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Suggested Citation:"Front Matter." Institute of Medicine. 1979. Pharmaceuticals for Developing Countries: Conference Proceedings. Washington, DC: The National Academies Press. doi: 10.17226/18441.
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Below is the uncorrected machine-read text of this chapter, intended to provide our own search engines and external engines with highly rich, chapter-representative searchable text of each book. Because it is UNCORRECTED material, please consider the following text as a useful but insufficient proxy for the authoritative book pages.

Pharmaceuticals for Developing Countries CONFERENCE PROCEEDINGS DIVISION OF INTERNATIONAL HEALTH INSTITUTE OF MEDICINE NATIONAL ACADEMY OF SCIENCES WASHINGTON, D.C. 1979 NAS-N/1E 71973 LIBRARY

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 competencies and with regard for appropriate balance. This report has been reviewed by a group other than the authors accord- ing 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. Supported by contract number 282-78-EJM from the Department of Health, Education, and Welfare. The Institute of Medicine was chartered in 1970 by the National Academy of Sciences to enlist distinguished members of the appropriate profes- sions 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. Publication IOM-79-001 Library of Congress Catalog Card Number 79-89015 International Standard Book Number 0-309-02891-4 Available from Office of Publications National Academy of Sciences 2101 Constitution Avenue, N.W. Washington, D.C. 20418 Printed in the United States of America

CONTENTS STEERING COMMITTEE MEMBERSHIP vii PREFACE Harold J. Simon xi OVERVIEW OF THE CONFERENCE Leighton E. Cluff xiii INTRODUCTION David A. Hamburg 1 KEYNOTE ADDRESS Edward M. Kennedy 3 HISTORICAL PERSPECTIVE Walsh McDermott 9 MAJOR DISEASE PROBLEMS OF DEVELOPING COUNTRIES—THE CURRENT STATUS OF PREVENTIVE, PROPHYLACTIC, DIAGNOSTIC, AND THERAPEUTIC AGENTS 29 Major Disease Problems in the Developing World 30 C. E. Gordon Smith The Pharmaceutical Industry's Perspective on Available Agents 49 Paul A. J. Janssen Denis Thienpont Assessment of Effectiveness, Safety, and Accessibility of Available Agents 59 A. Drugs Against Parasitic Diseases 59 Wallace Peters B. Accessibility of Vaccines William H. Foege 83 C. Vaccine Production Frank T. Perkins 95 Selected Pharmaceutical Developments Required for Prevention of Massive Blindness in Developing Countries 103 Barrie R. Jones Discussion 114 iii

CURRENT PROGRAMS FOR DEVELOPMENT OF PHARMACEUTICALS 121 UNDP/World Bank/WHO Special Program for Research and Training in Tropical Diseases Adetokunbo 0. Lucas 122 The United States Pharmaceutical Industry 130 Lewis H. Sarett The European Pharmaceutical Industry 136 Ernst Vischer Rudi Oberholzer Current Programs in United States Academic Laboratories for Development of Agents Against Selected Infectious Diseases 143 William S. Jordan, Jr. Discussion 166 INTERNATIONAL HEALTH: FROM THE PERSPECTIVE OF THE CARTER ADMINISTRATION 168 Gilbert S. Omenn PROBLEMS AND CONSTRAINTS 175 Introductory Comments for the Panel on Problems and Constraints, Conference on Pharmaceuticals for Developing Countries 176 William Hubbard Constraints on Expanding the Role of the U.S. Pharmaceutical Industry 179 A. United States Industry Perspectives 179 W. Clarke Wescoe B. Food and Drug Administration and Pharmaceuticals for Developing Countries Donald Kennedy 187 Constraints on Involvement of the U.S. Government and Academic Research Community, Including Manpower Considerations Richard M. Krause 192 Discussion 205 Problems of Distribution, Availability, and Utilization of Agents in Developing Countries 211 A. Industry Perspectives Max P. Tiefenbacher 211 B. Perspectives on the Distribution and Availability of Pharmaceuticals in Africa and the Middle East 228 Abdou M. Sallam lv

