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This conference summary was written by Eve Nichols for the Institute of Medicine's Roundtable for the Development of Drugs and Vaccines Against AIDS, chaired by Harold Ginsberg and Sheldon Wolff and directed by Robin Weiss. The document reports major themes of the conference discussions; these themes, however, do not represent policy statements by the Institute of Medicine.
The report has been reviewed by a group other than the authors according to procedures approved by a Report Review Committee consisting of members of the National Academy of Sciences, the National Academy of Engineering, and the Institute of Medicine.
The Institute of Medicine was chartered in 1970 by the National Academy of Sciences to enlist distinguished members of 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 advisor to the federal government, and its own initiative in identifying issues of medical care, research, and education.
The Roundtable is supported by the American Foundation for AIDS Research, the Merck Company Foundation, the Pharmaceutical Manufacturers Association, the U.S. Army, the U.S. Public Health Service, and the U.S. Department of Veterans Affairs.
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ROUNDTABLE FOR THE DEVELOPMENT OF DRUGS AND VACCINES AGAINST AIDS
HAROLD S. GINSBERG (Co-chair), Eugene Higgins Professor of Medicine and Microbiology,
Department of Medicine, College of Physicians & Surgeons, Columbia University, New York
SHELDON M. WOLFF (Co-chair), Physician-in-Chief,
New England Medical Center, and
Endicott Professor and Chairman,
Department of Medicine, Tufts University School of Medicine, Boston
DAVID W. BARRY, Vice President of Research,
The Wellcome Research Laboratories, Burroughs Wellcome Co., Research Triangle Park, North Carolina
JAMES S. BENSON, Acting Commissioner,
Food and Drug Administration, Rockville, Maryland
DONALD S. BURKE, Colonel,
Medical Corps, U.S. Army, and
Division of Retrovirology, Walter Reed Army Institute of Research, Rockville, Maryland
BRUCE A. CHABNER, Director,
Division of Cancer Treatment, National Cancer Institute, National Institutes of Health, Bethesda, Maryland
MAX D. COOPER, Investigator,
Howard Hughes Medical Institute, and
Professor of Medicine, Pediatrics, and Microbiology,
University of Alabama, Birmingham
MARTIN DELANEY, Co-Executive Director and President,
Project Inform, San Francisco
DANIEL DEYKIN, Chief,
Cooperative Studies Program. Veterans Administration Medical Center, Boston
R. GORDON DOUGLAS, Senior Vice President,
Medical and Scientific Affairs, Merck Sharp & Dohme International, Rahway, New Jersey
ANTHONY S. FAUCI, Associate Director for AIDS Research,
National Institutes of Health, and
National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland
GERALD FRIEDLAND, Professor of Medicine,
Department of Medicine, Albert Einstein College of Medicine, Montefiore Medical Center, The Bronx, New York
L. PATRICK GAGE, Executive Vice President,
Genetics Institute, Inc., Cambridge, Massachusetts
PETER BARTON HUTT, Partner,
Covington & Burling, Washington, D.C.
JAY C. LIPNER, Partner,
Silverstein Langer Lipner & Newburgh, New York, New York
DAVID W. MARTIN, JR., Vice President,
Research and Development, The Dupont Merck Pharmaceutical Company, Wilmington, Delaware
CATHERINE M. WILFERT, Professor of Pediatrics and Microbiology,
Department of Pediatrics, Division of Infectious Diseases, Duke University Medical Center, Durham, North Carolina
ROBIN WEISS, Project Director and Director,
SHARON BARATZ, Research Associate
RICHARD BERZON, Staff Officer
GAIL SPEARS, Administrative Assistant
The Roundtable for the Development of Drugs and Vaccines Against AIDS was established in 1988 by the Institute of Medicine. Composed of leaders from government, the pharmaceutical industry, academia, and the public, its mission is to identify and help resolve impediments to the speedy availability of safe and effective drugs and vaccines for human immunodeficiency virus (HIV) infection and acquired immune deficiency syndrome (AIDS). The Roundtable accomplishes its mission through regular meetings of its membership, during which urgent issues are identified and discussed, as well as through public conferences and workshops that explore scientific and policy matters central to the development of AIDS therapeutics. This publication is the report of a conference held March 12 and 13, 1990, in Washington, D.C.
The call for a "parallel track" for AIDS drug development—a proposal that would allow the early distribution of AIDS drugs to large numbers of patients in parallel with the conventional clinical trials that assess the drugs' safety and efficacy—has sparked controversy within the scientific community. Questions have arisen about the risks to patients of such a plan, about its potential effect on the successful completion of standard controlled trials, and about whether the parallel track will generate useful data. Larger questions have also been raised about whether the parallel track heralds fundamental changes in the philosophy underlying drug regulation in the United States, about the costs and financing of investigational therapies and associated medical costs, and about the role of expanded access mechanisms for drugs in reaching those whose health care generally is inadequate. The Roundtable sought to illuminate these issues by inviting knowledgeable speakers and the public to a two-day conference to examine proposals for expanded access to investigational drugs and possible repercussions of such an action.
Two months after the conference was held, in May 1990, the parallel track proposal was published in the Federal Register and comments were
sought. A meeting was held in September 1990 by the Public Health Service (PHS) to discuss the comments. As this report goes to press, the PHS is finalizing the document, which, when completed, will constitute a written PHS policy. As the policy takes effect, many of the issues raised in this report will serve as valuable guideposts in evaluating the parallel track experiment.
A note on terminology: Although the word effectiveness rather than efficacy was used by Congress in the Drug Amendments of 1962, we have chosen in this report to conform to the definitions of the two terms as they are commonly understood in the field of medical technology assessment. Here, the term efficacy refers to what a method (e.g., a drug) can accomplish in expert hands when correctly applied to a patient; effectiveness refers to its performance in more general routine applications.1 Therefore, most randomized clinical trials assess efficacy; the Food and Drug Administration, in reviewing the results of these trials, is evaluating the efficacy of the drugs under investigation.
This report seeks to summarize the conference presentations. It contains no recommendations or conclusions, and the Roundtable has neither altered nor commented on the views and opinions expressed by the speakers, except for purposes of clarity. The Roundtable and staff wish to thank Eve Nichols, whose capable hands crafted the transcript of the meeting into a smooth narrative. We also thank, once again, the conference speakers for their thoughtful presentations, and all participants for the lively and challenging discussions throughout the conference.