1
Introduction

The Institute of Medicine (IOM) was asked by the National Institutes of Health (NIH) ''to conduct a comprehensive study of the policies and processes used by NIH to determine funding allocations for biomedical research" in accordance with a congressional provision (U.S. Congress, 1997a). The U.S. House and U.S. Senate authorization and appropriations committees requested that IOM present findings, conclusions, and recommendations "for improvements in the NIH research funding policies and processes and for any necessary congressional action." Specifically, the congressional committees asked that IOM "assess:

  • the factors or criteria used by NIH to determine funding allocations for disease research,
  • the process by which research funding decisions are made,
  • the mechanisms for public input into the priority-setting process, and
  • the impact of statutory directives on research funding decisions."

IOM was asked to conduct the study and submit a report in time to inform congressional consideration of reauthorization legislation in 1998 as well as of fiscal year (FY) 1999 appropriations (that is, during the second session of the 105th Congress). To comply with this request, IOM agreed to deliver the report by July 1, 1998, 5 months after the study began. The committee process is described below, after the following review of the background of issues concerning research priority setting at NIH.



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--> 1 Introduction The Institute of Medicine (IOM) was asked by the National Institutes of Health (NIH) ''to conduct a comprehensive study of the policies and processes used by NIH to determine funding allocations for biomedical research" in accordance with a congressional provision (U.S. Congress, 1997a). The U.S. House and U.S. Senate authorization and appropriations committees requested that IOM present findings, conclusions, and recommendations "for improvements in the NIH research funding policies and processes and for any necessary congressional action." Specifically, the congressional committees asked that IOM "assess: the factors or criteria used by NIH to determine funding allocations for disease research, the process by which research funding decisions are made, the mechanisms for public input into the priority-setting process, and the impact of statutory directives on research funding decisions." IOM was asked to conduct the study and submit a report in time to inform congressional consideration of reauthorization legislation in 1998 as well as of fiscal year (FY) 1999 appropriations (that is, during the second session of the 105th Congress). To comply with this request, IOM agreed to deliver the report by July 1, 1998, 5 months after the study began. The committee process is described below, after the following review of the background of issues concerning research priority setting at NIH.

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--> BACKGROUND NIH is located in the U.S. Department of Health and Human Services (DHHS) with other health-related agencies of the federal government, including the Agency for Health Care Policy and Research, Centers for Disease Control and Prevention, Food and Drug Administration, Health Care Financing Administration, and Health Resources and Services Administration. NIH is the primary agency for research on health, accounting for 94 percent of DHHS's budget for health research and development (calculated from data from the National Science Foundation [NSF; 1997a:Table 8]). NIH, which traces its roots to 1887, has grown steadily in size and scope, especially since the end of World War II, when the National Cancer Institute (NCI) was the only separately organized entity and the budget was $3 million. Currently, in FY 1998, there are 21 institutes and centers addressing different aspects of health research and several other major organizational units, and the budget for all NIH institutes and centers (see Box 1-1) is $13.6 billion. BOX 1-1 Institutes and Centers of the National Institutes of Health John E. Fogarty International Center National Cancer Institute National Center for Research Resources National Eye Institute National Heart, Lung, and Blood Institute National Human Genome Research Institute National Institute of Allergy and Infectious Diseases National Institute of Arthritis and Musculoskeletal and Skin Diseases National Institute of Child Health and Human Development National Institute of Dental Research National Institute of Diabetes and Digestive and Kidney Diseases National Institute of Environmental Health Sciences National Institute of General Medical Sciences National Institute of Mental Health National Institute of Neurological Disorders and Stroke National Institute of Nursing Research National Institute on Aging National Institute on Alcohol Abuse and Alcoholism National Institute on Deafness and Other Communication Disorders National Institute on Drug Abuse National Library of Medicine According to the NIH Manual (National Institutes of Health, 1994b), "The National Institutes of Health is the steward of biomedical and behavioral re-

