Hal, I think that this very
broad-ranging effort on targeted look-back related to blood
transfusion is commendable. But what troubles me is that
it's been estimated that only somewhere between 4 and 7
percent of all of the living people with HCV got it from
transfusion. And the question is what percent of that
larger majority, around--round figures--95 percent, do we
think are reachable through general look-back strategies and
where are we on those?
DR. MARGOLIS: Well, as I'm saying, we don't have
any idea. The fact is we don't, and we don't have any
reasonable mechanism to look at that. We're currently, as I
say, I think some of these state-based surveillance systems,
once states can look at who these people are and figure out
how individuals acquired their infection, will be able to
help answer part of that question; in other words, in New
Mexico, what proportion of those 17,000, in fact, had
transfusion as their risk factor? And from that I think we
can then begin to estimate.
Basically, we have to get some population-based
approaches to looking at general look-back. And we are
sitting right now trying to figure out how best to do that
in a way we can afford it, and that's really our next step.
We put our major effort into looking at the effectiveness of
targeted look-back, and now we feel we need to do, as we
have done with the targeted look-back, is look at the
general look-back kind of in interval or, you know, looks.
So we need a baseline right now to see where we
are because everybody says hardly anybody knows they're
positive, yet you and I know there's a lot of HCV testing
that goes on each year, and I know there are a lot of
positive people in state health department records. So we
just need to figure out who they are and what their risk
factors are. So as I said initially, we don't know, and I
think that's the difficult part.
DR. KUHN: Dr. Margolis, there was a question I
had, and I was thinking about as you were talking about
methods and modes of communicating this to the public, and
the question that came to my mind, and I think it kind of
follows along with what Jay was speaking, is if I'm an
average American working person, I'm busy in my job and
perhaps I, I mean, I received either a blood transfusion
before 1992 or I had it through another mode, and I have
Hepatitis C, but I don't know it because I hadn't had any
need to go to a doctor because the disease is not presenting
in any way, shape or form, what is the best method or mode
of communicating or informing me that I may be at risk?
And I'm concerned because there are people out
there, and they have busy lives, and how are we
communicating to them that they are at risk?
DR. MARGOLIS: Well, again, I think we've taken a
lot of different approaches, from the surgeon general's
letter to those PSAs to what we have heard, and we've done
over a dozen focus groups in different parts of the country
with lots of different people of different races and
ethnicity. And they all say to us, "I'm not going to go,
most likely, if I'm--" and these were people who have had
transfusions, but they all essentially are saying, "I'm
probably not going to go to my physician just because I had
a transfusion. I expect my physician to ask that question
when I go there." And that we have heard over, and over and
over again. I mean, that was the very first data. And
that's why we think that, and people do go to physicians for
other reasons and as part of a standard health history.
Now, for instance, the American College of OB-GYN
has now put this in their health practices, their latest
health history. So they've been moving forward with this.
So what I'm saying is, and it's what this group told us, is
that the message has to be on both sides. We have to get it
to the public and get it to the public as close to their
health care setting as possible is what, again, the educated
approach was. And the other is that physicians have to ask
the question because if they don't ask it, it's not going to
happen. I mean, that we've heard both from the patient side
and from the physician side. So that's where we're trying
to go. I mean, I don't--it's difficult.
DR. KUHN: Yeah, and I understand it's a dilemma
right there. But, again, I kind of look back to where most
public citizens are all of the time is they are on the road,
they're driving. And I think one of the most effective,
that I saw, ways of a PSA were the yellow eyes, that poster.
That was a very effective way of getting people's attention
and maybe that needs to be looked at again, done on a
broader scale across the United States. I don't know how,
but there are people in marketing who have a way of being
able to reach the mass public on how to inform them that
they are at risk if they meet one of these entities.
DR. MARGOLIS: And just to assure you that we/CDC
aren't doing this alone. We're doing this with the big
marketing people. So that's why there's a lot of different
approaches out there. But, no, I agree, and again the
yellow eyes, which was done by the American Liver Foundation
I think is still running.
The big issue, though, is does it get somebody
into the physician to get the test? And that's where the
link has to occur. But you need both, and we agree.
CHAIRMAN CAPLAN: Keith?
DR. HOOTS: I'd like to congratulate you, at
least, from a couple of anecdotes that I've observed. At
least two patients over the last 2 months who clearly had
been told long ago they were HIV infected because they were
in an at-risk group and had been presumably educated, at
least we think we've educated them, but as you indicated,
sometimes it takes reinforcement over and over again. These
were two individuals who hadn't been seen in the health care
system for several years because they had done pretty well,
but came in not only asking about their Hep C and commenting
about, well, you know, it's been in the news a lot, and I
wanted to know, and they came with specific questions, which
indicated to me that some of this message is getting out
even to people whom you would think would be the ones who
wouldn't need it the most. But the fact that they
translated what they--just some peripheral knowledge that
had probably been long since forgotten into specific
questions suggests to me that, at least in that broad way,
and clearly what they said to me was that it was in the news
and that they had been looking, and it raised questions in
their mind.
CHAIRMAN CAPLAN: I have a question, actually,
three quick ones.
I had occasion to be talking recently to a group
of infertility clinics, and when they manipulate sperm, and
eggs and embryos, it turned out that not all of them, but
many, were routinely testing for Hepatitis C, among other
things. So in addition to the scripts, one question I have
is how is CDC working with the professional organizations
and societies to make them have, as part of their standards,
testing for risk behavior in the good standards and
practices area?
DR. MARGOLIS: We go to a lot of meetings. In
fact, I was just at the Infectious Disease Society of OB-GYN
and working also with, again, OB-GYN's Practices Committee.
Miriam, and I and others in my group spend a lot of time
working because the reality is the tough part of this is
getting this uniformly into medical practice.
And that comes from several ways, getting
professional organizations to say we agree or we're going to
rewrite the CDC recommendations for our own organizations,
and, again, that was part of in that February meeting, and
some groups are doing that. And then the subspecialty
groups within an organization has to do it. And let me tell
you, you have to knock them down one at a time. I mean,
there is no other way.
We actually now have a list, and we went back
after that February meeting to find out of the 150 groups
that were invited and for whom we even gave
dummy--dummy--but, you know, dummied up articles for their
newsletter. How many of them have done something with it?
Well, 25 have. So they've actually put it out in their
monthly newsletter or magazine, and then a much smaller
group are actually putting it into a committee to write
recommendations. And that's what we're tracking and working
with them.
And there is really no other way to do it because
that's our current medical system. But what it really all
starts with were the CDC recommendations and recommendations
from this committee and others that begin to say now we need
to move this forward.
CHAIRMAN CAPLAN: The second question I had for
you is at some--we spent a lot of time, and we'll probably
spend a lot of time today starting to or revisiting issues
of cost benefit, trying to achieve improvements in safety.
And in one sense, as Jay points out, Hepatitis C is not
primarily a blood transfusion issue. On the other hand, I
think the committee has always understood that there are
special obligations to notify, and look back and maybe spend
more because of the nature of the blood system. In order to
cement trust in the system, you may want to do that. And
also, to be blunt, it may be easier to carry some of the
public health factors about Hepatitis C on the back of the
blood issue. So there it is, and that's what it's going to
do.
At some point, however, in the expenditures,
cost-benefit expenditures, there comes a time, I suspect, at
which we start to say you ought to get a test if you're
older than 20 or older than 30--I mean, forgetting about the
individual risk factor thing. Do you envision a time, has
CDC been thinking about simply saying, look, anyone over the
age of "X," risk factors or not, we really should make that
part of routine testing, as part of what gets done? Cost
and so on I understand with the--
DR. MARGOLIS: We've not done cost analyses.
We've done it on kind of an identification effectiveness
analysis. And that, again, is the reason, for instance,
that while HCV is sexually transmitted, the number of people
who have the types of sexual practices that would put them
at risk of HCV is so incredibly large that the effectiveness
of identifying people, such as doing routine testing in an
STD clinic.
Now, we're looking at this in a number of the demo
projects, and there are data coming in. And, again, the
effectiveness of routinely testing, even in an age group,
turns out to have an incredibly low yield. And so we've not
done it as a formal cost analysis where, actually, it will
happen, but we've done it as an identification effectiveness
type analysis, and that's why we haven't used those
approaches because they just don't come close to any of the
things that we do in terms of other public health
activities.
CHAIRMAN CAPLAN: Last quick question. I see that
this letter probably, sounds like, is going out under some
disguise or stealth mechanism. So if the letter is going,
and there are PSA announcements in somebody's basement
waiting to be shown at 2:00 in the morning to various
people, and you have activities underway, can the committee
know or can we be assured that we could get an integrated
push?
This is a wonderful opportunity, it seems to me,
to really get the attention of our political candidates,
congressional candidates, all kinds of people, if we could
get a coordinated push around the appearance of the letter.
So is CDC thinking now about what to do when this letter
goes?
DR. MARGOLIS: Our problem has been we get the
sense this letter is not all going at one time. And, again,
I'm just talking about some of our--what little bit of
forecasting we can get. The other part is some of these
things which I've told you, such as the next piece to the
physicians, is in the pipeline. When that's going to happen
may not quite come together with the letter, to be honest,
in terms of just the lag time of the funding, and what the
contractor produces and what we all think we want. So we
are ready to be receptive; in other words, the hotline has
been geared up. There are more people.
We have tried to do some forecasting as to what
may happen, and if this upward trend puts another set point
in there, we can do that. In terms of other pushes, you
know, we can reissue some of what we have. Whether we'll
have a new rollout like we did at that May thing almost a
year-and-a-half ago, I honestly don't know. But it's a good
idea, and we'll sit down and strategize.
CHAIRMAN CAPLAN: Steve?
DR. NIGHTINGALE: If I can make one additional
comment in response to Dr. Caplan.
There is not necessarily a conflict, but there
should be a balance between an integrated approach and a
sustained approach. One of the things that I have taken
from Dr. L'Enfant and his very successful management of the
National Cholesterol Education Program is his very firm
belief, which I think is backed up by very extensive data,
that the duration of a public education campaign is of
comparable importance, if not more than comparable
importance, to its intensity. The primary purpose of the
Department's efforts is not to achieve a campaign that gains
intensity at a single point in time. In fact, there is
probably less enthusiasm for an intensity-driven campaign
within the Department than there is without. I think there
is more enthusiasm within the Department, and I believe I am
speaking for the agencies as well, of the duration of the
campaign.
What we're both looking for is effectiveness of
the campaign, whether or not intensity or duration is the
most important, perhaps will be debated. There is room for
both. But from within the Department, duration is what
we're looking for.
DR. PENNER: Two quick comments and a question.
The learning curve for the health professionals is directly
related to Board examination questions, and it would be a
strong suggestion, if it gets on the Board, it'll be picked
up very quickly.
Secondly, the PSA letters, as they come out,
require I think some attention on emphasis. When we get
letters that start out with substance abuse up top, for
example, the stigma of that suddenly turns off the rest of
the message. You say, well, if these are drug users, that's
fine, and forget about it. So if you emphasize that, and I
think this will bring back the question that was already
brought up, is it can be carried as a message and part of
the transfusion and blood which I think most of the
physicians, health care workers and the public respect. So
they'll look at it a little bit more carefully than if it
just comes, oh, another drug abuse situation and then just
discard it.
The question I have is that 3 years ago we dealt
with a look-back situation and made some recommendations.
And at this point, what percentage of the blood banks do you
believe have completed the look-back?
DR. MARGOLIS: The data that Dr. Alter presented
was that in April it was about 85--again, the estimates were
based on 85 percent. It was around 85 percent. And the
next survey, which will be done early in 2001, I mean, we
projected essentially it's, you know, it's done. That was
on 85 percent of the data reported back. Now, many of them
had already done all of their work, but didn't have the data
in terms of being able to report the numbers.
DR. PENNER: To 1990 or to 1992?
DR. MARGOLIS: Oh, excuse me. That was, actually,
that was back through '90, again, that was the--
DR. PENNER: The first-generation testing.
DR. MARGOLIS: No, that was not first--this was
only second-generation testing or second-version testing.
DR. PENNER: So what's happened on the
first-generation testing that we have asked for the
look-back to be reviewed with the specifications that we had
made with regard to cutoff period? What's happened on that
end?
DR. MARGOLIS: That we have done because at the
time when the survey was done, again, essentially last
January or February/March at that time, that wasn't in that
survey. That would then be put in the next survey. And so
we realize we're now going to have to look at version one
effectiveness data, which will probably carry us into 2002,
in terms of getting that data. Do you see what I'm saying?
DR. PENNER: Yes.
DR. MARGOLIS: In other words, all of the data
I've shown you so far is for version two, for the second
generation. And so for us, for the evaluation, it has to be
added to the questionnaire that will go out in 2001.
DR. PENNER: So you're really not sure what that
part of the look-back is--
DR. MARGOLIS: Don't have any data on that.
DR. PENNER: Mike, do you have any idea what that
might be, just offhand?
DR. BUSCH: Well, just I remember when we looked
at this there was data from Stanford that indicated that
their actual incremental pick-up with the version one-driven
look-back was quite low just because of the tracing issues,
the lack of records, the number of patients alive.
In terms of the first-gen look back, I think most
programs have implemented it. There's actually not a formal
final FDA recommendation, in terms of exactly how to conduct
it. But sort of verbally, I think BPAC meetings, et cetera,
FDA has indicated their position. So most programs have
initiated it and I think probably completed it.
DR. PENNER: With the first part, but the other
part--
DR. BUSCH: No, I'm talking about the
first-generation-driven, BIA-driven look-back programs have,
to my knowledge, been implemented and are probably well on
the way to completion.
MS. LIPTON: If I can just, the difficulty we
don't know. I mean, we're pretty confident about what the
blood centers have done. When you get into more problems is
trying to ascertain whether the hospitals have actually been
able to make the contact. I think most blood banks got on
it right away because if you were going to go through the
records once, you just wanted to accomplish it. But as to
how, you know, finding patients who were back much further I
think was a very different issue. And there are time lines.
I think everyone is working diligently. But, Kay, you don't
know, do you? I just don't think we know right now. And we
periodically send out surveys, but we don't have one on
the--and it's really the hospitals we need to hear from.
DR. GUERRA: Harold, a couple of public health
questions and concerns.
Most states now are doing universal screening for
HIV and Hepatitis B in their populations of women that are
being taken care of in labor and delivery room suites or
during their prenatal care, and it must be documented. It
is a requirement, at least prior to dismissal of the infant
from the hospital because of certain therapeutic decisions
perhaps that have to be made.
Are we anywhere close to considering that as a
universal recommendation for prenatal populations, given
what obviously is a tremendous increase or at least the
suspected increase in serum prevalence rates is one
question. And the other is within some of the efforts that
are taking place, are we also doing something to try to
dispel the myths and misperceptions about Hepatitis C? We
often get questions about household members being in contact
with somebody that is identified as being Hepatitis C. And
it's almost a real stigmatization that occurs in infants or
children in day care settings, where a parent has declared
themselves to be Hepatitis C, and it becomes a matter of
public concern.
CHAIRMAN CAPLAN: I just wanted to piggyback
something that I forgot to ask onto that question. We had a
big battle, which you're aware of, Hal, in Philadelphia
about firemen getting infected with Hepatitis C. And I got
a lot of calls about this casual contact transmission,
bleeding contact, that sort of thing.
DR. MARGOLIS: I would venture to say we spend 50
percent of our time, professional time, both public
inquiries and actual time "doing the studies," to dispel
some of the myths, actually maybe working from the fire
fighters back. Again, unfortunately, there are a lot of
things that go wrong out there, so incorrectly reported
data, which turns into somebody's political agenda, which
doesn't serve anybody. And as you saw, we moved pretty
quickly to test old data sets, analyze data, get an MMWR out
and try and answer and at least hopefully set the
information platform straight.
The same goes on with the question of
transmission. I think there are, again, good data, and
those get handled both through hotline and a lot of calls,
and, no, we don't have a pamphlet that says what do you do
in a day care center. That's on the list that needs to be
written. And so that's where, and again, we try and clearly
work with our partners to identify certain groups or
information areas in some of these cooperative agreements
that we do. I mean, that's frankly how we work is to let
them put that information together and get it out. And so
some of that type of, you know, those areas where we're
getting a lot of inquiries, we then try and generate, you
know, some written information or put something on the
website and go that way with it.
So it's kind of both, if we need the data--you
know, tatoos is, again, still one of the big issues. So
there are RFAs out there, there are awards that have been
made for looking at tattooing in various settings to, again,
look at risks. So sometimes we need to generate the data,
which is the research side. Other times it's just
communicating it that we try and deal with.
The question about perinatal transmission and
screening pregnant women, there is a CDC study that's being
presented now and is in the process of being written up for
publication that showed, again, that the rate of perinatal
transmission is low. It's about 3-3.5 percent.
Transmission only occurred in an HCV RNA-positive woman.
But, in fact, since this was actually a prospective
multicenter study, it showed two additional things:
One, it showed that there was an increased risk
for internal fetal monitoring--somewhat, you know, that's
kind of a logical thing, but it's never been looked at
before; and the other that there was increased risk with
prolonged rupture of membranes greater than 6 hours. And
actually those in the multi-variant analysis part of it
actually became the only dominant risk factors, in fact,
over HCV RNA titer in the mother.
It raises the question of, given that kind of
information, is there something we ought to be doing
differently in terms of identifying HCV-positive pregnant
women because in the past there wasn't anything you could
actually do as an intervention. And, again, going back to
how you make things happen in practice, we're currently
working with ACOG, and their Fetal Medicine Committee, and
their Infectious Disease Committee to review those data and
see if it warrants such a recommendation for screening
because that gets back into that issue of, you know,
identification of all of the issues and what one might do.
So those are new data that, as I say, have been
presented, and several meetings now are being put together
for publication and are being worked on by the groups that
would be most affected by it. So we are trying to move in
that direction.
DR. GUERRA: But beyond, obviously, the port of
transmissions or just they're serving the purpose of
identifying the Hepatitis C women in child-bearing years,
that obviously could then be put into a registry for
tracking and for doing the other kinds of preventive
measures.
DR. MARGOLIS: Again, it goes back to a bit of the
question that Dr. Caplan asked, which turns out that
actually in that age group, if you just did it uniformly,
you're identification rate is actually very low because
that's actually one of the lower-prevalence areas. We do
have recommendations that if women have risk factors, they
should be screened. And, again, if you go around and talk,
especially to large inner-city OB-GYN and delivery services,
many of them are screening, some of them now to the point of
routine. They're definitely asking questions.
And, again, ACOG has put that in one of their, you
know, in their newest screening questionnaire. So, again,
it's this issue of, you know, should you do everybody or
should you do some, and some of the new data on possible
intervention for the infant may change the equation in terms
of should we do all. And that's where are right now. And
as I say, this is just in the last couple of months. So
we're trying to deal with it with various advisory
committees.
DR. GILCHER: A couple of comments from a large
regional blood center that might be helpful.
With respect to first-time donors, it's 95 percent
of our HCV hits are in first-time donors. It's actually
95.4 percent at our blood center. We've gone back and
queried these individuals. There are not a lot, but we've
queried them. And what we have found is that, number one,
they were not seeking tests. They really did not know they
were infected. What we did find, though, was that about 50
percent of them did admit, even though they had denied this
at the time of the donor screening, they did admit that they
had tried IV drugs even one time.
We then added another question, and this is really
a comment that I'd like you to comment on. We said, "Do you
have any friends that are Hepatitis C positive?" And almost
every instance, they have friends. And, in fact, when we
query, it's those friends with whom they tried IV drugs even
one time.
The other interesting finding that we have now
from our NAP data, and I can only speak for my own center,
is that roughly--and this is a donor population, not a
general population, is that about 25 percent of the donors
who are RIBA-positive appear to have cleared the infection.
That's higher than what has been found in the general
population. But of that group, interestingly, about 30
percent of the women have cleared, but only about 17 or 18
percent of the men appeared to have cleared the infection.
I'd appreciate your comments.
DR. MARGOLIS: Starting with the last one, when we
looked at the NHANES data, 25 percent were, in fact, RNA
negative, realizing that's a one-time RNA test. But only 75
percent were positive. The younger you were, the more
likely you were to be RNA negative. And, again, that gave
us some pretty wide confidence intervals, but there are
other data that kind of keep coming out that way. And,
again, in that data set, as you recall that African
Americans had the highest chronic infection rate, at about
almost 90 percent. And, again, those data and other studies
have been seen that way. So that when you put that
together, I think that's probably what we're seeing, what
you're seeing, and it's part of, I guess, better knowing the
biology and the natural history of the infection.
The issue that an HCV-positive individual may have
another positive individual either in their family or close
to them, we're now seeing again, in a number of data sets
that we're analyzing, and where again I can just give you
from the NHANES that we're looking at, for instance, in
families actually close to 30 percent of the families had
more than one HCV-positive person in it. But when you
actually look genetically, these aren't the same viruses.
So it wasn't that they were transmitting to each other
because, unfortunately, NHANES didn't have all of the risk
factor data we would have liked in NHANES 3, but from what
we have, it seems they had similar risk factors. And,
again, I think that is beginning to be noticed, and we just
need to get a little more precise with how common that is
and how we can use it to identify individuals. It's not
that this is, you know, intrafamilial transmission, but it's
shared risk factors. And I think, frankly, we need to
figure out better how to use that information so we can
identify people.
But, yes, we've seen that in the NHANES, we've
seen it now in a couple of other data sets, and we're trying
to, again, figure out ways I guess really to best understand
it so we can use it for identifying people.
DR. DAVEY: Dr. Margolis, I think we can all
agree, and the comments have reflected this, that the
toughest group to approach is the IV drug-abusing community.
Probably over 50 percent, I believe, of infections are
thought to be by that route, as Ron and others have pointed
out.
Could you comment on the CDC's efforts to link
their control measures on Hepatitis C with other government
agencies that have responsibility for managing drug abuse.
And secondly, specifically, what's the CDC policy on clean
needles and providing clean needles for a prospective
management of this infection in that community?
DR. MARGOLIS: I think probably the best indicator
of what the Public Health Service--how we view this was this
meeting in May in Baltimore called Drug Use, Hepatitis, and
HIV--or I can't remember which way they had it--bringing it
all together, in fact, sponsored by the agencies who have
responsibility both for prevention, treatment, and control
of substance abuse.
When you then really start talking to drug
treatment center directors as well as the research
investigators and trying to--how do we figure this out, I
think everybody's well aware and I think now is beginning to
believe that the paradigm for prevention of infections among
injection drug users, while HIV has been the focus, it's
really HCV because it's there immediately, and, in fact, I
think it's made us all--and, again, based on data that was
done in syringe and needle exchange programs, actually
sponsored by CDC, show that it's probably things other--I
mean, the syringe and needle are extremely important, but
transmission also goes from the rest of the activity, and to
put it kind of quite bluntly and frankly, you need universal
precautions in a drug use setting, then reflect on that and
realize how difficult that is. But that's, in fact, what
the issue becomes. It's everything else that's
transmitting, and, in fact, we've done studies to ferret out
those other things, including blood on the hands and all
those things that go on with drug use.
So you still got to get at the prevention side.
You have to figure out how to interdict in terms of those
who are using. And let me tell you, one of the things that
I've seen--and I'm kind of new to this--is that in the
states where we have now seen hepatitis C coalitions from a
state perspective put together, it has heavily driven the
issue of drug treatment. So that, again, several states now
have moved drug treatment actually into clinical public
health settings instead of that "over there" kind of
situation. And these people are now talking to each other.
And, you know, there's been real changes in total
philosophy. Yes, CDC recommends that syringe and needle
exchange should be carried out. The Federal Government
doesn't fund it. So that's, you know, where there's some
divergence.
But, in fact, it's very effective, and most
recently some involvement we've had with vaccinating drug
users in exchange programs shows that it's highly effective,
can be done, and you can access people.
So I think things are changing, and the people who
are supposed to be dealing with this are talking to each
other a lot. Is there money? That's what Fernando asked
earlier. Unfortunately, no, there's not a lot of money
that's coming together with this. But I think at least
we're finally talking about it and realizing what the issues
are.
So, you know, that's kind of--this is new and this
is evolving, and evolving pretty rapidly.
CHAIRMAN CAPLAN: Maybe what we will do is take
one more question from the Committee. I might look to see
if there's one question out in the audience. Then I will
finally relieve Hal from standing up here so long.
Paul?
DR. HAAS: It's impressive the amount of work that
you have done, and I have to believe it's also very
frustrating in terms of how slow the information comes out.
And this part might be the wild part, but I'm listening to
this and thinking of watching these ads now showing up on TV
for different types of pharmaceutical drugs and the
awareness it has, I suppose, given the patient showing up at
the doctor's, probably asking for the drugs for the wrong
purpose, but at least they're aware that it's out there.
I'm just wondering, now that we do have treatment
for hepatitis C, whether some of those drug companies might
be willing to start funneling dollars in to help the
continued promotion that you're doing.
DR. MARGOLIS: In fact, the PSAs, you know, the TV
PSAs, which are very expensive, were funded through the CDC
Foundation and a drug company consortium. We just didn't
have the resources directly from what we had. And so we
haven't gone to the subliminal advertising yet, but we're
clearly partnering. And I think it's been working well.
And it's also including immunizations. So when I talk
about, you know--because again, as Dr. Guerra said, this is
the whole thing.
I mean, I don't know if you've heard me say it
here, but our buzz word around CDC now--and this is with the
AIDS groups and others--is one-stop shopping. I mean, the
reality is you've got to think of these all together, and
all the bloodborne infections are together, and we've got to
start talking about it that way. And so we've been trying
to do that with industry.
CHAIRMAN CAPLAN: Could I just ask you to identify
yourself for the record?
MS. JACOBS: Yes, Mary Beth Jacobs from FDA. I
have a follow-up question to the last one.
About a year ago, I saw an ad in the Washington
Post directed toward women from a company saying: The next
time you go to your gynecologist, why not ask if you should
be tested for hepatitis C?
Has CDC evaluated the effectiveness of that type
of direct ad to consumer, not the approaching of having
companies fund the PSAs, but the comparison between that
kind of approach in someone who has not yet been diagnosed?
DR. MARGOLIS: No, we haven't.
MR. CAVANAUGH: Dave Cavanaugh, Committee of
10,000. The occasion for this presentation is the letter,
which is stopped. There are several barricades to the
letter going out. We understood kind of elliptically from
Dr. Satcher that a sentence is being changed, and I don't
know if that means the letter's been pulled back or never
went to other Congressmen. And even with the ban, is there
any kind of assurance that something will happen that any
number of the 435 members will be sending it when they come
back in January?
Thank you.
DR. NIGHTINGALE: Just for the record, I spoke to
Mr. Slobodan of the House Commerce Committee yesterday. We
do have a plan, and we'll be in touch with you. There are
separation--because of the separation of powers, I'm not
going to say anything else right now, but I will say for the
record that we continue to work actively with the Congress
and fully respectful of their prerogatives.
CHAIRMAN CAPLAN: We will come back, undoubtedly,
to the letter with, I'm sure, other illuminating responses.
But let's stick with Hal for now. I'm going to take one
more question, if that's to Hal, and then we'll take--we're
getting ready for a break, although Steve has one comment to
make before we do.
DR. SAYERS: Merlyn Sayers from Carta (ph) Blood
Care, which is the community blood program for Dallas-Fort
Worth. Heaven forbid I should delay getting into the break,
but some comments that relate to remarks by Dr. Satcher, Dr.
Busch, Dr. Gilcher, and Dr. Margolis.
