Scientific Opportunities and Public Needs

Improving Priority Setting and Public Input at the National Institutes of Health

Committee on the NIH Research Priority-Setting Process

Health Sciences Policy Program

Health Sciences Section

INSTITUTE OF MEDICINE

NATIONAL ACADEMY PRESS
Washington, D.C.
1998



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--> Scientific Opportunities and Public Needs Improving Priority Setting and Public Input at the National Institutes of Health Committee on the NIH Research Priority-Setting Process Health Sciences Policy Program Health Sciences Section INSTITUTE OF MEDICINE NATIONAL ACADEMY PRESS Washington, D.C. 1998

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--> NATIONAL ACADEMY PRESS 2101 Constitution Avenue, N.W. Washington, D.C. 20418 NOTICE: The project that is the subject of this report was approved by the Governing Board of the National Research Council, whose members are drawn from the councils of the National Academy of Sciences, the National Academy of Engineering, and the Institute of Medicine. The members of the committee responsible for the report were chosen for their special competences and with regard for appropriate balance. The Institute of Medicine was chartered in 1970 by the National Academy of Sciences to enlist distinguished members of the appropriate professions in the examination of policy matters pertaining to the health of the public. In this, the Institute acts under both the Academy’s 1863 congressional charter responsibility to be an adviser to the federal government and its own initiative in identifying issues of medical care, research, and education. Dr. Kenneth I. Shine is president of the Institute of Medicine. Support for this project was provided by funds from the National Institutes of Health (Contract No. N01-OD-4-2139). The views presented in this report are those of the Committee on the NIH Research Priority-Setting Process and are not necessarily those of the funding organization. International Standard Book No. 0-309-06130-X Additional copies of this report are available for sale from the National Academy Press, 2101 Constitution Avenue, N.W., Box 285, Washington, DC 20055. Call (800) 624-6242 or (202) 334-3313 (in the Washington metropolitan area), or visit the NAP’s on-line bookstore at http://www.nap.edu. The full text of this report is available on line at http://www.nap.edu. For more information about the Institute of Medicine, visit the IOM home page at http://www2.nas.edu/iom. Copyright 1998 by the National Academy of Sciences. All rights reserved. Printed in the United States of America The serpent has been a symbol of long life, healing, and knowledge among almost all cultures and religions since the beginning of recorded history. The image adopted as a logotype by the Institute of Medicine is based on a relief carving from ancient Greece, now held by the Staatliche Museen in Berlin.

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--> COMMITTEE ON THE NIH RESEARCH PRIORITY-SETTING PROCESS LEON E. ROSENBERG* † (Chair), Professor, Department of Molecular Biology, and Woodwork Wilson School of Public and International Affairs, Princeton University JOHN ALDERETE, Professor, Department of Microbiology, University of Texas Health Science Center at San Antonio KENNETH B. CHANCE, Dean and Professor of Endodontics, Meharry Medical College School of Dentistry CARON CHESS, Director, Center for Environmental Communication, Cook College, Reuters University PURNELL CHOPPIN,* † President, Howard Hughes Medical Institute JAMES W. CURRAN,* Dean and Professor of Epidemiology, The Rollin's School of Public Health, Emory University DAVID CUTLER, Professor, Department of Economics, Littauer Center, Harvard University SUE DONALDSON,* Professor and Dean, School of Nursing and Professor of Physiology, School of Medicine, Johns Hopkins University BARUCH FISCHHOFF,* University Professor, Department of Engineering and Public Policy, Department of Social and Decision Sciences, Carnegie Mellon University SID GILMAN,* William J. Herdman Professor and Chair, Department of Neurology, University of Michigan ROBERT L. HILL,* † James B. Duke Professor, Department of Biochemistry, Duke University Medical Center RALPH HORWITZ,* † Chair and Professor, Department of Internal Medicine, Yale University School of Medicine THOMAS KELLY,† Boury Professor and Chairman, Department of Molecular Biology and Genetics, Johns Hopkins University ANNE PETERSEN, Senior Vice President, Programs, W.K. Kellogg Foundation SUSAN C. SCRIMSHAW,* Dean and Professor of Community Health Sciences, School of Public Health, University of Illinois at Chicago ROGER UNGER,† Professor of Internal Medicine, Center for Diabetes Research, University of Texas Southwestern Medical Center MYRL WEINBERG, President, National Health Council LINDA S. WILSON,* President, Radcliffe College ADAM YARMOLINSKY,* Regents Professor of Public Policy in the University of Maryland System *   Member of the Institute of Medicine. †   Member of the National Academy of Sciences.