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--> search for the nation. Its mission is science in pursuit of fundamental knowledge about the nature and behavior of living systems and the application of that knowledge to extend healthy life and reduce the burdens of illness and disability." As put more succinctly on its web page, the mission of NIH is "to uncover new knowledge that will lead to better health for everyone" (National Institutes of Health, 1998b). NIH carries out its mission by supporting research of many kinds, funding the training of new researchers, and fostering communication of research results to health care professionals and the public. How it allocates its resources among and within these activities—that is, how it sets priorities—is of great importance to the public, the media, organized disease-specific interest groups, health care providers, and researchers themselves because of widespread and deeply held interest in the impact of NIH on health and disease and on the productivity of the nation's health research enterprise (see Box 1-2 for a list of NIH constituencies). There are many ways to view the allocation of NIH's budget, reflecting its diverse portfolio of activities. Each cut or dimension emphasizes different aspects of NIH and priority setting. BOX 1-2 NIH Constituencies NIH interacts with various external constituencies who have a stake in research priority setting. These include: research scientists in universities, colleges, medical centers, and other research institutions outside NIH who conduct most of the research funded by NIH; clinicians who apply research results and who can help identify research needs (physicians, including specialized physicians, nurses, dentists, pharmacists, social workers, psychologists, public health practitioners, and other allied health practitioners) organized voluntary groups and individuals active in advocating for those with specific diseases or medical conditions; organizations and individuals who represent population groups with special health problems (members of particular ethnic groups, low-income populations, women, elderly people, children, etc.); Congress, which provides NIH with the authority and funding to carry out its mission, which oversees s effectiveness, and with which NIH must maintain good communication about priorities; and media (reporters, editors, and others) who communicate research results and NIH activities and who thus play an important role in helping the public understand the research enterprise.

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--> Although NIH is the largest single funder of health research in the United States, it is part of a much larger research enterprise in which industry—such as pharmaceutical companies and biotechnology firms—spends more (about $19 billion and $8 billion, respectively) on research (Pharmaceutical Research and Manufacturers of America, 1997; Biotechnology Industry Organization, 1998). Within this larger system, NIH supports the basic research to gain fundamental knowledge about health and disease processes that the private sector will not support because it may not be profitable, while companies focus their research on applications, such as new drugs, diagnostic tools, and medical devices that cure, detect, or prevent diseases. Funding Allocation by Institute and Center The categorical research institutes and centers (hereinafter referred to as "institutes"), which have been established one by one over the years, have a variety of foci. Some are organized by disease or organ (e.g., cancer; alcoholism; deafness; heart, lung, and blood; eye; and dental). Some are based on stages of human development (e.g., aging and child health and human development). Some are organized by field of science and medicine (e.g., general medical sciences, environmental health, nursing, and human genome research). Other entities include the National Library of Medicine, John E. Fogarty International Center, and National Center for Research Resources. All of these institutes and centers report to the Office of the Director (see Figure 1-1). There is a natural focus on the allocation of funding among the institutes and centers because each receives its own appropriation from Congress (the Office of the Director, Office of AIDS Research, and buildings and facilities accounts also receive separate appropriations; see Table C-1 in Appendix C).1 The institutes and centers are the main organizational units of NIH, with only the Office of the Director of NIH being above them, providing overall leadership and direction and cross-institute coordination.2 1   The Division of Research Grants (recently renamed the Center for Scientific Review), the Division of Computer Research and Technology (recently renamed the Center for Information Technology), and the Warren Grant Magnuson Clinical Center are funded through the NIH Management Fund, which is financed by "taps" on the institute and center appropriations. 2   Even the Office of AIDS Research, although located in the Office of the Director of NIH, operates in many respects as an "institute without walls" with a director, national advisory council with scientific and public members (Office of AIDS Research Advisory Committee), an executive committee of senior program officials, and an executive office for staff support (Office of AIDS Research) (Institute of Medicine, 1991:42).

