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First printing, March 1991 Second printing, November 1991
Committee to Study the AIDS Research Program of the National Institutes of Health
WILLIAM H. DANFORTH (Chair), * Chancellor,
Washington University, St. Louis, Missouri
LINDA H. AIKEN, * Trustee Professor of Nursing and Professor of Sociology,
University of Pennsylvania, Philadelphia
MARSHALL H. BECKER, * Associate Dean, School of Public Health, and Professor,
Department of Behavior and Health Education, The University of Michigan, Ann Arbor
VICTORIA A. CARGILL, Assistant Professor of Medicine,
University Hospitals of Cleveland, Case Western Reserve University, Cleveland, Ohio
JOHN M. COFFIN, Professor,
Department of Molecular Biology, Tufts University School of Medicine, Boston, Massachusetts
R. GORDON DOUGLAS, Jr., Senior Vice President,
Medical and Scientific Affairs, Merck Sharp & Dohme International, Rahway, New Jersey
JAMES EIGO, ACT UP,
New York, New York
Department of Biology and Center for Cancer Research, Massachusetts Institute of Technology, Cambridge
MELVIN M. GRUMBACH, * Edward B. Shaw Professor of Pediatrics,
Department of Pediatrics, School of Medicine, University of California, San Francisco
DONALD HOPKINS, * Senior Consultant,
Global 2000, Inc., Chicago, Illinois
C. MAX LANG, George T. Harrell Professor and Chairman,
Department of Comparative Medicine, College of Medicine, The Milton S. Hershey Medical Center of The Pennsylvania State University, Hershey
CURTIS MEINERT, Professor of Epidemiology,
School of Public Health and Hygiene, Johns Hopkins University, Baltimore, Maryland
NEAL NATHANSON, Chairman,
Department of Microbiology, University of Pennsylvania School of Medicine, Philadelphia
PHILIP S. SCHEIN, President and Chief Executive Officer,
U.S. Bioscience, Blue Bell, Pennsylvania
ARTHUR SILVERSTEIN, Professor Emeritus,
Institute of the History of Medicine, School of Medicine, Johns Hopkins University, Baltimore, Maryland
MICHAEL McGEARY, Study Director
ERIC DE JONGE, Research Associate
JEFFREY LEVI, Consultant
LEAH MAZADE, Editor
APRIL POWERS, Project Assistant
U. JANE SANVILLE, Staff Officer
GAIL SPEARS, Administrative Assistant
ROBERT A. WALKINGTON, Consultant
ROBIN WEISS, Director, AIDS Activities
Member, Institute of Medicine
Member, National Academy of Sciences
This report on the acquired immune deficiency syndrome (AIDS) research program of the National Institutes of Health (NIH) and the study on which it is based were requested by NIH's Office of AIDS Research (OAR). In only a short time, NIH has mounted a large, wide-ranging, complex program of AIDS research activities that involve every component of the agency. Because of the program's fast growth, scientific and public health importance, and high public expectations, James B. Wyngaarden, then director of NIH, asked Anthony S. Fauci, associate director for AIDS research and director of OAR, to oversee an evaluation of the program's research directions, management, and resources. OAR in turn asked the Institute of Medicine (IOM) to conduct an objective, unbiased review of the program, giving particular attention to its scope and content, management, results, and levels of budgetary and administrative support.1 IOM agreed to undertake the study beginning in August 1989 and to make recommendations for strengthening current efforts as well as for the program's future directions.
The president of IOM, with the concurrence of the president of the National Academy of Sciences, appointed a 15-member study committee to prepare the report. Individuals were selected for their knowledge and expertise in the areas of virology, immunology, neuropathogenesis, epidemiology, infectious diseases of adults and children, nursing, health behavior, drug and vaccine development, clinical trials, animal models development, research program administration, and health program evaluation. Academia, private industry, and the patient advocacy perspective were all represented. In addition, a special effort was made to appoint a chairman and a majority of members who were neither directly involved in NIH-supported AIDS research nor members of major NIH advisory groups overseeing AIDS research (such as the AIDS Program Advisory Committee, the executive committee of the AIDS Clinical Trials Group, and the national advisory councils to the institutes). Several committee members involved in AIDS research activities provided the grantee perspective on the NIH AIDS research effort, however. Adel A. F. Mahmoud and Howard M. Temin, members of the National Advisory Allergy and Infectious Diseases Council and the National Cancer Advisory Board, respectively, attended an early meeting of the committee.