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--> Committee Liaisons HARVEY R. COLTEN,* Dean and Vice President for Medical Affairs, Northwestern University School of Medicine JOSEPH T. COYLE,* Eben S. Draper Professor of Psychiatry and Neuroscience Chair, Consolidated Department of Psychiatry, Harvard Medical School JOHN D. STOBO,* President, University of Texas Medical Branch Study Staff ANDREW POPE, Director, Health Sciences Policy Program GEOFFREY FRENCH, Research Associate Consultants KATHI HANNA MICHAEL McGEARY Copy Editor MICHAEL HAYES Section Staff CHARLES H. EVANS, JR., Head, Health Sciences Section ROBERT COOK-DEEGAN, Director, National Cancer Policy Board LINDA DEPUGH, Administrative Assistant JAMAINE TINKER, Financial Associate *   Member of the Institute of Medicine.

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--> REVIEWERS This report has been reviewed in draft form by individuals chosen for their diverse perspectives and technical expertise, in accordance with procedures approved by the National Research Council's Report Review Committee. The purpose of this independent review is to provide candid and critical comments that will assist the Institute of Medicine in making the published report as sound as possible and to ensure that the report meets institutional standards for objectivity, evidence, and responsiveness to the study charge. The content of the final report is the responsibility of the Institute of Medicine and the study committee and not the responsibility of the reviewers. The review comments and draft manuscript remain confidential to protect the integrity of the deliberative process. The committee wishes to thank the following individuals, who are neither officials nor employees of the Institute of Medicine, for their participation in the review of this report: JOHN ABELSON,† California Institute of Technology DONALD D. BROWN,† Carnegie Institution of Washington MICHAEL S. BROWN,* † University of Texas Southwestern Medical Center JAMES EBERT,* † Marine Biological Laboratory, Woods Hole ROBIN ELLIOTT, Parkinson's Disease Foundation HARMON J. EYRE, American Cancer Society MARC W. KIRSCHNER,† Harvard Medical School S. ROBERT LEVINE, Juvenile Diabetes Foundation International ROBERT LEVY,* American Home Products Corp. MARSHALL LICHTMAN, Leukemia Society of America DOROTHY RICE,* University of California, San Francisco PAUL ROGERS,* Hogan & Hartson CHARLES SANDERS,* Glaxo, Inc. HOWARD K. SCHACHMAN,† University of California, Berkeley MORTON N. SWARTZ,* Massachusetts General Hospital SAMUEL THIER,* Partners Health Care System, Inc. LOWELL WEICKER, University of Virginia MYRON WEISFELDT,* Columbia University and past president, American Heart Association While the individuals listed above have provided many constructive comments and suggestions, responsibility for the final content of this report rests solely with the authoring committee and the Institute of Medicine. *   Member of the Institute of Medicine. †   Member of the National Academy of Sciences.