It's not all that long ago that community blood
banking was a lot simpler. All we really needed to do was
recruit donors, collect blood, test the blood, make
components, and distribute it. But whether we like it or
not, the role for community blood programs to become centers
of community and public health is increasing dramatically,
and that's understandable. Something like 40,000 volunteer
donors a day are scrutinized by an extensive health history
and extensive serological testing. So our role in community
and public health then immediately relates to counseling
those individuals that have been identified as potentially
worthy of counseling.
That community and public health role then gets
extended with targeted lookback, and I have no doubt that
that role is going to be extended even further when this
letter comes out announcing to the nation at large what the
risks are of hepatitis C, the silent epidemic. And whether
we like this or not, I have no doubt that even though
individuals will be cautioned against going to their blood
program to donate to get tested, I would not be at all
surprised if we do not see a small surge in individuals who
are found to be reactive in HCV because simply they're one
of the many millions that do not have health care coverage.
So at every point, the cost of doing business at
blood programs is increasing, and a significant element of
that increased cost has to do with the fact that we now have
a community and public health role.
Now, we cannot exactly pass those costs,
understandably, on to the individuals that we sell blood to.
Goodness knows they can't get reimbursed for the additional
testing that we're doing. So, Dr. Margolis, you posed the
question just a couple of minutes ago, is there the money?
And what I'm wondering is, if there is the money which is
going to recognize that community blood programs are now a
very valuable and very important source of community and
public health, could there be creative ways to fund these
public health programs which are centered at the blood
centers?
End of sermon.
DR. MARGOLIS: Well, let me just tell the group,
the Committee, because it's public record, you know, our
budget through this year, the end of this fiscal year, is
about $13 million. So that pays for all these things you're
hearing about and some things you're not hearing about, like
the sentinel counties, the NHANES, all of those things, and
also pays for some staff. Congress, in the current
President's budget for 2001, the estimate mark is an
additional $5 million, and that's kind of where we are, and
that's how we put it together.
There's been testimony in a number of hearings as
to what the estimates might be for a program that would fund
counseling and testing and support of the various community
activities that you're describing, and that's in the range
of $40 to $60 million per year. So we're a long way and,
you know, we're trying to be as creative as we can.
Yes, we think it ought to be a part of the mix,
and if you look at HIV, you know, some of those types of
things do occur. But we don't have that for hepatitis C.
DR. GILCHER: Merlyn, in response to your
statement, something that we have done for over 12 to 15
years is offered what is called non-donor testing. We
clearly found out that there is a segment of the population
who wants anonymous testing not at a doctor's office but are
willing to pay for it. And we believe that that has
actually enhanced the safety of our blood supply and has
removed test seekers from donating blood because we now
offer this kind of program through our system, which is
really in a sense a public health maneuver.
CHAIRMAN CAPLAN: Okay. Thank you, Hal.
Steve, you wanted to say a word about the WHO
issue that the Secretary brought up, I think.
DR. NIGHTINGALE: A word or two, and about several
subjects. I am taking over Dr. Snyder's obligation to be
brief before the break. I'm not sure that I will succeed.
Hal said that you had to hear a message seven
times before you really understand it. This will be the
tenth time that the Committee will have heard either in full
or slightly abbreviated fashion the conflict of interest
statement.
Relative to what we're up to, I would ask you to
listen to it at least as carefully as you have on the
previous occasions because there are some very important
things in it, and it reads as follows:
The following statement is made as part of the
public record to preclude even the 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 Committee members
present no conflict--potential conflict of interest when
evaluated against this agenda.
In particular, specified in Title 18 of the United
States Code at 208(b)(2), a special government employee,
which all Advisory Committee members are, may participate in
a matter of general applicability, for example, advising the
government about its policies on the 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--and here is the key point--provided that the
matter will not have a specific or distinct effect on the
employer or the employee other than as a member of that
class.
The example give in 5 C.F.R. 2640.203 is as
follows: A chemist employed by a major pharmaceutical
company has been appointed to serve on an Advisory Committee
established to develop new standards for AIDS vaccine trials
involving human subjects. Even though the chemist's
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's
employer will be affected by the new standards only as 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 in which a member has a financial
interest, that member should exclude him- or herself from
the discussion, and that exclusion should be noted for the
public record.
With regard to the other meeting participants, we
ask in the interest of fairness that they disclose any
current or previous financial arrangements with any specific
product or any specific firm on which they plan to comment.
The point here, the tenth time, is that conflict
of interest is an extremely important issue in a democracy.
The process of regulation--it's very important that we do
this right on several different levels. We have and will
continue to talk about issues where the advice--we cannot
get the advice we need as a government unless we get it from
people who will have a conflict of interest. This Committee
is intentionally much more inclusive than some of the other
Advisory Committees, and I think that has been one of its
strengths. And one of the things that--perhaps my personal
agenda is to try to make other committees stronger by being
more inclusive.
I believe that we have in the statement that I
just read, and, as you can see, I deeply respect, a
principle analogous to the principles that we will be trying
to elucidate--to gather from you today on the broader issue
of blood safety that goes part of the way but not all of the
way. And that's why I wanted you to focus on it. The
principle for conflict of interest works extremely well, I
think, for individuals. Does it work as well for aggregate
committees? That's where we fall short.
I saw the lawyer to my left give a knowing smile.
I think there are a few others.
For example, it is perhaps not quite in our
technical capacity yet to clone the chemist that I mentioned
earlier and several times previously, but clearly a
committee that was made up of 10, 18, or 24 clones of the
chemist would not be an ideal committee. We really don't
have the principles quite yet for aggregates as we do for
individuals in regard to conflict of interest. How we
develop a committee that is not collectively biased is
something we haven't figured out yet. We have some
standards, though, and I think you should look--when we make
our disclaimers or our proactive statements that we
encourage members of minorities, women to apply, that
geographic diversity is indeed a criteria for membership on
the committee, these are perhaps sentinels that we would use
to see whether--as a first pass, but they're clearly not
sufficient.
So as you are thinking today about either the
certain matters or the principles, however we formulate the
statement, I would encourage you to consider that we've come
part of the way towards that but not all of the way.
Now, in that context, one other thing that I think
we have that we don't use perfectly but I think use well is
the charter that we have for this committee identifies
people--the class from which we wish to draw nominees, and,
in fact, this is my segue to the comments on our request for
nominations for membership in the committee.
Our charter does not identify specific people who
have chairs on the Committee other than that there are six
non-voting governmental representatives. There is somewhat
of a split between people on the left, people on the right,
and people in the center. That is not cast in stone.
That's something we're still working on, and comments on
that either now or sometime in the future from the Committee
would be helpful. But as we actively solicit nominations
for membership in the Advisory Committee, we would be
interested in nominations that make the whole Committee
stronger rather than individuals.
One final point, of course, is that the Committee,
while it might be the people around the table, we have made
and will continue to make a deliberate attempt to act as
much as we can as a Committee of the Whole. Dr. Caplan has
been superb--and I would like to make that compliment for
the record right now--in including members of the audience.
The audience has been equally superb in their contribution,
and for the record, thank you for your continued support.
With that, then, on May 31st, notice was published
in the Federal Register, Volume 65, No. 105, page 34705,
soliciting nominations of individuals to serve on the
Advisory Committee in accordance with its charter. As Dr.
Satcher noted, the terms of five members will expire on
September 30, 2000. Appointments will be made for a term of
four years, and it is now necessary to renominate
individuals previously nominated.
In accordance with the Committee's charter,
persons nominated for membership should be from among
authorities knowledgeable in blood banking, transfusion
medicine, bioethics and/or related disciplines. Members
shall be selected from state and local organizations, blood
and blood products industry including manufacturers and
distributors, advocacy groups, consumer advocates, provider
organizations, academic researchers, ethicists, private
physicians, scientists, consumer advocates, legal
organizations, and from among communities of persons who are
frequent recipients of blood and blood products.
Membership is by secretarial appointment, and I
can assure you that it is by secretarial and not by staff
appointment.
A copy of the announcement is available at the
back of the room. We have attempted to make the nomination
process as simple as possible. We need to know who the
nominee is, how to reach her or him, which of the very broad
categories I just mentioned the nominee fits into, and that
is only because of the requirement of the Committee charter,
and a written statement of the nominee that, if appointed,
he or she will serve.
We need a copy of the nominee's C.V. Additional
supporting materials are welcome but not necessary.
Individuals may and are encouraged to nominate themselves.
In accordance with well-known Department policies regarding
nondiscrimination and diversity, women and members of
minority groups are encouraged to apply. If someone other
than the nominee is the nominator, we need that person's
name and address. If the nominator is a corporation, we
need a human contact in that organization.
Finally, we need to receive the materials by 4:00
p.m. on August 31, 2000, which will be the 92nd day since
the notice was published on May 31, 2000.
If anyone has any questions, please contact CAPT
McMurtry. His direct telephone line is 202-260-1351, and it
will not be changed until after the close of business on
August the 31st.
[Laughter.]
DR. NIGHTINGALE: Now, to the statement of issue
for the meeting, at your request you are meeting to discuss
the role of various considerations in decision making
related to new and additional safety measures. Dr. Satcher
suggested the alternative but not contradictory title of
what are the principles on which a blood policy to assure a
safe, available, and affordable supply should be based.
This is meant to encourage rather than to limit comment. I
think one of the uses, perhaps the immediate use of the
comments will be as the Department considers how best to
support the efforts of Dr. Emmanuel, who you met at the last
meeting, and certainly Dr. Epstein, who has made huge
contributions to this process, to the meeting in Geneva on
November 13-17 by WHO, the first Global Collaboration for
Blood Safety. A copy of the announcement was distributed
with your briefing memoranda.
We approach this with an open mind, with, as you
heard from Dr. Satcher, a desire to participate in a
constructive manner both in regard to the needs and the
policies and the political conflicts of developed countries
as well as developing countries.
Finally, having made that comment about the
immediate purpose, that is, of course, not the only purpose
of the meeting. The Department is aware, in part because of
its response to your previous recommendations, of the issue
of reimbursement for blood products and reimbursement for
the services that are incorporated into the provision of
blood products, many of which economists would call
externalities. The issue of what the market recognizes and
what it doesn't is one of which we are aware, but do not
mind being reminded once again at this meeting.
The agenda for today's meeting is much less
structured than it has been in the past and much less
structured than I anticipate it will be in the future. The
reason we have done this is to provide the members of the
Advisory Committee and the members of the public in
attendance an opportunity to say whatever they want to say
on this issue and to do so in an unbiased context as
possible. The one request I have, although I may not have
honored it myself, is to keep it short.
CHAIRMAN CAPLAN: Steve, just for the record, some
members of the Committee have asked me, having perhaps not
been as intimately acquainted with the charter of this
Committee as you are, with a new election how does that
affect if there's a turnover at HHS, with the Secretary, how
does that affect our business, our charter, what goes on?
DR. NIGHTINGALE: On November 9th, all bets are
off. At the same time--I've actually--I've never gone
through a government transition, particularly not being kind
of in the--I don't want to call it "the bunker," but in the
Secretary's office.
What I do anticipate, however, is that there would
not be a complete change of administration. I am aware of
very broad bipartisan support for this Committee. It's been
incorporated into report language in the past. It's been
expressed to me privately, and I communicated it to you
because, folks, it's your work that has been recognized.
Where are we going? The speech that Dr. Satcher
said was this Committee, I believe very successfully, has
addressed six very complex issues. The burden of Dr.
Satcher's--at least the text of his message was that these
problems, while addressed, remain, have not gone away, and
if we let them drop, they could become problems again very
soon.
To give a little bit of my political feel for
this, it is that the establishment of this Committee and the
maturation of this Committee was a very labor and
emotionally intensive process. I know of no one in the
government or I think in the private sector who wants to
start all over from scratch.
DR. DAVEY: Steve, one quick question about
membership. Assuming the Committee continues, I would
recommend--and I think we've discussed this--that we include
a representative from the Health Care Financing
Administration as a representative on the Committee joining
our other government representative colleagues.
DR. NIGHTINGALE: Noted. Also, I think membership
on the Committee by a nongovernmental individual who may
have expertise in that area would also merit consideration
and will be considered.
Dr. Davey did remind me to say one of the many
things that I wanted to say and didn't say when I was
reading my text real fast: Membership on this Committee is
a membership of individuals in accordance with the charter.
We nominate, the Secretary approves individuals for
membership in the Committee.
Having said that, I would say something else. The
Committee needs new blood. You get tired of listening to me
read that conflict of interest after a while, and you get
tired of listening to some of the other things. At the same
time the Committee needs continuity. These are issues for
which we don't have good guidelines. We make it up as we go
along. But I think in a government, the more, first of all,
that the public participates in the process and the
more--the better the road map, the easier it is to govern
with the consent of all who are being governed. That's what
we're looking for.
CHAIRMAN CAPLAN: Okay. Unless there are any
other questions about our membership and what to do to put
people forward, why don't we take a 15-minute break? Then
what I would propose is we come back and follow up on the
issues raised by Dr. Satcher and Dr. Margolis in terms of
hepatitis C lookback, notification, that area. Then I think
we have some public comment to do, which I suspect is going
to take us toward the values and principles issue about
increasing efforts at safety relative to cost and
practicality, which I know members of the Committee want to
talk about, and that should get us up through lunch.
So let's plan on thinking about what we heard this
morning, going to the lookback issue and notification and
public awareness, and spend some time with that and then
move on to the public testimony as a way to move us toward
the consideration of continued efforts to push for safety.
DR. NIGHTINGALE: Could I have a show of hands
about how many members of the Committee wish to make
statements? One, two, three, four, five, six, seven, eight,
nine, ten, eleven, twelve, thirteen.
CHAIRMAN CAPLAN: Well, forget it.
[Laughter.]
CHAIRMAN CAPLAN: Okay.
DR. NIGHTINGALE: On that note...
CHAIRMAN CAPLAN: We have time, so we will go
there. Did you want to do that sort of pre-public
testimony? You want to go around that way?
DR. NIGHTINGALE: Yes.
CHAIRMAN CAPLAN: All right. We'll do it that
way. Maybe we'll get us into that and then move into the
public testimony side after lunch, if that's how that's
going to work. Okay.
[Recess.]
CHAIRMAN CAPLAN: Could we take our seats back
here, please?
What I'd like to do is have the members of the
committee spend whatever amount of time they want, but just
a few minutes on the presentations we heard this morning on
hepatitis C on the look-back. I know we've had discussions
about, and requests about this matter of sending the letter.
I understand that this is a subject under discussion, and
has legal and even constitutional issues in play, but you
may want to say a bit more about that in terms of advice to
the Secretary.
One idea that I had which I can toss up in front
of the committee is that we might want to say if something
hasn't happened by a certain date, than we would hope that
monies would be appropriated to make sure that something
happens by another date, but that's just my particular
thinking about letters.
But let me open the floor and see if, based upon
the presentations, we have any discussion, comment that you
want to make about the hepatitis C, the look-back, the
notification issue.
One thing I think we might want to urge, if not
recommend, is that there be, with acknowledgement to Dr.
Nightingale's comment about intensity versus duration, some
effort to coordinate the letter with CDC efforts, patient
and foundation activities in private sector activities. It
seems to me, whether you want to commit to an intense effort
or make sure that something is there that's going to go on
over time, it's just important to get the ducks lined up so
that you get a maximum impact of sending the letter. Plus,
if the Secretary--if you remember what he said, he said in
part, we're not sure we're ready for a response that might
be large, to sending out a letter. Other organizations and
other groups need to be on board here in terms of knowing
what's going on, being ready to both field questions, but
also perhaps to bring pressure to bear to make sure that the
infrastructure for an adequate response, if not right after
the letter goes back, that at some point in the future is
there. So that is of concern to me, that we try to push
that they coordinate on this kind of thing, and that we urge
everybody to try and work together, all the interested
parties here. Jim?
DR. AuBUCHON: I certainly agree with you that
coordination would appear to be helpful, although we have no
data to know exactly what is going to work, and that is an
unfortunate impediment, as Dr. Margolis identified, but even
beyond coordination, I would urge the federal government to
identify resources to put toward this problem.
If I could make a--what will probably be perceived
as a cynical observation--when this committee began its
existence several years ago, there was a great push on from
the Congress that we had to address this hepatitis C problem
immediately, and we have addressed it from a transfusion
point of view, but as Dr. Epstein pointed out, that is a
very small piece of the puzzle. The larger piece of the
puzzles is not getting the attention and the resources it
deserves. One might suggest that some in the federal
government--and I do not mean those around this
table--directed attention to hepatitis C through transfusion
as a cheap means of addressing what they thought was the
hepatitis C problem, because the federal government didn't
have to pay for hepatitis C look-back, primarily. They
foisted it off on someone else. I think it's time, and the
situation deserves additional resources from the federal
government to address the larger part of the problem.
CHAIRMAN CAPLAN: Fernando?
DR. GUERRA: I think as we continue to take the
hepatitis C national effort to the scale, we need to
somehow--and I'd be interested in Dr. Margolis's
response--we need to build that onto the efforts that have
been in place for HIV/AIDS, the national campaign, and not
take away from that, because that continues to pose a very
significant threat in communities.
CHAIRMAN CAPLAN: Well, perhaps we can come back
after we listen to committee presentations up until the
lunch period, to what we want to say, if anything, about
both budget resource commitment and coordination in general.
Since we've been around for the three years, I continue to
find it somewhat surprising that the total budget to send
out the letter is bigger than the CDC Hepatitis Office
budget, but all right.
Okay. I guess the way to go on this, we have a
lot of people who took the request for comment very
seriously, and that's great, and I am now in the unfortunate
position of trying to steer a group, in terms of time, that
I have learned to be unruly about these matters. But I
think we have a 5-minute aspiration on presentations and
statements, and what I'd like to do, is instead of
organizing us by height or alphabet or something, I'm going
to start down with John over there. I tend to--these people
have accused me of looking to the left first, so let me go
over there, and then we'll just move right around the room
for committee members to make their presentations. I didn't
watch all the hands, so not everybody has one, but if you do
have one, let's start there and just come down.
So, John, I give you the first 5-minute
opportunity.
MR. WALSH: Thank you. I'm used to being at the
end of the alphabet.
First of all, I'd like to express the appreciation
on behalf of the alpha one community to this committee for
taking the shortage situation with respect to A1PI products
as seriously as we have. The Alpha One Foundation is
dedicated to providing the leadership and resources that
will result in increased research, improved health, and
worldwide detection, and ultimately a cure for alpha one.
AAT is a single-gene defect leading to loss of one serum
protein, and there is currently only one product available,
manufactured by one manufacturer, naturally, to provide
augmentation therapy for patients with alpha one.
The resolutions that I would like to report on in
the form of an update that helped us through a crisis that
lasted some 24 months with supplies decreasing for
allocation between 40 and 80 percent throughout our
population.
On April 28, 1998, resolution: "The Department of
Health and Human Services should explore, in collaboration
with industry, health care providers and appropriate
consumer groups, methods to optimize and standardize
allocation of available products in an equitable manner,
including management of emergency supplies and programs that
distribute products directly from manufacturers to
registered consumers."
This distribution issue was addressed by the Alpha
One Association, the alpha one community, in conjunction
with the foundation and the medical and scientific advisory
panel. We did address it, and a direct distribution
strategy was developed to respond to the request for direct
consumer allocation, and deliver prolastin directly to the
consumer. Prolastin is no longer sold to distributors. It
is allocated directly from the manufacturer to the consumer,
ensuring that the fully-prescribed dosage is available to
each alpha as long as there's product available, which
includes issues related to lot releases and ultimate supply
issues.
Everyone enrolled in the program has received
their full prescription at 28-day intervals, resolving the
need for reduced dosages or increased intervals at this
time. In addition, over 200 consumers that did not have
access prior to November '99, are currently on augmentation
therapy. So a controlled direct distribution in the case of
a one-product-one-manufacturer community like ours has
definitely resolved the inequities and directly related to
decrease and severe shortages.
April 28, 1998, the committee made a
recommendation that industry should explore with the FDA
strategies for reallocating partially processed plasma
materials from one manufacturer to another, you know, to
optimize production of alpha1-antitrypsin deficiency and
other plasma derivatives.
We are able to work very closely with industry,
and again, on behalf of the Alpha One community, I'd like to
thank the American Red Cross and Baxter for working very
closely with Bayer in a cooperative relationship that
ultimately provided enough 401 paste to optimize production
capacity at both of Bayer's manufacturing facilities.
So for now we have enough raw paste to produce at
maximum capacity. That does not mean we're going to have
enough end product or throughput to be able to satisfy
demand.
April 28, 1998, again the NIH and industry should
immediately evaluate alternative dosing schedules and
alternative delivery systems for alpha one therapy,
including prophylactics strategies and strategies for
treatment during acute exacerbations of disease, and
accelerate the development of gene-based products and
gene-directed therapies for alpha one.
This definitely supported the evaluation of new
delivery technologies. The advisory committee
recommendations for expedited development of new and
non-plasma derived options helped break the logjam for
aerosol or inhaled development of A1PI, and we currently
have three manufacturers that are in various processes of
development for an aerosol product, including one transgenic
product, will be the first recombinant product available to
our community, potentially available to the community,
obviously, pending trials and licensure, but it's already
gone through a Phase I.
The current situation, with only one manufacturer
and one product, with limited production capacity, it is
inevitable that demand will exceed supply. In fact, I
believe we're there right now, where we're going to see,
within the next two months, the inability to be able to
dispense 100 percent of prescribed dosages to patients, so
we've already hit the wall in optimizing the distribution.
We need another product. This will ultimately affect every
consumer. The product will continue to be shipped in
sequence with no prioritization given to reimbursement
issues, which was one of the problems in our distribution
that's corrected by this, effectively, or clinical triage.
There's been no determination by medical and scientific
advisory committee that clinical triage is possible, let
alone appropriate in relationship to augmentation therapy
for alpha one. It failed in Europe. The European
Respiratory Society tried to implement one in 1999.
The future IV products--there are two IV products
currently under different stages of development. One is
finished the Phase III and delayed in PLA application for
numerous reasons. The other is in Phase III now. It's
fully recruited for enrollment in the trial and should be
completed, and once their data is analyzed, will obviously
get to the FDA for expedited licensure.
There are three initiatives, as I said, with
respect to the aerosol delivery, which would--we hope, would
make a more efficacious process to deliver the drug directly
to the lung. It's hoped that aerosolized products will
provide increased access to more consumers, being able to
take care of up to five times as many consumers with the
same raw material, and also be more cost effective.
The Alpha One Foundation will continue to work
closely with industry and the FDA to promote the development
of new therapies. Alpha One has a meeting scheduled with
the FDA and CBER, which has been very cooperative in
addressing the issues related to clinical trial design and
the IND issues. One of the issues we're discussing is a
recommendation to form a working group between the FDA, the
NIH and the Alpha One Foundation Medical and Scientific
Advisory Group, to be able to actually look at some of the
surrogate marker endpoint issues and other impediments to
design of clinical trials and approval of products.
And the foundation is organizing and HR CT scan,
using high resolution CT scanning to look at the progression
of lung disease as a potential surrogate marker, and also an
animal model study workshop, which will be conducted in
Sienna in Italy.
There's just a couple other comments that I would
like to make to the committee. Regards HCFA, the
committee's action, I think HCFA had more response to the
outpatient prospective payment issues. The APC code change,
where there would be a direct path to plasma derivatives,
definitely made it possible so that all of our HCFA
consumers would not be cut off from product and create a
real problem for access. So we thank the committee for the
support in that.
We've taken the responsibility to communicate
directly with all of the distributors, all of the providers
for HCFA treating alpha one patients, and let them know how
to actually do the billing, and we're working closely with
HCFA to accommodate that.
The transition to recombinant is a result of this
committee's strong recommendation, although I notice that
the--in the final recommendation that was published, it only
mentioned hemophilia factor products. I believe the
committee discussed that in the broader context of plasma
derivatives, and I'd like to state for the record that I
think that very much helped the PPL Therapeutics out of
Scotland to focus their attention on alpha one, and not just
cystic fibrosis.
Finally, I'd like to thank the members of this
committee for their support for the Five Points of Life,
speaking of donor awareness, and the Surgeon General's
remarks earlier, the Five Points of Life event is to raise
donor awareness for organ tissue, DNA, plasma pheresis,
obviously, donations country-wide. Dr. Gilcher, his group
has a rider involved with that program that raises a lot of
awareness, and I think it's just a ripple and we need to do
more, as much as possible, we're committed to. ARC, AABB,
ABC and ABRA have all contributed to sponsor the event, and
there will be an activity here in Washington, a reception
for congress people. And even the insurance industry is
involved. State Farm, I think, is the only one I'm aware of
so far that's embraced this, but I think it's important for
us all to take on the responsibility to get involved with
creating more donor awareness.
I would also like to thank the organizer of the
ride, Life South, for all their efforts, and to thank our
committee's Executive Secretary, Dr. Steve Nightingale, for
his ongoing participation and support throughout the
process. Dr. Nightingale is actually riding the first week,
from Bar Harbor, Maine to Boston, Massachusetts.
So again I thank the committee on behalf of our
community, and look forward to making more progress in the
future.
CHAIRMAN CAPLAN: Thank you. Unless it seems--I
mean if someone--I'll take a minute to see if there's a
question or a comment after each speaker, but you don't have
to feel compelled to do so, comment or--that's a polite way
of--
[Laughter.]
CHAIRMAN CAPLAN: All right. Jerry?
DR. WINKELSTEIN: Well, I was unprepared. I'm
usually last alphabetically, so thank you very much.
Well, I wanted to make two comments. The first is
in the form of a thank you, which is heartfelt, and the
second is in the form of a proposal for a future agenda
item.
Now, the first is a thank you, and if you will
remember, one and two years ago I presented information to
this committee when I was not on the committee, but a member
of the audience, about the shortage of IV gamma globulin and
the many patients with primary immune deficiency diseases.
I'll remind you that well over 10,000 patients receive IV
gamma globulin, and it is the only thing for primary immune
deficient patients which is therapeutically beneficial.
There's no substitute for the IV gamma globulin. But during
the past few years there had been a very significant
shortage of IVIG for these patients, which was impacting
very significantly on their health status.
Now, the part of the thank you is that through the
efforts of this committee, a similar committee advising the
FDA, the Immune Deficiency Foundation, and under the
leadership of Dr. Epstein, the FDA itself, all of these
groups developed a realistic and achievable clinical
protocols which will speed the licensing of new IV gamma
globulin preparations from a number of old manufacturers and
new manufacturers. One such protocol, my understanding is,
nearing completion, if not been completed, from the
patients' point of view at least. Another clinical protocol
has begun to enter patients over this summer, and two other
manufacturers are developing clinical protocols as we speak.
I believe that the availability of these new preparations
will help very significant in alleviating the shortage. So
my thank you is that I would like to publicly thank both
this committee and the FDA for their efforts to alleviate
the shortage. I do believe it made a difference, and I
think we're seeing the results of that even as I speak. So
if you ask yourself if the committee has done anything
worthwhile, which I had to ask myself before I joined the
committee, rather than sit around, I can give you evidence
that you made a significant difference for these patients.
Now, my second point relates to asking your advice
as to whether or not there should be consideration for a new
agenda item over the next number of months or year, and I'd
like you to consider whether this is an appropriate agenda
item. Now, as you know, patients with primary immune
deficiency diseases are the only long-term users of IV gamma
globulin, one of the plasma products. Many of the patients
have been on for decades. In fact, there are patients
entering their third decade of use of licensed IV gamma
globulin in this country, and most of them will remain on IV
gamma globulin for a period of four, five or six decades if
the therapy is as good as we think it is. From that point
of view they're quite unique, and represent, if you will, a
canary population for patients receiving IV gamma globulin
and many other disease states in the short term. These
long-term patients receive it for decades, as I said.