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--> FIGURE 1-1 Organization of NIH. SOURCE: National Institutes of Health (1997d:13).

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--> Each institute supports research, training, and information programs in its area. After research proposals from the nation's biomedical and behavioral scientists and engineers are peer reviewed for scientific merit, they are assigned to an institute for funding and administration. Most institutes and centers also have intramural programs. Each institute is headed by a director who has strong research and leadership skills in the areas addressed by his or her institute. Each has a national advisory council with members representing the scientific community and the public to advise the institute director on policies and programs and to review and approve research grant applications. Institute directors are also advised on the intramural program by a Board of Scientific Counselors, consisting of nongovernment scientists with the appropriate expertise. The institutes are organized internally by research area, and the program heads also advise the directors on research priorities; in turn, they typically have standing and ad hoc advisory committees of nongovernment experts and, sometimes, public representatives (the standing advisory committees are listed by institute in Appendix A). Funding Allocation by Mechanism In its budget documents and presentations to Congress, after a table showing funding by institute, NIH usually presents a table or figure indicating funding by mechanism (see Table C-2 in Appendix C).3 It indicates what share of NIH's budget goes to different mechanisms of support—for example, research project grants (RPGs), research and development (R&D) contracts, intramural research, and so forth. In FY 1998, for example, NIH expects to spend 56.7 percent ($7.7 billion) of its total budget on nearly 29,000 RPGs. RPGs are scientific studies or experiments proposed by researchers based in more than 1,700 universities, medical schools, and other research institutions in the United States and abroad.4 About 8.8 percent of NIH's budget will go to support interdisciplinary research centers focused on a particular disease or other aspect of health, and 4.7 percent will go to support other types of research grants. R&D contracts will account for about 6.4 percent of NIH's budget, and intramural research at NIH itself will account for 10.4 percent of NIH's budget. Training of researchers will account for 3.1 percent of the NIH budget. The remainder will support program management and administration in the institutes, including the scientists and engineers who review research proposals and administer the extramural 3   See, for example, the overview section of NIH's budget request to Congress (U.S. Department of Health and Human Services, 1998:62) or NIH's press release for its FY 1999 budget request (National Institutes of Health, 1998c). 4   Almost two-thirds of RPGs are R01s, which support a single project proposed by a single principal scientist, NIH's most traditional form of support.

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--> grant programs (3.6 percent), the Office of the Director of NIH (1.8 percent), the National Library of Medicine (1.2 percent), cancer control activities funded by NCI (1.9 percent), and construction of research facilities (1.7 percent). NIH leaders monitor the allocation of funding among mechanisms because the levels of funding for different mechanisms of support help NIH to discern patterns in the nature of scientific work as well as detect changes in the balance between investigator-initiated and NIH-initiated work. As NIH's press release for its FY 1999 budget request put it, "NIH's highest priority is the funding of basic biomedical research through research project grants. The emphasis on peer-reviewed and competitively awarded RPGs allows NIH to sustain the scientific momentum of investigator-initiated research while providing new research opportunities" (National Institutes of Health, 1998a:7). NIH leaders focus particular attention on the number of new grants that can be awarded each year, because new grants constitute the part of NIH's budget that can be used most flexibly to address shifts in priorities, such as promising opportunities for new scientific advances, emerging health problems, or public health emergencies. Grant awards average 4 years. This multiyear funding is essential to the planning and conduct of successful investigations. It means that most of the annual RPG budget goes to continuing existing grants, which NIH calls its commitment base. In FY 1998, for example, more than 75 percent of the funding for RPGs is already committed and not quite 25 percent ($1.9 billion) is available for new projects.5 Other mechanisms, such as grants for research centers, clinical trials, and R&D contracts, tend to be more directed in nature and more tied to an institute's mission. They are usually solicited by NIH through requests for applications (RFAs) rather than initiated by extramural scientists, are reviewed by peer-review committees in the institutes rather than in the Center for Scientific Review, and are often funded as cooperative agreements in which NIH officials have more of a say in project direction than they do with RPGs. They also tend to be used more in problem-oriented research efforts, such as disease-specific programs, especially in their beginning stages (for example, in the early years of the War on Cancer and of research on AIDS and Alzheimer's disease). Because of their directedness, such mechanisms tend to be specified by Congress in legislation or report language when Congress concludes that NIH should move more quickly to attack a particular disease or other problem. If NIH's budget is increasing slowly, such mandates or set-asides for research centers, RFAs, or other specific mechanisms could impinge on the share of funding for investigator-initiated research projects. 5   Some agencies that may fund multiyear grants (e.g., NSF) fully fund each grant up front rather than fund each grant incrementally year by year. Thus, 100 percent of each year's grant budget is potentially available for new priorities rather than the continuation of existing lines of research.

