The Children's Vaccine Initiative

Achieving the Vision

Violaine S. Mitchell, Nalini M. Philipose, and Jay P. Sanford, Editors

Committee on the Children's Vaccine Initiative: Planning Alternative Strategies Toward Full U.S. Participation

Division of International Health

INSTITUTE OF MEDICINE

NATIONAL ACADEMY PRESS
Washington, D.C.
1993



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The Children's Vaccine Initiative: Achieving the Vision The Children's Vaccine Initiative Achieving the Vision Violaine S. Mitchell, Nalini M. Philipose, and Jay P. Sanford, Editors Committee on the Children's Vaccine Initiative: Planning Alternative Strategies Toward Full U.S. Participation Division of International Health INSTITUTE OF MEDICINE NATIONAL ACADEMY PRESS Washington, D.C. 1993

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The Children's Vaccine Initiative: Achieving the Vision 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. This 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 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. Support for this project was provided by the Agency for International Development; the Department of Health and Human Services; the Pharmaceutical Manufacturers Association; the United Nations Development Program; and the World Health Organization, Children’s Vaccine Initiative. Library of Congress Catalog Card No. 93-84552 International Standard Book Number 0-309-04940-7 Additional copies of this report are available from: National Academy Press 2101 Constitution Avenue, N.W. Box 285 Washington, D.C. 20055 Call 800-624-6242 or 202-334-3313 (in the Washington Metropolitan Area) B179 Copyright 1993 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 serpent adopted as a logotype by the Institute of Medicine is a relief carving from ancient Greece, now held by the Staatlichemuseen in Berlin. COVER: Samantha Edington (age 4 years) provided the cover drawing for this report. Thanks are also due to Stefanie Hairston (5), Mira Kelada-Antoun (4), Stephanie Howson (6), Nadia Scott (4), and Anna Stoto (6).

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The Children's Vaccine Initiative: Achieving the Vision COMMITTEE ON THE CHILDREN'S VACCINE INITIATIVE: PLANNING ALTERNATIVE STRATEGIES TOWARD FULL U.S. PARTICIPATION JAY P. SANFORD (Chair),* Dean Emeritus, Uniformed Services University of the Health Sciences, Dallas, Texas MARY LOU CLEMENTS, Professor and Head, Division of Vaccine Sciences, Department of International Health, Johns Hopkins School of Hygiene and Public Health, Baltimore, Maryland CIRO de QUADROS, Regional Advisor, Pan American Health Organization, Washington, D.C. MICHAEL A. EPSTEIN, Partner, Weil, Gotshal & Manges, New York, New York RONALD W. HANSEN, Associate Dean for Academic Affairs, William E. Simon Graduate School of Business Administration, University of Rochester, Rochester, New York DONALD E. HILL, President, Don Hill & Associates, Silver Spring, Maryland JOHN LLOYD HUCK (Retired), Former Chairman of the Board, Merck & Co., Inc., New Vernon, New Jersey DAVID T. KARZON, Professor, Department of Pediatrics, and Department of Microbiology and Immunology, Vanderbilt University School of Medicine, Nashville, Tennessee THOMAS D. KILEY, Attorney, Hillsborough, California RICHARD T. MAHONEY, Vice President and Director, Technology Promotion, Program for Appropriate Technology in Health, Seattle, Washington WENDY K. MARINER, Professor of Health Law, Boston University School of Public Health, Boston, Massachusetts DAVID C. MOWERY, Associate Professor of Business and Public Policy, Walter A. Haas School of Business, University of California, Berkeley. MARK NOVITCH, Vice Chairman of the Board, The Upjohn Company, Kalamazoo, Michigan SURYANARAYAN RAMACHANDRAN, Immediate Past Secretary, Department of Biotechnology, New Delhi, India ANTHONY ROBBINS, Professor of Public Health, Boston University School of Public Health, Boston, Massachusetts JERALD C. SADOFF, Director, Division of Communicable Diseases and Immunology, Walter Reed Army Institute for Research, Washington, D.C. *   Member, Institute of Medicine

