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Blood Banking and Regulation: Procedures, Problems, and Alternatives IV Congressional Oversight and Regulatory Initiatives Editor's Note: In July 1995, as the Forum on Blood Safety and Blood Availability was preparing the agenda for its final meeting, the Institute of Medicine (IOM) report HIV and the Blood Supply was issued. That report was a retrospective discussion of the events of the early and middle 1980s concerning transfusion-associated human immunodeficiency virus (HIV) and AIDS, with a special focus on decision-making. The authors of that study offered a number of recommendations to the Department of Health and Human Services (DHHS), to the blood banking industry, and to the public. The Forum therefore invited Anne Marie Finley and Mary Pendergast to address the closing session of the September 1995 meeting, to describe the work of the Congress and of the DHHS with respect those recommendations. Their presentations continue the theme of regulatory effectiveness and regulatory reform.
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Blood Banking and Regulation: Procedures, Problems, and Alternatives This page in the original is blank.
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Blood Banking and Regulation: Procedures, Problems, and Alternatives Congressional Oversight of Blood Safety Issues Anne Marie Finley On behalf of Chairman Christopher Shays, I am pleased to have the opportunity to discuss with you the work of the Human Resources and Intergovernmental Relations Subcommittee. The subcommittee has oversight jurisdiction over the U.S. Department of Health and Human Services (DHHS) and its agencies, including the Food and Drug Administration (FDA), the Centers for Disease Control and Prevention (CDC), and the National Institutes of Health (NIH), for the House Government Reform and Oversight Committee. Our oversight responsibilities in the blood safety area are taken very seriously not only because of the loss of life in the HIV epidemic but also because of our oversight obligations with regard to government reform. THE WORK OF THE SUBCOMMITTEE In May of 1995 the subcommittee began a general oversight investigation into blood safety issues and FDA's management of the Blood Products Advisory Committee. This investigation resulted in a letter from Chairman Shays to FDA Commissioner David Kessler requesting that the agency not accept the June 23rd recommendation of the Blood Products Advisory Committee (BPAC) but, rather, that FDA license a short-incubation HIV monoclonal p24 antigen test for donor screening. The Chairman's concerns included prevention by up to 40 individuals per year from exposure to antigen-positive, antibody-negative blood products; continued incentives for the development of further screening tools to close the window period during which HIV infected persons test negative on the current antibody based test; and a determination ''that cost estimates should not outweigh scientific evidence of the effectiveness of antigen testing to save lives." The Chairman also objected to the agency's failure to approve or disapprove the product licensing applications (PLAs) for the short-duration antigen tests within the 180 days mandated by the Federal Food, Drug and Cosmetic Act.
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Blood Banking and Regulation: Procedures, Problems, and Alternatives PLAs were initially filed in 1990 and remain unapproved and pending at the agency. Furthermore, Chairman Shays asked Dr. Kessler to disband the Blood Products Advisory Committee immediately and to replace it with an advisory committee on the safety of the nation's blood supply. He felt that the critical mission of this advisory committee should be reflected in its title and that its mission should be clear. Consistent with HR 1021, legislation introduced by Representative Porter Goss, Chairman Shays stated that at least one third of the membership of the new advisory committee should be individuals who have received blood products or who are representatives of consumer organizations with expertise in blood products. The subcommittee was very pleased with FDA's August 10, 1995, announcement, including the recommendation that blood establishments should begin screening all blood and plasma donors with the antigen test kits within 3 months after FDA's approval of the initial test kit. On October 12, the Subcommittee will conduct an oversight hearing on DHHS's management of the risk of infectious agents in the blood supply. This will be a culmination of not only the antigen investigation but continuing work that we will be doing in blood. We expect testimony from DHHS officials, U.S. Public Health Service agency representatives, consumers, clinicians, and individuals from the regulated industries to address prospective problems in the regulatory scheme for blood and blood products. We do not expect to relitigate the incidents of the early 1980s, but we do anticipate testimony on the DHHS's response to the IOM recommendations. (Editor's Note: The IOM recommendations referred to by Ann Marie Finley are not those of the present IOM Forum, but of the IOM report HIV and the Blood Supply.) Additional issues that concern the subcommittee and that may come up at blood investigations and hearings include emerging infectious agents in the blood supply, the status of antigen test kit reviews, reconstitution of the Blood Products Advisory Committee, and hepatitis C virus transmission through blood and blood products. There is a strong possibility that the way in which we regulate blood and blood products will change not only at the agency and department level but possibly through congressional and public interest as well. Chairman Shays and the subcommittee share the commitment of the regulatory agencies, consumers, and the regulated industries to the safety of the blood supply and welcome efforts to develop responses to the recommendations of IOM so that the blood supply remains as free from infectious agents as strong leadership and good science can ensure.