C. Problems of Distribution, Availability and Utilization of Agents in Developing Countries: An Asian Perspective 236 Sanjaya Lall D. Developing Country Perspectives—An Overview 250 Vittorio Fattorusso Innovation and Availability in the United States of Drugs for Tropical Diseases Jean DiRaddo 260 William M. Wardell THE SCIENTIFIC BASE: OPPORTUNITIES FOR RESEARCH 281 Introduction Joshua Lederberg 282 Comparative Biochemistry and the Design of Chemotherapeutic Agents for Infectious Disease Seymour S. Cohen 284 Cellular Regulatory Processes in Parasitic Helminths: Significance in Drug Development 298 Tag E. Mansour Rational Development of New Drugs for Trypanosomiasis 312 Anthony Cerami Steven R. Meshnick Immunology and Parasitic Diseases 318 John R. David Molecular Biology and Genetics as Basic Approaches to the Problems of Parasitism and Specific Chemotherapy 326 Joshua Lederberg Methods of Drug Administration John Urquhart 329 Clinical Trials in Developing Countries 349 A. Methodology of Clinical Trials in Developing Countries 349 Alvan R. Feinstein B. Facilitation of Clinical Trials in Developing Countries 359 Edmund de Maar J. M. Kofi Ekue Opportunities for Research—American Industry 367 Pedro Cuatrecasas Discussion 389 INTERNATIONAL HEALTH POLICY INITIATIVES 391 Jacob K. Javits

STRENGTHENING INCENTIVES AND OVERCOMING DISINCENTIVES 399 Panel Presentations James Henry 400 Ivan L. Bennett, Jr. Barry M. Bloom William N. Hubbard, Jr. James Lee Max P. Tiefenbacher W. Clarke Wescoe Kenneth Warren General Discussion 416 UNITED STATES INTERNATIONAL HEALTH POLICY: ROOM FOR PUBLIC AND PRIVATE INITIATIVE Richard S. Schweiker 419 CLOSING REMARKS Leighton E. Cluff 425 INVITED CONTRIBUTORS 427 vi

NATIONAL ACADEMY OF SCIENCES INSTITUTE OF MEDICINE DIVISION OF INTERNATIONAL HEALTH Steering Committee Conference on Pharmaceuticals for Developing Countries Chairman: Leighton E. Cluff Vice President The Robert Wood Johnson Foundation Princeton, New Jersey Sune Bergstrom Rector Karolinska Institutet Stockholm, Sweden Barry M. Bloom President of Central Research Pfizer, Incorporated Groton, Connecticut Seymour S. Cohen Distinguished Professor Department of Pharmacological Sciences School of Basic Health Sciences Health Sciences Center State University of New York, Stoney Brook Long Island, New York John U. Farley Professor Graduate School of Business Columbia University New York, New York Robert J. Gerraughty Associate Vice President of Health Sciences Creighton University Omaha, Nebraska vii

Raymond Gosselin President Massachusetts College of Pharmacy Boston, Massachusetts James F. Henry President Center for Public Resources, Incorporated New York, New York William Hubbard President The Upjohn Company Kalamazoo, Michigan Michael Katz Chairman Department of Pediatrics College of Physicians and Surgeons Columbia University New York, New York Tag Mansour Chairman Department of Pharmacology Stanford University School of Medicine Stanford, California Kenneth Warren Director of Health Science Programs The Rockefeller Foundation New York, New York Alejandro Zaffaroni President and Director of Research ALZA Corporation Palo Alto, California viii

Staff Harold J. Simon Study Director Senior Visiting Scholar Institute of Medicine Professor of Community Medicine School of Medicine University of California, San Diego David Tilson Director Division of International Health Karen Bell Staff Associate ix

PREFACE In the summer of 1977, Senators Kennedy, Javits, and Schweiker of the Senate Subcommittee on Health and Scientific Research expressed concern that neither component of America's large biomedical research establishment — the pharmaceutical industry's research laboratories and those in academic institutions — was devoting enough attention to the enormous unsolved health problems of the developing countries. The Senators informally requested the Institute of Medicine, in cooperation with the Department of Health, Education, and Welfare and the Pharma- ceutical Manufacturing Association, to organize a Conference on the problems associated with development and distribution of the pharmaceu- ticals needed by developing countries. They hoped the Conference would enable the Institute of Medicine to make recommendations that would encourage serious attention by American biomedical scientists to the health problems of developing countries. The Department of Health, Education, and Welfare subsequently decided to sponsor such a Conference. An Institute of Medicine Steer- ing Committee, convened in June 1978, proposed that the Conference explore four sets of inter-related issues: (l) the principal health problems of the developing world and the effectiveness, safety, and utilization of those preventive, prophylactic, and therapeutic agents now available; (2) the factors responsible for the paucity of research effort on the disease problems of developing countries by the biomedi- cal research establishments of the American pharmaceutical industry and universities; (3) relevant scientific opportunities; and (4) policy initiatives required to strengthen incentives and overcome disincen- tives for industry and the academic community to do more to help solve these problems. Knowledgeable persons from American governmental agencies, pharmaceutical firms, and academic institutions, complemented by repre- sentatives from the European pharmaceutical industry, academic science, and the World Health Organization, were asked to prepare papers and par- ticipate in discussions on these matters. The Conference was held at the National Academy of Sciences on January 29-3l, 1979. Senators xi