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The Children's Vaccine Initiative: Achieving the Vision GEORGE R. SIBER, Director, Massachusetts Public Health Biologic Laboratories, Massachusetts Department of Health, Jamaica Plain, Massachusetts JANE E. SISK, Professor, Columbia University School of Public Health, New York, New York Former Member WILLIAM A. PACKER, President, Virus Research Institute, Cambridge, Massachusetts, and former Senior Vice President and Director, SmithKline Beecham Biologicals & Strategic Projects, Philadelphia, Pennsylvania Institute of Medicine Staff VIOLAINE S. MITCHELL, Study Director (from September 1992) STEPHANIE R. SAGEBIEL, Study Director (until September 1992) NALINI M. PHILIPOSE, Research Assistant DEE SUTTON, Administrative Assistant (from July 1992) AMY STRONG, Senior Secretary (until July 1992) POLLY F. HARRISON, Director, Division of International Health SUSAN M. WYATT, Financial Associate MICHAEL EDINGTON, Managing Editor Consultants ROBERT D. CRANGLE, President, Rose & Crangle, Ltd., Lincoln, Kansas MICHAEL K. HAYES, Contract Editor GREG W. PEARSON, Consultant Editor/Writer

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The Children's Vaccine Initiative: Achieving the Vision Preface Vaccines are among the most cost-effective health interventions known. Indeed, the development and widespread use of vaccines in developed and developing countries have contributed greatly to the prevention of many devastating childhood diseases. Progress has been particularly impressive in the two decades since the establishment of the Expanded Program on Immunization under the leadership of the World Health Organization. Unfortunately, a significant percentage of children, most in the poorest and most remote regions of the world, are not adequately immunized with existing vaccines. Underimmunization is also a problem in the United States, particularly among economically disadvantaged children living in rural and urban areas. Furthermore, no effective vaccines exist for a number of important infectious childhood illnesses. The Children's Vaccine Initiative was launched at the World Summit for Children in New York City in September 1990 to address these and other concerns related to childhood immunization. This Institute of Medicine report, which addresses the central question, "How can the United States participate fully in the implementation of the Children's Vaccine Initiative?," provides important background information about the status of childhood immunization in this country and abroad, the available resources and infrastructure for producing vaccines, the supply of and demand for new and improved vaccines, the multistep process of vaccine research and development, and the dynamics of developing and manufacturing new and improved vaccines. In developing our conclusions and recommendations, the Institute of

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The Children's Vaccine Initiative: Achieving the Vision Medicine Committee on the Children's Vaccine Initiative has drawn on the expertise of individual committee members and has sought the participation and input of many individuals connected to the research, development, procurement, and supply of vaccines both domestically and internationally. The committee recognized early on in the study process that effective and efficient vaccine distribution and delivery systems are critical to ensuring the ultimate goal of disease prevention, but because this was not included in the charge to the committee, it is discussed only briefly in this report. It is the conclusion of the Institute of Medicine Committee on the Children's Vaccine Initiative that the current system of vaccine research and development in the United States, which leads to the development of high-quality vaccine products for the domestic market, is unlikely to produce the majority of vaccines required by the Children's Vaccine Initiative. In addition, the committee believes that although the combined resources and expertise of the public and private sectors in the United States for the development and production of vaccines are both significant and impressive, they are not integrated and are not focused effectively on meeting public health goals. These conclusions led the committee to its major recommendation: the need for a National Vaccine Authority. The committee believes that a National Vaccine Authority, through a dynamic partnership between the public and private sectors, will offer the United States an extremely powerful tool to ensure the development of novel vaccines and vaccine technologies for use in immunization programs in the United States and around the world. Publication of this report has been preceded by considerable national discussion about the desirability of having the U.S. government take a greater role in the purchase and distribution of vaccines recommended for use in U.S. children. The Institute of Medicine Committee on the Children's Vaccine Initiative did not study, and has not taken a position on an expanded federal purchase of vaccines. I believe I speak for the committee, however, when I say that certain sections of this report have relevance to the on-going discussion. The committee forwards its recommendations having recognized that the curtailment of the burden of disease and death in the twenty-first century throughout the world, including within the United States, is another step toward the goal of a peaceful future for ourselves and our children. Jay P. Sanford, Chair Committee on the Children's Vaccine Initiative