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--> Funding Allocation by Type of Research NIH also classifies its funding according to whether it is basic research, applied research, or development work, using definitions supplied by the federal Office of Management and Budget (OMB) for government-wide reporting purposes. Basic research is defined as "systematic study directed toward greater knowledge or understanding of the fundamental aspects of phenomena and of observable facts without specific applications towards processes or products in mind" (Office of Management and Budget, 1997). Applied research is "systematic study to gain knowledge or understanding necessary for determining the means by which a recognized and specific need may be met." Development is the "systematic application of knowledge toward the production of useful material, devices, and systems or methods, including design, development, and improvement of prototypes and new processes to meet specific requirements.'' In FY 1998, excluding training, administration, and facilities, NIH expects about 57 percent of its budget to go to the support of basic research, 31 percent to applied research, and 12 percent to development (see Table C-3 in Appendix C).6 Taken literally, these percentages mean that the majority of NIH funding is not immediately diseasespecific, because the research is so fundamental that the investigators do not know for certain which diseases their results might affect. No matter how esoteric their research may be, however, most if not all scientists are motivated by the fact that extending the frontiers of knowledge about basic biological and behavioral processes has greatly increased the current capacity to diagnose, treat, and prevent illnesses and enhance health and that it is virtually certain to do so in the future (although how or when cannot be predicted). One should not take these percentages to be very precise, because in practice it is often difficult to classify research as basic or applied. For one thing, it involves determining whether the researcher has an application in mind. Even as rough estimates, however, the data indicate that, like other mission agencies of the federal government, NIH supports a substantial amount of basic research on the grounds that a growing science base will undoubtedly lead to better applications in the future.7 History has proven this to be so. As noted in the previous section, however, NIH generally prefers to monitor the balance between research projects proposed by scientists and those proposed by NIH, as indicated by the mechanism of support. The rationale is that, on average, scientists working in laboratories across the country will have better ideas than NIH officials about where progress might be made, whether in basic or applied research. 6   Calculated from information provided by NIH's Office of Financial Management. 7   The percentages of R&D funding expected to go to basic research at other agencies in FY 1997 were 92 percent at NSF, 35 percent at the U.S. Department of Energy, 20 percent at the National Aeronautics and Space Administration, and 3 percent at the U.S. Department of Defense (National Science Foundation, 1997b:Table C-7).