Kennedy, Javits, and Schweiker all participated as did Dr. Gilbert Omenn from the Office of Science and Technology Policy of the Executive Office of the President. Their statements provided both Legislative and Executive Branch perspectives on the American government's policy concerning the health problems of the developing world. These Proceedings contain the commissioned papers, prepared remarks of the invited speakers, and the summaries of the general discussions. A diversity of backgrounds and viewpoints is represented. The Proceedings were used by the Steering Committee to develop an Institute of Medicine policy paper issued under separate cover, and con- taining an analysis of the issues and recommendations for action. That paper is entitled, "Pharmaceuticals for Developing Countries: Issues and Recommendations." Harold J. Simon, M.D., Ph.D. Study Director Senior Visiting Scholar Institute of Medicine xii

OVERVIEW OF THE CONFERENCE ON "PHARMACEUTICALS FOR DEVELOPING COUNTRIES" Leighton E. Cluff Disease has often interfered with the master plans of strategists who wish to improve the economic, social, and political development of a nation. Many historical examples show that disease has had devastat- ing effects upon the population of a particular region, upon achieve- ment of commercial, economic or political objectives, and upon personal and social well being. The conquest of yellow fever, which made construction of the Panama Canal possible, was followed by elimination of this infection from the Southern United States and elsewhere, and illustrates one of the greatest practical triumphs of scientific medicine and the far reaching commercial and political consequences of controlling a single disease. Today, infectious and parasitic diseases in developing coun- tries adversely affect social, economic, and political development. If these diseases were controlled, the consequences could certainly be as dramatic as those following control of yellow fever. The health and standard of living, particularly of the poor people in these countries, could be advanced impressively. Poverty contributes to the occurrence and prevalence of many diseases, including those attributable to infection. Some of these diseases, however, can be controlled despite coexistent economic depri- vation. For example, smallpox has now been largely eliminated from the world, including the poor and developing countries. The ravages of poliomyelitis have been eased, and death rates from infantile diarrhea and cholera have been significantly lowered in both poor and affluent segments of several countries without concomitant changes in socio-eco- nomic conditions. These and other infectious diseases were controlled by widespread application of specific, effective medical interventions. Other specific and effective interventions exist and could be used to control some important infectious diseases which are particularly preva- lent in developing countries. In these instances, we must find ways to remove obstacles to and provide incentives for their use. In other instances, we must encourage and support the research needed to under- stand the diseases and the development of appropriate interventions. xiii

Some specific interventions that effectively control diseases can be administered to large populations, or to particular groups of people at risk of becoming sick. Such interventions may not require continu- ous access to personal health services such as are provided by physi- cians or other health professionals. For example, a vaccine can be administered to all children in a community or population to control a particular disease, even though there is no one regularly available to care for individual children when they are sick. In other circumstances, effective interventions can be made available only to individuals who have been evaluated by a personal health professional who can prescribe a particular treatment. Then, the entire population has to have access to personal health services if the intervention is to affect signfi- cantly the public health. Many developing countries lack effective systems and sufficient numbers of trained providers to deliver personal health services to the population at large. In addition, the public health systems in many of these countries are ill prepared to meet the needs of the total popula- tion. Therefore, even available interventions that could be effectively used to control specific diseases cannot easily be deployed. These countries will need assistance to enlarge and strengthen their personal health care services and their public health systems to distribute and use effectively interventions that are now available or that could be developed. The occurrence and prevalence of particular infectious diseases vary among the developing countries. Nevertheless, some infectious diseases affect poor and deprived people wherever they live. For exam- ple, infantile diarrhea and respiratory diseases occur commonly among poor children in every country. Other diseases, such as schistosomia- sis and cholera, are largely confined to particular geographic areas of the world. Most diseases occurring mainly in developing countries affect the poor and rural populations. As many people move from rural areas to the cities, such diseases may become less prevalent, but the expanding urban populations suffer from diseases common to areas of poverty anywhere. Most technology is difficult to apply under conditions of extreme poverty unless directed against specific problems. It is impossible to control all diseases, and it makes much more sense to direct efforts at the control of one or a few diseases at a time. Appropriate employment of available and effective interventions, and continuing search for weak links in the epidemiological and pathogenetic chains of specific diseases are important aspects of practical and successful efforts at disease control. Smallpox was controlled by a specific focus on it, not through efforts aimed at disease control in general. Although per- sonal hygiene and environmental control measures would significantly reduce the burden imposed by many infectious and parasitic diseases on the people in the developing countries, the widespread application and use of specific drugs and vaccines that can effectively control the xiv