Now, in recent years, there's been a growing
suspicion that long-term consequences have not been well
documented. There are at least two articles of anecdotal
case reports of patients with primary immune deficiency
disease, who have received IV gamma globulin, presenting
with unexplained central nervous system diseases. In
addition, there are some other problems associated with the
use of this material, as there would be with any
pharmacologic or biologic material. Renal disease,
immediate adverse events, and the consequence of long-term
use have been looked at, but not in a formal way.
And so what I'm wondering is whether or not this
committee should consider adding a future agenda item which
would include presentations on the value of a formal
assessment or prospective study of the long-term and
short-term adverse effects of IVIG. Obviously, I would hope
then that they would endorse such studies because these
studies are not being done currently. Whether that's a role
for this committee or not, I'm young on the committee, and
so I'm not sure, but I did want to bring it to your
attention.
CHAIRMAN CAPLAN: Thank you.
DR. NIGHTINGALE: I would comment briefly that
there are many ways to view the role of the committee and
many readings that you can make of the charter, but one that
I think that would encompass not only your statement but the
statements that I anticipate from the other side of the room
here, would be that this committee very fundamentally
addresses what economists call externalities. For those of
you who are not either economists or children of same--and I
am in category B--and externality is a cost of producing a
good or product that the market for one reason or another
does not recognize. So I think I've answered your question
constructively.
DR. WINKELSTEIN: Then my impression would be that
this would be an appropriate agenda item, depending on
priorities of the committee.
CHAIRMAN CAPLAN: Jane.
DR. PILIAVIN: I was under the impression that our
remarks were supposed to be addressed to the precautionary
principle. So of course, as soon as I start to talk, my
voice acts up. And I guess I'd like to start by referring
to the article that you included in our voluminous materials
for this meeting, entitled, "Will Blood Transfusion Ever Be
Safe Enough?"
And that's the point that I guess my remarks are
all oriented around that. Whether that puts me on the left
or on the right, I don't know, because I don't know where
that dimension goes from to. I also want to note that we
were just handed out a bunch of new stuff, one of which is a
memo from Merlyn Sayers about leuko-reduction, which just
ties in with this same issue.
First I want to say that I read everything you
gave us about the precautionary principle and I still don't
know what it is. That's because very early on in the
material it says that it's not going to give a definition of
it, and then as you read the examples, clearly, the
precautionary principle goes everywhere from saying if
something has any kind of potential harm to the environment,
to human health, you shouldn't do it, to a very basic kind
of risk cost benefit analysis on the other hand, that says
when it comes to new technological things, you should be
really, really careful and think about the concerns for
human health. So I don't know what the precautionary
principle is.
I think we all have been trying to be careful, and
if the precautionary principle says "Be careful", then we've
been following it. I get the sense that because of the
examples they use about the European community, there's more
attention in regard to this principle to new developments
than to long-standing situations, and I'm wondering whether
that's appropriate. Is it only about new technological
things or is it about any kind of dangers? And I'm going to
deal with it more as if it's any kind of dangers.
Okay. I kind of liked one of the things I read in
here which said that when dealing with data under the
precautionary principle, you need less than a preponderance
but more than a scintilla of evidence of harm. I just love
the word "scintilla" anyway. And I'm wondering to what
extent we in the past and other committees have been dealing
with scintillas or less rather than with preponderances, and
that we should take that into account. I'm specifically
referring to the NBCJD issue, which strikes me as having
less than a scintilla of evidence. As you all know, I have
been opposed to that regulation regarding people who have
been in Great Britain.
Also in the materials on the precautionary
principle, one of the things that they said--and this was
clear--is that you should deal comparably with things, that
you should not have one sort of reaction to one problem and
a different kind of reaction to another problem, that things
should be dealt with in some equitable kind of way. And
again, referring to the response to NBCJD, I would like to
ask why did we respond that way to that issue and have not
responded to what struck me as more evidence about Chagas
Disease, and certainly lots more evidence about problems
with errors, simply blood matching and other kinds of
errors. Now, these--I mean, the latter is of course not
technological in the usual sense and it's not new, but if
we're going to apply this principle to concerns about risk,
I think we have to look at having comparable responses to
issues, and certainly there are far more people who die from
errors of the sort we talked about last time than have,
obviously, NBCJD--nobody's died--and Chagas Disease, I don't
think anybody's died from transmission, and certainly
there's all sorts of things that could come down the pike.
In terms of equity issues of a different sort--no,
I think I'm going to skip over that because I know I don't
have that much time here.
I want to get back on my usual hobby horse of
safety equals availability. At the level of infectivity
that we now have in the blood supply, the idea of adding
leukocyte depletion and individual NAT testing strike me as
inappropriate use of money for a variety of reasons. Of
course it's going to drive the price up, which will lead to
both a decrease in the number of people who can afford the
product, and a decrease in money that's available for other
things. Certainly it will lead to an increase in medical
premiums. It will put an excessive drain on the public
funding, Medicare, Medicaid, and from what I saw in terms of
the estimated dollars per year of quality adjusted life that
will be added by the individual NAT testing, this struck me
as really inappropriate use of money.
I want to think, in terms of other uses of money
and how much life could be saved, things of the sort of
child immunization, sex education and HIV prevention,
prenatal care, visiting nurse programs and even cancer
research, and certainly putting in funds for getting more
information to more people about Hepatitis C would come
under better uses of money.
In regard to availability then, these things also
have the impact of further decreasing the availability of
blood, but also the possibility of bankrupting the blood
centers. Because it's more or less guaranteed, I think we
all know, that reimbursement for the Medicare/Medicaid
patients won't be sufficient.
As a social scientist, I was thinking about this
as I was reading the materials, I would love to get a survey
of all of the blood centers in the United States to see
where they are financially. I mean, it's conceivable to me
that all of a sudden a good proportion of these
organizations, which are mainly nonprofit, are just going to
go belly up, and they're not going to do it one at a time so
we can see a trend going. There's going to be a bunch of
them who are going to say, "This is the straw that breaks
the camel's back. We can't do it. We have to quit." And
that can be a disaster.
It would be very nice to know where they are, how
close to that point these organizations are. Because if
anything like this disaster scenario were to happen, we
would be in deep trouble.
It's clear, also, from the materials that this
message we've been trying to send, that we want a no-fault
coverage for people who do, indeed, get injured by these
very, very, very few people who get injured by infectivity
in blood, that nobody is paying any attention to this. The
secretary says they can't do it, and I'm sure that they
can't. And the Congress, who mandated this committee, seems
uninclined to do anything. I think the best precaution
against the very small known risk would be accepting that
risk at this point and providing that kind of relief for
people who are, indeed, harmed, as we do for people, the
very few people who are harmed by vaccines.
I'm trying to think of a comparison that would be
apt in terms of the number of people who are injured in this
arena, as compared to other arenas. Unfortunately, I was
late reading my material, and therefore did not have access
to even the Internet to check anything. But I know that we
lose many more people on the highway in one day than from
transfusion-transmitted diseases in the last 15 years. And
so that struck me as apt. Why aren't we putting a lot more
money into educating people about safe driving and so on?
Undoubtedly, we lose more mothers and babies from
inadequate prenatal care, to put it in the same arena, but
the people that we're losing are probably, for the most
part, from groups that aren't powerful enough to lobby
Congress for their needs.
Why aren't we doing cost-benefit analyses
comparing the things that we spend our money on? Now,
obviously, this committee as a committee can't do that. But
you asked us to give you our opinions, and this is my
opinion. I think that the next HHS secretary should ask
Congress to appoint a committee to assess how our medical
dollars are spent across the spectrum of things that
endanger the health of our populations--how much bang for
our buck are we, indeed, getting?
I want to end with sort of the old simile about,
in terms of where we're spending money, are we like the
drunk who drops his keys while trying to open his front
door, but looks for them under the streetlight because he
can see better over there? Are we pursuing technological
and bureaucratic fixes for smaller and smaller risks very
expensively simply because we can, while ignoring much more
critical problems because we can't?
CHAIRMAN CAPLAN: Comments?
DR. NIGHTINGALE: I need to make a couple of brief
procedural comments. First of all, Jane asked, very
legitimately, what is the precautionary principle. It was
included in your packet because it is one of the many
approaches to the answer of the question not because it was
a preferred approach. If it has any primacy, it is because
it is, in fact, the law of a very large chunk of land with
which we have trade, in which we have biologic trade, and it
is not something that I think that we are free to ignore,
although we are, by no means, required to accede to it. And
if my own personal take on it snuck in, in that last
comment, so be it. I realize, also a comment to Jane's why
was the precautionary principle put in, in something this
big, it was put in because this is a very complex question.
And I think one of the things that the government
can do wrong when it runs a committee like this is to try to
dumb it down so you can get an answer. And one of the
things we have not done here is to try to dumb this
committee down. And along those comments, when Jane finally
asked are we getting our bang for our buck, this meeting
costs about $25,000 to put on. Most of it goes to plane
tickets and most of the rest goes to the Hyatt. Not much
goes to food, as you tell.
[Laughter.]
DR. NIGHTINGALE: But I would say, so far, we are
getting advice at about a thousand bucks a pop, and so far
it is the judgment of the government that we are very much
getting our money's worth, and I thank you.
DR. PILIAVIN: Steve, I wasn't talking about this
committee. I was talking about the medical budget of the
United States.
DR. NIGHTINGALE: No, no. But I was trying to say
thank you.
[Laughter.]
DR. NIGHTINGALE: It's easier to say that to some
people than others.
[Laughter.]
CHAIRMAN CAPLAN: I do think the precautionary
principle is used by some outside the United States, and
their policy is for new things, so it doesn't really set
itself up for comparative risk assessment.
John?
DR. PENNER: It seems to me that the recurring
theme of our deliberations is what price safety. And I'd
like to reemphasize the fact that I don't believe blood is
an industrial product. I don't think it's comparable to a
drug or a medication. I think it's unique, it has no
alternative, and you do not have a dose ratio of efficacy to
safety. In other words, exposure and the occurrence of any
viral effects to that exposure are not reversible. It's all
or none. You can't just reduce the dose and reduce the side
effects or complications associated with this item, which we
call blood.
If we had a situation whereby a medication I think
was put up for approval and would produce a death rate of,
say, 1 percent or 1/2 percent or so on in every recipient
that received it, I don't think that drug would be expected
to be approved. And we do have that situation with blood,
that it does, once it's administered, produce an effect that
is not going to be reversed in those individuals who are
exposed to whatever the contaminants are. And since there
is no alternative, I think we're put into a very unique
situation that is not comparable to what we find with other
manufactured medications.
And then lastly, I think the public expects safety
with this product. The donor population is particularly
sensitive to this issue and always will respect the fact
that if the blood is not considered to be safe, we will
probably continue to have problems on recruitment of donors.
It will have an impact. So it would seem, therefore, to me
that cost will have to respond to these requirements. And
despite the fact that the cost effect may be considerable, I
don't think we really have a lot of choices if we want the
public to respect the fact that we are providing them with
something that they don't have to fear.
CHAIRMAN CAPLAN: Good. Karen?
MS. LIPTON: I just had a few short comments. And
we're going to have more of an opportunity later on the
precautionary principle, so I was going to leave that aside
for now.
I wanted to let you all know, some of you knew, I
actually had an opportunity to participate as a blood safety
and availability committee member in the fourth annual
meeting of GAIN. And GAIN is the Global Aviation
Information Network. It's a very interesting organization.
It sort of takes off from where we talk domestically about
air and accident reduction. And the whole purpose of GAIN
is to really improve aviation safety worldwide by promoting
the exchange of information by and among members of the
aviation community.
When you get into the details of what GAIN is,
it's truly a remarkable partnership between government, FAA
in particular, but other government regulatory agencies
around the world, and the airline industry globally. And
the whole purpose is really to share information about near
misses, and deviations and problems that come up--I mean, to
share it in a way that really is a no-fault exchange and
really does improve safety.
The FAA provides technical support, but the
aviation industry is truly in partnership with the FAA
because, together, they have constructed a data base that
everyone has access to. Now, interestingly, they have the
same barriers that we identified in our last meeting, the
principle barrier, of course, being legal obstacles.
There's fear of discovery of the information that's in the
database, and there's also fear of the punitive action that
might be taken as a result of the information in the
database.
It's interesting that they've been able to solve
those problems. They've been able to solve it both through
international treaty and through an international
organization that's very interested in promoting this and
has gotten different countries to sign on to their
principles. FAA itself I think we saw by the regulations
has agreed that they will not use the information that's
reported through the system to take punitive action.
Some of the other obstacles they reported will
sound familiar--lack of support from people within their own
profession, that cultural shift that needs to be made, and
also from the CEOs' perspective in the airlines,
demonstrating cost-effectiveness. But they are making some
headway there.
I wanted to comment about this conference and tell
you about it because, at the end, the conference came up
with five guiding principles. They were consensus
statements. And I only want to talk about the first two
because they do relate to our discussions later today,
whether it's on precautionary principle or blood safety
issues. One is that the first guiding principle is that the
safety of passengers and workers is of paramount concern to
this industry. My guess, as they stated, it's because
generally the airline pilot is first at the scene of an
accident. But that being said, I think there are some
things that we can take into our own community from that.
But the second was very, very intriguing to me,
and that is that the public perception of safety, of airline
travel, is as important to them as absolute safety. And
this is a principle that, excuse the pun, they live and die
by. They don't have a problem incorporating that into the
actions that they take to improve safety, even when they
think that airline travel, if you look at it, is safer than
it's ever been. We've heard that before. You know, your
chances of really dying in an airline accident are quite
small. But they don't care, they are continually trying to
drive down the number of accidents. So I just wanted to
bring that up, and perhaps we could get back to that again
when we talk about our own decision making.
The second issue is to talk about reimbursement,
and again to thank this committee for its support, and HHS
in particular. We did achieve some beneficial changes to
outpatient reimbursement. We're now really working
diligently and very hard on trying to change reimbursement
for inpatients for people who will require transfusions in
the hospital.
We've been greatly encouraged by the interest of
congressional members in this issue. They clearly
appreciate that both safety and availability, as Jane
pointed out, are really tied to reimbursement issues.
I think, at the same time, we've frankly been I
guess challenged and maybe somewhat disheartened by the lack
of uniform appreciation and support within the health care
community itself for the need to fix reimbursement for blood
and blood products. There's a general need to say we need
to fix hospital issues. But getting the attention focused
on blood and blood products continues to be a dilemma.
After all, we compete with a lot of other issues that
hospitals are concerned about, and we're critical, but we're
small. But, again, we are critical, and I think that we
lose a real opportunity if we don't keep this committee's
attention on the reimbursement issue and if we don't keep
pushing.
I guess we're going to hear a little bit later
from Paul on some adequacy data. But, again, related to
reimbursement, adequacy remains a critical issue in this
country. But to my mind, adequacy, again, it's not related
to a lack of good ideas, it's not related to the fact that
we don't have good professionals or very motivated people.
The real issue is it all comes down to costs. It costs
money to recruit donors, and we have not been paying enough
attention to putting money into that side, and that will
raise the cost of blood.
I think, frankly, as a government and as a
society, we've been kind of content with supplying just
about enough blood--80 to 95 percent. We could easily get
to 100 percent, where there aren't shortages, where there
aren't surgeries that are postponed. But it all comes down
to reimbursement and comes down to the cost of what that
last unit will be. And it really isn't, in most cases, to
the individual patient, it's what we as a society and we as
government or, you know, part of the government today are
willing to pay for.
CHAIRMAN CAPLAN: Jim?
DR. Au BUCHON: If I could just second Karen's
note about the importance of public perception of safety and
commend to the committee's attention an article that appears
in this month's issue of Transfusion, entitled, "Public
Perception of the Risk of Blood Transfusion." And in that
article they note that about two-thirds of individuals in a
national telephone poll noted that blood transfusion was
associated with images of safety or high levels of safety,
but one-third did not. And they regarded blood transfusion
as risky or very risky.
And the authors went on to note that the
perception of an individual about the degree of safety in
transfusion today was closely correlated with their
perception of safety of other technologic matters and that
even for those individuals who did not regard blood as being
unsafe today, it wouldn't take a whole lot to sway them in
the other direction should something pop up.
And in their conclusion they note that it is
better to spend resources prophylactically on this issue
because the consequences of not averting potential concerns
about real or imagined risks, and thus losing public
confidence, can be extremely costly. We've seen that in
blood banking many times over.
CHAIRMAN CAPLAN: Dana?
DR. KUHN: Mr. Chairman, I really don't have a
comment unless we're not going to revisit the "Dear Citizen"
letter. I still had some questions that have been left
unresolved, and hopefully, I'm not sure if we're going to
come back to address that issue.
CHAIRMAN CAPLAN: You can do it now. We can come
back. We should say something more about it.
DR. KUHN: I think that I understand that there is
a dilemma about allocating resources to inform the public
versus the resources to handle the requests for testing,
once that letter is out. And that brings me to the question
has that letter gone out. I seem to have, maybe I'm
interpreting things wrong, but it seems to me what I have
been hearing is there's kind of some elusive answers to
whether or not it has officially gone out, the letter. Has
it gone out? In what capacity has it gone out to the
public? Has it been funded? And to what extent has it been
funded to go out? Did the House Administration Committee
request the funding for it? Has it in this budget year? Is
it there?
And the other question I have, has it perhaps not
gone out because of the 90-day rule, and will it go out
after the 90-day rule is complete? The question I have, I
was hearing that it had gone out, is that I happen to be a
constituent of Congressman Bliley, and I have not seen a
letter come to me notifying me of anything of the kind. The
time I saw it, the first time, was when it was sent to us in
a packet. So there are a lot of questions I think are still
unresolved that I would like to hopefully have answered to
know more about whether or not or what this committee may be
able to do. If the funding is not there, does the committee
have the power to request the secretary somehow, some way,
maybe--as we all know, the elusive budget or member of the
budget is usually the HHS bill. And I'm not sure if it
could be attached onto there, the funding to do it
adequately in getting out this letter.
I'm still, there are a lot of questions that I
would like to see if there are ways, perhaps in the time we
have today, these could be answered.
DR. NIGHTINGALE: Let me try to do a better job
than I did the last time. The letter had been the subject
within the Department long before I arrived. When I
arrived, it got a formal hearing. In fact, after Dr. Kuhn
asked Dr. Margolis a question in February of 1999, is this
still on the table, and Hal said, yeah, it's still on the
table. And following that meeting, I made a second
investigation of the cost. I had made one actually in
August of 1999, and the cost that I had gotten some bids
from private manufacturers was far in excess of $30 million.
What I found was that the post office charges 13.5 cents to
send a letter. And if you're going to send $100 million
letters, that's going to be $13.5 million.
The printing of a letter--if it was going to be on
official stationery, something that would make you look at
it, would probably be two-color with the surgeon general's
letterhead, it's blue, it says, "Public Health Services,"
it's the letterhead we use--would probably be in the range
of 11 cents. And one of the issues, of course, is you put
it one side English and one side Spanish, where do you stop,
where do you start? Probably low end will be about 11.5
cents for English only, two color.
In addition to that, there are costs for sorting
the letters. You've got to put 100 million labels on there.
That's going to be about 4 cents a letter. And you've also
got to deliver it. So while I might, until right now,
privately peg the thing at about 26.5 cents per letter sent
out, I don't know if we could do it for 26.5 cents. A
hundred million is a round number, but that's the number
I've gotten from a couple commercial suppliers. So there is
real money involved in that.
This is 20th century technology. There are other
ways of delivering letters right now. They may be in the
process of development, but the Internet is not necessarily
the best way to deliver mail to the targets of this--to many
of the targets of the letter. But a third way that came up
in private conversations that went on for several months
between the Department and the House was inclusion of this
into a congressional mailing. The idea was not to have a
separate letter, you know, a separate envelope on with
Bliley's frank on it, but Congressman Bliley or anybody else
in the House who had a teenager who knew how to use Adobe
Pagemaster--I do, that's presently unemployed--and I think
there are others in that group that what we thought we could
do is get the same message out at less than $30 million.
I think that there are the problems, the objection
to the separation of powers, was not one that was
anticipated by either side at the time that we had the press
conference. We weren't going to do the press conference.
The thing came up. Hey, I was in Ely, Minnesota, when I got
word of it, came back and have this being dealt with. Max
has been dealing with it, and we'll be dealing with the
issue next week. We did review this with Dr. Satcher on
Monday morning. Max had the conversation with Al, and I've
had a conversation with Mr. Slobodan. And we're going to
proceed as quickly as we can to resolve the issues.
The one thing that I have to emphasize, and this
is a very direct order from the surgeon general of the
United States to his employee, is respect the Constitution,
the autonomy of the House. And that is the sole limitation
on what I have to say right here. I think I've probably
said as much as I can. But if you want to try to pry some
more out of me, go right ahead, and I'll try to open up.
CHAIRMAN CAPLAN: The issue still, however,
remains, and we can get back to it later, of whether we want
to say anything about how to proceed with the letter in
terms of should things not go well, in terms of the autonomy
and privileges of the Congress looming larger than the
surgeon general's mail opportunities. Do we want to say
something about what should happen then or, as I said, put a
date forward and say if it doesn't happen by X, then let's
ask for Y, appropriate monies, et cetera, et cetera.
DR. KUHN: Yeah, I think we need to be specific in
asking for a date. I guess what I'm hearing you say, Steve,
is that the letter is not going to go out.
DR. NIGHTINGALE: Did I say that?
DR. KUHN: Has it? Well, I guess the question I'm
asking, has it gone out?
DR. NIGHTINGALE: If you go to
www.surgeongeneral.gov, you will find the letter. It's one
of the four little blue lines up at the top. It's on the
CDC's website. Before we received the concern of the House
Management Office, a photo-ready copy of the letter that was
underneath, not on top of, but underneath the press release
by Congressman Bliley was hand-delivered to every office of
the House of Representatives. I believe that the American
Liver Foundation was provided with some copies of the
letter, as well, these being at taxpayer expense. This was
what I was doing between the time the letter went out and
between the time I mailed it to you guys. So it was
something being done, yeah. It's being done.
DR. KUHN: And I understand it can go out on the
Net, but then there's not a whole lot of access to the
American public to the Net and then even finding where it is
on the Net. I guess, and I'm understanding also that it
probably went to each member in Congress, this letter?
DR. NIGHTINGALE: It was a copy in a white
manila--well, in a manila-colored manila folder--was, in
fact, delivered by--we hired a temp, a really good guy.
CAPT. McMurtry: It went to every member of
Congress, every voting member of Congress, every nonvoting
member of Congress, the representatives from the trust
territories and the District of Columbia. If there was an
office in any House building, it got a copy.
DR. NIGHTINGALE: And it got a copy the day after.
And that copy--actually, it was 2 days after, because we
spent a day having the temp run the letter through the
autopen, so it would have the same signature--the original
legal signature and not just a rubber stamp.
CHAIRMAN CAPLAN: I'm going to go over to Larry in
a second. But so far we know that the Congress has been
notified about Hepatitis C. So anybody else besides them?
MR. ALLEN: I guess a couple of things that I
wanted to know about. First of all, it's been passed on to
members of Congress. Do they have an obligation, from that
point on, to send a letter out?
DR. NIGHTINGALE: No.
MR. ALLEN: Beyond the fact that the surgeon
general has written this letter, who is actually in charge
now? Who do we go to to find out where the next steps are
going to be taken for this?
DR. NIGHTINGALE: Well, I think it was suggested
earlier that the Advisory Committee might wish to make some
statement on the subject. I have not been told by my boss,
the surgeon general, to discourage such a statement. Did
you see me wink?
[Laughter.]
CHAIRMAN CAPLAN: Mike?
DR. BUSCH: I think this is an interesting
discussion because all of us, at the initial discussion, the
idea of sending out a letter to notify the public, said it
sounds like a reasonable thing to do. But then hearing the
costs to me, $100 million, in the context--
DR. NIGHTINGALE: Thirty.
DR. BUSCH: In the context--or $30, depending on,
you know, how it's structured, et cetera--is extraordinary.
And I really would question whether the concept of a letter
to the public, particularly one focused, in part, on
transfusion recipients or even on HCV is an
appropriate--this is an appropriate mechanism to raise this
issue. I think you've done the correct thing in putting the
brakes on this and asking is this the mechanism. And I'm
not so sure it is. And certainly if there is going to be a
letter, I think it perhaps should be framed with much
broader implications than certainly transfusion recipients
or Hepatitis C specifically. I think 99 percent of these
are going to end up in the trash can.
DR. NIGHTINGALE: Dr. Busch raises--we didn't come
to the 99-percent in the trash can letter--but if I could
quote, without hopefully misquoting Dr. AuBuchon in another
context, I think I heard him once at a public meeting say,
"If this cost five bucks, we wouldn't be talking about it."
That was in September of 1998. Jim recognizes the allusion
and doesn't fight me too bad.
There are a lot of things that we could do with
$30 million. Dr. Margolis just talked about a couple of
them. If time was not an issue, I could go on for a number.
There is a--yes, I'm saying it--a finite amount of money
here, and what we're trying to do is to make the best use of
the available money that we have. In that context, however,
I would say one thing that gives me an opportunity to
respond to Jane and perhaps anticipate a response that I
might want to make to some of the other members of the
committee, there is a very common assumption among
noneconomists that many economic issues are zero sum gains.
We spend so much money on a letter, we don't have
the money to spend on something else. That assumption is
not, in many economic domains, true. For example, those of
you who got through EC 1 heard of the multiplier effect.
Hopefully, you remembered it when it came to the final exam.
The recruitment of funds from a government source to a
nongovernmental source is another. In a simple world, we do
simple things. At the risk of dumbing down to the
committee, this ain't a simple world.
CHAIRMAN CAPLAN: Does anybody care to comment on
the impact/effectiveness of the previous Koop letter? I
mean, we did have an experiment on letter writing from the
surgeon general about something.
DR. PENNER: I think that issue that Koop
proceeded with on the tobacco? No. On the HIV? Or the
HIV--yeah, both had surfaced very effectively I think in the
media. So I don't think it would be as startling as the
Hepatitis C. maybe we should send this out with the income
tax forms. That would be cheap.
DR. MARGOLIS: There were several formal studies
done of the America Responds to AIDS letter. Basically,
about half of the households recalled getting the letter.
Those who recalled getting the letter, and this was again
with some pretty wide sampling, of those who recalled
getting the letter, it was probably around half of those
individuals used it to discuss issues about HIV. Now that
letter was focused on transmission issues, not a
call-to-action to do anything. And, in fact, it was quite
variable of what was looked at as to whether anything
happened.
Now, you have to realize that what was in place at
that time were hotlines not only at the CDC or the national
hotline, but also essentially every state was funded for an
HIV hotline. And some states saw increases, some didn't see
any change. So it was kind of inconclusive as to whether it
changed anybody's behavior, but it was clear that it did
change or had people discussing it. I mean, that was kind
of the focus group or the context of it. But, again, it was
a very different at least thought-to-be-purpose. That was
clearly to talk about transmission issues. And I know much
of the issue for this letter is to, again, get people to do
something; namely, to get tested. And so, again, we have no
experience with whether these types of things do that.
MR. ALLEN: I hear a couple of things. I hear Dr.
Busch say something about it might have been right to put
the brakes on. I know I missed the morning session here,
but have we put the brakes or has someone put the brakes on
this letter? That's the first part.
DR. NIGHTINGALE: No.
MR. ALLEN: Okay. Secondly, in my opinion, I see
this committee having an obligation to at least continue
what we've done, you know, up until now, which is continue
to push for some form of notification. I don't see how we
can get around that being on this committee, that we go
ahead with that beyond things like the Internet or PSAs. I
think we need to continue to push for some type of a letter
to go out and find a means of doing it, so that if it is $30
million, maybe there's a way of getting it out cheaper than
$30 million. But I just see that that is an obligation of
this committee to make sure that as many people as possible
are notified in the proper manner.
