STRENGTHENING A

WORKFORCE FOR
INNOVATIVE
REGULATORY SCIENCE

IN THERAPEUTICS DEVELOPMENT

Workshop Summary

Steve Olson and Anne B. Claiborne, Rapporteurs

Forum on Drug Discovery, Development, and Translation

Board on Health Sciences Policy

INSTITUTE OF MEDICINE
OF THE NATIONAL ACADEMIES

THE NATIONAL ACADEMIES PRESS
Washington, D.C.
www.nap.edu



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STRENGTHENING A WORKFORCE FOR INNOVATIVE REGULATORY SCIENCE IN THERAPEUTICS DEVELOPMENT Workshop Summary Steve Olson and Anne B. Claiborne, Rapporteurs Forum on Drug Discovery, Development, and Translation Board on Health Sciences Policy

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THE NATIONAL ACADEMIES PRESS 500 Fifth Street, N.W. Washington, DC 20001 NOTICE: The project that is the subject of this report was approved by the Govern- ing Board of the National Research Council, whose members are drawn from the councils of the National Academy of Sciences, the National Academy of Engineer- ing, and the Institute of Medicine. This study was supported by contracts between the National Academy of Sciences and Department of Health and Human Services (Contract Nos. N01-OD-4-2139 TO #158 and HHSF223001003T), American Society for Microbiology, Amgen Inc., Association of American Medical Colleges, Bristol-Myers Squibb, Burroughs Wellcome Fund, Celtic Therapeutics, LLLP, Critical Path Institute, Doris Duke Charitable Foundation, Eli Lilly & Co., FasterCures, Foundation for the NIH, Friends of Cancer Research, GlaxoSmithKline, Johnson & Johnson, Merck & Co., Inc., Novartis Pharmaceuticals Corporation, and Pfizer Inc. Any opinions, find - ings, conclusions, or recommendations expressed in this publication are those of the authors and do not necessarily reflect the view of the organizations or agencies that provided support for this project. International Standard Book Number-13: 978-0-309-22214-3 International Standard Book Number-10: 0-309-22214-1 Additional copies of this report are available from the National Academies Press, 500 Fifth Street, N.W., Lockbox 285, Washington, DC 20055; (800) 624-6242 or (202) 334-3313 (in the Washington metropolitan area); Internet, http://www.nap.edu. For more information about the Institute of Medicine, visit the IOM home page at: www.iom.edu. Copyright 2012 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 Staatliche Museen in Berlin. Suggested citation: IOM (Institute of Medicine). 2012. Strengthening a Workforce for Innovative Regulatory Science in Therapeutics Development: Workshop Summary. Washington, DC: The National Academies Press.

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“Knowing is not enough; we must apply. Willing is not enough; we must do.” — Goethe Advising the Nation. Improving Health.

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The National Academy of Sciences is a private, nonprofit, self-perpetuating society of distinguished scholars engaged in scientific and engineering research, dedicated to the furtherance of science and technology and to their use for the general welfare. Upon the authority of the charter granted to it by the Congress in 1863, the Academy has a mandate that requires it to advise the federal govern - ment on scientific and technical matters. Dr. Ralph J. Cicerone is president of the National Academy of Sciences. The National Academy of Engineering was established in 1964, under the charter of the National Academy of Sciences, as a parallel organization of outstanding engineers. It is autonomous in its administration and in the selection of its mem - bers, sharing with the National Academy of Sciences the responsibility for advis - ing the federal government. The National Academy of Engineering also sponsors engineering programs aimed at meeting national needs, encourages education and research, and recognizes the superior achievements of engineers. Dr. Charles M. Vest is president of the National Academy of Engineering. The Institute of Medicine was established in 1970 by the National Academy of Sciences to secure the services of eminent members of appropriate professions in the examination of policy matters pertaining to the health of the public. The Institute acts under the responsibility given to the National Academy of Sciences by its congressional charter to be an adviser to the federal government and, upon its own initiative, to identify issues of medical care, research, and education. Dr. Harvey V. Fineberg is president of the Institute of Medicine. The National Research Council was organized by the National Academy of Sciences in 1916 to associate the broad community of science and technology with the Academy’s purposes of furthering knowledge and advising the fed - eral government. Functioning in accordance with general policies determined by the Academy, the Council has become the principal operating agency of both the National Academy of Sciences and the National Academy of Engineering in providing services to the government, the public, and the scientific and engineer- ing communities. The Council is administered jointly by both Academies and the Institute of Medicine. Dr. Ralph J. Cicerone and Dr. Charles M. Vest are chair and vice chair, respectively, of the National Research Council. www.national-academies.org