The committee met five times between October 1989 and September 1990 to define its task, develop a study plan, receive information, analyze the issues, and develop recommendations. The charge from NIH focused on questions of program balance, organization, management, and
Appendix A lists specific issues of program organization, management, staffing, and funding to be addressed in the study.
resources. Included in the charge was a review of the appropriateness of the scope and content of NIH's AIDS research program. This phrase might have justified a detailed evaluation of the AIDS research agenda, but the committee decided against doing so. Such an effort would require a large-scale consensus development process involving groups of experts from many scientific fields and medical disciplines, a project beyond the resources and time available for the study. The group concluded instead that, given the long-term nature of the epidemic of HIV and AIDS, it would be most useful to review and make recommendations for improving NIH's institutional capacity to develop and implement an appropriate AIDS research agenda in the coming years. Thus the study emphasized programmatic issues, with the intent of advising NIH on ways in which it might improve its ability to identify and pursue the most important research questions. The committee did assess the appropriateness of the current NIH role and scale of effort in each area of research and addressed the appropriate balance among the areas and the potential impact of AIDS research on other research areas.
At the first several meetings, the committee heard presentations by NIH officials from the Office of AIDS Research (Anthony S. Fauci), the National Institute of Allergy and Infectious Diseases (Daniel F. Hoth, H. Clifford Lane), the National Cancer Institute (Bruce A. Chabner), the National Heart, Lung, and Blood Institute (Elaine M. Sloand), the National Institute of Child Health and Human Development (Antonia C. Novello), the National Institute of Neurological Disorders and Stroke (Carl M. Leventhal), the National Center for Nursing Research (Ada Sue Hinshaw), and the Division of Research Grants (Bruce Maurer, Gilbert W. Meier). The committee also benefited from presentations by the following: James R. Allen, Director, National AIDS Program Office, Public Health Service; Frank E. Young, Deputy Assistant Secretary for Health/Science and Environment, Department of Health and Human Services; Robert Wittes, Executive Vice President for Cancer Research, Bristol-Myers Squibb; Lawrence Corey, Professor, Department of Laboratory Medicine, University of Washington; and Edward M. Connor, Associate Director, Department of Immunology and Infectious Diseases, University of Medicine and Dentistry/New Jersey Medical School. Corey and Connor, respectively, chair the Executive and Pediatric Committees of the AIDS Clinical Trials Group. Committee members and staff also learned much from attending meetings of the AIDS Clinical Trials Group, the NIH director's AIDS Program Advisory Committee, the NIH AIDS Executive Committee, and the National Institute of Allergy and Infectious Diseases ' (NIAID) AIDS Division advisory committee.
The committee invited testimony from AIDS-related groups, and representatives of many of them testified at a hearing held by the committee in conjunction with its second meeting on December 4–5, 1989. Others submitted written statements. The following organizations responded to the committee 's invitation: National Organizations Responding to AIDS, American Association of Physicians for Human Rights, Project Inform, National Association of People with AIDS, Physicians Association for AIDS Care, American Nurses' Association, National Minority AIDS Council, National Organization for Rare Diseases, AIDS Treatment Registry, and the Pediatric AIDS Foundation. The committee also monitored testimony from these and other groups, as well as from federal AIDS officials and academic researchers, at congressional hearings on AIDS and before the National Commission on AIDS.