CHAIRMAN CAPLAN: Well, one thing we could do, if
we're amenable, is ponder this a bit more and return to it
this afternoon. It's somewhat disquieting to me that we may
get the letter rolling without coordination, and the
involvement of other things that might want to accompany the
mailing of the letter. I know the Surgeon General would
like to probably put his health indicators thing into any
mailings that he does so you can find out you should get
tested and you're fat, too.
[Laughter.]
CHAIRMAN CAPLAN: But that's my personal animus
about this thing. The fact is, without coordination, I
worry about impact here. It is expensive, although one
thing we can also ponder if we're going to return to what we
want to do on the letter business is: Has Congress really
taken hepatitis C seriously, the challenge of it in terms of
budgeting and so forth? I remind you, again, we're spending
more on the letter than the CDC office budget, so that's an
issue. And maybe we should spend a lot more on both, but we
may want to say something about rising to the challenge here
in terms of adequate funding for this public health problem.
That was a way of saying why don't we table the
discussion, but come back to it, and go to Keith.
DR. HOOTS: Well, even though it's the heat of the
summer, this is kind of, as I understood it, our January
meeting and the fact that we were looking backwards where
we've come from and perhaps forward where we ought to go.
And so I thought it might be an appropriate time both in
reality and philosophically to look at a couple microcosms.
John has already looked at one. I thought I'd look back at
the hemophilia issue, particularly in the context of what
Jane alluded to, which we've been over many times, which is
that safety is intrinsically linked to adequate supply, and
also alluding to what Karen was talking about with regards
to the precautionary principle.
Perhaps implicit in that is being ever vigilant to
not only do what we perceive to be the precautionary way,
with all the precautionary pattern, but, in fact, to
continue to monitor the fact. And that, of course, leads
into the principles of aviation safety, error management,
which we spent the last two meetings talking about.
So I thought I would give you just a little bit of
an example of how I think some things have worked in this
Committee related to one microcosm, supply of clotting
factor concentrates, but also to point out the fact that it
is an ever present danger and it's always hanging on the
precipice, and give you some examples in real time of things
that could actually influence that availability versus the
demand for that product.
I should start by saying that if--I'm putting
myself in the ever present proverbial pharmaceutical chemist
role, that some of the things I will say, which I
won't--first of all, time won't permit, but I won't offer
opinions about what the solution should be. But were I to
do that, I would have to change my lab coat about five
different times for conflict of interest reasons. But I
want to point out some of the things that have been going on
behind the scenes, and before I do that, I would be remiss
if I didn't acknowledge really a lot of people who worked
very hard on all the elements we talked about today, but
particularly this microcosm: the FDA, the pharmaceutical
industry, the consumer groups and interests, this Committee,
and particularly its Executive Secretary and its staff, all
of whom have been involved on all these issues on both very
high-level, superior sort of policy roles, but also at the
very grassroots level.
If I could have the first--I have two slides just
because these issues are so complex that I thought if I
tried to even remember them all, I'd probably use up all my
time. And I'm not going to go into great details, but what
I want to tell you is that right now, as you all know, as a
direct outgrowth of the discussions that took place on May
8, 1988, and August of 1988, we have had consecutive reports
from PPTA on clotting factor concentrate availability. We,
all members of this Committee, plus all the consumer and
governmental agencies, get these data. We've been following
them very closely, and I think it's fair to say that we made
it through some stormy times in late 1988, but now in
early--or mid-2000, entering late 2000, we're probably back
very close to where we may have been in 1988. And that's
the reason I thought we should visit some issues.
So what could--I should tell you the data shows
that at the very best of times between those two periods,
we've had about 9 weeks' worth of inventory, up to 9 to 11
weeks. We're now back down to 4 weeks of inventory for
clotting factor concentrates overall.
Why is that? Well, these are the issues, at least
as I can identify them, having spent some time about it.
First is production. There have been, as you
know, as we've talked about, new production capacities that
are coming online, new bioreactors by certain pharmaceutical
companies that are scheduled to come on. Overly optimistic
projections on the time line in terms of FDA approval in
some cases have compressed our more optimistic projections
and put us a little bit more precarious than we thought we
might be if everything had gone according to the best
projections. But, again, those are moving along.
As recently as yesterday, we got notification that
one of the newest products is now FDA licensed, and, again,
to be applauded is the FDA for having moved this along very
quickly. But because of some of the last issues I'll talk
about, which are international relationships and the
companies' inability to really meet their own projected time
lines, we won't have this product available next month as we
had originally hoped. And, in fact, it's now projected to
be available in 2001. So all these things are part of what
go into making a projection model in terms of supply.
Reimbursement issues are continuously, as with all
the issues this Committee has taken up, are howling around
us all the time. Most recently, one that has particular
imminent import is actions related to reimbursement of HCFA
programs, particularly--specifically Medicaid and Medicare,
and the reaction by states' attorneys general to average
wholesale pricing, a collective impression on their part
that these AWPs on which reimbursement was based were too
high and, therefore, categorical decisions but not--many
times they were unilateral, those categorical decisions, to
modify those adjustments.
What has been the outcome of that is that certain
of the private sector distributors, like home care
companies, have been put in tenuous binds about being able
to get adequate reimbursement for their services, and in
cases of patients that they supply who are HCFA-supported,
Medicaid or Medicare, have at least to my knowledge on a few
cases actually said they could no longer do it because they
were losing money. So, clearly, even though that's not a
direct supply issue on a national basis, it has localized
geographical significant import.
Similarly, there have been--to say discussions is
probably to be euphemistic. There have been discussions
going on about a prime vendor program which we discussed
once upon a time, and I don't expect anybody around this
table to remember that discussion unless they're in
hemophilia. But essentially HRSA was charged by the
original act that created lower federal pricing for factor
concentrates to look for a way to make sure that all this
pricing could be accessed by all hemophilia treatment
providers and patients around the country.
Well, that also has created discussions about
whether that really is anti-trade and all kinds of issues.
If that were to be--if that were to really go at this
particular point in time to a confrontation between, say,
PPTA and HRSA, to where both threw down the gauntlet and
said we're going to fight this out, whether legally or
otherwise, that could have an imminent impact on supply.
It's particular important, I think, to draw from
the analogy of the aviation industry where we learned very
quickly that when weird things happen in multiples, things
that shouldn't happen, that's when they happen. I mean,
that's when you are most vulnerable, and that's the reason I
decided to point out these issues, because at this point in
time I think we're vulnerable to each of these issues.
The next slide, please?
Distribution. There have been proposals. Some of
the newest technologies that are coming on, recombinant
technologies, are just--one of which has been delayed, as I
just told you, but another one that's coming online
imminently, there have been proposals that it be provided on
an allocation basis based on certain individuals who opted
that they wanted to just go that route and put their names
in, it would be a first-come, first-served basis.
A number of us have argued strongly that that
takes the flexibility out of the system in terms of the
end-user level, if I've allocated Product A for little
Johnny and little Sammy has a bleed that's acute and little
Sammy is not part of that allocation system, if they do
that, I can't borrow from little Johnny to help little
Sammy. So it takes our flexibility as treaters out of play.
So those are all issues, I think, that in a time
where a shortage is imminent or where supply is marginally
adequate, where we have to continue to argue all the facts,
because keep in mind that these decisions in many cases are
made irrespective of issues of supply. They're made for
economic reasons. They're made for political reasons or any
other kind of reasons. And so it's our job collectively,
both as, you know, members of this constituency but also as
broader--this whole Committee, I think, to have people
around who are continually--and we do have them--continually
monitoring along with the federal agencies like the FDA.
The other thing about it, as everyone knows, we
recommended on this Committee that clotting factor
concentrate use in the United States moved to an all
recombinant supply. Well, clearly, the biggest impediment
to that is the supply itself, and the less available
recombinant there is, the more likely that people who would
otherwise move are not going to be able to.
Which leads me into the last point, and I think
I'm probably right at the end of my five minutes, to say
that as we've talked about before, that's not just a U.S.
issue. That's an international issue because these are
multinational producers, and they're also chains of
suppliers that are multinational as well, along with supply
routes. And so I'll take the example of the most recent
product that got delayed until January 2001.
In that case, that product was licensed in Europe
first, and right now the demand in Europe is so great that
there has been a reluctance, not to divert because there was
never any intention to divert from that supply, but I'm sure
it's influencing the availability, how quickly they can come
online to provide a North American supply to this, which
would help ameliorate the recombinant shortage, which then
finally kicks down to the plasma-derived supply piece, which
I want to end up with by saying--to remind ourselves--and
it's already been alluded to multiple times--that this is a
domino effect. So that if we don't have the recombinant
supply in the developed countries, we're going to draw on
the plasma-derived supply that is intrinsic to providing the
care for the developing world.
So all of these issues are exceedingly complex, as
you all know or you wouldn't be here, but I think they're
things that indicate why we need to be ever mindful as we go
along not only what we should recommend for our government
and our policymakers to do, but to kind of stay on the case
to make sure that, as we go along, as new contingencies
occur, which they inevitably will, that we're there ready.
Because the one thing I, as a member, personally--as a
member of this Committee, I would want to think that when
someone says, well, why didn't somebody think of this, we
can say we did think of it and we tried. Even if we fail,
we at least thought of it and we tried.
CHAIRMAN CAPLAN: Comment?
[No response.]
CHAIRMAN CAPLAN: Thanks, Keith.
Let's go to Paul.
DR. HAAS: I guess I'm going to ask you to indulge
me as an academic for five minutes. And as I was putting
these thoughts together, I had several titles running
through my head, but I guess I ended up with "Economics and
Blood Products: Do They Mix?"
The two most common economic terms, supply and
demand, are probably the most commonly misused terms.
Supply and demand reflect dimensions of both price and
quantity. Supply and demand flow from the concepts of
competition, which is built on the assumption that there are
a large number of sellers of a homogeneous product, that
buyers and sellers are well aware of the properties of that
product, and that the entry into a market is free and easy.
Furthermore, it is assumed that products are
manufactured in a single plant producing a single product.
Right away we should understand or see the complications
with these assumptions related to the so-called market for
blood products. While there's a significant homogeneity
within various blood products, there are multiple products
included in the blood product category. Depending on the
product and the timing, there may or may not be many
sellers. And we heard from John that there's one seller of
his product. And the concept of a single plant producing a
single product doesn't exist.
Entry is not free and easy. While some of the
general forces of competition might exist, the distinction
of real conditions from those in the competitive market
cause alterations, often significant alterations, in the
expressed outcome.
In addition, the so-called market for blood
products is complicated by the role played by government.
Also, the term demand depends upon a large set of
assumptions that rarely are met in medical economics. For
example, the demand concept is compromised by the existence
of third-party payers, which themselves are compromised by
the existence of privately purchased insurance,
fee-for-service and managed care, government-provided,
insurance and charity.
When demand for blood products is considered, are
we really talking about, one, the willingness and ability of
patients to pay for the product? I don't think so. Two,
the decision to provide blood products with or without the
payment by individuals or insurance? Or, three, simply a
measure of quantity devoid of any concern about price?
The first option is the definition of demand under
conditions of a free market, which, of course, do not exist
in the market for blood products. The last view doesn't
make any economic sense because the cost to provide the
products always exists. Even if the society were to supply
the blood products free of charge, there are still the taxes
and the opportunity costs associated with using the
resources to supply that blood and not something else.
Obviously I could become very pedantic, but the
fundamental point is that economics is very much a
discipline that's concerned about choices, but its models
must be used carefully or the choices offered by the model
will not be equitable or efficient.
Economic reasoning applied to the public sector
must use supply and demand reasoning very cautiously. Since
society has determined that one's income should not
determine one's access to health, then health care and blood
products move out of the private domain of demand and supply
and into the public sector. Yet this movement doesn't avoid
the problems associated with cost or rationing, as those of
us on the Committee read that article about rationing.
Rationing is there because there's a trade-off.
Every economic decision is a rationing decision.
The controversy isn't about rationing but how the rationing
should be accomplished, and Jane and Karen were really
saying similar type things.
Economics has given us cost/benefit analysis and
cost-effectiveness analysis to approximate the forces of
demand and supply. Cost/benefit analysis attempts to
determine the most efficient way of accomplishing a given
activity. Cost-effectiveness analysis attempts to broaden
the scope of the analysis by including other dimensions such
as pain, suffering, and disability, that is, value
judgments, again referencing another article we have in the
packet.
Cost-effectiveness is the more appropriate
analysis because value judgments are an essential part of
the process. If we could rely upon the existence of a
competitive market, then we could rely more heavily on the
concepts of demand and supply or cost/benefit analysis,
which under conditions of competition provide results that
are both efficient and equitable. Since the competitive
market for blood products does not exist, use of
cost/benefit analysis is likely to provide deceiving
results.
As the article "Cost-Effectiveness Analysis in a
Setting of Budget Constraints--Is It Equitable?" points out,
lack of measures of demand and supply leads to a form of
cost-effectiveness, equity does matter, equity matters in
competition. But the competitive assumptions are
unrealistic in the blood market. If we are not careful,
then efficiency concerns dominate equity concerns in
economic decision making.
The supply problem associated with the blood
supply are complicated by the desire to provide only the
best--and I think that means safest--blood product. If we
truly believe in market forces, then blood products with
many different degrees of safety would exist at a variety of
prices. The safest blood products--and that includes
getting the blood product safely to the bedside--would have
the highest price. Because of equity concerns, society
would want blood to be as safe as possible, but often
forgetting the costs associated with that desire.
Yet we don't know what safe really means. Is a
blood product safe if the probability of transmitting the
disease to a typical blood product recipient is one in a
million? What if that same product is given to a heavy user
of the product, such as hemophilia, alpha 1, immune
deficient patients, would the probability of transmission of
one in a thousand or one in ten thousand be considered safe?
Is it possible to provide a variety of varying
qualities of blood to different groups? I don't think so.
Yet, as an economist, I'm supposed to present options. I'm
supposed to--but as soon as we move away from the conditions
necessary to permit an effective use of the concepts of
demand and supply, economists are subject to concerns other
than efficiency and profitability. In a perfectly
competitive market, efficiency, profitability, and equity
happen naturally. No human interference is necessary or
even desired. Unfortunately, no one has ever seen that
competitive market, certainly not in the health and blood
areas.
Unless we or society are willing to define what is
meant by safe blood and what is meant by the demand for and
supply of blood products, then this Committee will always be
facing the impossible task of deciding what error we are
willing to accept: error one, very safe blood but
inadequate supply and high cost of production; or error two,
less safe blood, more supply, and lower cost. And, yes, I
know there are many other options, but I'm not going too far
over my five minutes.
In the article "Cost-Effectiveness Analysis in a
Setting of Budget Constraints--Is It Equitable?", the
options are clear. Of course, with a little bit of creative
thinking, there are additional options, such as increasing
the budget. And, again, a couple of other people have
mentioned that. This approach is harder to achieve, but
failure to think outside of the so-called box limits our
ability to assess all options.
If we and society want an adequate supply of the
safest blood products, then more resources need to be
devoted to this area, and some other area--who knows
what?--education, defense, environment--again, Jane
mentioned some of these--will lose resources.
The economic refrain of Tans Stoffel (ph), "There
ain't no such thing as a free lunch," never goes away. With
this thought in mind, I personally prefer to push the
envelope on safety and search for resources to accomplish
this goal. I take this stand in spite of Dr. Klein's
editorial in JAMA pointing out the trade-offs between safety
and equity. I argue that we need to set the safety standard
according to the needs of those who use the product on a
regular and continuing basis. I do not argue that blood
products need to be 100 percent safe. I know that goal is
unrealistic. But I do, however, argue that until the blood
industry restores its reputation, safety has to be the
dominant concern.
CHAIRMAN CAPLAN: Okay. Thank you. Let's go to
Fernando. Then we'll get to me, and then I guess we'll get
to lunch and then we'll pick up with Ron and go around. I'm
sort of hopping over our ex officio and non-formal members
in the presentations, and I thought we could hear them, open
that up for discussion, and then swing back around to
reconsideration of anything that others who are observers or
ex officio members might want to say, and then come back to
the letter issue and whatever else is on the table. So
that's my rough tactic here.
DR. GUERRA: I think Ron had a comment to make. I
saw his hand up. No? Okay.
Thank you for the opportunity, and certainly being
privileged to serve on this Committee has given me a
real-time opportunity to see for the first time what I
consider some interfaces of public health and the blood
industry and blood banking and those enterprises, especially
as that interface plays out in large urban centers.
But I think that in the first instance there are
clearly some overlapping obligations, responsibilities, and
opportunities, and those are certainly for surveillance, for
prevention, for screening, for testing, for communicating,
for tracking, for doing a lot of things that maybe we have
not always thought about in the context of that interface,
and where I think the collaboration certainly in the future
could open up some incredible opportunities for how we
convey to the general public those issues, those concerns
related to the transmission of disease beyond hepatitis C,
HIV/AIDS, but any number of other conditions that perhaps in
one way or another, directly or indirectly, come very close
to the nation's blood supply.
I think that there is a real need for being able
to better communicate what is real versus perceived risk,
which, again, is very much a part of what public health does
on a regular and ongoing basis because of so many conditions
that we're faced with and that we have to somehow
communicate to communities that today are very diverse and
that at times their grasp of information is limited, and so
one has to develop some very creative types of strategies,
especially for dealing with populations at risk, as some of
the targeted populations, the populations that are homeless
or those that engage on a regular basis in a variety of
risk-taking behaviors.
I think that an area where there is a real
strength in trying to more clearly define that
interface--and I hope that it is an opportunity for the
future in terms of some of the agenda items--that public
health probably has a much greater access to population
data, linking data sets, looking at vital records, what
birth and death rates are, what the number of C-sections
are, how or what the utilization of blood is, what inventory
is. I mean, some of that is tracked because of other public
health responsibilities that one has.
But there is an emerging natl information system
called the Health Alert Networks, where I would think that
it is possible to link into some of those information
technology networks, some of the issues, concerns, and
opportunities related to the blood banking industry and the
populations that are served.
Public health in the instance of accessing the
political arena, in particular elected public officials,
especially in local communities and at the state level,
where that access could perhaps be a very powerful one for
the blood banking enterprise in both the public and the
private sectors. On a regular basis one is faced with
having to access that arena for either allocation of
resources, for concerns for development of legislation, for
ordinances, et cetera.
There is hopefully--even though I think we heard
from Dr. Satcher on any number of occasions, and certainly
we've heard it in some of our discussions, we don't always
have the capacity and/or the support for that capacity for
tracking populations. But there is some expertise that
could be expanded, and I think that there perhaps could be
some additional support brought into it for such things as
outreach and case management. The DIS staff, for example,
in public health departments that track--that does the
contact investigation when there are a number of infectious
diseases could certainly be called into play for some of
these other efforts.
I think the interface would allow really for the
expansion on both sides of the leadership role in first
responder type of activities. There's no question but that
the nation's blood supply faces the same threat that
especially large population centers face related to weapons
of mass destruction, bioterrorism. Contamination of the
nation's blood supply could be a tremendously serious
situation that I think all of us would be concerned about.
I think public health has at times had some
special initiatives, some that are just emerging related to
environmental concerns. This is an area that really needs
to be linked very closely to both the supply of blood, the
donor supply, et cetera, related to environmental pathway
exposures. We don't always understand, for example, the
exposures to potentially toxic chemicals, insecticides,
pesticides, or dietary substances or heavy metal
exposures--any number of things, radiation, et cetera, that
I think perhaps could pose some additional threats that
certainly have to be looked at and addressed.
I think if there is an opportunity to deal with
some of the issues of disparity and social justice, it is in
the interface of public health with the blood banking
industry. We've learned in public health, in keeping with
some of the finest traditions--and I think we've seen it
certainly in the list that Dr. Satcher handed us. But
there's an even longer list that deals with disparities
across population groups and communities by race, ethnicity,
and socioeconomic status, and I think we see that play out
every day within the blood banking industry and the
populations that are served or not served or those that have
particular conditions that have to be recognized as part of
a greater social justice discussion.
Then, finally, in the instance where there is
potential harm to large numbers of people, it is public
health in keeping with, again, the authority that is placed
within that system, and in particular those individuals that
carry the designation of local health authority that have to
sometimes issue quarantines and/or shut down operations if
they pose a threat to groups of individuals. And so I think
that it is very clear, as I have been on this Committee now
over several years, that somehow we need to better define
and forge the relationship at the interface between public
health and the blood banking industry.
Thank you.
CHAIRMAN CAPLAN: Thank you, Fernando.
I think I'm last up and headed for lunch, so I
will keep to my five minutes, although somebody can yell at
me if I start to lurch over.
I tried to think hard at the beginning of this
summer about values and principles. I got asked to write
something about leukocyte reduction and some of the issues
that came up there, and I never got it done because I got
caught up in brokering this peace treaty between the private
sector and the public sector on who is going to figure out
what to do with the human genome. But I still have been
thinking about it, and I hope to write about it, and it led
me to reflect on a couple of things which, I guess, in the
spirit of Paul's remarks, I could call it bloody ethics.
Sometimes ethics looks like a nuisance here. I
think overall when I try to think about competing social
goods that we have to wrestle with, spending money on blood
versus other things, what I find myself thinking about this
Committee is that our mandate is blood safety and
availability, and the advice we should give, as I see it, is
to figure out how to get a safe and available supply of
blood, but it is not always to weigh other things that are
important to do or good to do.
If I had more confidence in the political system,
I'd feel better about that. But, nonetheless, I find myself
thinking the job is to figure out, given many choices and
options and expenses and opportunities, what do you do to
get a safe and available blood supply?
I have always found it interesting that our
Committee is called Blood Safety and Availability. It's not
Blood Availability and Safety, and a lot of our debates go
have we gone too far down the safety road and jeopardized
the availability part. So we sort of took safety as our
starting point. Safety in some ways triggered our
existence. Safety has been the driving force behind a lot
of what I think the congressional or the public concerns
are. And, ironically, availability often takes second
place. I'm not sure that is true. But I don't worry as
much overall in a sense of justice about trying to weigh
childhood vaccination or a better highway system or more
seat belts or better tires. I think it is our mandate to
think about blood safety and availability, and then Congress
and the politicians will think about all the rest of the
things.
So that's how I sort of think about the large
picture of competing goods when we ask questions about $30
million letters or is it worth doing that testing or
leukocyte reduction. It may be that we shouldn't even have
a Blood Committee. We should have the Surgeon General send
out a letter saying: Do you know you don't have health
insurance? And that would be the most politically adept
thing we could with mailings, but, all right, it's not about
blood so there we are.
Within that context, then, I always find myself
thinking not so much about principles as about values. And
one thing I know--and Jane's knows this better, but I have
read her book and I've talked to her about it off and on--is
that this is a wacky area because it is not about economics
since the provision of the substance that is at issue is a
gift, and it always is rooted in this ethics problem of how
do you cement public altruism to give this substance, blood,
that then gets a cost put on it and then gets into
distribution.
I do know for sure that if the public perceives
that the distribution is not fair and that the cost or price
that's being charged is too high, then altruism as the basis
of collecting the substance is in jeopardy. It just becomes
tremendously put at risk. I know that because I've studied
for a long time the organ and tissue worlds, and nothing
dries up altruism faster than a perception of inequity in
the distribution of a gifted item.
I also know that this is a weird area, as Paul was
hinting at, because in terms of values, if we are going to
commit to altruism, we have certain obligations to watch
distribution that aren't market-driven because we are trying
to get everybody to give, in theory, and that makes for a
problem as opposed to those who could pay.
The second thing I know is that the use of the
substance is involuntary. So it's not something that you go
and get; it's something that you have to--very few people
have said to me, Boy, I hope I get to use blood anytime
soon. Most people, the majority of users, I would venture,
don't want to. They have to for various reasons.
That, of course, puts them in a position then of
feeling exposed to risks or dangers that might be different
if somebody said I'm going to drive real fast and have many
deaths on the highway every day, but I chose to do that, as
opposed to here's the safety that I have to face if I must
use this thing, which I don't want to, but it's foisted upon
me.
I think sort of the first value that's out there
that we have to be alert to is altruism. The second is the
involuntary nature of the use and what that means in
thinking about safety. I think it means something different
than when you voluntarily assume risk or voluntarily do
certain things vis-a-vis behavior that can be pretty risky.
Another factor that plays a role here is what I
would call privacy and stigma. If you look at blood and
blood transfusion, blood product use over the years, we
can't even talk about hepatitis in this country without a
lot of people worrying that they're going to be typed as a
particular lifestyle or having a particular background. And
HIV certainly has plenty of that, and most of the
infectivity in the blood supply carries stigma. It also
carries the very real prospect of discrimination. So that
values about privacy and confidentiality loom large because
of the connection to stigma and discrimination.
So here we have to not only account for what's
efficient, we have to make sure we're building privacy and
confidentiality protections in as we think about safety and
availability at all points, just because of the nature, the
controversial nature of the substance.
I have to say, too, even though it makes me sound
almost like Martin Boober (ph) for a second, that blood is
symbolic. There are a fair number of people who still say
that the way they define kinship with one another is through
blood. And I could go on about the symbolism here, but when
you have an entity that is not seen as an oxygen-carrying
fluid but a mystic substance of life or other types of
things that different cultural and ethnic groups have, then
it imposes even more limits on what you can do vis-a-vis
safety and purity.
I once mentioned to someone in the biotech
industry that were I to start making genetically modified
foods, my first one would not have been milk. Blood has
this problem, too. It's just fraught with symbol, and,
therefore, people have expectations about purity and safety
not consistent with what they might expect from other arenas
of their lives. That's a reality that we have to struggle
with.
I'd say one other thing. As I watch this group
function and listen to different voices, I come to
understand that blood is one of the few ways Americans can
seem to be a community. There's almost nothing that makes
them seem to be a community. We don't even have baseball
anymore. But we do have blood collection and blood drives,
and not-for-profit voluntary organizations that pull the
community together, and we make appeals to one another to
pull society together.
This may be a funny social glue, but there it is.
And we could shift the ideology of it. We could be less
concerned about gifting. We could be less concerned about
altruism. We could be less concerned about community. But
if you look at the blood organizations, the blood banks, the
kinds of things that they talk about, it is very clear that
the value of community is trying to be exemplified at all
points in what takes place with respect to blood.
We get disappointed when we hear about blood
donation rates dropping, not just because it means that
we're at risk, it's because it says something about society.
I've had that said to me as I've left this room, whenever we
see that little curve, somewhat in dispute about how bad the
dip is. But when you put up a curve that looks like it
might be a dip in supply, people sort of say, Boy, this
country's going to hell. They don't say I'm at risk. They
say this is bad. This is a bad thing about America.
So the value of community is there, and I might
add it's reflected in another two special areas: the
liability on the product is different from what happens in
the market normally, so it's got special status and
protection because of this altruism community set of values;
and you've got in play something about the modulation of the
cost since a good deal of the blood industry moves under the
banner of not-for-profit. We can dispute whether that's
true. There are certainly segments that are for-profit, I
understand, but generally speaking, I think the American
people have said a gifting, altruistically driven,
community-based system should try to reflect a
not-for-profit ethos in terms of what makes it tick and
function.
So what I would say by way of my pre-luncheon
reflection is I don't know how yet--I still don't understand
exactly how issues of fairness, compensation, precaution,
and risk avoidance play out. But I would say this: It
becomes very tough, I would agree with Paul, to just take
cost/benefit or cost-effectiveness analyses or efficiency as
the sole value here because you've got this other set of
values that you're constantly trying to mix with this notion
of efficiency.