incidence, severity, and outcome of particular diseases is also of vital importance. Production and supply of several vaccines and drugs that would effectively control specific diseases are insufficient to meet global requirements. Without planning and investment, and without a better data base on which to gauge the need, it is not possible to meet pro- jected rises in demand for these pharmaceutical agents. Systems for distribution and administration of agents to control a disease must be developed, and the required resources provided. Specific vaccines and drugs are needed in the developing countries, and such needs should per- haps be better reflected in their domestic budgetary priorities, but the cost of obtaining these pharmaceuticals cannot be met by these coun- tries without help — international cooperation and assistance will be necessary. Multinational cooperative efforts by the more affluent industrial- ized countries and by the developing countries will have to be increased if the health problems of the latter are to be dealt with effectively. The populations of industrialized countries, and particularly the United States, are insufficiently aware of the health problems of peo- ple in developing countries to make positive and rational commitments in response to their need. The legitimacy, importance, and self inter- est of Americans in the health of people in impoverished countries should be given greater attention and visibility. Popular support is necessary to strengthen public and private institutions concerned with international health, and to expand national efforts and international cooperation to address the health needs of developing countries. Never before in history have the opportunities for rational scientific and technologic approaches to health problems of developing countries been so great as they are now. The research, development, and production capacities of the pharmaceutical industry in the United States have achieved a commanding position in practical and theoretical efforts to perfect available drugs and vaccines, and to generate new agents for use in developing countries. Nonetheless, current con- straints and regulation of the pharmaceutical industry in the United States function as disincentives to capitalizing upon such opportuni- ties and accepting the challenge to develop new and improved drugs and vaccines which may find their greatest, and perhaps even their sole applicability beyond the borders of the United States. Furthermore, the amount of encouragement and support of research by academic scien- tists in the infectious disease problems of primary concern to develop- ing countries has been less than that necessary to exploit emerging scientific opportunities and challenges. Academic scientists provide most of the fundamental information needed for the practical develop- ment of new or improved medical interventions. The pharmaceutical industry, on the other hand, has organized the multidisciplinary approaches required for development and production of these agents. Natural linkages exist between the efforts of academic scientists and xv

the interests of the pharmaceutical industry. These linkages must be strengthened to optimize the efforts of both communities. Neither academic scientists nor the pharmaceutical industry can confine their activities to the United States in conducting all the studies needed to investigate diseases which occur only in developing countries. If the expertise in the United States is to be directed towards the study and practical development of agents to control dis- eases principally occurring in developing countries, arrangements must be made for some of these efforts to be conducted in developing coun- tries. Political, financial, and scientific cooperation between devel- oping countries and the United States is essential if academic scien- tists and the pharmaceutical industry are to contribute effectively toward control of disease in the developing world. At present, legal, regulatory, fiscal, political, and social constraints compromise imple- mentation of such efforts. They must be modified or removed before much can be accomplished. Improving international health requires better partnership arrange- ments between government, industry, academic, public, and private insti- tutions — supported by popular understanding and encouragement. Any- thing less will compromise optimal contributions to the health needs of the poor and sick in the many developing countries. Furthermore, failure to implement intensive efforts to control diseases prevalent in developing countries will significantly impair any and all other efforts toward facilitation of socio-economic advancement in impoverished and heavily populated countries. xvi

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