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PLANNING COMMITTEE FOR THE WORKSHOP ON STRENGTHENING A WORKFORCE FOR INNOVATIVE REGULATORY SCIENCE IN THERAPEUTICS DEVELOPMENT1 BARRY S. COLLER (Co-Chair), The Rockefeller University, New York, NY ELAINE K. GALLIN (Co-Chair), QE Philanthropic Advisors, Potomac, MD GAIL H. CASSELL, Harvard Medical School (visiting), Carmel, IN GARRET A. FITZGERALD, University of Pennsylvania School of Medicine, Philadelphia JESSE L. GOODMAN, Food and Drug Administration, Silver Spring, MD HARRY B. GREENBERG, Stanford University School of Medicine, Stanford, CA STEPHEN GROFT, National Institutes of Health, Bethesda, MD SHARON HESTERLEE, Parent Project Muscular Dystrophy, Tucson, AZ PETRA KAUFMANN, National Institute of Neurological Disorders and Stroke, Bethesda, MD JACK D. KEENE, Duke University Medical Center, Durham, NC FREDA LEWIS-HALL, Pfizer Inc., New York, NY MICHAEL E. MENDELSOHN, Merck & Co., Inc., Rahway, NJ AMY PATTERSON, National Institutes of Health, Bethesda, MD CARL PECK, University of California, San Francisco NANCY SUNG, Burroughs Wellcome Fund, Research Triangle Park, NC LESLIE D. WHEELOCK, Food and Drug Administration, Silver Spring, MD JANET WOODCOCK, Food and Drug Administration, Rockville, MD Study Staff ANNE B. CLAIBORNE, Forum Director REBECCA A. ENGLISH, Associate Program Officer ELIZABETH F. C. TYSON, Research Associate ANDREW M. POPE, Director, Board on Health Sciences Policy ROBIN GUYSE, Senior Program Assistant 1 Institute of Medicine planning committees are solely responsible for organizing the workshop, identifying topics, and choosing speakers. The responsibility for the published workshop summary rests with the workshop rapporteurs and the institution. v

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FORUM ON DRUG DISCOVERY, DEVELOPMENT, AND TRANSLATION1 JEFFREY M. DRAZEN (Co-Chair), New England Journal of Medicine, Boston, MA STEVEN K. GALSON (Co-Chair), Amgen Inc., Thousand Oaks, CA MARGARET ANDERSON, FasterCures, Washington, DC HUGH AUCHINCLOSS, National Institute of Allergy and Infectious Diseases, Bethesda, MD LESLIE Z. BENET, University of California-San Francisco ANN BONHAM, Association of American Medical Colleges, Washington, DC LINDA BRADY, National Institute of Mental Health, Bethesda, MD ROBERT CALIFF, Duke University Medical Center, Durham, NC SCOTT CAMPBELL, Foundation for the National Institutes of Health, Bethesda, MD C. THOMAS CASKEY, Baylor College of Medicine, Houston, TX GAIL H. CASSELL, Harvard Medical School (visiting), Carmel, IN PETER B. CORR, Celtic Therapeutics, LLLP, New York, NY ANDREW M. DAHLEM, Eli Lilly and Company, Indianapolis, IN TAMARA DARSOW, American Diabetes Association, Alexandria, VA JAMES H. DOROSHOW, National Cancer Institute, Bethesda, MD GARY L. FILERMAN, Atlas Health Foundation, McLean, VA GARRET A. FITZGERALD, University of Pennsylvania School of Medicine, Philadelphia MARK J. GOLDBERGER, Abbott, Rockville, MD HARRY B. GREENBERG, Stanford University School of Medicine, Stanford, CA STEPHEN GROFT, National Institutes of Health, Bethesda, MD LYNN HUDSON, Critical Path Institute, Tuscon, AZ THOMAS INSEL, National Center for Advancing Translational Sciences, Bethesda, MD MICHAEL KATZ, March of Dimes Foundation, White Plains, NY PETRA KAUFMANN, National Institute of Neurological Disorders and Stroke, Bethesda, MD JACK D. KEENE, Duke University Medical Center, Durham, NC RONALD L. KRALL, University of Pennsylvania, Center for Bioethics, Steamboat Springs, CO FREDA LEWIS-HALL, Pfizer Inc., New York, NY 1Institute of Medicine forums and roundtables do not issue, review, or approve individual documents. The responsibility for the published workshop summary rests with the work - shop rapporteurs and the institution. vii