I think it's also true that even though it
irritates us all the time about trying to say how important
is it to get marginal safety or marginal increases on risk
avoidance, to some extent the symbolism, the history, the
sort of moral status that blood donation plays for us, is
reflected in what people are willing to pay to get that
marginal utility. It doesn't make sense. If you just toted
up the numbers, there's no sense at all. But it's probably
playing roles other than that, and to change that--I'm not
saying that's the correct ethical outlook, but if you want
to change it, you've at least got to say you want to modify
these other value dimensions I've been talking about, you
want to shift them, they don't make sense, or they're not
worth preserving. They cost too much, literally, if you
want to put it that way. But I think that's why we're not
so efficient in what we do. That's because we're doing a
lot of other things with blood that have nothing to do with
who's going to get a safe unit transfused to them, or at
least a reasonably safe unit, and it's why people I think
are so concerned and maybe even why Jim's article comes out
the way it does that you've got one of the safest things
going, and yet a third of the people who hear about it are
still not sure that it's safe enough, and a lot of others
could shift their attitudes about it. It probably tells you
what they think about their neighbors, what they think about
stigma, what they think about symbol, as much as it does
what they think about somebody's safety analysis.
So there are some big thoughts to launch us off to
lunch. I will take no questions on these truths, and--
[Laughter.]
CHAIRMAN CAPLAN: And then the game plan is,
however, to swing back around over to this side, stop, talk
about what we've heard from the members add other people in
who are here, who may want to say something, observers in
the ex officio role, and then revisit what we need to
revisit.
I think the dream today--I mean, I'm amenable to
hanging around as long as people want to hang around, but
the target goal might be to maybe get us out by 4:30, but I
mean, if we go longer, I'm here. However, if we're going to
do that, you really have to get back from lunch in an hour
or less, so one hour. It's now about 12:30, so I'm going to
try to kick this off at 1:25, and that should give us enough
time to keep moving here. Okay.
DR. HOOTS: Can I have a autographed copy of the
stone tablets?
[Laughter.]
[Whereupon, at 12:30 p.m., there was a luncheon
recess.]
A F T E R N O O N S E S S I O N
CHAIRMAN CAPLAN: Can we reconvene?
You will recall the moving final statement by the
Chair before lunch left us in a position to now move to a
more concrete insight. Ron, I think you're up next if you
want to, for comment.
DR. GILCHER: Thank you. It is interesting that
the comments about--which I will make in a moment from a
prepared statement that I prepared before coming to the
meeting--are very similar to comments that have been made
already by Dr. Penner, by Karen, supporting those views, but
in somewhat of a contradistinction to what Jane Piliavin
said. And the statement relates to blood safety, blood
supply and cost, and it is from my perspective as the
director of a large regional blood center and the impact
that the initiatives that we have put in place have had in
our system. So I'll read this statement and try to stay
below 5 minutes, Mr. Chairman.
The safety of blood products is regarded by the
recipient patient as being of such critical importance that
the inherent and perceived risks should approximate zero
risk as closely as possible. The receipt of a blood
transfusion is analogous to flying as a passenger in an
airplane, which we've heard from you, Karen. Specifically,
zero risk or zero defects, even though realistically not
possible, remain the goal.
In transfusion medicine we have approached the
safety of blood products in three general ways, and these
are not the only ways. Number one is pathogen detection.
Number two is pathogen removal, and number three is pathogen
inactivation.
Very briefly, pathogen detection, that is,
testing, now uses the state-of-the-art testing, nucleic acid
testing, to approach zero risk for specific pathogens,
again, HCV and HIV. In a low-risk system for HIV, such as
in our system in Oklahoma, the estimated cost to detect and
prevent a window transmission for HIV will probably be
around $7 million, with one detection estimated to occur
over and above our current systems, using that about once
every nine years. For HCV this cost is about $1 million per
detection in our system. Theoretically, these are the costs
to detect one HIV or one HCV positive unit, yet nobody knows
which unit that is until it happens.
Number two, pathogen removal, which includes macro
and micro-filtered blood for removal of clots, aggregates
and other debris, has been used for decades to prevent
theoretical embolic risks and other risks, and now we are
using leukocyte reduction, which has the ability to remove
cell-associated pathogens for which we do not, cannot, or
will not test. At the Oklahoma Blood Institute we adopted
universal leuko-reduction as of July 1st, 2000 for all red
cells and platelet transfusions. We had had that in place
for platelets already for over five years. From hospital
surveys which we did, we estimated that approximately 40
percent of all red cell transfusions in our system went
through a micro-aggregate filter, for which the cost of that
was about $380,000 per year, and average cost of $9.85 per
filter. Universal leuko-reduction has removed the need for
micro-aggregate filters, but has added about 1.9 million or
a net increase, if you look at that in our system, of about
1.54 million in 96,000 red cell transfusions, and
interestingly, that comes out to about $16 per red cell
unit, and that is applied to every single blood product that
goes through the system, in contradistinction to NAT, which
is only picking up the two.
Longer term cost effective studies are now in
progress at two of our major hospitals, and the preliminary
data is very interesting because one of the hospitals was a
nay-sayer, and in fact, they have the data that supports
leuko-reduction even more so than the hospital that was in
favor.
Number three, pathogen inactivation is the newest
safety initiative for blood products with much of it being
research based, although it has been used for plasma
derivatives, and now a pool form of fresh frozen plasma,
where it has doubled the cost, with some questions regarding
safety enhancement in the face of the advanced pathogen
detection technology, that is, NAT.
These three approaches, pathogen detection,
pathogen removal, and pathogen inactivation, at times
clearly overlap, and there's a significant cost to that
overlap. Identification and subsequent removal of the
unnecessary overlap can result in cost savings so that the
goal of zero risk and the approaches toward zero risk, can
be economically, ethically and medically justified.
The perception and reality or working towards zero
risk or zero defects for blood products is--and this is in
my experience and opinion--also very important to blood
donors, and ultimately influences the donation rates and
availability of the blood supply. We have clearly focused
on that in our system, and as you know, it is one of the
systems that generally always has enough blood.
In summary, zero risk, although not achievable,
should remain the goal in transfusion medicine. To do less
will continue to fuel the fears of patients and the
distrust, mistrust of both donors and patients. The end
result is then an inadequate blood supply and ultimately a
less safe blood supply. Thank you.
CHAIRMAN CAPLAN: Questions, comment? Thanks,
Ron. Rick?
DR. DAVEY: Well, Steve, I took you at your word
fairly concretely when you suggested that we as a committee
identify a set of intellectually consistent and politically
acceptable principles on which a coherent and durable policy
to assure safety, availability and affordability of blood
and blood products can be established. And so I did look
over some of the readings, do some thinking about this, and
thought that I might just have a condensation of some of the
principles that were in the precautionary principle paper,
as well as some of the thoughts on papers by Ubel and
others, and maybe suggest a template that might have been
useful for us to use in the past on some of our
deliberations, that might be also useful for us to consider
when further actions of this committee might be considered,
and again, just putting this in the context of some of the
comments that Art made about our special gift relationship,
the altruism, that we have to consider the confidentiality,
the privacy, also the political, scientific and financial
environment that we specifically have to deal with in blood
and transfusion medicine.
But with those--in that context, I'd like to
suggest seven evaluation parameters from the readings and
some elaboration that I've had on them that we might want to
consider. I guess I would consider them evaluation
parameters, and maybe think of these seven things almost as
a template. Whenever a new action is considered, are we
going to think about each of these seven items and consider
them before we decide on what to do.
Again--and this is in some of the readings--but
the seven are as follows. Number one: Whenever we want to
consider an action that affects the blood and blood supply,
is it proportional to the chosen level of the protection
that we want to have? For instance, we knew we were going
to need to tailor measures to be appropriate for the level
of protection that we're looking at, and again, we might
want to think of the appropriateness of the UK ban here. Is
that proportional to the level of protection? That's number
one.
Number two: Is the action equitable? Is it
something like in consideration of what Jane was saying
earlier and in consideration of the paper by Ubel, is it
something that can be equally applied to all people that the
action affects? I think the paper by Ubel is very
interesting, if you read it, where there was a evaluation of
two different ways to screen for colon cancer. One was more
cost effective and one was more equitable, if you will, and
it was the equity that carried the day in terms of when
ethicists and medical professionals reviewed the two
opportunities.
So proportional to the chosen level of protection,
equitable. And three--I think this is what Jane was looking
at--is it comparable? Can comparable situations be looked
at comparably? Can we look at Chagas Disease and New
Variant CJD and say, "Look, are we dealing with these two
issues in a comparable fashion or not?" So is our
action--is the consideration of the action comparable?
Fourthly: Is it consistent with other similar
measures that have already been taken? The actions we might
want to consider should be of the scope and nature of those
that have already been done, that we're not going off on
some kind of tangent.
Fifth: The actions should be based on cost
benefit analysis. There should be a evaluation, not only
maybe of the economic cost benefit analysis of a particular
action, but maybe some of the non-economic considerations
also. Is this an acceptable action for the public to
consider? So cost benefit analysis for sure. That's five.
Six: Is the action really subject to review? Is
it something that can be periodically reevaluated, and if
necessary, reviewed in the light of new scientific progress,
amended if necessary? Can it be reviewed?
And lastly, and maybe related to the subject of
the review, can responsibility be assigned for getting the
scientific evidence so that it can be reevaluated and
amended? Can we assign responsibility to follow up on these
issues?
And I think when we think of 100 percent leukocyte
reduction and HCV look-back, many of these, I think,
principles, could have been perhaps better utilized in our
deliberations. So, again, I would very briefly suggest that
we have a template, that we think of these seven things when
we review our actions, that they might have been good in
some cases in the past.
Again, let me quick review them again. Number
one: Is the action proportional to the level of protection?
Is it equitable? Is it comparable to other situations that
have occurred in the past? Is it consistent with similar
measures we've already taken? Is it based on cost benefit
analysis? Is it subject to review? And can we assign
responsibility for producing that evidence?
Simple, but it might be seven little principles
that would be useful as a template whenever we think of
something new. Those are my comments.
DR. BUSCH: Just a follow up. I know both Steve
and Jay and I were involved in a WHO program that I guess is
going to play out in the next meeting as well, but similarly
attempted to define a process, you know, a really on-paper
consensus process of issues that needed to be addressed in
terms of making decisions. I know Jay has been much more
involved than I, but I think what you've done in terms of
trying to lay out concrete elements of decision making is
what I hope we end up talking a lot about and working a lot
on.
DR. HOOTS: I just wanted to make a comment in
terms of one of the articles. I think one of the--where the
rubber meets the road and where the complexities comes in,
as is pointed out in the cost efficacy versus equity
discussion. And perhaps what we need to do in addition
to--and I applaud your effort to organize the thought
process--but to look at really where the conflicting
components almost inevitably come into play, and then also
then further sub-stratify or sub-strategize, perhaps is a
better word, how you deal with those issues, because that's
really where we always kind of end up.
DR. PENNER: Maybe one other comment, and that is
a question. Is it relevant with respect to other costs that
are involved in therapeutic and medical care? As I'm
recalling, we were paying, I think, something like about $45
20 years ago for a pint of blood at that point, and now it's
something like about, what 92? So it's--over 20 years it's
increased, doubled. And what's happened to the antibiotics
and the other therapeutic agents that we're using cost-wise?
I think I'd still make the point that we are probably under
selling or under costing our blood products in comparison to
many of the other products that are being used as
therapeutics, and that kind of mindset has been hard to
dislodge, that we should just maintain that level of pricing
for those products just because they're, supposedly,
initially, voluntary. I think we have to really look at it
in terms of market, and market value on everything else has
gone sky high. If we're paying $1,000 a day for some
antibiotic regimens in our intensive care unit, and we're
still giving two units of blood at maybe $140. It sounds
like there's a discrepancy there in relationship to the
significance of the product that we're giving that is being
overlooked.
DR. BUSCH: Yeah. I presented about two months
ago a talk that Steve was at at CDC that he thought maybe
some of this would be useful, and a lot of this is stuff we
kind of know, but I think could make some points sort of
relevant to the decision process, particularly as it's
evolved in my area of focus, which is in the testing side of
blood safety.
If you could turn on the first slide, please.
Yeah, this is just sort of a strong backdrop of how much
safety we've achieved, and I think there were earlier
comments about sort of the perception of the public. And I
remember a colleague from CDC, who worked in blood safety
only for a few years, Lyle Peterson, five or six years ago
made the comment that, "You guys are completely missing the
aim. You should be out there emphasizing what an incredible
success story the elimination of transfusion-transmitted
infections is to the public." Instead, all the focus is
down on the incredibly minute residual risk, and we're not
adequately educating the public about the extraordinary
safety of blood.
Next slide. One of the other things that I think
we've struggled to do, and it's kind of bit us, is we've
spent a lot of time and a lot of energy trying to estimate
risk, the residual risk. And it's required--you know, we've
moved away from simple ways to measure the risk to
recipients, following recipients and measuring
sero-conversion rates or doing special testing of donor
units to find rare residual infections. We've moved away
from those because you just can't do studies big enough to
quantify the extremely low levels of residual risk.
So what we've done in the last, you know, five to
ten years is develop model strategies that estimate the rate
of window-pair period donations, the potential sources from
variant viruses, unusual people who don't sero-convert, and
testing errors, and this has been a sort of building process
over the last, you know, ten years, that have come down with
numbers that are extremely low and essentially unmeasurable
in the range, for example, of HIV of, you know, 1 per
million anticipated transmission event. And the problem
again is how do people weigh, in a risk analysis sense,
these extremely low numbers? In general, my sense is that
people take any quantifiable number as a problem, instead of
having some way to balance the risk of these remote
transfusion events compared to other risks that are faced.
But in any event, we have put numbers on risks and
have developed strategies that we thought were pretty
reasonable to estimate risk. These model projections of
risk and value of new tests have been criticized in the
past. For example, after the debate over antigen testing,
where we presented estimates of the yield of antigen
screening, Congress came back and criticized those as gross
underestimates of the yield without a lot of data. So I
think one of the questions that is important is, as
suggested a few minutes ago by Rick, is coming back to our
estimates and trying to benchmark and confirm that those
were reasonable. And we'll come back to that in a minute,
because I think the introduction of nucleic acid testing has
really very adequately confirmed these estimates that are
laid out here.
Next slide. Because the window period, as you saw
on the prior slide, has been the major theoretical source of
residual risk, there's been a lot of work that's gone on to
estimate, understand the dynamics of virus during the window
period, and estimate the ability of various new tests, be it
antigen, NAT, either applied to small pools or single
donations, to further close the window period. We don't
have time to go into that, but I think there's an enormous
amount of strong data that allows us to estimate how many
additional days of closure could be achieved by adding this
test or that test, and that's both for HIV, and on the next
slide, for hepatitis C there's a similar large body of data
that's over the last few years been generated to understand
the duration of the viremic period prior to antibody, and
the ability of various test strategies to interdict these
window period donations.
And using that--next slide--we've been able to
estimate how much these nucleic acid tests, which are now in
place, have been able to reduce the window period, and one
of the big issues that I'm focused on is what the potential
additional benefit that we would achieve in terms of window
closure or yield, and at what cost would the transition from
mini-pool screening to individual donation NAT achieve?
And you can see here that the mini-pool screening,
because of the dynamics of the window period for each virus,
detects the vast majority of the overall window period for
HCV, and a less proportion of the window period for HIV and
HBV. But it's--what I'm trying to emphasize is I think the
scientific efforts to get empiric data to help make these
decisions is a first step in the decision process.
Next slide. And the other point that I wanted to
emphasize is, again, that I think we have to always ask
whether our prior predictions, be they in this arena for NAT
yield, or risk, or issues like in HCV look-back, where there
was all the controversy over how effective would it be, how
many infections would we actually identify? And I think
it's very valuable and important that CDC undertook the
effort to try to quantify what the real impact was, so that
we can then use the knowledge, and I think in HCV, as with
HIV--I'm sorry--in HCV look-back as with NAT, what we've
discovered is that our estimates were really very accurate.
The yield of HCV look-back was a very small fraction of the
total proportion of infected recipients, let alone infected
people. And it's important to go back to that, because I
think that question of whether those estimates were accurate
was a major driver in the decision to do something that some
of us argued would not be very effective.
Here again we say--if you just look at the yield
of nucleic acid testing over the first year of screening, we
picked up 42 HCV and 4 HIV window-phase infections. Some of
these actually would have been detected by other screening
measures like ALT, but these were all antibody-negative
yield cases. And if we then estimate--derive from this
yield the observed rate of detection, we can actually then
compare the yield that was observed with the predicted yield
from those model estimates I presented earlier. And again,
the big message here really is that our predictions were
really pretty close. We actually slightly over estimated
the yield, implying that we probably over estimated the
risk.
In addition, for HCV, we can look at what kind of
yield we picked up in terms of window-period versus error
versus these silent carriers, and again, it's really quite
comparable to our predictions.
So, to me, I think we have in place now a strong
surveillance program and strategy for making these
projections, and I think it's very important that we use
that to reassure the public that we know what we're talking
about and use these data to frame future questions.
Next slide. Just in terms specifically about
moving to individual donation testing, we can then use these
window-period closure estimates, the further closure that
could be achieved by taking this next step of testing every
unit individually, and given the incidence in the donor
pool, we can estimate the yield, and these are per 10
million donations. So these are the projected number of
additional infected donations that we will detect
essentially per year in the United States. So we're talking
really about two or three additional HCV and HIV infections,
and with HBV, the incremental yield of individual donation
NAT would be greater, but HBV is much less important in
terms of most patients who are exposed clear the infection,
resolve it completely.
And the next slide is coming to a conclusion, just
puts this into the cost effectiveness, and these are numbers
that were generated with Jim, and for important comparison
purposes--and I'll come back to this at the end--what I've
done here is to juxtapose the cost effectiveness, kind of
the cost, the number of infections detected, and then the
cost effectiveness in the formal dollars per quality
calculation, of the basic antibody assays with these, you
know, sort of next-generation high-tech window closure
detection methods. And what you can see is the basic
antibody test for HIV and HCV, that because they detect the
vast majority of infected donors, the prevalent infections,
and are relatively inexpensive, they're actually extremely
cost effective. In fact, for HCV, it's actually a negative
dollars per quality, which means that we save a lot more
health care cost by screening the donor units and preventing
the transmission than it costs to screen.
So these are very--you know, these are sort of
"gimmes." And one of the things I'll close with is the big
problem is that these tests are not being done in many parts
of the world. In strong contrast to that, we can see that
these window closure assays, because the yield of additional
infections that are being detected, are so low, and the
costs are relatively higher, you know, the cost per quality
is just--from a cost effectiveness, economic perspective,
you know, out of control. Whether these still justify, in
terms of safeguarding the US blood supply and public trust,
et cetera, is, I think, perhaps being defined.
Next slide, just final. This is just a
compilation again with Jim of the cost effectiveness of
various blood safety initiatives. These are the mini-pool
and individual donation NAT testing. You have some of the
viral inactivation methods over here, and you know, basic
antibody testing and other generally accepted medical
practices come in under 50,000 per quality. We're talking
about measures in blood safety in the millions of dollars
per quality, which there's nothing comparable, I think,
perhaps, except for airplane safety in terms of public
health initiatives.
And finally, next slide. Just a point that
although, you know, you can argue negatively that these
direct virus detection methods like NAT are perhaps
inappropriate or cost ineffective. I think they are
important for one reason, which is that the perpetual
discovery of new viruses that are coming now every year, one
of these is eventually going to prove to be clinically
important, I suspect, and having a direct virus detection
platform like NAT in place is going to be a very important
safeguard at some point in the future, and should cause us
to revisit the whole paradigm of screening in terms of, you
know, perhaps getting rid of some of the older antibody
tests, antigen tests, obviously P-24, but probably surface
antigen, and relying more on the high-sensitivity direct
virus assays. Certainly as we look at new agents, I think
our first decision or first choice will often be to add a
system to the NAT screening program.
Next slide. Just wanted to make one comment about
international safety and close. I mentioned this earlier.
These are slides from Luke Noel at WHO, but what we sort of
ignore is the fact that a large proportion of the world, and
nearly, you know, a third of the global blood supply, is
collected and transfused in developing countries, and in
these settings, almost half of the blood is untested.
Next slide, again from WHO. They estimate that in
the range of 10 million units that are transfused annually
that are not tested for HIV, 15 or so million for HBV, and
probably in the range of 25 million not tested for HCV. And
much of this is in regions of the world where the prevalence
of these viruses are extraordinary compared to here.
So, finally, the last slide is sort of a new slide
that we built as part of a group that I'm involved with,
which is called the International Consortium for Blood
Safety, which is trying to get resources toward the
international safety arena. And what this just illustrates
is the point that that sort of cost effectiveness slide also
emphasized, which is that the basic first generation--or
relatively standard antibody tests, are able to interdict,
you know, probably at least 95, and probably closer to 99
percent of all potential infectious donations, and that
these technologies are really relatively inexpensive on a
per donation basis and very cost effective, because they do
detect such a high proportion of the transmitting units, and
you know, what's sort of our of equilibrium on a global
level is the extraordinary expenditure that's being applied
to these sort of asymptotic residual risk infections in the
US and other developed countries. And although, clearly
there's a mandate to this committee and to us to try to do
what we can to maximize safety, I think we also should
consider our responsibility globally to make sure that basic
screening tests are brought into play throughout the world.
That's it.
CHAIRMAN CAPLAN: Questions? Comments
MS. LIPTON: Just one comment. Mike, when you
were talking about risk perception, you were really focusing
on, well, you know, we have a good story, and we do, and so
we should try to change the public's perception. And that's
really not my point, and I just want to make that clear.
From my dealings with the FAA, they accept what people's
perceptions are, and they're not trying to change them. You
don't hear the FAA talk about how safe airline travel is.
What they say is any accident is bad, and our goal is to get
rid of those.
And so I think that we think that if we just
educate the public enough they're going to get the point.
And I think what we have to do is say to ourselves, if we
educate ourselves enough, we will get their point. And so I
would turn it a little bit on its head.
I absolutely agree with you, too, about the
international issue. And you know at AABB we've been
working a lot in the international field. I think testing
is critically important and technologies. But the other
thing that we've found in working in countries is it's not
just the testing technology. You must work to build
infrastructures there and systems that can actually use the
tests appropriately, maintain them on an ongoing basis and
training. And I think there are a lot of initiatives going
on right now that are very good and I think are coordinated
more and more. And I know, again, our obligation is here,
and I don't bemoan what we spend in this country, but I do
think we could look outside of our borders more often.
DR. HAAS: Mike, were you using cost benefit or
cost effectiveness? Because at least as I was hearing you,
I was hearing cost, and I was hearing prevention of disease.
And that would put, in my mind, more the cost benefit rather
than cost effectiveness.
DR. BUSCH: Well, these are numbers that, in terms
of those specific numbers that Jim derived, and he's saying
cost effectiveness.
DR. HAAS: I'd like to see some background on--not
today.
DR. Au BUCHON: The process by which societies,
their governments and skilled technocrats make decisions
about safety have fascinated me for many years. I'd like to
make a few preliminary comments on the issue in general and
then proceed to discuss a specific illustrative example.
This committee has considered many issues of
pressing medical, social and political importance over the
first 3 years of its existence. Although a more rational
approach might have been to first lay out the framework by
which decisions related to blood safety should be made, I
can readily understand that action was first required on the
issues of the day before we could take a collective step
backwards, draw in a deep breath and develop a plan by which
future issues should be addressed. I'm glad that we're now
at that point.
In considering how this committee should address
the often conflicting issues of safety and availability, I
believe it would be prudent to recognize that health care
resources are limited. It was the federal government that
first created a prospective payment system for senior
citizens in our country, and it is that same largest of all
health care payers that has, on multiple occasions over the
last 10 years, reduced payments to hospitals for care that
is increasingly more complex, sophisticated, costly and
successful.
This committee was specifically charged to
consider the cost of its recommendations, along with other
important implications. While efforts through and beside
this committee to obtain additional resources for
hemotherapy are of course welcomed and encouraged, we cannot
kid ourselves into thinking that the pot of gold from which
these resources come is boundless.
I believe it is the task of this committee to
identify the risks, benefits, costs and alternatives in
critical blood safety decisions. We should highlight what
can be done with the resources available and highlight where
the commitment of additional resources will lead to further
improvements in hemotherapy. Where the people indicate a
desire to pursue efforts beyond a point that may make sense
from a health benefit point of view, implementation of these
strategies should be pursued if, one, the commitment of the
public is reflected in the commitment of additional
resources to the effort so that, two, no net detriment to
health will rise through the implementation.
This committee will, hopefully, continue to
provide insights into how we can improve transfusion in this
country. However, I believe the decision-makers should be
made well aware of the facts about yields and costs of
proposed programs and that such considerations are an
important part of our deliberations. When resources are
limited and choices have to be made, this committee may be
of invaluable assistance by saying to society or its elected
or appointed representatives that certain issues deserve
attention first and that more resources are necessary to
accomplish all that could be done to make transfusions safer
or to improve availability.
Now I would like to turn my attention to the issue
of universal leuko reduction as an example of the kinds of
situations that we will find ourselves in in the future, an
example of the dilemmas that our intentions and our
limitations lead us. I do not do this to speak for or
against universal leukocyte reduction, but merely to use it
as an illustrative case.
As a former medical officer at the national
headquarters of the American Red Cross and as a physician
dedicated to providing the best hemotherapy to the patients
I serve, I'm always on the lookout for new means to achieve
safer transfusion. For example, my institution uses only
apheresis platelets and donor retested plasma in an attempt
to reduce the inherent risks of allogeneic transfusion. And
to my knowledge, we are the only hospital in the country
that routinely cultures all platelet units to detect
bacterial contamination and one of the few to employ a
mechanical barrier system to prevent mistransfusion.
What is best for our patient should always be
paramount in the minds of health care providers, and our
institution has an admirable record of directing its
resources to that end. I would hope that those around this
table and in this audience can accept that my intentions are
those supposed by Hippocrates and not Adam Smith.
[Laughter.]
DR. Au BUCHON: Some would apply the precautionary
principle to require that something be done just in case
leukocytes are harmful to transfusion recipients. I do not
believe the data are yet available to conclude from an
academic point of view that the safety of transfusion will
be advanced by universal leukocyte reduction. In fact,
there's a reasonable possibility that diversion of
resources, including our attention, toward universal leuko
reduction will prevent transfusion services from addressing
issues that have already been documented as causing more
facilities than the presence of leukocytes in allogeneic
blood components; namely, mistransfusion and bacterial
contamination. That would be a mistake.
I have been told by more than one blood center
official in confidence that the push by many blood centers
toward universal leukocyte reduction is driven by the
economics of their finances and the inability of many
facilities to maintain operations with their current red
cell pricing structures. If this is the case, it should be
addressed directly and not through diversion of additional
funds, amounting to approximately half a billion dollars per
year, to an effort of unproven consequence.
We need, as a society, to pay for the safety
improvements already implemented. And the push toward
universal leukocyte reduction, at least by some blood
centers, may be derived really from a perverse approach to
the problem of economics and is a sad commentary on how we
have attempted to pay for past improvements in blood safety.
Is it wise to force hospitals to spend precious
limited resources on an unproven technology? When the
potential for harm is great, the trigger for action should
be easier to squeeze. However, in my medical opinion, there
does not appear to be the horrendous threat to transfusion
recipient safety, about which some are raising the alarm
with respect to allogeneic leukocytes. If clinical harm
were indeed evident, then it should be prevented.
If the public wishes to spend additional resources
on an unproven technology, I will be happy to implement it,
providing it does no harm. But to ask me to direct limited
resources to an arena where I can predict no benefit and in
doing so deplete the resources needed to address more
well-documented and larger problems, is to ask me not to
deliver the best health care I can to my patients. As a
physician, I have difficulty accepting that directive. A
federal directive to implement universal leuko reduction
takes the decision how to treat patients out of my hands.