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The Children's Vaccine Initiative: Achieving the Vision Acknowledgments Being asked to serve on, or in my instance to chair, an Institute of Medicine (IOM) committee is not a role that one assumes lightly. The Institute of Medicine does not address issues which are not of significant importance and social consequence, and the challenges of serving on an IOM committee are great and demanding. At the same time, one can be assured that one's colleagues will be dedicated to the task, expert in their fields, and collegial in their approach. One can also be confident that support by the IOM leadership and staff will be exceptional. On behalf of the Institute of Medicine Committee on the Children's Vaccine Initiative, I would like to express our sincere appreciation to the numerous individuals and organizations who gave of their valuable time to provide information and advice to the committee. The committee would like to thank all the individuals who participated in the two working group sessions in June 1992 and who are listed in Appendix I . In addition, the committee would like to thank the following individuals who provided valuable information and advice to the committee: Kenneth Bart of the National Vaccine Program; Kenneth Bernard and Linda Vogel of the Office of International Health; Frank Cano and Jane Scott of Lederle-Praxis Biologicals; Glenna Crooks of Merck and Company, Inc.; George Curlin, Carole Heilman, John LaMontagne, Pamela McGinnis, Regina Rabinovich, and Dale Spriggs of the National Institute of Allergy and Infectious Diseases; Elaine Esber, M. Carolyn Hardegree, and Amy Scott of the U.S. Food and Drug Administration; Scott Halstead and Bruce Gellin of the Rockefeller Foundation; William Haussdorf, Pamela Johnson, Caryn Miller,

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The Children's Vaccine Initiative: Achieving the Vision and Michael White of the Agency for International Development; D. A. Henderson of the Department of Health and Human Service; Phyllis Freeman of the University of Massachusetts at Boston; Lance Gordon of ORAVAX; Sharon Mates of North American Vaccine; William Packer of Virus Research Institute; Ok Pannenborg of the World Bank; Philip K. Russell of Johns Hopkins University; Roy Widdus of the National Commission on AIDS; and Douglas Williams of Connaught Laboratories, Inc. The committee gratefully acknowledges those who provided tables, graphs, funding data, and other information critical to the committee's deliberations: Janice Babcock of the Vaccine Project at the University of Massachusetts at Boston; Col. William Bancroft of the United States Army Medical Research and Development Command; Amie Batson, Peter Evans, and David Magrath of the World Health Organization; Matthew Berry and Geoffrey Evans of the Health Resources Services Administration; Timothy Brogan and Thomas Copmann at the Pharmaceutical Manufacturers Association; John Gilmartin and Terrel Hill of the United Nations Children's Fund; Tore Godal of the United Nations Development Program/World Health Organization/World Bank Special Program for Research and Training in Tropical Diseases; Akira Homma of the Pan American Health Organization; Major Robert Lipnick of the Department of the Army; Joseph McDade of the National Center for Infectious Diseases and Bill Nichols of the Division of Immunization at the Centers for Disease Control and Prevention; Robert Myers of the Michigan Department of Public Health; Lt. Col. Willis Reid of the Walter Reed Army Institute for Research; and Chester Robinson of the National Vaccine Program Office. Finally, and in particular, the committee would like to express its appreciation of the Institute of Medicine staff who facilitated the work of this committee. We especially thank Violaine Mitchell, who assumed responsibility for the study midstream and was responsible for translating on-going discussions and debate into prose for further committee discussion; Nalini Philipose for her substantive and procedural contributions to the committee's work; and Dee Sutton, for her superb logistical support over the course of the study. The committee gratefully acknowledges the support and contribution of Stephanie R. Sagebiel who launched the study. Others within the IOM and National Academy of Sciences who were instrumental in seeing the project to completion were Michael Edington, Claudia Carl, and Betsy Turvene of the Reports and Information Office; Laura Baird, research librarian; Scott Jones, computer analyst; Sue Wyatt, financial associate; and Carolyn Hall, Dana Hotra, and Sharon Scott-Brown of the Division of International Health. Special thanks are due to Greg Pearson who edited and wrote sections of the report; Michael Hayes, editorial consultant; and Robert Crangle who facilitated the working group meetings