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Blood Banking and Regulation: Procedures, Problems, and Alternatives THE QUESTION OF COSTS Question from the Audience: We have many health crises in this country and very limited resources to deal with them. When does cost become an issue? Anne Marie Finley: Under the current statute FDA has no authority to consider and make regulatory decisions based on cost effectiveness. Their only criteria are safety and efficacy. We were concerned that the antigen testing issue at BPAC was, in fact, decided on some other basis. Whether that resulted from improper instruction with regard to what the criteria were or whether the agency felt that it needed to address the concerns about costs, we cannot say, but we did not feel that it was appropriate for the agency to allow the decision to be based solely on an advisory committee process that we thought reached its result on the basis of cost. It is rare that the agency does not follow the recommendations of the advisory committee, which is the reason that the Chairman sent the letter, and we were pleased that the agency took the actions that it did. We want to be supportive publicly of that decision. We also think it is critical to work toward eventually closing the window. This is the goal that the FDA Commissioner himself stated at a November NIH conference. We must encourage companies to take incremental steps to get there. Clearly, the short-duration monoclonal antibody test was the first step that we needed to take in that direction. We, as a society, talk about controlling medical costs, but we have not come to the point where we are making global decisions. With respect to the immediate decision about antigen testing, the criteria were that AIDS is a fatal disease, that we were able to stop some of its transmission, that there was a commitment on the part of the commissioner and the agency toward closing the window, and that we needed to take incremental steps. There are definitely concerns with regard to the question of overall cost, because the statute does not address it and we have not yet reached the point in Congress, at least with the industries and the consumers approaching FDA, to say that one should consider changing the statute. It is a good question. I do not have a definitive answer, except to say that at the moment we have not addressed it as a society and, therefore, that the Congress has not addressed it. Thomas Zuck: I would take issue with the observation that we have not addressed the health care cost question. Maybe the Congress has not, but those of us who are suppliers to hospitals and to the managed care organizations have been pressed to offer discounts and reduced prices. At the same time we are being asked to follow drug manufacturing practices, rationing and the like. So, mandating a test that costs 50 million or 60 million dollars for very little quality-life-years in gain is marginal at best. It is a very expensive endeavor,
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Blood Banking and Regulation: Procedures, Problems, and Alternatives of nonspecific tests—replaced, that is, with new donors who are 13 to 16 times more dangerous than a repeat donor who has been screened multiple times. The cost is enormous, and the blood banks must absorb it. William Sherwood: The American Red Cross's revenue as a supplier of blood products to the health care system is rapidly becoming capped. When new interventions come about and costs go up, they come as unfunded mandates. As a result, our revenues are squeezed, and so, to comply with the unfunded mandate, we must stop doing something else. In the selection of these mandates one must be very careful about whether it is going to be worthwhile in the larger view. The Red Cross recently explained the new HIV antigen test to a group of hospital blood bank administrators. In the city of Philadelphia, with a usage of 350,000 units of blood per year, we will find one positive test result every 2.5 years. The hospitals' reaction was that they were not going to pay for that. As a result, we must fund the unfunded mandate. As a result, we will not do something else that we could have done. Question from the Audience: Your remarks focused on the Blood Products Advisory Committee and its decision-making process. Can you describe how an advisory committee ought to work? Anne Marie Finley: The Chairman recommended in his letter to Commissioner Kessler that BPAC be disbanded and that we start again with a national advisory committee on the safety of the blood supply. I personally believe, and here I am not speaking for Mr. Shays or for the committee, in greater representation; for example, a permanent voting member from CDC would be not a bad thing. We may want to raise the profile of BPAC beyond the level of just FDA. With regard to whether we want to make it a department level function, following through to some extent on the IOM recommendations, would be an option. We are interested in hearing the DHHS's response to those issues, recognizing that it has extensive technical expertise as well as a long history with BPAC. The Chairman is a cosponsor of legislation that would require one-third of the voting membership of BPAC to be consumers or consumer representatives. That bill contains criteria that define consumers. Another bill, introduced by Porter Goss, defines consumers as individuals who have received blood or blood products or who represent blood or blood product consumer organizations. This is important; it was missing in the early 1980s. I do not think that anyone can say definitively that it would have made a difference, but Congress perceives that as something that many people feel comfortable with. That,
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Blood Banking and Regulation: Procedures, Problems, and Alternatives again, goes to the consumer confidence issue. Perhaps it could be addressed by a greater voting membership on the part of the public.