We have an opportunity today to gather the
technical resources of those trained in the field with the
representatives of society charged with overseeing them and
the associations of those who are the beneficiaries of the
efforts of the first two groups to define how this society
should make decisions about blood safety in the future.
Although a substantial part of my academic career
has been based on cost-effectiveness analyses, I would be
the first to acknowledge that there is more to decision
making than determining the
cost-per-quality-adjusted-life-year saved. At the same
time, we as a society cannot continue to demand a risk-free
blood supply at Wal-Mart prices. This committee is the
perfect forum for considering options and opportunities, and
I hope that we will explore them honestly and openly.
Much has been accomplished over the last 15 years
to make transfusions safer. We have many significant and
important opportunities open to us to improve further the
safety of a transfusion. To the extent that existing or new
resources are available to advance toward the mutual goal of
safer transfusion, let's direct them towards the biggest
problems. Although universal leuko reduction has garnered
the limelight recently, this is not the biggest problem we
face.
The patients that I treat every day expect me to
do my best to make their transfusion an event they will not
regret. To meet their expectation, I need to direct the
resources available to me toward the largest risks that they
face. Although emotion and rhetoric can be powerful,
helping patients should be our goal. And directing
resources, time and money toward the largest problems is the
way to improve the health of patients.
We do need more resources to make transfusion as
safe as it could be, and we need to direct these to the
problems that cause the greatest morbidity and mortality.
CHAIRMAN CAPLAN: Comments? Keith?
DR. HOOTS: In light of what you said and what
Karen said earlier, it seems to me that one of the things we
don't have that might be useful here is cost utility because
one of the things that's critical for this whole decision
process is what are people willing to pay if they're given
all the facts. And that's what cost utility is supposed to
measure.
And I wonder, maybe you can--I mean, you would
know, I'm sure, if anybody is doing cost utility related to
transfusion events and are they doing general population or
are they starting--if they are doing it, are they starting
with more people--people who are more sophisticated in terms
of risk analysis? But it seems to me that that's part of
the equation that we've always been missing.
But as Karen was saying in terms of aviation
safety, the perceptions are incredibly important. And if
people, I mean, it's obviously a major undertaking just to
educate this committee to all of the subtleties of the data
that might just show, much less the whole population, but it
seems like, at some point in time, we have to at least try
to get some data on that side of the equation that's
collected in a scientifically appropriate fashion and also
probably could be put side-by-side with cost benefit and
cost efficacy.
DR. Au BUCHON: A willingness to pay I think is a
problem in health care analyses often because the patient
usually does not see the bill for what their health care
really is. And when their health is on the line or when
they're about to step into an airplane, they want an
absolute assurance of safety. But yet when taken out of
that immediate threat context and put in the framework of do
you want to pay more income tax in order to make sure that
there's enough resources in the system to provide the very
safest blood, well, then people say, well, what am I really
getting for this? And that question is answered differently
when they're about to be transfused versus some unknown
person at the other end of the country is about to be
transfused.
DR. HOOTS: Yes, and I understand that. And maybe
the targeted population initially then should be people who
are getting ready to undergo the transfusion and also have
an experiential base for other risks that they're
undertaking related to their medical care as well. So at
least they'd have some basis of comparison to what risks on
other medical-related issues compared to transfusion-related
issues and relative costs of each.
DR. HAAS: If I may just jump in very quickly, I
agree with the concept that the ability to measure that is
almost nonexistent because you are playing with two
different fields of information. You're asking someone to
say what would I do if, and there have been some economic
studies on uncertainty, and it comes out saying people have
to make decisions. And I think that's why these discussions
around here become very important. Where we have slight
differences, one of our jobs I think is to say, as a
committee, what do we recommend here because we're not going
to get the data.
DR. EPSTEIN: Jim, although you discussed
universal leuko reduction, for example, you kind of took a
position on the issue. And I would just ask you, just at a
technical level, do you feel that there has been an adequate
assessment of cost effectiveness or cost benefit for
universal leuko reduction? And if so, where do you put it
on a scale on the kind of graph that Mike displayed?
Because I think that a lot of the underlying debate, even if
we can put aside the reimbursement problem, which is, of
course, real, it really focuses on the difference between
immediate benefit to patients without known indications
versus long-term benefit to the population as a whole. And
one of my concerns is that that issue has not been framed
adequately scientifically, and it tends to confound the
debate and is the underlying reason why opinion is so
polarized on this.
So could you just comment on whether there has
been an adequate cost-effectiveness or cost-benefit
assessment and what did it yield.
MR. ALLEN: I don't feel we have seen that yet in
the literature. There have been several economic analyses
of the effects of providing leukocyte reduction, in terms of
potentially reducing length of stay or antibiotic usage or
other such benefits. The studies to which those analyses
have been attached have had some difficulties in terms of
their structure and in their interpretation, although some
adherence of the concept of universal leukocyte reduction I
firmly believe that it will be cost saving. It's very
difficult to document that because most of the studies have
been retrospective. And with the rapid change in health
care and the pressures to reduce length of stay, it's hard
to construct a longitudinal study to answer the question.
Like many others, I'm anxiously awaiting the
results of the study that has recently been concluded at
Massachusetts General Hospital, where for the first time in
a U.S. hospital situation patients were prospectively
randomized between receiving leukocyte-reduced blood, when
they had no specific indications, versus receiving
nonleukocyte-reduced blood. And specifically the end points
that are being sought in that study are economic or have
economic implications, such as length of stay.
So I'm hoping that that study will provide some
very useful information to try to guide decisions as to
exactly what economic benefit we will see. In order to
really conduct a cost-effectiveness analysis, one would need
to determine what the effectiveness is, and I have great
difficulties projecting what the yield of universal
leukocyte reduction will be.
MS. LIPTON: I think, and then to add, you know,
if you look at this from a different viewpoint, and some
people are struggling with another concept, and that is that
if you accept that there are certain subsets of patients who
would benefit from this, we are unfortunately in a situation
in our society where I don't believe we can effectively
identify accurately, in every situation, those patients who
do need them.
And the way the DRGs are structured, and the way
you go into a hospital, and the limited medical history you
get, and you go in on a specific service, and it's their job
to get you off of that service as fast as possible, really
doesn't have us treating patients, you know, from birth to
death. We treat events. And I think that there is some
anxiety on the part of people who think if we accept this
principle of selective, you know, doing selective leukocyte
reduction, that really, in the long run, as Jay said, when
you look at a lifetime of these issues, that you don't have
a way of measuring that, but you've totally discounted that
as a potential good effect.
DR. NIGHTINGALE: Jim, I'd like to follow up on
Karen's question with the following one. One of the things
that skilled technocrats do, and I think there's several
people around the table who would self-identify themselves
as skilled technocrats do when confronted with a problem and
having the resources, is to throw money at the problem. And
I said that not just--I wanted to get a laugh out of it to
diffuse the situation, but to ask you a very, very serious
question. One of the frustrations of us
technocrats--skilled or otherwise--when dealing with issues
of blood, is whether or not money that we would throw at a
problem would actually reach it.
Do you have any suggestions as to how any such
money that we might throw at this problem, which is the
problem of limited resources for blood establishments,
broadly, limited reimbursement at the level of the blood
establishment, how would you suggest rectifying that
problem? If you, in fact, feel it is one.
DR. Au BUCHON: I could think of a number of
approaches, not all of them, probably none of which,
actually, are politically viable, at least in this country.
But, for example--
DR. NIGHTINGALE: Yeah, but don't stop trying.
DR. Au BUCHON: Okay. No. For example, the blood
collection and delivery service could be directly funded by
the government. And, in fact, in most countries, most
developed countries, that is indeed what is done. So if the
society then decides that they want a new test, society
would couple that decision with putting more money into the
blood supply system. It would directly go to those who are
expending the money to buy the test kits and do the test.
That would be one way to do it.
Another way that has been discussed recently, as
we've been considering how best to try to get society to
reflect their interest in increased safety with a
willingness to pay for it, would be to have a separate
payment category for blood transfusion. Right now blood
transfusion is rolled into the diagnosis-related groups
through which federal government pays for Medicare patients
and that there is, indeed, no guarantee that increasing
reimbursement through the Medicare system as a whole will be
identified by hospitals as due to increased costs of blood.
If one were charging out blood directly, then it
would be easier to have that recognized. It would at least
be hoped that with the amount of attention, amount of press
at least within the health care community that the cost of
blood is currently getting, that any increased reimbursement
through Medicare to hospitals would be able to be used by
hospital transfusion service directors when internally
negotiating with their administrators to say this additional
money should be allocated to our budget in order to bring
about additional blood safety initiatives in the hospital.
DR. NIGHTINGALE: If I could have a follow-up
question then, one of the problems that we have in this
country in dealing simplistically with this issue is that we
don't have a national blood supply, we don't have a
nationalized payer. I think it's 22 percent of health care
expenditures, last I checked, and it's in the packet that I
prepared for you, are Medicare. So simply if the government
were to set a price for blood, it would only account for 22
percent of the benefits.
I guess the question I would ask you to consider
is, is there a way that the government could set a price for
blood without nationalizing the blood business? We've
tried, as you know, with the outpatient--APCs.
MR. ALLEN: I do not have a direct answer to your
question. Maybe I could defer to the economist among us,
but I would say that the federal government, while obviously
not having control over all health care expenditures or
paying for all health care, has incredible power to cause
things to happen in the marketplace and particularly in
health care reimbursement. So if the federal government
were to, for example, indicate that a particular avenue was
appropriate and the federal government were willing to put
more resources into that to pay for that, I believe that
private insurers would probably follow suit.
DR. HAAS: Steve, can I just add on to that? I
agree with what Jim has said, but I don't think there's any
reason to assume the federal government would put more
money. They could put less, as Jim already indicated in his
comments, so that's a pretty dangerous route.
DR. NIGHTINGALE: The federal government has
stated on April 7th that the price it is willing to pay for
a unit of leuko-reduced blood, when used in an outpatient
hospital setting, is $137.21. That's a provocative
statement by the government. It's a policy that's in place
right now. It's clearly not an adequate policy by itself.
It can be viewed, at least as I view it, as a noble
experiment, but it is one of the potential responses to the
very legitimate issues that you just raised.
MR. ALLEN: My understanding is we will get back
to the "Dear Citizen" letter, so I basically just have a
comment. As was mentioned earlier about the blood industry
and the issue of safety insofar as the perception goes,
basically what I wanted to say was that we do have a long
way to go in terms of educating consumers about how low the
risk is now through transfusion, as far as contamination.
However, when consumers, such as some of us here at the
table, go out into our communities, we don't talk about the
numbers like we do here. We talk about the individuals and
the effect that these issues have on their families and
these people.
So I'd just, because of some of the things I hear
and we're talking about numbers, I just think it's important
that we remember that we are talking about people here and
that these issues have a great impact on not only their
lives, but their families' lives. And we also heard earlier
about the possibility of blood banks going under. I mean, I
just want to emphasize that we have families that go under
as well. So we just need to, I think, keep all of that in
mind when we have these discussions.
CHAIRMAN CAPLAN: What I hear or take away from
the committee comments might be lumped into four categories.
There's an interest in talking about correspondence that
people might send around. That's come up. There's interest
in talking about do we want to have a template of principles
or considerations that we might agree are relevant, even if
we don't agree on their application or how to interpret any
one of them, but just factors to consider when you're
looking at an issue and sort of leave saying, yep, that's a
rough outline of what we'd like to have.
We were reminded by a number of people that there
are groups who rely heavily on blood who are still concerned
about supply and equitable access, whether it was IVIG or
alpha or hemophilia and asking whether we could put that on
the agenda or keep maintenance going on those areas to make
sure that they get done. And then there's a discussion out
there about what we want to say and do on issues of safety,
continued safety, both here--I'll call it the marginal
safety issue--and then overseas, the sort of impact on
global safety. I may have not lumped them up completely.
But, anyway, those are areas that I heard kind of presented
to us.
I was going to suggest to sort of not illuminate
it, but I think it is our responsibility to keep an eye on
the supply and access issues for the populations heavily
dependent. So on that one I suspect we can just direct
ourselves to return to these issues. We may want to go to
IVIG and hear about that in terms of a sentinel study or
something, but to devote parts of future meetings to that
topic. So I don't think there shouldn't be too much
controversy about that I would imagine.
And then I'm open to going in whichever direction
you all want to go to. Are people--well, let me throw open
the floor and see. Do people want--what they would like to
pursue, if I've got those categories straight, is there an
interest in discussing the letter a bit further and trying
to do that or--
DR. EPSTEIN: Art, were you going to entertain
5-minute presentations from--
CHAIRMAN CAPLAN: What I was going to do is let
them comment a bit on this and then go to the 5 minutes.
But it sounds to me like you think your 5 minutes is
germane.
DR. EPSTEIN: I think before we, you know, cone
down on one of the four topics, it might be helpful. At
least it speaks to one of them. It speaks to one of them.
CHAIRMAN CAPLAN: All right. We'll do your 5
minutes because it may be relevant to one of them. All
right.
DR. EPSTEIN: Okay. Well, as a hopefully skilled
technocrat, I took literally Steve's charge for this
meeting, that we hope to identify a set of intellectually
consistent, politically acceptable principles on which a
coherent and durable policy to assure the safety,
availability and affordability of blood and blood products
can be established.
And so what I sought to do in my 5 minutes was to
look at the different paradigms of decision making that have
been in use as a framework for trying to think about
underlying principles. And my first observation is that I
think that there are two large categories that tend to get
confounded. You need to go backwards to the first slide.
One is how we deal with known threats, and the other is how
we deal with potential threats.
So with respect to known safety threats, some of
the paradigms that have been in operation, and we could talk
about which cases were addressed when and how, have dealt,
particularly in the post-AIDS era, with a zero risk concept,
which is intervene at any cost. And what I've tried to do
is rank these in some order of what one might call
stringency, and I would say that that's the least stringent
because it requires the least reflection about benefits and
costs or tradeoffs.
Another principle, which I think is a little bit
less more stringent than zero risk is looking at whether
there's a favorable benefit-to-risk ratio. However, that's
also a limited framework because it implicitly is at any
cost. As long as sort of the gains outweigh the losses,
then it's good and you should do it. But that's independent
of whether it costs too much.
I think that a larger framework which begins to
encompass cost has been the concept of cost-effectiveness is
favorable and that that should drive decision. But there's
been criticism that at least the FDA shouldn't look at the
world that way. We should be cost neutral from our purview.
Another principle that has been utilized in other
domains, not particularly in the blood area, but has been a
successful paradigm, is the notion of trying to have risk as
low as reasonably achievable, so-called ALARA, and that's
yet another way that one can look at the problem integrating
all of the different ways of framing benefits.
And then lastly I think the most global point of
view is something that's been called public health or
wellness, which is to try to look at a possible intervention
and rate its value for wellness amongst all alternatives,
including things that aren't of a like kind; in other words,
tradeoffs for, say, different parts of public health--money
into the vaccine versus marginal blood safety, for example.
Now, as hard as these decisions obviously are
dealing with known risks, they usually are made in the face
of unknown risk and/or unknown benefits of particular
interventions. And yet once again I think it's possible to
recognize that there have been a range of strategies with,
again, varying stringency as to how much scrutiny is applied
as the basis for decision.
And so in the approach for potential safety risks,
I think that the most stringent framework, and here I'm
going in reverse, has been to withhold all action pending
scientific certainty. And I think that that's the paradigm
that we had in place approximately 20 years ago, where we
waited until the data were conclusive and essentially all
fair-minded scientists were convinced. Short of that was
the idea that we would withhold action pending a scientific
consensus, which basically meant that the decision-makers,
whoever they happened to be, tried to figure out if there
was sort of a dominant scientific view and then they would
go with it.
Less stringent than that is what has been called
the precautionary principle. And here I'm pretty much
describing it as it has been explained in the recent
document from the European Commission, which is to adopt
precautionary measures based on a consideration of options
considering the underlying factors of proportionality,
nondiscrimination, consistency, and cost benefit.
And I think we need to recognize that we have also
had operating in some situations a precautionary principle,
but one which has been even less stringent than as
articulated by the European Commission, which is to adopt
precautionary measures, but in the absence of a full
consideration of impacts and alternatives, and that part of
the problem has been that some of our decisions have been on
that paradigm.
Now, I guess I had hoped that at this meeting or
perhaps as possible topics for future meetings, we might try
to examine some of the upcoming decisions in blood safety
and availability or, alternatively, I suppose we could adopt
the retrospective model, look at cases where we've already
made decisions and ask what paradigm were we on and did it
have value or didn't it have value.
But I thought it useful to at least note some of
the pending decisions. And these fall also into two bins:
potential added safeguards and potential relaxations of
existing standards. In the area of potential added
safeguards, we have, you know in the near horizon, things
such as implementation of the minipool nucleic acid test for
Hepatitis C, for HIV, which of course are well along in
development. But then a big question mark about Hepatitis
B, which is a subject of current debate.
Shortly over the horizon, lies the question of
whether there should further be single-unit nucleic acid
testing. And there's a question of whether that should
supersede minipool NAT or whether it should be seen as
following minipool NAT as an additional incremental safety
benefit. And Mike showed us what that would mean in terms
of yield and cost and likewise for the same agents.
Again, echoing Ron Gilcher, we look forward to an
era where there will be infectious agent and activation
technologies for cellular components. And the current
paradigm would layer them onto existing testing, and I think
that the challenge is whether we can deal with the overlap
and perhaps not do both always.
Likewise, we have the challenge of dealing with
emerging infectious agents, and Chagas disease has been
mentioned, as well as perhaps the inconsistency of how we've
been approaching Chagas versus other things. But the list
doesn't stop. It goes on: Human herpes virus type 8, human
parvovirus B19, Hepatitis A virus, and other things which
are less-well characterized.
Then, of course, we have the question of whether
to expand the exclusions for TSE risk not only related to
Mad Cow disease, but potentially related to other TSEs of
mammals that may also be consumed in the food supply, and as
has been already mentioned, the questions of routine use of
leuko reduction.
So under the heading of "Relaxation of Standards,"
again, I would echo Jane Piliavin's comment that we
shouldn't only be looking at the new technology and the new
safety threat. We should concurrently and perhaps always be
reexamining existing policies and standards and reassessing
their benefit in a current scientific light. And I would
put on this list such considerations as the voluntary
discontinuation of ALT, the movement toward relaxing the
restriction on donation by persons with hereditary
hemochromatosis as to the current requirements for unit
labeling and restricted donor frequency, and we've already
discussed the fact that FDA has moved toward permitting
exceptions, whether to discontinue HIV 1P24 antigen testing
in the era when we can approve minipool NAT and perhaps
single-unit NAT, relaxing potentially the current exclusion
criteria based on behavioral risk of males who have sex with
males, whether there is continued utility of the serologic
test for syphilis and what is the continued utility of the
test for antibodies to the core antigen of Hepatitis B.
These are just ones that are already under consideration,
not even getting into the question of things that may come
down the road.
So what are the key issues then in terms of the
decision-making process? My concept is that the issue of
the day is to talk about a process by which blood safety
availability and affordable cost decisions can be made in
the post-AIDS era based on some appropriate set of
underlying principles that should govern that process.
My own notion of those principles is shown on the
next two slides, and fundamentally they conform with the
recent statement of the European Commission regarding
application of the precautionary principle. However, again,
I would point out that the underlying principles really are
not limited to the situation of threats of unknown risk or
unquantified risk; that the same principles really should
underlie consideration in the case of known or quantifiable
risk, as well as reconsideration of existing measures.
So what are these principles? Well, I would put
way to the top of the list that I think acceptance of risk
is a political decision. And what I mean by political is
that ultimately it's a matter of social choice, however the
society goes about that. Related to that, I would agree
with what was stated, at least by Dr. Gilcher, that zero
risk should remain a goal, but it cannot be viewed as a
mandate. Regarding it as a mandate in any given situation
may lead to irrational actions, even if we continue to
embrace as a reality public fear and the public goal of zero
risk.
Secondly, I would say that just as acceptance of
risk requires political decision, acceptance of cost is a
political decision. Why? Because in the end there are
finite resources, and it is up to the society where to place
its resources and for whatever gains or perceived gains.
Thirdly, in a democracy, I think it's inevitable
that decision making must be transparent. But I think that
this is also a necessary feature to obtain a public
endorsement, both of the process and its outcome.
Fourth, I concur with the EC statement that any
effective decision-making process in this domain must be
structured and must include the elements of risk assessment
or risk management; that is to say, well-defined strategies
of known or presumed effectiveness, and additionally, risk
communication, however effective or ineffective that might
be.
Further, I believe that there has to be ongoing
scientific input; that this has to be both prospective and
retrospective in any situation.
And I would add two dimensions that are part of
the WHO thinking, but are not really echoed in the EC
document, which are that in this global village, blood
safety decisions need to be considered in an international
framework because they have international repercussions
bi-directionally from the U.S. point of view, and that
additionally it's my personal view that wherever possible,
the decision makers nationally should attempt to
independently articulate the scientific basis, economic
basis and political basis of blood safety decisions because
I think that that effort contributes to clarity and creates
increased freedom for national governments and societies to
make different decisions in the face of the same available
scientific data.
And so that's my stab at trying to frame what I
think are the underlying problems in examining decisional
paradigms, as well as my view of the basic principles we
might wish to discuss.
CHAIRMAN CAPLAN: All right. We can move toward
principles and a matrix discussion or we can move toward
some discussion of the correspondence issue. Anybody want
to take us in a direction? I'm going to see which way the
comments go here.
DR. KUHN: I'd like to give a stab at the
correspondence issue, and hopefully it can be addressed
pretty quickly.
After listening to much of the discussion this
morning, it seems like we do have a correspondence that was
sent out to congressional members. They did get it in some
kind of a packet, but they are not under any obligation to
mail it to their constituents. And it seems like there
probably is--funding is probably not available in the 2000
budget for trying to address the correspondent issues.
But I believe that in the spirit of what the
committee accepted and their recommendations of August 13,
1997 and November, 24, 1998, regarding Hepatitis C look-back
notification and education, and also seeing that the surgeon
general deemed the "Dear Citizen" letter most important, I
would offer a recommendation, hopefully, that could be
considered, and that would be that the committee actively
supports the surgeon general's effort to inform the American
public of the risks that Hepatitis C or HCV infection and
transmission. Moreover, the committee strongly urges the
secretary to seek adequate supplemental fiscal year 2001
funding in order to provide the CDC with the most
efficacious and widespread means of informing the American
public, including the consideration of sending a "Dear
Citizen" letter to the American public by March 31st, 2001.
CHAIRMAN CAPLAN: That sounds like a proposal.
Why don't we have a little discussion.
MR. ALLEN: Would it make sense to consider
possibly asking HHS to allocate the funds directly to the
surgeon general? No? Why not?
DR. NIGHTINGALE: I think--we don't--not that much
money passes through us, and there's a reason for it. I
don't think that where the money goes is essential the
perf--is material to the performance.
MR. ALLEN: I guess what I'm concerned about is
obviously I'm one of the people that would like for this
letter to go out. But my concern, one, is if it comes from
a congressperson who may feel obligated to send it out, but
really doesn't want to send it out, and then once the
constituents get this letter, they're more than likely going
to call that congressperson's office, and they may or may
not have the right information to give them. So I'm a
little concerned about doing it that way. So that's one of
the issues.
If this is a letter that the surgeon general feels
strongly about, I want to back him in getting this letter
out.
DR. NIGHTINGALE: I apologize. Larry, while you
were talking, I was looking over my shoulder to see if the
window that was blocking the text of the statement had been
obliterated. So I misunderstood what you said.
Could you repeat it once more for me. I know it's
already in the record.
MR. ALLEN: I just wanted to know whether or not
we could ask that funds be given directly to the surgeon
general for the purpose of sending out this letter directly.
DR. NIGHTINGALE: Oh, here, I just have to speak
personally, and this is from the heart. The administrative
staff of the surgeon general is limited. You are looking at
them.
[Laughter.]
DR. NIGHTINGALE: I think, given that reality,
there are more administrative staffs in agencies where such
programs are usually based, and there is a reason for that.
MR. ALLEN: Okay. Well, then besides letting
Congress do this is what I guess I'm getting to here. I'm
just concerned about the letter coming from congresspeople
that might, you know--
DR. NIGHTINGALE: If the money were to be
allocated, I cannot anticipate problems spending allocated
money. But if I could anticipate a problem, it is one
person, contrary to public belief, in the government can
only spend so much money so fast. It takes more of us to
spend all of that tax money.
CHAIRMAN CAPLAN: Paul?
DR. HAAS: I guess, listening to some of the
discussion before lunch, I guess before the discussion I
wanted a letter out. Listening to the discussion, I'm not
so convinced. I just don't know. And the way the proposal
is written, it doesn't say, "Send a letter." It says,
"Let's get appropriate information out there." And in the
spirit of some of the discussion we have just had, I guess
I'm a little reluctant this afternoon to say the letter is
the absolute most important thing to do or the best way to
do it. So I guess I'd prefer to keep it the way it is, and
then figure out a way to put the nice gentle pressure and
say, "This is extremely important", and the intent of that
letter's got to go out there whether it's the letter or not.
So, again, I guess my fundamental point is I'm not right now
in favor of tying anybody's hands.
DR. NIGHTINGALE: Again, I'm just speaking as a
grammarian here, not as an official of the government, if
that is possible--and it probably isn't. But I don't see
anything up on the board that would tie our hands.
DR. HAAS: I agree. That's what I'm saying, I
like it the way it is.
MR. WALSH: Mr. Chairman, on the contrary, I think
our hands are shackled right now without this.
And Larry, maybe that you weren't here earlier
this morning for the discussion of--the entire discussion,
but the letter is a letter signed by the Surgeon General on
his original letterhead. It's not as if it's coming from a
member of Congress, advising a constituent of a potential
health problem, so I think that hopefully they would look at
the Surgeon General's letterhead and not the Congress
person.
DR. GUERRA: I certainly agree with trying to get
information to the greatest number of people in the shortest
period of time, but I think incumbent in the process is also
the need to have some capacity in place to serve the general
public that is going to have the questions, and that is
going to need to be counseled and screened and tested, and
then connected to resources, and I think that that is a
moral and ethical obligation that we will have, but I think
it's part of this process.
DR. NIGHTINGALE: Again, I have to phrase this
more carefully than perhaps I phrased some of the things
that I've put into this microphone. But there are several
proposals currently before Congress, of which I am aware,
that address the point that Dr. Guerra just raised.
What are they?
DR. GUERRA: Yeah. What are they and how far
along are they in the process, because there are very few
resources out in the field right now.
DR. NIGHTINGALE: I understand. Rather than just
telling everybody to go to Thomas dot LOC and search for
hepatitis, that's one way to do them. To answer your
question--and I haven't done that search recently--I am
aware of one proposal that is an authorization rather than a
dollar amount to spend money for this purpose--a children's
health insurance plan. Just kind of a for instance, because
that's the one that I looked at yesterday.
CHAIRMAN CAPLAN: Mary?
DR. CHAMBERLAND: Just for the record, I haven't
really had any involvement with the development of the
letter at CDC. That's something that Hal Margolis and
others, as you heard this morning, have been working on.
I guess before we vote on anything, my sense from
the comments that I heard this morning, Steve, is that the
letter is not a dead end, and that as I understand it, there
has been a very public announcement by the Surgeon General
and Congressman Bliley to put out a letter using Congress
and its mailing capacity to do so, to its individual
constituents. And, yes, there is no obligation for each
individual congressman or woman to do that. And it has hit
a snag, a couple of snags, in terms of the separation of
powers issue and this 90-day moratorium on mail-outs prior
to an election.