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The Children's Vaccine Initiative: Achieving the Vision in June 1992. The committee would also like to thank Kenneth I. Shine, IOM President; Enriqueta C. Bond, IOM Executive Officer; and Polly F. Harrison, Director of the Division of International Health, for their ongoing support of this study. The committee would also like to thank the study sponsors for their willingness to provide both support and input without attempting to influence the deliberations of the committee in any way. Jay P. Sanford, Chair Committee on the Children's Vaccine Initiative

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The Children's Vaccine Initiative: Achieving the Vision Contents 1   EXECUTIVE SUMMARY   1 2   WHY A CHILDREN'S VACCINE INITIATIVE   19     Progress Toward Universal Childhood Immunization   19     Limits of the Expanded Program on Immunization   20     The Children's Vaccine Initiative   21     Characteristics of CVI Vaccines   24     Concerns about the CVI   30     Relevance of the CVI to the U.S. Immunization Efforts   31     Status of Immunization Efforts in the United States   31     A Role for the CVI in the United States   32 3   RESOURCES AND INFRASTRUCTURE   36     Resources in the United States   36     U.S. Federal Agencies and Programs   37     U.S. State Vaccine Manufacturers   47     U.S.-Based Pharmaceutical Firms   48     Development-Stage Companies   51     Nongovernmental Organizations   53     International Resources   55     Multilateral Organizations   56     Public-Sector Resources   60     Private-Sector Resources   61     Nongovernmental Organizations   63

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The Children's Vaccine Initiative: Achieving the Vision 4   VACCINE DEMAND AND SUPPLY   66     Global Demand and Supply   66     Demand   66     Supply   68     Innovation   74     Demand and Supply in the United States   75     Demand   75     Supply   76     Vaccine Innovation   82 5   INVESTING IN NEW AND IMPROVED VACCINES   98     Market Considerations   100     Market Size   101     Intellectual Property   103     Liability   106 6   STAGES OF VACCINE DEVELOPMENT   109     Priority Setting   110     Basic and Applied Research   111     Clinical Evaluation   111     Licensure   114     Production   115     Pilot Production   115     ScaleUp and Full-Scale Manufacture   118     Vaccine Production in Developing Countries   119     Recommendations for Use   120     Procurement   122     Distribution and Delivery   123     Surveillance   123     Immunization Status   123     Monitoring Effectiveness of Vaccines   124     Adverse Reactions   124     Setting Priorities for Vaccine Use and New Vaccines   125 7   A STRATEGY TO ENHANCE U.S. PARTICIPATION IN THE CHILDREN'S VACCINE INITIATIVE   128     A National Vaccine Authority   129     Mission   130     Functions   132     Management and Organization   138     Funding   138     Location   140

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The Children's Vaccine Initiative: Achieving the Vision     APPENDIXES         A Relevant Intellectual Property Rights Law   149     B National Vaccine Injury Compensation Program   160     C Regulatory Aspects of Vaccine Development, Manufacture, and Distribution   164     D Strategies for Achieving Full U.S. Participation in the Children's Vaccine Initiative   170     E Public Health Service Act (1944)   184     F National Vaccine Program Legislation   189     G Immunization Schedules   193     H Historical Record of Vaccine Product License Holders in the United States   196     I Working Group Participants   205     J Committee and Staff Biographies   210     K Acronyms   220

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The Children's Vaccine Initiative: Achieving the Vision Children represent the most vulnerable segment of every society... they are our present and our future. Declaration of New York, September 1990

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The Children's Vaccine Initiative: Achieving the Vision The Children's Vaccine Initiative Achieving the Vision

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