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--> Funding Allocation by Disease NIH also keeps track of the annual amount of funding related to specific diseases and other particular purposes (see Table C-4 in Appendix C). These data have been kept for many years to enable NIH to respond to requests from Congress or others about how much it spends on "programs of interest." Most of the items are disease related, such as research on cancer, heart disease, stroke, AIDS, Alzheimer's disease, Parkinson's disease, diabetes, hepatitis C, and tuberculosis. Some focus on other categories of research, such as gene therapy, health and behavioral research, immunology, nutrition, prevention, rehabilitation, and vaccine development. Some concern research related to particular groups, such as research related to aging, pediatrics, the health of the rural populations, and women's health. NIH tracks about 250 programs of interest and reports annually on about 50 of these. Some of the items included in the 50 change from year to year, depending on information requests. For example, in the FY 1999 appropriations hearings, NIH was asked about funding for research on a half-dozen programs (e.g., autism, food-borne illness, obesity, uterine and ovarian cancer, behavioral research, and international activities), and they will likely be included in the list next year. The data are reported by each institute to NIH's Office of Financial Management, which means that it is possible to track funding by institute (see Tables C-5 and C-6 in Appendix C for funding for research on cancer and diabetes by institute, respectively). In some cases, institute-by-institute tables are reported regularly by the lead organization for a particular disease (e.g., the Office of AIDS Research and AIDS research or the National Institute on Aging and Alzheimer's disease). In each institute, scientist-administrators review each grant and contract award and decide whether it is related to one or more of the programs of interest. In the case of a disease, they include more than just those awards that mention a disease in the title or in a list of key terms for inclusion in the computerized database system containing information on research projects and programs supported by NIH and other DHHS agencies. They also include basic research that is related because it is studying a phenomenon implicated in the disease. For example, research aimed at trying to gain a better understanding of the basic processes of neuronal death might be considered related to Alzheimer's disease. It would also probably be considered related to Parkinson's disease and some other neurodegenerative diseases marked by the progressive loss of neurons. At the same time, much basic research is too fundamental to be considered related to any specific disease. This means that some research is counted twice while other research is not counted at all in the data on funding by disease, even though the latter kind of research is helping to build the science base that will lead to the disease-specific advances of the future (see Box 1-3).

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--> BOX 1-3 Basic Research Crosses Disease Boundaries When establishing research priorities it is important to keep in mind that therapeutic advances frequently emerge from basic research that crosses disease boundaries. For example, much of the progress in AIDS treatment—including the blood test for AIDS and the protease inhibitors used to treat the disease—can be traced to the fundamental work of NIH-supported scientists who were researching cancer. In the 1960s and 1970s these scientists discovered certain viruses called retroviruses that cause cancer in chickens and mice. They learned an enormous amount about the biology of these viruses. Once it was discovered that the AIDS virus resembles a retrovirus, two decades of knowledge in the effort to study cancer could be applied to the understanding of AIDS. Building upon this base of knowledge derived from NIH-supported basic research in cancer. three companies simultaneously developed protease inhibitors for the treatment of AIDS. Another crossover occurred between heart disease and cancer research. University scientists, supported by the National Heart, Lung, and Blood Institute. learned the fundamental mechanism for the regulation of cholesterol levels in blood. This allowed companies to develop drugs called statins that lower cholesterol levels and prevent heart attacks. Basic biologists, working on cancer, used the statins to discover a new property of cancer-causing proteins called Ras proteins. Pharmaceutical companies seized upon this information, and in 1998 they began clinical trials with new drugs that inactivate Ras proteins and thus may be useful in the treatment of cancer. All of this progress in cancer treatment is directly traceable to the discovery of statins through NIH's program of basic research in heart disease. In the table of spending by disease in its budget submission to Congress and on its web page, NIH does not provide population-based information such as the number of people who are afflicted or who die annually or cost-of-illness estimates. The institutes with lead responsibility for particular diseases sometimes collect and publish information on burden of disease and economic costs, but they use different databases and methodologies, which limit the comparability of their results. Despite limitations, these data are used by health groups and advocates to help assess how much effort NIH is putting into research on particular diseases, that is, as a measure of NIH priority setting. Disease-specific interest groups usually report on the rate of increase in funding for research on the disease in which they are interested and compare it with those for research on other diseases, and they may compare spending per afflicted person to argue that NIH is neglecting the disease in which they are interested. Some members of Congress have also used the data to judge whether the allocation of NIH funding matches