But what I heard this morning is that what is
under way is--actively under way, is to try and work through
those two implementation issues, and--
DR. NIGHTINGALE: And furthermore, if you--excuse
me--to search for alternative means of distribution that
would not subvert the purpose of the message, changing the
letterhead, say, to a commercial entity signed by the
Surgeon General is not on the table, but--
DR. CHAMBERLAND: Exactly.
DR. NIGHTINGALE: We could discuss it, I suppose.
DR. CHAMBERLAND: Right. So--and I understand
there's this whole other discussion that's been going on
this morning about the merits of a letter and how effective,
whatever it could be. And I guess I'm just wondering if
such a vote is a little premature. Perhaps we should--you
know, if the committee wants to make some statement,
recommendation about their support for such a letter and
that it be sent out, and that Congress, individual members
of Congress, support this effort, because I guess I'm
believing that this is eventually going to be worked out,
that there will be a way for Congress and the Surgeon
General to work together to get a letter out, but it can't
be until after this 90-day period that has to be paid
attention to. And that, really, we should maybe be
supporting efforts to see that that does happen once these
sort of implementation issues can be worked through.
DR. NIGHTINGALE: This is Steve Nightingale. I
agree with Dr. Chamberland. Let me restate that while I
can't predict the future, I can state my own intentions, and
I can state the result of a conversation that we had on
Monday with the Surgeon General, was to proceed on the basis
of our private discussions with congressional staffers that
the issues were prudentially resolvable, and also to proceed
with other avenues for distribution of the letter. Also in
the context of support for the overall CDC program report.
I think it is important here for me to state that it is not
our view--this is not the only component of the CDC's or the
government's effort in this area. It is not our intent to
get into a discussion of the relative importance of this or
other initiatives, because, frankly, we don't care. We want
to get the job done, and successful as possible, with
the--and make--I should find a better word--the minimum
amount of mess in the process of doing so. This is a messy
issue. At the same time, since we are certainly open to
alternatives to distribution, we are certainly open to
getting the maximum amount of effect for the money that we
spend. I think it's fair for me to say that I don't--I
didn't see anything up here, as I said earlier, tied our
hands on this issue, but that's because I'm not looking at
it right now. I'm looking at the committee, and if somebody
else sees something that would tie our hands, please let me
know.
CHAIRMAN CAPLAN: I see something that will tie
our hands. If anybody thinks--this is my opinion--that
they're going to sneak a $30 million expense under the
franking privilege around the lawyers that are now paying
attention to this, forget it. So my hunch is that unfunded,
this may not fly. You betting me? All right. I'll bet the
lawyers. I'll take it.
So if you want to be risk averse and follow the
zero risk principle, you might want to look at something
like that as a ace in the hole of the letter, finding a way
to send out a efficacious letter. I'm assuming here, that
as we deliberate about this, it might turn out that putting
it all on TV was better than sending a letter, but that's my
outlook on things. But whatever, some way to get that
message out. That may be our safest course, it would seem
to me.
DR. EPSTEIN: I just wanted to propose some
rewording that might be a little bit better received by the
Secretary. What I'm suggesting is that the part directing
the Secretary what to do simply be omitted, and that the
date certain be omitted because that would increase
flexibility. So if we do that, it would read, "The
Committee actively supports the Surgeon General's efforts to
inform the American public of the risk of the HCV infection
and transmission", and then jump down, "including the
consideration of sending a "Dear Citizen" letter to the
American public as soon as feasible."
I think the virtue of that is that you have the
committee endorsement, but you really have not dictated how
the message should be conveyed. You've suggested the sense
of urgency, but you've not, you know, put a burden on the
Secretary should it not be feasible by that particular date.
So it's a little bit softer, but I think it gets our message
across. Let me just reread what I'm saying.
Basically what it would be, deletion from the word
"moreover" through "American public." So it would say--yes.
And then I would strike "by March 31st", and substitute "as
soon as feasible." So this version would read: "The
Committee actively supports the Surgeon General's efforts to
inform the American public of the risk of HCV infection and
transmission, including the consideration of sending a "Dear
Citizen" letter to the American public as soon as feasible."
And that way we're not telling the Congress what
to do, we're not telling the Surgeon General what to do.
We're just saying that something should be done and as soon
as feasible.
DR. KUHN: I thought about the wording very
carefully, and I think it--and that the logic or rationale I
had behind that was, number one, I think it fulfills
Congress's concern that they have had on this issue for
quite some time. I think it addresses that, number one.
Number two, I think it also addresses the urgency of many of
the decisions that this committee has made, and also the
encouraging ways that the CDC is trying to get this
information out to persons. I think, number three, it
supports the Surgeon General's--I believe his wishes of
trying to get the communication out as quickly as possible
in a widespread manner. And also when you're dealing with
Congress, I think we all know, you have to have money. And
I think it has to be mentioned in there in order for this to
fly.
And I also thought about putting--when I put this
together, that we needed to make it flexible enough--a
directive, but a soft directive, and flexible enough so that
there is latitude to be able to accomplish the objective.
It doesn't mean that it has to be a "Dear Citizens" letter.
That is the direction that the Surgeon General proceeded in
in having that letter written, but it doesn't necessarily
mean that is going to be the most effective way to
accomplish it. I think the goal is accomplishing it. How
do we make the American public aware in a widespread way?
And it's going to take funding, and I don't think--I think
if we ignore mentioning the funding, I think this will get
lost someplace in the budget process that's going to be
taking place in the next month here.
MR. ALLEN: I agree with what Dana's saying. My
concern is leaving it open-ended like that. I think maybe
if we could ask that they get--give us some response back by
the next meeting in terms of where we're at with--where
they're at with this letter, the timeline that they feel
maybe that will give them enough time to have some answers,
because right now, you know, if we leave it the way it is
now, look at how long it's taken us to get to this point.
By the time they finally get a letter out, it might not mean
anything.
So I'm concerned about that. I would like some
kind of language in there that at least ask someone within
HHS to get back to this committee by the next meeting with a
timeline on what's happening with this issue here.
CHAIRMAN CAPLAN: Being somewhat befuddled about
my parliamentary procedure here, we have two proposed
statements with nothing moved yet, so somebody could move
one or the other, or we can have a little more discussion.
DR. KUHN: Mr. Chairman, I move that the first
proposal or recommendation that I had submitted by put on
the floor.
CHAIRMAN CAPLAN: Did you save that one, Mac, in
your--
DR. AuBUCHON: Second.
CHAIRMAN CAPLAN: Discussion? About amendments to
this short of Jay's amendment. We could do it that way.
Any other language clarification as soon as it reappears?
DR. BUSCH: Just that the "infection and
transmission", I'm trying to understand what those--how
they're different. One thought would be to, instead, have
"infection and disease" instead of "transmission."
DR. EPSTEIN: I thought the point of that was to
deal with the issue of secondary transmission. In other
words, your own infection and the risk to transmit to
others. Your modification would be correct, of course, but
would lose that conflict. That's how I read it.
CHAIRMAN CAPLAN: Jim?
DR. AuBUCHON: From time to time the Executive
Secretary of this Committee offers us valuable insights into
the workings of the government. Might he offer some
comments as to which two versions that have been discussed
would provide the intended outcome? And I do not perceive
that there is really any difference around the table in
terms of what we would like to see happen, but how best to
make it happen is not entirely clear.
DR. NIGHTINGALE: I can obviously live with either
one, because it's my job to live with each other one,
appearances sometimes to the contrary. I--this is a gut
feeling, something that comes from the committee,
particularly if it is very strongly endorsed by the
committee, will have more force, because believe me, if they
want to get a hold of me or Jay, they have our phone numbers
and they can get our advice--well, not for free, but at a
relatively discounted price to the market.
So for that reason, I suspect that the more potent
one would be the one from the committee. Did I walk the
fine line?
DR. AuBUCHON: Very ably.
MR. ALLEN: Steve, what would be your opinion then
on how long of a time before we got a response from the
Secretary?
DR. HAAS: Maybe we could ask Steve to turn off
his mike.
[Laughter.]
DR. NIGHTINGALE: I think that the--obviously, the
Secretary's FY 2001 budget is in. At the moment, what
happens with the budget that begins on October 1st is in the
hands of Congress. Yes, there is a lot of discussion going
back and forth, but really, most of that discussion at this
point is White House, sort of is White House coordinated.
I'm not speaking in absolute terms. I'm trying to give the
committee information based on what I know general processes
are, but specifically, what I know the processes for the
health budget are.
The Secretary has not had problems in the past
saying, "Thanks, but no thanks" or "Thanks, but reconsider."
And if she or the senior staff were to disagree, we're not
in a confrontational mode here because we're all trying to
do the same thing. That's why I made the suggestion that I
did. I wouldn't sweat the small stuff here right now,
because it's not going to offend anybody, I think, or put
anything off track. I think you've heard from me what our
consensus within this administration is, which is we
consider that a component of what we're trying to do, we're
trying to make it happen. We've also--there's a lot of
other things that we would like to do with 30 million bucks,
and if we can do this for less than 30 million or get
somebody else to pick up the tab, we'll be--we could still
use the money.
So I mean, given that bias, if yours is not--if
yours, the committee, is not identical to that--and I think
to some members of the committee, it is not identical to the
one that i just articulated--then if you wish to articulate
your own position very clearly, this would do it. Again,
I'm really--and not just for the record. I think people
understand, I'm not trying to talk you into one position or
another. These are easy sells for me one way or another
because the distinctions--I mean, these are tactics rather
than strategy. Everybody wants to get to the same place
here.
CHAIRMAN CAPLAN: Jane?
DR. PILIAVIN: There is some discussion. I just
heard in fact one mention of there being lots better ways to
spend $30 million. Speaking now as a social scientist,
which I don't get to do specifically very often here, I
think we should think about the response that was made by
the gentleman who's no longer here, to how effective the
last Surgeon General's letter was, and it didn't sound as
though it was very effective.
You mentioned the use of television. I think you
mentioned it, perhaps not seriously, but you could get a lot
of spots in very high-profile places. John mentioned the
Super Bowl. It only costs $500,000 for a spot in the Super
Bowl, which in comparison to--I always used to think that
was a lot of money, but in comparison to $30 million, it's a
drop in the bucket.
In terms of the persuasive power that we know
different media have, television has a whole hell of a lot
more than a letter that comes in the mail, that if it has
got a congressional stamp on it, is most likely to end up in
the waste basket, because a lot of people don't think very
highly of the Congress, and a communication from them is
unlikely to be seen as terribly valuable, whereas a
communication--
CHAIRMAN CAPLAN: I hope you have the data to back
that up.
[Laughter.]
DR. PILIAVIN: I don't, but I could probably find
it.
But seriously, it's clear that this is what the
Surgeon General would like to do. I think a spot with him
saying this on television, that could be put on 15 times for
a third of this money, even in extremely high-profile
places, would reach more people with more effect, and I
would suggest that maybe it would be good to have some
social scientists who are more expert in persuasion than I
am--and I could give you a list of people--speak to him
about alternative ways of doing the very same thing.
CHAIRMAN CAPLAN: Well, just two quick comments.
One, there will be many television marketing people
available after November 9th who might want to work on this,
and there are also--I think the language that I like about
this is to try and figure out the most efficacious and
widespread means of informing, including the letter, because
it does make a window there to have it taped, read,
broadcast, and, as I was trying to indicate, my concern is
coordinated. There are a lot of other things going on, and
I hope that the way that language reads would get a
coordinated effort going with the letter. I too am not so
sanguine about the fate of the letter actually, even though
I was joking about it, but I'm not impressed that a letter
alone is going to get us where we want to be, and I think
that was the lesson of the Koop thing.
So now, how about a call for vote? Yes, I see
some sentiment for that. All in favor of the Kuhn proposal
on encouraging correspondence?
[Show of hands.]
DR. NIGHTINGALE: Eleven votes in favor.
CHAIRMAN CAPLAN: All opposed?
DR. NIGHTINGALE: I see no votes.
CHAIRMAN CAPLAN: Abstain?
[A show of hands.]
DR. NIGHTINGALE: I see--
CHAIRMAN CAPLAN: I got two abstain.
DR. NIGHTINGALE: We have two abstentions? The
motion has 11 votes in favor and no votes against, two
abstentions. Motion carries.
CHAIRMAN CAPLAN: All right. Now, I'm in a mild
dilemma here. I want to leave some time for public comment,
and I want to leave some time for consideration or talk
about further comment about the matrix principles, whether
there's something we can do. I know we don't have to settle
that one, but I'd like to at least--since we had people kind
of turn in homework assignments that covered some of this,
it would be useful to give some opportunity for a little bit
more comment on that.
So my proposal is this: How many people do we
have who want to make public comment? If I could get hands
way up. Okay. What I'd like to do then is ask you, as I
often do, to limit it to two minutes, come to the mike.
We're going to do it in the order in which you arrive at the
microphone. There's someone going first. I like that. Yet
again, let me ask you to identify who you are, and give me
your last name and that will give me the clue about who's
coming next.
MS. HAMILTON: Okay. My name is Jan Hamilton.
I'm Executive Director of Hemophilia Federation of America.
When the IOM gave its report on crisis and
decision making in July of 1995, it considered the nation's
blood supply a unique, life-giving resource. They said the
safety of the blood supply is a shared responsibility of
many organizations, including the plasma fractionation
industry, community blood banks, the Federal Government, and
others. The precipitating event for the findings of the IOM
was contamination of our nation's blood supply by the HIV
virus and all that it entailed.
In the ensuing years, we've heard much rhetoric
about who failed to do what and when and what should be done
about it. We've also discussed the same things today.
Thousands of lives have been lost, and even more families'
lives turned upside down because of the lack of a systematic
process in the past.
The charge from the IOM was that we learn from the
past and prepare for the future, and that's what I hope I
hear coming out of this Committee.
We would like to urge that we continue the charge
from the IOM. Several of the charges have been addressed by
Dr. Satcher as he mentioned them this morning. There are
still some areas that need to be visited and some that need
to be revisited.
Please continue to monitor foreign bodies in the
blood supply: CJD, HIV, hepatitis C, hepatitis XYZ,
whatever, and even such things as bacteria and/or mold in
the manufacturing process.
We appreciate the proactive stands in these areas,
including, but not limited to, NAT testing and leukocyte
reduction. Continue to monitor problems of supply. Dr.
Hoots mentioned it earlier today, and we have been very
concerned about the decline in the recombinant supply and
are speaking with industry directly. But we could use a
little help from our friends.
Continue to address errors and accidents. Some of
these have proven to be disastrous and cost dear lives.
We've been pleased about the Committee's stand on the
prospective payment system issues, and now we would request
some assistance in addressing looming inequities on AWP for
coagulation products, and especially recombinant products
for Medicaid and Medicare patients. There should not be a
double standard for these patients, or for anyone.
The establishment of this Committee has been a
bright light on the horizon, and we're very pleased to be
able to attend, learn, and put in our two cents' worth of
comments on issues heavily impacting the coagulation
disorder population. We hope you will continue to be
vigilant in the areas of blood and blood product safety and
continue your strides towards excellence.
Thank you for allowing us a few minutes to
comment.
CHAIRMAN CAPLAN: Thank you. Questions? No?
Okay.
Again, I'll ask you to state your name for the
record.
MS. FOSS: Thank you. I'm Mary Foss from Mayo
Clinic, Rochester. Dr. Brendan Moore and I would just like
a few minutes to talk about costs and benefits related to
leuk reduction.
I would like to start with just a little bit of
data to give you an idea of the scope of our practice in
Rochester so that you can kind of compare our costs with our
size.
In 1999, we transfused about 39,000 red cells,
about 46,000 platelet unit equivalents, close to 14,000
fresh frozen, and about 6,000 cryoprecipitate.
If I could have the next slide, please?
Our blood inventory comes from the blood we
collect at Mayo Clinic, Rochester. We collect, test,
process, as well as transfuse, and we also receive
supplemental blood supplies from the St. Paul Red Cross. So
about 56 percent of the red cells that we transfuse at Mayo
Clinic, Rochester, are collected at Mayo. And the other 44
percent come to us from St. Paul Red Cross.
Red Cross provides 29 percent of our platelet unit
equivalents, and about 71 percent are collected by us at
Mayo. Of those 71 percent platelet unit equivalents that we
provide, about half of them, a little more than half come
from apheresis donations and the other half are random donor
platelets made from whole blood donations.
So you can see we rely heavily on the random donor
platelets made from whole blood donations. So,
consequently, we feel that if and when we go to 100 percent
leuko reduction, leuko-reduced blood supply, we will need to
continue to manufacture random donor platelets from whole
blood donations.
If I could have the next slide?
You can see that when we calculate what that's
going to mean to us when we switch from our current
situation, which was that we were at 17 percent of our red
cells being leuko-reduced and 70 percent of our platelets.
If we go to 100 percent, we will go from a supply cost of
$15.50, approximately, to a supply cost of $40. This is
exclusively due to the need to switch than to a new
collection set with the in-line filter.
If I can have the last slide, please?
I wanted to just show you the incremental cost for
us to switch from our current situation to 100 percent leuko
reduction at Mayo Clinic, Rochester. When we look at the
increased cost for us for the blood we collect as well as
the increased charges from the St. Paul Red Cross for all of
their blood to be leuko-reduced, for us at Mayo Clinic,
Rochester, the incremental cost is $1.2 million. And when
we saw those big costs, we knew we needed to step back and
better define the benefits before we made the final
decision. We thought we wanted to go to 100 percent leuko
reduction. We saw a lot of reasons why we probably should.
But $1.2 million in our institution is a lot of money, and
we needed to be able to justify that decision.
So now I'd like to turn it over to Dr. Moore to
have him give you some of his thoughts and ideas about those
benefits and this conundrum that we find ourselves in in
making the decision.
DR. MOORE: I'm Dr. Brendan Moore. I'm the
Chairman of the Division of Transfusion Medicine at Mayo
Clinic.
Next slide, please? Next?
This is a figure that's been shown recently
looking at the total number of blood collections in the
United States, and it's available to anyone to look at, but
there's clearly a down trend, which is a national problem.
Next, please?
If you just look at the question of benefits and
concentrate on certain areas of benefits for leuko
reduction, one of the areas that stands out in my mind as
being potentially where the money is is post-operative
infections. If you look at the number of surgical
procedures annually in the United States, there are 18
million, and post-op infections are either surgical site,
catheter-related, pulmonary, or general sepsis.
Now, if you just look at surgical site infections
alone, by the best estimate about 2.7 percent, or 486,000,
surgical site infections per year. Approximately 50 percent
of the transfused blood in the United States goes to
surgical patients, and there are data to support this.
You'll see where I'm going with this in a moment.
Next, please?
These are the data from the Vamvakas' study of
surgical versus medical use, and they're approximately the
same in Olmstead County. So we think it's probably the same
nationally, about 50 percent. And, of course, if you're
going to use surgical site infections, then you're only
looking at the benefit from half of the blood that you
leuko-reduce.
Next, please?
Here's a study by Kirkland, et al., from Duke, I
believe. They did a matched cohort study of surgical site
infections on 255 pairs in inpatient surgery. Now, the main
point of this slide is to show that mortality was different.
Those requiring ICU admission was different. The median
hospitalization, translate that to dollars, and median
direct costs were different if they got a surgical site
infection or they didn't. And we've all known this for
years.
Next, please?
If you then looked at the survivors requiring
readmission, you now look at 41 percent of those with
surgical site infection versus 7 percent, excess
hospitalization of 12 days, and in this one study alone of
just 255 matched pairs, the incremental cost attributable to
surgical site infections alone was $1.9 million--255
patients, $1.9 million, surgical site infection alone.
Next, please?
So if you extrapolate from that--and I know that
economists and maybe epidemiologists would shudder if they
saw how I did this, but if you just take those figures and
extrapolate them to the national data, one is potentially
looking at a cost of surgical site infection alone in this
country annually of $3,764.1 million. Now, that's real
dollars.
However, the proportion of inpatient surgery which
is associated with transfusion is not known, so it's not
clear that all inpatient surgery, even major surgery, is
indeed now associated with transfusions. You can see where
this leads.
Next, please?
Multiple studies have demonstrated that there are
various risk factors for surgical site infection, depending
on the type of surgery, the age of the patient, whether it's
spinal surgery, whether they have a high admission
hematocrit, and whether they're allogeneic transfusions, and
those contain white cells. This is non-leuko-reduced. So
transfusions are only one of the factors that may well have
affected the surgical site infection or other infections.
But it probably is one.
Next, please?
The relative contribution of allogeneic
transfusion to the risk of SSI is actually unknown for most
surgical operations, and this is clear--and I'll come back
to this point. We don't know enough yet.
If you look at the study of post-op infections,
localized or systemic, in 120 patients--this is from Heiss'
study--having colorectal cancer surgery, those getting
allogeneic transfusions had an odds ratio of 2.84 by a
multivariate regression analysis. So, in other words, in
that study it appeared to make a difference.
Next, please?
If you look at Vamvakas' study, retrospective
analysis of nearly 1,000 colorectal cancer patients at MDH,
allogeneic transfusions were associated with a relative risk
of 1.07 for post-op infections. In other words, there was a
7 percent increase in those transfused with allogeneic
blood. And another study from Vamvakas in the same
institution, this time looking at just CABG and pneumonia,
which is a common occurrence after CABG, the allo-exposed
versus the unexposed had a clear difference, and the risk
was related to the length of storage of the blood. None of
these were prospective, randomized studies.
Next, please?
If we weren't looking at this unfortunate
situation, none of us, as was mentioned earlier, would even
bother discussing this question. We'd all have universal
leuko reduction a long time ago. But, unfortunately, we are
dealing with large dollars.
Next, please?
So, in summary, what I would like to say is that
allogeneic transfusion exposure appears to be one of many
significant factors in post-op infections. The result of
multiple studies, in fact, appear to implicate the white
cells in that blood, hence, leuko reduction. The potential
savings to society are enormous if the post-op infection
rate can be significantly reduced. And, remember,
transfusions may only be one factor, but it may be an
important one.
The final comment I would make is really a plea
that before we make significant decisions--and this
Committee makes significant decisions relative to blood
safety in the United States--that there be more data
obtained so that the decisions that are made are based on
some sort of analysis that gives you a balance. And I think
most experts would agree that there really is a need for
large, multi-center, prospective, randomized clinical trials
to assess the role of white cells, red cells, and even
plasma, free hemoglobin, and maybe even iron content in
post-op infections, because there are data supporting the
role of each one of these, but we don't know the full answer
yet. So before we spend the money, let's figure out what
it's likely to--what the bang is likely to be.
Thank you.
CHAIRMAN CAPLAN: Questions? Larry? Dr. Moore,
do you want to stay up there?
MR. ALLEN: I was just curious. In the best of
both worlds for you, what would you prefer to have? What
would you like to see happen?
DR. MOORE: Well, to make life very simple, I
would like the FDA to mandate leuko reduction. Number two,
I would like the government to then recognize this is going
to cost an enormous amount of money, and before the mandate
is done, more data are obtained, and they're obtained in
decent studies with large numbers in a fashion similar to
what the TRAP study attempted to do and did very well some
years ago for a totally different issue. So that everybody
would arrive at the same point and say we have a consensus,
we don't need a mandate that people fight over, it's very
obvious that we have cost-effectiveness, let's do it.
Because there are multiple medical reasons for giving
leuko-reduced blood, but not necessarily to everyone.
That's the problem.
COL. FITZPATRICK: Dr. Moore, on your cost
estimates, do you use a micro-aggregate filter at the ward?
And did you eliminate that as part of your cost estimate?
DR. MOORE: No, we don't use micro-aggregate
filtration, and, in fact, if you look at the data on
micro-aggregate filtration over the years, it really hasn't
been effective in taking out the numbers of white cells that
one needs to get to. And, in fact, recent data from Europe
particularly would indicate that the level that we should be
aiming at is not 5 by 106 per unit, it's really 1 by 5 by
10--1 times 106 per unit. And that is achievable with
modern filters, but not at the bedside and not
micro-aggregate. They'd have to be done in the lab with
proper QC.
COL. FITZPATRICK: I'm not sure--the question was
more pointed to how you're currently using them, and would
you eliminate them with ULR?
DR. MOORE: Oh, no, we don't--we have never used
them. For those patients who need leuko-reduced products in
our practice--and you saw the figures for the numbers--we
use third and fourth generation filters. They're not
micro-aggregate filters.
CHAIRMAN CAPLAN: Last one to Jerry.
DR. WINKELSTEIN: I noticed that the increment
between pre- and post-leuko reduction cost estimates was
incrementally in terms of percent greater for the Mayo
Clinic than the American Red Cross. I think the Mayo Clinic
went from 400,000 to 900,000, and the American Red Cross
from 1.6 million to 2.1.
Is that a real difference? Are there cost savings
that differ from provider to provider, or what?
DR. MOORE: Mrs. Foss might want to make a comment
about this since she pulled the data together and put it
together. But it seems to me that what we did was a fairly
rough and ready estimate as to what our own cost difference
was going to be based upon the materials--not the blood
itself, the materials that we would have to buy, the
filters, for example, the bags, the new bag systems, in
order to do it in our establishment. We really can't
compare that to anybody else's cost where scale may be
different, for example, at Red Cross, et cetera.
The contracts that we have with the manufacturers
of blood bags and filter systems might be quite different
from the contract that the Red Cross would have. So it's
apples and oranges, and I wouldn't like to compare more than
that.
Mary, do you want to say anything?
[No response.]
DR. WINKELSTEIN: Thanks.
CHAIRMAN CAPLAN: Thank you. I'm going to move us
along here. Please tell me who you are for the record.
DR. FANG: Thank you, Mr. Chairman and members of
the Committee. My name is Chyang Fang, and I am Director of
Scientific Affairs of Chiron Corporation's Blood Testing
Division.
The purpose of my comments today is to update you
on the impact of Chiron's genomic Nucleic Acid Testing (NAT)
capabilities.
Since March of 1999, Chiron and its strategic
partner Gen-Probe, Inc., has been supply NAT reagents,
instrumentation, training, and technical support to U.S.
blood centers performing NAT under FDA-approved
Investigational New Drug (IND) protocols.
The Chiron HIV-1/HCV assay is currently utilized
to screen approximately 75 percent of all volunteer blood
donations in the U.S. under IND. Blood banks from the
civilian blood collection sector and, most recently, the
Armed Services Blood Program are now routinely screening
blood donations using this highly sensitive and new
technology.
Scientific studies estimate that NAT may reduce
the window periods of potential HCV infection by 70 percent
and by nearly 50 percent for HIV.
Working with all blood centers in the United
States, by June of this year NAT assays have identified 62
HCV-positive donors and four HIV-positive volunteer donors
whose infectious units would have been transfused but for
NAT testing.
In addition, NAT assays are responsible for
identifying three HCV-positive donors that were initially
HCV EIA negative due to laboratory errors.
In addition to interception of infected donors,
NAT has demonstrated other public health benefits. NAT can
identify infected persons at the early stage of infection
for more effective antiviral treatment.
In addition, NAT results are an important tool for
counseling of volunteer blood donors and also paid plasma
donors. NAT can enhance diagnosis and treatment and prevent
secondary infections.
Outside of the U.S., the Chiron NAT assay has been
specifically approved for blood screening in France,
Germany, Spain, Australia, and Singapore. The Australian
Red Cross began routine screening in May 2000. Various
European countries are finalizing plans for full
implementation of nucleic acid testing.
Blood is a national resource that must be
preserved and maintained. Each person in the U.S. shares
equally in the potential need for blood and the resulting
potential for contracting transfusion-associated illness,
such as hepatitis and AIDS. Therefore, we must strive to
protect and maintain both the safety and availability of the
nation's blood supply. There are still cases of
transfusion-transmitted HIV and hepatitis in the United
States and other countries. We can substantially reduce
these infections by adopting new technologies, such as NAT,
in order to improve detection of these infectious agents.