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--> Preface The United States is preeminent in medical research. Since World War II this country has fashioned a medical research system—with government, academia, and industry at its core—that is a source of great national pride and the envy of the world. The federal government is the single largest sponsor of this research, committing more than $16 billion of public funds in the current fiscal year (1998). Of this total, the vast majority—$13.6 billion—is appropriated to the National Institutes of Health (NIH). Given the size of this public investment and the likelihood that it will be increased significantly in the immediate future and given the mission of NIH—"to uncover new knowledge that will lead to better health for everyone"—it should come as no surprise that there is intense interest in how NIH sets its priorities, that is, how it allocates its sizable budget. How could it be otherwise? Every one of us wants to live a long and healthy life. Every sick person—woman, man, or child—wants researchers to find new ways to make him or her well or to improve the quality of life for those who are disabled, regardless of whether the ailment is common or rare, acute or chronic, life-threatening or self-limiting. We must acknowledge that setting priorities at NIH is an awesome task. Not only must the leadership of NIH answer to the executive branch and to the U.S. Congress, it must work with all of its constituencies—scientists, health care providers, patients, voluntary health groups and patient advocates, and industry executives—before making its fateful decisions. The quality and quantity of excellent science that it has supported, the widespread respect for it in and out of government, and its favored position in the annual congressional appropriations process signify that, over time, NIH must be doing many things right. Yet, we must also acknowledge that the recent request from Congress that the Institute

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--> of Medicine conduct an independent assessment of research priority setting at NIH and that the evaluation be completed within 6 months signifies, just as clearly, that there is at least a perception that some things are not right. It is apparent that some segments of the public, in general, and its representatives in Congress, in particular, are dissatisfied enough to ask for ways to improve the current process. This committee was charged with examining four issues related to setting priorities at NIH: allocation criteria, the decision-making process, mechanisms for public input, and the impact of congressional directives. Despite the nearly impossible constraints imposed by the study's time line, we took this broad charge seriously. To grasp the approach that NIH currently uses, we heard in person from the director of NIH, most of the institute directors, and many of the directors of offices housed within the director's office. To understand the tensions surrounding public input, we held a full-day public meeting at which we received verbal and written testimony from patients, advocacy and interest groups, foundations, and professional societies. To understand Congress's rationale for requesting the study, we interviewed legislative aides from key offices. To expand our collective knowledge base, we reviewed many current and past publications relevant to our charge. As we listened and deliberated, several things became clear to me. First, the country has extraordinarily high expectations of NIH. To some, NIH has become a virtual surrogate for the U.S. Department of Health and Human Services, being encouraged to expand its purview well beyond research. We heard from no one who wants to dismantle NIH; we heard from many who wanted their "cause" to be embraced by NIH; we heard from all that NIH must excel at everything it does because what it does is so important to the hopes and aspirations of people everywhere. Second, there is a sense that NIH has evolved mechanisms for judging scientific opportunity and merit that surpass its capabilities for assessing and being influenced by public health needs. Estimating research spending by disease and developing metrics for spending according to disease burden (e.g., incidence, mortality, disability, and cost) must be done more systematically and more thoroughly than they are currently done because not doing so leads some to conclude, incorrectly I believe, that NIH cares more about curiosity than cure, more about fundamental science than clinical application. Third, there is frustration on the part of some groups about not being listened to and heard by NIH. We heard repeatedly that some institutes, and particularly the Office of the Director, lack mechanisms for orderly, regular public input and outreach. As the authority of the director over priority setting has increased, the demand to influence that office has become louder. Fourth, there is a lack of understanding about how NIH priority setting "works." NIH has not crafted simple communications that make its priority-setting processes as transparent as possible to its many publics. NIH has not developed

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--> sufficient communications tools to satisfy people that allocation decisions are made on the basis of equity and justice, as well as scientific opportunity. Each of these observations has been converted to recommendations that reflect the committee's consensus. Our recommendations address each of the committee's charges, but there is a single theme that runs through them. It is that NIH must revamp its approach to public input and outreach—at every level—without delay. This will strengthen the priority-setting process in many ways. It will underscore that openness is as important to the process as such other valued qualities as expertise, innovation, and objectivity. It will provide NIH leaders more ways to demonstrate that they share the public's view that NIH exists to improve health through research. It will enhance the public's understanding of the complexities of decision making at NIH. Finally, it will give Congress additional confidence that it can delegate priority setting to NIH leadership knowing that a broader range of views will be sought and welcomed before decisions are made. I would like to thank the many people who have made it possible for this report to be completed on schedule: first and foremost, the members of the committee who met and overcame the many challenges of our difficult task with a commendable blend of experience, energy, collegiality, and wisdom; second, the staff of the Institute of Medicine, without whom we would have foundered; third, the leadership of NIH, who educated us about this agency; and fourth, and perhaps most important, the public, who reminded us of the purpose of NIH and of the democratic ideals that must permeate effective stewardship of a federal agency. Leon E. Rosenberg, M.D. Chair