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--> the burdens and costs of diseases in the population. These issues are discussed in Chapter 2, where there is further analysis of the reporting system and recommendations for improving it. RECENT TRENDS AND ISSUES NIH is the leading federal agency supporting research related to improving the nation's health. By various key measures it has been very successful in fulfilling its mission. The scientists and clinicians whom it has helped train and whose research careers it has supported have consistently been in the forefront of research discoveries that have advanced fundamental knowledge of human biology and of better ways to treat or prevent disease and promote good health. Over the past 50 years NIH as an institution has played a substantial role in contributing to the explosion of knowledge that has amounted to a revolution in biology. One indicator of NIH's contributions to science is the number of Nobel Prize-winning scientists and physicians whom it has supported. Over the years NIH has funded 93 Nobel Prize winners for work ranging from deciphering the genetic code to determining the cause of diseases to developing better techniques to diagnose, treat, or prevent diseases. There are many examples of how NIH-supported work has contributed to better medical care and health. They include the decline in the rate of mortality from heart disease, the leading cause of death in the United States, by nearly 50 percent over the past 20 years. Death rates from stroke decreased by 50 percent during the same period. Dental sealants have sharply reduced the number of cavities in children. Despite tremendous progress, however, there is still a long way to go in conquering disease. At the same time, the benefits of past research have not reached everyone, as indicated by significant differentials in disease rates and outcomes among members of different socioeconomic and ethnic groups. Demographic trends will have an impact; for example, the aging of the population will substantially increase the individual and social burdens of certain diseases and conditions. Also, about half the nation's health care costs result from unhealthy behaviors and environments, which pose major research challenges (McGinnis and Foege, 1993). NIH's success has earned it steady budget increases despite a tight overall federal budget in recent years. The NIH budget is $13.6 billion in FY 1998, and the administration has proposed an increase of 8.4 percent to $14.7 billion in FY 1999. Some members of Congress have talked about a larger increase. Despite the favorable budget trend (NIH's budget has increased by 80 percent since 1990, compared with an increase of 48 percent in the rest of the nondefense discretionary budget), concern about research priority setting at NIH has grown in recent years. For example, since 1995 there have been at least eight instances in which members of Congress or congressional committees have asked about

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--> priority setting in some aspect of NIH's research program, not including the one leading to this report.8 Concerns about priority setting in the allocation of NIH research funding come from several sources. First, some members of Congress believe that there should be more of a correlation between the allocation of funding by disease and the distribution of disease burdens and costs in the population. They point to widely different amounts of research funding per afflicted person from one disease to another and ask why NIH is spending much more per death from HIV/AIDS infections compared with spending per death from cancer, from heart disease, and from stroke. They also note that the largest amounts of NIH funding do not always go toward research on diseases that cost the federal government the most through the Medicare program (see, for example, the charts and discussion in U.S. Congress [1997a:36-42] and Agnew [1996]). Second, more and more disease-specific interest groups have begun campaigning for increases in NIH funding for research related to the particular diseases in which they are interested. They are urging Congress to set aside specific amounts by disease or to boost the funding for the institute that supports most of the research on their disease of interest relative to that for other institutes. They point to the amount of funding related to their disease of interest, compare it with the larger amounts being devoted to other diseases, and question the priority-setting process that led to that result. Traditionally, disease-specific interest groups have avoided open competition with each other for NIH funding. Rather, they supported increases generally for NIH, expecting to benefit from the overall increase, perhaps lobbying quietly to win a larger than average increase for their program or institute. The success of the direct approach for some groups, however, has put pressure on all groups to make more specific demands and to compete openly with other groups for more resources. Third, the leadership of the health committees in Congress has become increasingly uncomfortable with intervening in research priority setting at NIH, for example, by mandating specific funding set-asides, new programs or institutions focused on a specific disease, or the use of particular research mechanisms or by trying to push research advances in specific areas in other ways. In part they may be reacting to feedback about the unintended effects of disease-specific earmarking in the late 1980s and early 1990s that added up to more than the overall increases and thus cut into the funding for other programs (Congress has always included earmarks in report language or less formally, but until recently, they rarely added up to a substantial share of the total budget increase that NIH received in any given year and so did not affect other research areas directly). They are also facing a large and growing set of groups pushing for earmarks, making it even harder to satisfy such demands within the overall 8   From a list provided by the NIH Office of Legislative Policy and Analysis.