In addition to presentations and testimony, the committee reviewed a series of reports on AIDS research by previous IOM committees, the Presidential Commission on the Human Immunodeficiency Virus Epidemic, NIH advisory groups, and NIH itself. It also reviewed current internal NIH planning and budget documents and the documents justifying NIH 's budget request to Congress for fiscal year 1991. The staff then synthesized information on the NIH AIDS research program and the AIDS activities of other Public Health Service (PHS) agencies (Centers for Disease Control; Alcohol, Drug Abuse, and Mental Health Administration; and Health Resources
and Services Administration) in a series of background papers that were discussed at committee meetings and formed the basis for the report's findings. These papers reflected not only pertinent documents and program data but also more than 150 interviews, primarily with NIH officials, program managers, and researchers, and with other PHS officials, university-based researchers, community-level clinician-researchers, and congressional staff (see the list in Appendix B).
Rather than offering a lengthy description and analysis of all facets of NIH's large, rapidly changing AIDS research program, the committee chose to present its conclusions and recommendations in a brief report that addresses the major issues of research and management. The report concentrates on the broad content areas of NIH's program, on the balance among those program areas, and on the overall management system needed to plan, set priorities, coordinate, and monitor a research effort being implemented in every institute, center, and division of NIH. Although the committee chose not to organize the study specifically around the nine study issues suggested by NIH, each of the issues is addressed in the report at some point (see Appendix A for the list of study issues and the report sections that address them). The report does not comprehensively review AIDS-related activities of other PHS agencies but only presents those activities in their relation to NIH and its research mission.
During the preparation of this report, the committee was constantly reminded that it was assessing a moving target: NIH continues, appropriately, to add to and revise the AIDS program. Consequently, this report presents the committee's evaluation as of September 1990. Important developments since then are noted in footnotes at appropriate points in the report. The number and detail of the committee's conclusions and recommendations vary with its judgment of the importance and complexity of the issues raised. Thus, for example, the committee spent a great deal of time assessing the AIDS clinical trials program and made a number of recommendations because the program is large, important, and controversial and because at the time of the study it was undergoing several major changes.
The committee concluded that, because of the scientific opportunities and the importance of controlling the burden of illness, it is wise to increase substantially the funding for AIDS research. The source of these additional funds remains an issue, however. Research on AIDS is part of and dependent on the total biomedical research enterprise. Given the current serious shortage of funds for new and competing research project grants, it would be unwise to shift funds from other areas of biomedical research and risk setbacks in the orderly development of the nation's research effort. Maintaining the total biomedical research effort must be a high priority for all those concerned with specific diseases.
Important among the AIDS-related issues that lie outside the purview of NIH is financing of care for AIDS patients. The health care burden of AIDS is large and still growing, and it has already begun to impose severe stresses on America's health care system. Moreover, human immunodeficiency virus (HIV) infection and disease are spreading rapidly among those who have inadequate or no insurance. Much-needed research is difficult among these groups because many of them have no regular source of care and hence are difficult to enroll and retain in research protocols for new treatments or therapies. The committee is further concerned that inadequacies in the health care system will slow or prevent some populations from adopting the products of AIDS research, for instance, new drug therapies or early interventions that might delay the development of disease in presymptomatic HIV-infected individuals. The committee could only flag the problems associated with health care financing for persons infected with HIV and recommend that the administration and Congress resolve them.
The committee wishes to thank the many individuals and organizations that advanced its work, many of whom are named already in this preface (or listed in Appendix B). Special thanks go to Jack Whitescarver, deputy director of the Office of AIDS Research, and Marc Horowitz, of the OAR staff. As project officers for the study, they facilitated the committee's access to people, documents, and data at NIH. Donna Adderly of the Division of Financial Management answered many requests for budgetary and program data with unfailing cheer, as did Linda Jackson, administrative officer of the Office of AIDS Research. John James, Bruce Maurer, and Gil Meier, all of the Division of Research Grants, provided data about grant applications and awards. The committee also deeply appreciated the support of its capable, hard-working staff, who conducted scores of interviews and reduced mountains of scattered data to a manageable form. Finally, I should like to thank the other committee members for their time and effort. Their contributions are an important element in the effort to address a problem that will continue to challenge NIH and all of society for at least the next decade and probably well beyond.
William H. Danforth
Chair, Committee to Study the AIDS Research
Program of the National Institutes of Health