NAT testing ensures that our nation's blood supply is truly
as safe as it can be.
Thank you.
DR. NIGHTINGALE: Do we have any questions? Are
there any questions from the committee members?
[No response.]
DR. NIGHTINGALE: If not, thank you very much,
doctor.
Merlyn? I'm sorry, Merlyn, could you identify
yourself in the microphone?
DR. SAYERS: I still am Merlyn Sayers. I'm going
to be speaking on behalf of America's Blood Centers, and we
really do appreciate this opportunity to address the
committee.
America's Blood Centers is an association of some
75 diverse community blood programs that are responsible for
about half of the nation's volunteer donor blood supply.
ABC has many concerns that really reflect the
division of opinion, both within the organization and
nationally, regarding the justification for universal
leuko-reduction. While some ABC blood programs have
embraced universal leuko-reduction as appropriate, those who
point to the lack of medical consensus on the topic also
consider that the selection of such components is an
expression of the practice of medicine. ABC members are,
however, in agreement that if FDA does go forward with a
recommendation for universal leuko-reduction, the agency
must proceed with formal rule making, rather than allowing
implementation to proceed until a standard of care has been
established, followed by regulation under CGMP. Only
through formal rule making will there be consistent quality
control and licensure requirements.
Furthermore, with regard to licensing, this
ponderous process must be streamlined before the expected
surge of universal leuko-reduction linked applications, and
to these ends we ask this committee to urge the FDA to
continue considering creative, effective and efficient
methods, as the agency outlined in a June concept paper, to
assure the quality of leuko-reduced components and the pace
of their licensing.
We're concerned that as we move towards universal
leuko-reduction some other unforeseen issues are going to
arise, such as the problems currently being seen with
leuko-reduction of red cells from donors with sickle cell
trait.
And finally, we ask this committee to exhort the
FDA, even as that agency proceeds with rule making, to
cooperate with PHS, health care agencies and hospital
organizations to help remedy reimbursement deficiencies.
And while we recognize that FDA is not charged with solving
reimbursement problems, it no longer is acceptable for
regulation to be divorced from reimbursement. And in this
context, America's Blood Centers applauds members of this
committee for their resolution of April the 26th, which
recommended legislation to provide funding for blood-related
costs, and we respectfully solicit this committee to persist
in encouraging steps that put an end to unfunded mandates.
Thanks.
DR. NIGHTINGALE: Thank you. Before Ms. Gregory
approaches the microphone, are there any questions from the
committee for Dr. Sayers?
[No response.]
DR. NIGHTINGALE: If not, Kay.
MS. GREGORY: Some disadvantage to being short.
My name is Kay Gregory, and I'm here with two
separate statements. First of all, I want to speak--when I
get the microphone fixed--on behalf of the new multiple
organizational task force that the AABB has developed, to
evaluate and develop recommendations to simplify the uniform
donor history questionnaire, including consideration of an
abbreviated version for repeat donors.
The task force appreciates the opportunity to make
this advisory committee aware of its activities. We hope
that this meeting will also provide an opportunity to let
the public and those with a special interest in donor
screening know of our activities. So we're here to tell you
about a concern, but a way in which we're going to address
the concern, instead of just letting somebody do something.
The task force was formed in response to
information from the FDA that the agency would like to see a
simplified questionnaire developed, perhaps sometime in
2001, and would prefer to have a single initiative supported
by the entire blood banking community. The task force,
chaired by Dr. Joy Friday, is composed of representatives
from the AABB, ABC, ABRA, ARC, CDC, and the FDA, and we will
shortly be joined by representatives from NHLIB and the
Armed Services Blood Program Office as well.
The task force is intending to be the core group
guiding the effort, but each organization will be active
participants, assisting the task force with things such as
identifying member centers to pilot questions and generate
additional information or data as needed. It is vital that
we include everyone in this effort including donors.
The task force charges are to reevaluate the
scientific validity of all FDA required infectious disease
questions in view of the most recent scientific data,
including current testing technology, to identify and reword
questions for which the wording may represent comprehension
difficulties for average individuals, to identify questions
that can logically be grouped together and simplified, and
to reorder questions as appropriate, and finally, to
evaluate methods and develop recommendations for
administering the questionnaire. These would be things like
oral, written, computer-based questioning methods, how to
handle recurring questions, et cetera. And then lastly,
when we complete our task, submit it to FDA to be sure that
it meets their approval.
It's an active task force. It was only organized
in June. We've already had three conference calls where the
entire task force and numerous conference calls for the
subcommittees that are working.
I just want to list for you some of the activities
that are currently under way. First, we're working with the
FDA, planning a joint workshop, which will be held October
the 16th, and information about that will be posted on both
the AABB and the FDA web page and will be announced in the
Federal Register shortly. Secondly, we've distributed a
survey to obtain information about questions currently in
use to selected blood centers, hospital blood banks and
plasma collection facilities. Third, the AABB members have
specifically begun to review which of the AABB added
questions need to remain and which we might do something
about. We're not going to blame it all on the FDA. The
AABB has made this questionnaire somewhat complicated as
well. And then, finally, the FDA is busy compiling
information about questions that are most frequently cited
in error and accident reports, and they're also looking to
give us some direction about those questions or those errors
where they're non-negotiable, that is, they're going to need
to stay on the questionnaire in some format, maybe not the
exact wording we use now, but at least so we're not spinning
our wheels on things that we can't do anything about.
I thank you for the opportunity to speak today.
The task force is excited about the opportunity to
accomplish meaningful change, and we plan to keep giving you
regular, progress reports.
DR. NIGHTINGALE: Thank you, Ms. Gregory. Are
there any questions? Dr. Penner?
DR. PENNER: Just a quick question. Will there be
a first-time donor questionnaire as opposed to those
repeated donors, or is all going to be strictly a global
thing?
MS. GREGORY: We really haven't gotten quite that
far into it yet. That is one of the things we're
considering, is, you know, can the first-time donor be
different from the repeat donors? Are there local issues
that need to be addressed? So I can't really give you an
answer yet.
DR. PENNER: Yeah, because I'm sure it's a
nuisance to continue to answer the same--
MS. GREGORY: That's one of the things that we
hear is, "I've already answered this, and if I said I never
did it, why do you keep asking me?"
DR. NIGHTINGALE: Are there any other questions
from the committee for Ms. Gregory? Is there anybody else
in--I'm sorry. Kay, you've got another one. You don't have
to identify yourself again.
MS. GREGORY: This time I'm speaking on behalf of
the American Association of Blood Banks, and we would like
to take this opportunity to recognize the activities of the
advisory committee on blood safety and availability.
We note, however, that the committee charter
indicates that the committee would cease to exist on October
9th unless renewed by appropriate action prior to its
expiration date. Even though we've not always agreed with
all of the committee recommendations and conclusions, we
believe the committee has served, and should continue to
serve, a vital function. Although there are other public
forums for discussing scientific matters, this committee is
the only public forum for addressing other important issues
such as availability, cost, other economic issues,
reimbursement. Further, the committee has been remarkably
successful in obtaining action in response to its
recommendations. Clearly, your voice is being heard in the
right places.
In keeping with the general approach of the
committee to enunciate positions in the form of a
resolution, the AABB makes the following recommendation:
Whereas, the Advisory Committee on Blood Safety and
Availability provides a public forum for discussing issues
of concern to the blood banking community and the public,
and whereas the committee has successfully formulated
recommendations presenting potential solutions to the issues
it has discussed, the AABB recommends that the committee
charter be renewed and that the committee continue to
provide this valuable service to the blood banking community
and the public. In short, we urge the Secretary to take
appropriate steps to allow for the committee's ongoing work
to provide patients with a safe and available blood supply.
Thank you.
DR. NIGHTINGALE: Thank you, Ms. Gregory. Would
you state for the record that my mother did not write that
statement?
[Laughter.]
MS. GREGORY: I will state that I wrote the
statement.
DR. NIGHTINGALE: Then I would state more formally
for the record, that we have completed the process of
applying for a two-year continuance of our charter, and that
we anticipate a favorable response from the Secretary. The
process is such that she does not sign the reauthorization,
I believe, until after the conclusion of the current
committee, but CAPT McMurtry is the administrator who
understands this far better than I. Comment, Mac?
CAPT. McMURTRY: We expect the Secretary's
signature right around the end of September.
DR. NIGHTINGALE: We, nevertheless, appreciate the
kind words very much.
CHAIRMAN CAPLAN: We have approximately, I would
say, 30 minutes before we start losing airplane travelers.
My suggestion would be that we take advantage of that time
to think about--and this is just a suggestion; I'm open to
other suggestions--but my suggestion is that we take
advantage of that time to think about not resolving the
issue of what matrix or dimensions to use, but perhaps to
direct the staff to take under advisement what Dr. Epstein
presented, what Rick presented, some other comments, in the
formulation of a matrix of points and factors to consider.
The reason this becomes important, that we give
some direction to a matrix, if you will, is that with this
international meeting coming up, the Secretary, I suspect,
could use some advice about what framework to bear into that
meeting in Geneva. What's that, November?
DR. NIGHTINGALE: November 13th--14th, 15th, 16th
and 17th, I believe.
CHAIRMAN CAPLAN: I mean, I may be wrong, but I
think we're going to today, arrive at the framework, but I
think we probably have enough material here to assemble what
starts to look like a series of points to consider or
principles to consider, and sort of use that as the outcome
of our trying to struggle to do this on a case-by-case
basis, but saying that it jumped out of there. So anyway,
that's my thinking about where we might go with principles,
but the floor is open for other business and other
approaches.
DR. AuBUCHON: I am pleased to see that we're not
going to try to come up with that framework in the next 28
minutes. I was very impressed with Dr. Epstein's
enumeration of the different approaches, and I think Dr.
Davey's distillation of similar concepts to a template
format could indeed be useful.
I would like to suggest that we consider hearing
from Dr. Salmi [ph], who produced the template that was
utilized at the WHO meeting in March, which now has had some
practical application, at least within that meeting, to see
another approach from another country, but one that has had
some international application, and it may help move us
toward understanding the factors that really push us to make
certain decisions.
COL. FITZPATRICK: In light of Dr. Caplan's
comments, I'd like to add some input for the staff when we
start formulating that or they start working on that.
We have the advantage of being one of the largest
organizations in the nation that collects, manufactures,
processes, and transfuses blood products, and so we get to
see the global perspective. We now have more U.S military
deployed throughout the world than ever before. In every
instance of that deployment during this time of peace, we
have had to collect blood, fresh whole blood, and transfuse
it untested to treat either U.S. casualties or local
national casualties or accident victims.
This presents us a unique problem in safety and
availability challenges because the charter of our office is
to continue to maintain and supply safe, compliant blood to
all U.S. forces deployed throughout the world. Each
additional test makes it more difficult to do this.
We now have multinational forces supported by
multinational medical forces and a multinational hospital
being built in Kosovo. Adhering to the policy of the United
States that U.S. deployed forces will receive FDA-licensed
or equivalent blood becomes much more difficult in these
situations.
Timing is everything, and it appears we have
actually timed something appropriately. November 9th and
10th we will have the NATO Civil-Military Blood Committee
meeting here in Washington, D.C., immediately after the
American Association of Blood Banks meeting and immediately
prior to the meeting in Geneva.
The topic and focus of the NATO meeting is
interoperability and harmonization of international blood
supplies. In order to achieve those goals, we need our
federal regulatory agencies of each nation and our blood
committees and blood bank organizations of each nation to
work together in Geneva to try and harmonize the
requirements and the donor selection criteria and infectious
disease testing criteria in order to make support for these
multinational forces more possible, because as each nation
draws its military down, we will have more multinational
events.
Costs continue to be a major factor, and as I have
mentioned before at this Committee, every time a new test is
required, it takes us two years to accomplish budgeting to
do that test. Because of that, reallocation of resources is
required, and in the military medical health care system we
do have to take from some other pot to put into the
infectious disease testing pot, and some factor of health
care suffers for those two years.
So I would just ask the Committee to continue to
keep in mind the aspect of funding for the Department of
Defense blood program when a new test is required, and that
we feel the development of a template or cost/risk/benefit
model which will provide a framework for making these
decisions and discussions extremely important in our
strategic planning and the world's strategic planning in
keeping a safe and available blood supply.
I want to thank HHS for allowing DOD to be a part
of this Committee, and we hope that we can continue to
contribute. Thank you.
DR. DAVEY: I have a couple of comments, first
about the international meeting coming up, Mr. Chairman. I
spent a year with WHO working with their blood program and
spent a lot of time in Africa, and I can only second I think
what Karen was saying earlier for those who are going to be
at the meeting that the testing techniques have been there
and have been available for 10, 15 years, mini-pool testing,
cartridge tests. The technology has been there, and the
funds, almost, to provide the testing has been there. What
is lacking, again, which Karen pointed out, is the training
and infrastructure and also the importance--and, again, this
was brought out--of each country developing their own blood
program based on their own needs and their own culture and
their own customs.
There have been a lot of mistakes about moving
technology in in a Western fashion with Western technology
and Western thinking, and it hasn't worked. So there's been
a lot of experience. We're at another threshold, I think,
of consciousness raising, and perhaps we can move this
forward now with the experience, and I think the program at
WHO has really gotten funded with some good people, so
there's a good opportunity now to move it forward.
On another topic, I fully support the Chairman's
recommendation that staff go ahead and work on a matrix and
see what we can come up. I think Jay has had some good
ideas. Perhaps my seven points might be considered.
Also, I think it would be useful as maybe a
preamble, since this is a turning point for the Committee,
we're looking backward, looking forward, that a listing of
some of the pending issues and actions--again, Jay had a
good one, I think Mike and others have pointed out some very
key issues--that that be kind of a preamble. These are
things that are facing this Committee and the country, and
this is a template that we can use to address them in the
future. It would be a useful piece to put in the minutes.
CHAIRMAN CAPLAN: We did mention that one of the
things that might be useful to add to our agenda was a
consideration of changes of unnecessary or out-of-date
safety measures, and we haven't sort of gone at that
systematically, and that might be an important agenda item
to flag for the future, too, using the matrix approach to
help us think about that.
DR. NIGHTINGALE: Yes, I appreciate Dr. Davey's
comments. In response to the anticipation of future agenda
items, while the process might appear somewhat undirected, I
hope it's appeared to be transparent. We have tried to use
the Committee to identify problems that, bluntly, we thought
might be resolvable. This is a very useful instrument of
government, but it's by no means the perfect--better still,
it's not the only instrument of government. You saw that,
for example, when the TSE scientific debate went to the TSE
Advisory Committee rather than to this Committee, not
something that everybody agreed with but, nevertheless, if
I'm a little reticent to commit to the next year or even to
the next meeting's agenda, it is because blood is a
complicated business. And it's also because blood is a
high-level business. I think there were some kind words
spoken about what happens after resolutions are made here,
but that's really only possible when there is consensus
within the Department about the topics and the expected
outcomes before the decisions are made.
I mean, we can perhaps--an analogy I've used
elsewhere--keep the river from flooding the village, but we
can't make the water go back uphill here. And I think that
that's been one of the strengths of the Committee.
So while I take it under advisement, there is a
clearance procedure that I would have to go through, and
I'll at least try to keep that clearance as transparent as
possible to the members.
MS. LIPTON: My only comment, I absolutely support
looking at what we could do to reduce or eliminate some of
these things, but I think there is a process here, and the
process begins with FDA. And if they have that on their
agenda--I mean, I don't think--I certainly don't feel
qualified to sit here and evaluate scientific data. So
anytime something comes up to this Committee, I would hope
that we have, you know, some kind of deliberation before
BPAC or some other body that we can then add those other
factors. I would hate to see us bypass that route before it
got here.
DR. NIGHTINGALE: And if I may second that, I
believe I'm on the record repeatedly as supporting that, and
possibly to simplify what Ms. Lipton just said, you know, if
there's science on which to base a decision, it shouldn't be
here. We don't get the hanging curves. So far we haven't
got them, and if a hanging curve shows up here, it probably
should have been somebody else who had a chance to hit it
out.
CHAIRMAN CAPLAN: Jim?
DR. AuBUCHON: I understand what you're saying,
Steve, but at least today in blood safety I don't see many
issues where the scientific conclusion or the scientific
consensus alone creates a slam-dunk situation. And there
are clearly other factors, particularly economic, that need
to be factored in. Furthermore, Blood Products Advisory
Committee or other Advisory Committees may not be in a
position to take into account all of the availability
discussions, which are part of an ultimate conclusion of an
issue.
So I would like to, as we look at how decisions
are made, discuss not only the factors that should be
considered in making a decision but which arms of the
Federal Government in its Advisory Committee structure can
be utilized and how they should interact with one another.
CHAIRMAN CAPLAN: It's fair to say that the Chair
occasionally gets from the people that--even though we
sometimes think of ourselves as advising the Secretary,
occasionally e-mails arrive at my desk asking about things
that the public wants to know about. Some I pass to Steve.
Some which are more acerbic I don't. But one thing that
comes up a lot is the issue of male sex and the rule about
donation. I've heard a lot of communication about that.
People are interested in that.
My point in getting us to some issues that might
be considered by us was not so much to jump ahead of where
the science might be but to act as a prod to make sure that
the science gets done if it doesn't seem to be getting done
because we've grandfathered in a lot of things that may make
no sense or may be sort of up for reconsideration.
Jay's list is pretty good. I didn't get the sense
that the FDA was asleep about these things, but it's sort of
pushing more than it is saying, oh, okay, well, we can set
the standards here about all those things.
DR. EPSTEIN: Yes, I think--I presented a list of
things that are in the hopper. I mean, these are happening
now. We are considering them. But I resonate very strongly
to both what Karen Lipton said and what Jim AuBuchon said,
which is, you know, we start with an FDA process, but we
have an underlying problem of divided responsibilities. And
the reason for that is that the lines have been drawn in a
certain way over areas of responsibility. I mean, FDA is
not supposed to look at cost. We're supposed to look at,
you know, safety, effectiveness. We can go as far as to
look at, you know, public health, risk/benefit, but not the
C word.
You know, that creates a problem in an era of
limited resources and very difficult trade-offs. And on the
one hand, I think that we have evolved to a system where we
do have fora to vet related issues, global concerns. But
the process by which we decide which issues surface, how
they surface, where they go, when the decision should get
made, what is linked to what, that's the part that's
ill-defined.
I mean, we recognize, for example, that the issue
of safety advances and how to pay for them is linked to the
reimbursement system. But, on the other hand, it's been
pointed out many times, and correctly, that that isn't
really FDA's charge. But then whose charge is it, and what
system do we have to ensure the integration? And I think
that's really where the challenges lie. I think we do have
a pretty good idea what the particular concerns are, but we
don't have a system that decides which is the correct
paradigm to address that issue.
You know, I guess we aren't going to do it at this
meeting, but my notion was that we might want to go through
that list and ask ourselves what paradigm are we applying
now and is it the right one, kind of issue by issue. Now,
that doesn't mean that we want to take on all issues in this
forum, but I think thinking along those lines is what's
needed.
You know, there are some people who would say,
well, you know, you're doing the right thing about HHVA
wanting to have proof of transmission before you attempt to
intervene. And there would be other people who say, But
that's not following the precautionary principle. You know,
you've already got enough reason to be concerned because
it's leukocyte-associated.
So, you know, the whole problem is where should
you be and how do you decide where to be. How do you
measure the relative importance of the different issues? I
think part of the problem is that at least to the FDA, they
each present as an entity in their own right, because we're
really not charged with trading them off against each other.
We're charged with dealing with them all at once.
And so, again, my feeling is, yes, it starts at
FDA and, yes, we don't bypass FDA. But there is a dilemma
related to divided responsibilities.
DR. NIGHTINGALE: To follow up that, I think that
the issues we've had over the last three years, the six that
I enumerated--hepatitis--or the Surgeon General did, I'm
sorry--hepatitis C, CJD, plasma shortages, blood shortages,
reimbursement, and error management--were issues that came
to us.
We're at a nice time right now, as the Surgeon
General said this morning. We may not have put them to bed,
but we've made constructive progress on them. It may very
well signal a transition not only for the Committee but for
the blood community as a whole that we are in a position to
start acting proactively rather than reactively. And if
that, in fact, is the case, that's a good thing.
I also think if that is the case, we've made a
really good start this afternoon. We do not give door
prizes out at the meeting for the best talk, and that's good
because, not to offend anybody, but the last two speakers
would have had to fight over the door prize, not because
they happened to have the right words, I think, but because
I think both of them--and if you'll forgive me, particularly
Dr. Epstein--captured where the Department is right now.
Dr. Epstein's is not exhaustive--it was not an exhaustive
analysis. It was a useful framework, and what he said there
very much reflects what we were--the internal discussions of
the Department over the last month after we set on this
agenda.
This is a process, a discussion--what we wanted to
do was find a place to start where hopefully there was some
consensus outside not only of the Humphrey Building but
outside of the FDA, CDC, Humphrey, NIH axis. And that's the
reason why I brought up the analogy of the door prize
because that was a tricky thing to do.
It is my impression from reading the body
language, if you will, that Dr. Epstein's formulation of the
position has fairly wide support. And if it does--for what
it was intended to be, a formulation of talking points, a
set of principles that we realize are not the final answer
to a very complex question, but a point of departure. And
if that is--if my perception is shared, it would be of some
assistance, I think, to Dr. Epstein and the others involved
in planning for, say, the Geneva meeting to have some
expression of that support, if, in fact, that's there.
CHAIRMAN CAPLAN: John?
DR. PENNER: Jay brought up the "C" word, which I
presume is cost. And at this point I'm getting a little
confused inasmuch as I pay something like a thousand dollars
a day for antibiotics on a patient in the intensive care
unit, and $3- or $400 worth of blood may be life saving.
And I don't quite understand how the price of blood is being
set by HCFA, at this point, which then sets the pace for the
rest of the reimbursement situation.
Our movement from some $48, as I mentioned, 20
years ago to the 90-some-dollars now seems to be rather
negligible in comparison to other drugs that are being used
and employed very effectively at very, very high levels of
cost. Does this need more attention or have we spent as
much as we can on our energies in getting that area taken
care of?
DR. NIGHTINGALE: No. I think the one last point
that I would want the committee to recall is something I
said earlier, on November 9th it's a new ball game. The
issue of reimbursement for blood products I suspect we have
taken it as far as we can take it in the isolated context of
blood alone. And while I'm not suggesting that we have
failed because I don't think we have, I think that to get
beyond where we have gone with our recommendations and
actions resulting from them, we're going to have to look
more carefully not only as to how blood fits into the
current reimbursement framework, but perhaps into
limitations of the current reimbursement framework. And a
new administration is a convenient place for that to happen.
I said far more right there than I perhaps should have.
DR. PENNER: Blood is cheap in comparison, and I
think we all fail to look at that because we are left over
with the years of getting blood, and it's donated, and
therefore it should be free concept in our minds, and that I
think has got to be altered.
CHAIRMAN CAPLAN: Just to respond to Steve's point
and see if we can get agreement perhaps on a recommendation
or suggestion to the staff on this one. Jay's framework I
don't think is a basis for consensus about decision making.
It's a good matrix, however, of points and approaches to
consider. I'm not a particular fan of the precautionary
principle, for example. So I know it's on the list, it's a
way to go. It wouldn't be my way to go. I can't imagine
capitalist-mad America deciding that it's going to be risk
averse in the way that the precautionary principle suggests,
but maybe--I doubt it. That's why we'll be eating GMO and
everybody else won't.
However, there is enough there to set out a matrix
of points to consider ways to approach, and that's what I
would like us perhaps to recommend that the staff develop
for us. Can we perhaps even take a vote so I can get a
formal or a second or a motion or something? A motion. I'm
making a motion that we direct the staff to develop a matrix
out of the presentations we have of points to consider. We
then might move on with that matrix to some weighting or
what we in the trade would call a prescriptive position
about which is better or which is worse or which we want to
go with. But first we need to get the whole thing in front
of us.
DR. NIGHTINGALE: Since I would--Mac and I would
have to be the people to do what you're directing us to do,
are you directing the staff to develop a matrix from the
presentations--plural--made at the committee as a basis for
further discussion about principles--
CHAIRMAN CAPLAN: Right.
DR. NIGHTINGALE: --that would underlie safe,
effective and portable blood supply?
CHAIRMAN CAPLAN: That's my idea.
DR. NIGHTINGALE: That's eminently doable, and
thank you for the opportunity if that's what you choose to
do.
DR. HOOTS: Kind of implicit in that, but would
you consider, as a friendly amendment, that essentially this
draft would then--we would kind of commit ourselves to
applying it, as it's already been implied, that we apply it
to certain models, whichever ones we prioritize in the next
few meetings, to see how it works; in other words, we
actually commit ourselves to seeing if we can use this
paradigm to develop policy?
DR. NIGHTINGALE: While my own enthusiasm for Dr.
Epstein's and Dr. Davey's presentation is there, I think
that the presentations require further public discussion
before there is a commitment to them. And I think not
because of any inherent weakness in them, but because these
are new ideas to get into the public--
DR. HOOTS: I didn't mean to imply that we would
do it for all times. I mean just experiment with trying to
use that thought process as we go through a problem and
learn as we go. That's what I meant, not that it would be
the end all and be all.
DR. NIGHTINGALE: Beta testing, in other words.
DR. HOOTS: Correct.
DR. DAVEY: So moved.
DR. GUERRA: Second.
CHAIRMAN CAPLAN: All in favor?
[Show of hands.]
CHAIRMAN CAPLAN: Opposed?
[Show of hands.]
CHAIRMAN CAPLAN: Abstained?
[No response.]
CHAIRMAN CAPLAN: That's a fairly innocuous
recommendation I suspect.
I'm going to take the last few minutes here to
thank the people who have served on the committee whose
service is now over. When is our next meeting? January
what?
DR. NIGHTINGALE: I was afraid you'd ask. Mac?
The next meeting of the committee? The last Thursday and
Friday in January. We've had a mix-up with days before.
But if anybody has a 2001 calendar, it's the last Thursday
and Friday.
CHAIRMAN CAPLAN: Of January.
DR. NIGHTINGALE: Of January 2001, right.
CHAIRMAN CAPLAN: January 25 and 26; is that the
last? January 25-26?
DR. NIGHTINGALE: Let me state for the record that
you will be informed through the Federal Register, and
personally and on the committee's website of the next
meeting of the Advisory Committee. We do have a contract
with this hotel, but to the extent that we can minimize
conflicts with our I don't know if I want to say sister
organization, we will certainly strive to do so.
CHAIRMAN CAPLAN: So still hang onto the last
Thursday and Friday date until you're told otherwise.
The other thing that obviously is going to happen
is elections come, Steve has used the word "new ball game" a
couple times. There could be changes, not by the time we
meet next, but changes could come in the administration, and
the people that we talk to at HHS and so on, and the
secretary's office. So it'll be an interesting time for us
to transit. My hope is, however, that our work has been
such that we will sail on, despite new winds in the air,
potentially, or for certain--actually, there will be no
winds for certain no matter what goes on--and that whoever
is in Congress, whoever is in the White House, whoever winds
up at HHS is going to see the value of having this group
here.
Remember, please, that the floor is open for
nominations to membership. And I think you have to get it
in by the end of the month, and Mac will accept by phone or
e-mail or letter names and ideas, and so that is very
important that we get the right people to replace the good
people that we're losing.
Unless there's new business, I'm going to suggest
that we now adjourn.
[No response.]
CHAIRMAN CAPLAN: Okay. We stand adjourned.
[Whereupon, at 4:19 p.m., the proceedings were adjourned.]
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