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Blood Banking and Regulation: Procedures, Problems, and Alternatives DHHS Task Force on HIV and the Blood Supply Mary Pendergast When the Institute of Medicine (IOM) issued its report, HIV and the Blood Supply on July 13, 1995, the Secretary of the U.S. Department of Health and Human Services (DHHS) accepted it and stated that she agreed with the principles stated in the report and that details were to follow. She then asked Phil Lee, the Assistant Secretary for Health; David Satcher, the head of the Centers for Disease Control and Prevention (CDC); Harold Varmus, the Director of the National Institutes of Health (NIH); and David Kessler, the Commissioner of the Food and Drug Administration (FDA), as well as each of their deputies (Claire Broom at CDC, Ruth Kirchstein at NIH, and me at FDA), to convene to study the IOM report, to think about what it was saying, to take a hard look at what had happened between the time when the IOM report left off and today, and then to think about what, if anything, needed to be done in addition to or differently from what we had been doing in the past. THE TASK FORCE CHARGE Our charge is to respond to each of the recommendations made by IOM that might have moderated some of the effects of the AIDS epidemic. Because IOM had also urged the government and private organizations responsible for blood safety to evaluate their current policies and procedures to see if they fully addressed the issues raised by the recommendations, we are doing that as well. As of the date of the Forum meeting, the task force had not completed its work. We have not yet prepared a report to the Secretary, although because the Secretary will soon testify before Congressman Shays on these very issues, I predict that we will finish the report sometime before she testifies. We have spent quite a bit of time looking at the existing blood safety system. It comes as no surprise to any of you that things have changed in the 11 years since IOM saga left off. We are also looking at these very significant
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Blood Banking and Regulation: Procedures, Problems, and Alternatives changes. We will be meeting with roughly 30 entities, including blood organizations, blood-related organizations, and persons or organizations interested in blood safety issues. These discussions will be part of our deliberations. At some point we will then make our recommendations to the Secretary, who will present them in testimony to Congressman Shays. BLOOD PRODUCTS ADVISORY COMMITTEE In the meantime, we have changed the composition of the Blood Products Advisory Committee (BPAC) somewhat, although it is important to recognize that we changed the composition of BPAC in response not to the IOM report but actually to a much earlier one. We had asked IOM to help us some years ago on our advisory committees, because one of the things David Kessler and I have been trying to do is make the agency more of a unitary organization. We have different product centers that sometimes operate with different plans, projects, and attitudes. They run their advisory committees very differently, so that we were not presenting a coherent picture to the outside world. It was causing some confusion. Therefore, working with IOM, we addressed our advisory committee structure, and one of the things that we realized is that BPAC is constituted in a way that was very different from the way that the remainder of our advisory committees were structured. We began then to think about how we could bring BPAC into line with the rest of our advisory committees. It is seen as being reactive to IOM, but the goal actually was to make sure that there is more of a consumer voice on all of our advisory committees, to figure out ways to get the other federal agencies at the table, and to recognize that it is very hard to ask the people who are being regulated to make decisions that affect their economic interests. We had to come up with a new paradigm, and we have been striving to do that. We are also trying to find other ways to develop a broad public conversation about the tough issues of the day. It is interesting to note how opinions about these things go in cycles. FDA used to have CDC and NIH on its advisory committee, until we were criticized as being too government focused and insular. Now we hear the call to put them back on again. We value their input. We agree that they must be at the table. We have been trying to figure out different ways to get CDC and NIH at the table. We do not want to be acting inconsistently, nor do we want to be acting alone. We need their advice and we need their consultation, but it is very challenging. Every agency has a full agenda; it is a great demand to ask them to take on not just their issues but ours as well.