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--> Acronyms ACD Advisory Committee to the Director DHHS U.S. Department of Health and Human Services FIC John E. Fogarty International Center FY fiscal year IOM Institute of Medicine NCI National Cancer Institute, NIH NCRR National Center for Research Resources, NIH NEI National Eye Institute, NIH NHGRI National Human Genome Research Institute, NIH NHLBI National Heart, Lung, and Blood Institute, NIH NIA National Institute on Aging, NIH NIAAA National Institute on Alcohol Abuse and Alcoholism, NIH NIAID National Institute of Allergy and Infectious Diseases, NIH NIAMS National Institute of Arthritis and Musculoskeletal and Skin Diseases, NIH NICHD National Institute of Child Health and Human Development, NIH NIDA National Institute on Drug Abuse, NIH

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--> NIDCD National Institute on Deafness and Other Communication Disorders, NIH NIDDK National Institute of Diabetes and Digestive and Kidney Diseases, NIH NIDR National Institute of Dental Research, NIH NIEHS National Institute of Environmental Health Sciences, NIH NIGMS National Institute of General Medical Sciences, NIH NIH National Institutes of Health NIMH National Institute of Mental Health, NIH NINDS National Institute of Neurological Disorders and Stroke, NIH NINR National Institute of Nursing Research, NIH NLM National Library of Medicine, NIH NSF National Science Foundation OMB Office of Management and Budget PHS Public Health Service R&D research and development RFA request for application RPG research project grant

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--> Contents     EXECUTIVE SUMMARY   1 1   INTRODUCTION   13     Background,   14     Funding Allocation by Institute and Center,   16     Funding Allocation by Mechanism,   18     Funding Allocation by Type of Research,   20     Funding Allocation by Disease,   21     Recent Trends and Issues,   23     IOM Committee Process,   26     Organization of the Report,   28 2   CRITERIA FOR PRIORITY SETTING   29     Why Criteria Are Important,   29     NIH's Criteria for Priority Setting,   30     Criterion 1. Public Health Needs,   31     Criterion 2. Quality of Research Supported,   34     Criterion 3. Scientific Opportunity,   36     Criterion 4. Portfolio Diversification,   37     Criterion 5. Adequate Infrastructure Support,   38     Conclusions and Recommendations,   38 3   PRIORITY-SETTING PROCESSES   43     Priority Setting Through the Budget Process,   44

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-->     NIH Director's Role in Priority Setting,   46     Transfer Authority,   47     Discretionary Fund,   47     NIH Areas of Research Emphasis,   48     Advisory Committee to the Director,   48     Priority Setting at the Institute Level,   49     Conclusions and Recommendations,   51 4   MECHANISMS FOR PUBLIC INPUT   53     Public Input into Priority Setting,   54     Recent Changes,   55     Two-Way Communication Between NIH and the Public,   57     Conclusions and Recommendations,   61     Offices of Public Liaison: Rationale, Roles, and Responsibilities,   62     Director's Council of Public Representatives: Rationale, Roles, and Responsibilities,   64     Policy and Program Advisory Group Membership,   66     Summary,   68 5   CONGRESSIONAL ROLE   69     Congress and NIH,   69     How Congress Communicates Priorities,   70     Conclusions and Recommendations,   76     REFERENCES   81     APPENDIXES   85     A Federal Advisory Committees of the National Institutes of Health,   87     B Acknowledgments,   95     C National Institutes of Health Funding Tables,   101     D Committee and Staff Biographies,   111

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--> Scientific Opportunities and Public Needs

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