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--> increases. NIH leaders and the scientific community also began to object strongly to overly detailed prescriptions about priority setting and funding allocations from Congress. The director of NIH and the institute directors have testified on a number of occasions that congressional directives "distort" the priority-setting process because they do not allow NIH to adjust for the different probabilities of scientific progress against each health problem and because they do not properly weigh the need for basic research for progress in the long run (the director and several institute directors made this point to the IOM committee at the committee's first meeting on March 6, 1998) (see also Varmus [1997] and the testimony of Harold Varmus, director of NIH [U.S. House of Representatives, 1997a; U.S. Senate, 1997a]). In the markup of the Senate reauthorization bill, the NIH Revitalization Act of 1996, members of the authorization committee began to debate whether Congress should be directive by authorizing more set-asides for specific diseases or should let NIH determine the direction for research funding. They were considering a number of amendments that authorized specific funding amounts and mandated certain mechanisms for particular diseases (U.S. Senate, 1996:10; also see the section on the 1996 Senate Reauthorization Bill in Chapter 5). The 1996 reauthorization effort had many members of the Senate authorization subcommittee saying that they "should not micromanage biomedical research by establishing legislative mandates for specific areas" (U.S. Senate, 1997a: 10). One result was that the subcommittee held an important hearing in May 1997 on research priority setting at NIH (U.S. Senate, 1997a).9 The subcommittee heard testimony from the director of NIH and representatives of the scientific community in universities and industry who strongly encouraged Congress to resist pressures to become involved in how much NIH should spend on research targeted to specific diseases (see, for example, testimony by John W. Suttie, president of the Federation of American Societies for Experimental Biology [U.S. Senate, 1997a:59-60]). Representatives of voluntary organizations promoting health research testified that one reason that disease-specific interest groups appeal directly to Congress for earmarks or set-asides is the perceived lack of opportunities to be consulted in the planning and priority-setting process at NIH (see testimony by Myrl Weinberg, president, National Health Council [U.S. Senate, 1997a:63-64]). Questions about the desirability of congressional micromanagement were also being raised on the House side. In an oversight hearing held by the House authorization subcommittee in September 1997, Congressman Greg Ganske discussed the issue (U.S. House of Representatives, 1997b:4): I do have significant concerns about whether Congress has played politics with the NIH budget and micromanaged difficult scientific questions.... While 9   Two more Senate hearings were held on specific issues (see U.S. Senate, 1997b,c).