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Blood Banking and Regulation: Procedures, Problems, and Alternatives DECISION-MAKING Personally, I believe that it is critically important that we continue to talk about the issues, that we get all of the different points of view, recognizing that there is no such thing as a right or a wrong point of view, and that we at FDA know that many different vectors influence what we do. We must be very careful that we are not the "thought police," that we do not overvalue ideological purity, and that we have public forums and opportunities where people can say what they need to say in an open and forthright manner. FDA has a tough job. The head of the FDA blood program must make specific decisions on a case-by-case basis when society does not have an agreed-upon paradigm for how to make those decisions. Therefore, the case-by-case decision-making drives the public policy, although ideally it should be the other way around. Blood is not alone in this. It is not uncommon for the case-by-case decision-making to outstrip the public policy, but we do not have a common set of values that is fully accepted by everyone. As a consequence, no one decision is perceived as being the right decision. As somebody said, "Which way are you going? Do you want more regulation or do you want less? Do you want the heavy hand of FDA to be greater or less? Do you want more safety or fewer costs?" I think that as a society we do not know what we want. COST AS A FACTOR With respect to whether cost is a factor, I do not think that it is, but it is also naive to think that absolute safety can be a reality. No product that FDA regulates, whether it is milk or human genome products, biotechnology, medical devices, or old-fashioned chemical drugs, is absolutely safe. If we strive toward absolute safety, we will drive the products off the market. We must accept that fact. The fact is, blood is not and is never going to be perfectly safe. It is as safe as we can get it, perhaps. It is as safe as blood any place in the world, but it is not absolutely safe. We are kidding ourselves, and we are kidding the public whenever we say that. Therefore, given that fact, what do you do about it? Where do you draw the line? For every single product and every single therapeutic about which FDA makes a decision, we draw that line. In the summer if 1995 we had a situation in which a drug that prevents epileptic seizures in some people who are refractory to any other drug also gave 10 percent of them aplastic anemia. It is not safe, but it really makes a difference in some people's lives. Do we approve it or not? It is not safe, but it provides therapy, and we approved it. We approve things that are not safe every day of the week, and we always will; blood is not going to be any different. We must think of a new way to
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Blood Banking and Regulation: Procedures, Problems, and Alternatives talk about blood, a way that is not kidding ourselves and kidding consumers. It is a risk-versus-benefit criterion, not a cost criterion. If you apply absolute safety, you are going to apply unlimited costs, and if you apply unlimited costs, then the heavy hand of FDA will make these products disappear. I do not know anyone who wants to see the United States without a blood supply, although that, in fact, is where we are headed if absolute safety is the paradigm that we are going to choose. THE PUBLIC VIEW Question from the Audience: The language of safety is somewhat misleading. Everyone knows that when we talk about automobile safety we are not completely safe. I am not sure we are as well informed about blood. Mary Pendergast: For some FDA products the standard of safety is higher. For pesticides for foods that kids eat, the U.S. consumers want absolute safety. As for blood, U.S. consumers want absolute safety. We as regulators and you as industry and academics have helped to perpetuate that. It may be time to change the debate. There has never been an FDA official who has stood on Capitol Hill and said, ''This drug is safe." Nobody ever says that. They always say that the benefits of this drug outweigh the risks. I know that the statute says "safe and effective," but you can only think of safety relatively. We are able to make the debate in some areas, but we have not made it in all areas. Question from the Audience: You mentioned both the value of diverse opinions being expressed and an interest in continuing the dialogue. In what venues should that dialogue occur other than through the new BPAC and the coalition? Mary Pendergast: It is fair to say that this has been our most fully debated issue. It is the nub of the question and is the toughest thing that we must deal with. I cannot yet tell you what we are going to do. Any suggestions are welcome.
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