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--> promoting an increase in funding for one disease or another, I am concerned that these lobbying efforts are turning the floor of Congress into a scientific peer review panel. We are being asked whether to spend additional millions on breast cancer or diabetes or Parkinson's, and the list goes on and on.... I have concerns about whether members of Congress have the scientific expertise to determine where the most promising areas of research are. By September 1997, the campaign by groups advocating for increased research on Parkinson's disease succeeded when the Senate voted to include the Udall Bill authorizing a $100 million program consisting of research centers, training grants, a patient information center, and other mechanisms in the FY 1998 appropriations bill. Advocates had argued that NIH had neglected Parkinson's disease research in terms of the burden and cost of the disease and scientific opportunities and that Congress should take active steps to correct the disparity (see, for example, funding comparison charts and written testimony by Joan I. Samuelson, president, Parkinson's Action Network [U.S. Senate, 1997a:76-80]). In speeches on the Senate floor when the amendment for the Udall Bill was being debated, leaders of the authorization committee expressed concern over the process by which Congress mandated programs piecemeal in response to organized lobbying efforts. They proposed that IOM conduct an independent study of NIH's priority-setting process that would recommend any changes needed so that Congress could rely on NIH to make the allocation decisions (see speeches by Senators Dan Coats and Bill Frist [U.S. Congress, 1997b:S8714-S8716]). That proposal led to the present study and asked IOM to pay particular attention to two features: NIH's mechanisms for public input and the role of Congress in directing the allocation of funding among areas of research. IOM COMMITTEE PROCESS The IOM committee proceeded by assessing NIH's priority-setting process in light of the agency's mission and objectives and the changing policy environment. The committee addressed the following questions in the charge: Are the criteria adequate? Is the process for implementing them working? Given the objective of responding to health needs as well as scientific opportunity, in conjunction with the expansion of organized disease-specific interest groups, are the mechanisms for public input adequate or can they be changed to increase the complementarity between NIH's goals of responding to health needs and scientific opportunity? Can Congress, the holder of the public purse strings, be assured that NIH has a good process for priority setting in which the full range of considerations is taken into account in planning programs and allocating funding? In an effort to be as comprehensive as possible in the short time allowed, the committee pursued several mechanisms for collecting data and receiving

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--> input. At the committee's request, NIH provided a large amount of data regarding its research programs and budgetary and priority-setting processes. These data included organizational information, funding histories, and descriptions of the priority-setting processes in each institute at NIH. The committee also held organized panel discussions with the NIH director and deputy director, the directors of institutes and offices at NIH, and congressional staff. In addition, the committee solicited and received public comment through presentations and panel discussions at a public meeting, via a questionnaire mailed to more than 1,000 NIH constituency groups, and through the committee's web site. The committee felt that it was important to receive as much input as possible from public groups involved with or seeking involvement in NIH's research priority-setting process. To do so, the committee convened a public meeting on April 3, 1998, to gather information and hear from groups or individuals, especially those identified by the NIH institutes as being part of their constituencies. The committee made every effort to include as many groups as possible, given the short time available. Twenty-nine patient-advocacy groups and professional societies appeared before the committee (see Appendix B) in panels and an open comment session. Although each participant had a unique perspective, several themes became evident: Many presenters felt that research funding should reflect, among other factors, the burden of disease in society. The difficulty of this approach becomes apparent, however, when considering rare diseases. Although NIH indicates that it takes disease burden into account, several panelists felt that the criteria that NIH uses to set research priorities are unclear or ambiguous. Many advocacy groups are familiar with NIH and its organizational structure, but for issues that cross institute lines or for which there is no precedent, groups can find themselves at a loss about where to turn. There is a need for greater patient or public representation on NIH advisory bodies. There is a need for better communication between NIH and constituency groups. The committee received written comments from 56 individuals and organizations (including those from panelists at the committee's public meeting [see Appendix B]). These comments came from a spectrum of individuals involved with NIH—from researchers and advisers to patient-advocates and patients themselves—on issues ranging from education and training to disease-specific allocations of funding for research. The comments addressed many different topics or elements of the priority-setting process. Many called for more research funding allocations for specific diseases; others requested added emphases on certain types of research, such as environmental, interdisciplinary, clinical, or

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--> basic research. Still others commented that Congress should play a different role in priority setting, including some who believed that it should have greater involvement and some who believed that it should have less involvement. Many commentators noted the importance of increased inclusiveness in the identification of health research needs and highlighted the importance of open communication. ORGANIZATION OF THE REPORT The remainder of this report is organized into sections that address issues related to criteria for priority setting (Chapter 2), priority-setting processes (Chapter 3), mechanisms for public input (Chapter 4), and the role of the U.S. Congress (Chapter 5). Several appendixes (A to D) are included, as follows: Appendix A, Federal Advisory Committees of NIH; Appendix B, Acknowledgments; Appendix C, NIH funding tables; and Appendix D, committee and staff biographies.