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MARK B. McCLELLAN, The Brookings Institution, Washington, DC CAROL MIMURA, University of California-Berkeley ELIZABETH (BETSY) MYERS, Doris Duke Charitable Foundation, New York, NY JOHN ORLOFF, Novartis Pharmaceuticals Corporation, East Hanover, NJ AMY PATTERSON, National Institutes of Health, Bethesda, MD MICHAEL ROSENBLATT, Merck & Co., Inc., Whitehouse Station, NJ JANET SHOEMAKER, American Society for Microbiology, Washington, DC ELLEN SIGAL, Friends of Cancer Research, Washington, DC ELLIOTT SIGAL, Bristol-Myers Squibb, Princeton, NJ ELLEN R. STRAHLMAN, GlaxoSmithKline, Research Triangle Park, NC NANCY SUNG, Burroughs Wellcome Fund, Research Triangle Park, NC JANET TOBIAS, Ikana Media and Mount Sinai School of Medicine, New York, NY JOANNE WALDSTREICHER, Johnson & Johnson, Raritan, NJ JANET WOODCOCK, Food and Drug Administration, White Oak, MD Study Staff ANNE B. CLAIBORNE, Forum Director RITA S. GUENTHER, Program Officer REBECCA A. ENGLISH, Associate Program Officer ELIZABETH F. C. TYSON, Research Associate ANDREW M. POPE, Director, Board on Health Sciences Policy ROBIN GUYSE, Senior Program Assistant viii

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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 institution in making its pub- lished report as sound as possible and to ensure that the report meets institutional standards for objectivity, evidence, and responsiveness to the study charge. The review comments and draft manuscript remain confidential to protect the integrity of the process. We wish to thank the following individuals for their review of this report: William W. Chin, Harvard Medical School H. Clifford Lane, National Institute of Allergy and Infectious Diseases, National Institutes of Health Michael Manganiello, HCM Strategists, LLC Brian L. Strom, University of Pennsylvania School of Medicine Although the reviewers listed above have provided many constructive comments and suggestions, they were not asked to endorse the final draft of the report before its release. The review of this report was overseen by Hugh Tilson, University of North Carolina at Chapel Hill. Appointed by the Institute of Medicine, he was responsible for making certain that an independent examination of this report was carried out in accordance with institutional procedures and that all review comments were carefully considered. Responsibility for the final content of this report rests entirely with the authors and the institution. ix

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Contents ACRONYMS xv 1 INTRODUCTION 1 Scope of the Workshop and Organization of the Summary, 3 2 THE IMPORTANCE OF INNOVATIVE REGULATORY SCIENCE 5 Perspective from the Food and Drug Administration, 6 Perspective from the National Institutes of Health, 11 Perspective from the Pharmaceutical Industry, 13 Perspective from Academia, 15 Patient Perspective, 16 Principles and Themes, 17 3 DEFINING A DISCIPLINE OF REGULATORY SCIENCE AND CORE COMPETENCIES FOR ITS WORKFORCE 19 Defining Regulatory Science Through the Lens of Translational Science, 20 Defining Regulatory Science as Science of Evaluation of Regulations, 22 Case Studies: Regulatory Science in Practice, 24 Core Competencies of Regulatory Science, 24 Defining Regulatory Science, 28 xi

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xii CONTENTS 4 EDUCATION AND TRAINING OF A REGULATORY SCIENCE WORKFORCE 31 An Overview of Existing Training Programs, 32 Developing Education and Training Programs in Regulatory Science, 34 Models for Education and Training, 36 Fellowships and Exchange Programs, 37 5 CAREER PATHS WITHIN ACADEMIA AND INDUSTRY 43 Career Paths in Academia, 44 Career Paths in Industry, 47 6 INTERNATIONAL APPLICATIONS OF REGULATORY SCIENCE 51 Maintaining a Robust Global Therapeutics Pipeline, 52 Therapeutics Development for Global Neglected Diseases, 54 7 COLLABORATIVE MODELS AND NEW PARADIGMS FOR SUPPORTING REGULATORY SCIENCE RESEARCH AND PRACTICE 57 Creating a Collaborative Environment in an Academic Setting, 58 A Collaborative Model for Research, Training, and Business Development, 61 Regulatory Science: Solving for a Larger Context, 61 Closing Panel, 63 REFERENCES 65 APPENDIXES A WORKSHOP AGENDA 67 B PARTICIPANT BIOGRAPHIES 81

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Figures and Boxes FIGURES 2-1 Many discoveries fail to traverse the “valley of death” from discovery to commercial product, 8 4-1 PharmaTrain has a three-tier program of postgraduate training with optional extension after completing each level, 37 5-1 Trends in the pharmaceutical industry led to a “perfect storm” leading to increased regulatory science demands, 48 7-1 The New Drug Development Paradigms (NEWDIGS) initiative involves design teams that progress through established modules to engage in innovative regulatory science activities, 59 BOXES 2-1 FDA Strategic Plan for Regulatory Science, 10 3-1 Collaboration in Cystic Fibrosis Research, 25 3-2 Drug Safety, 26 3-3 Disciplinary Components of Regulatory Science, 27 5-1 A Nonexhaustive List of the “Big Questions” Identified by Participants, 47 xiii

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Acronyms CBER Center for Biologics Evaluation and Research CDC Centers for Disease Control and Prevention CDER Center for Drug Evaluation and Research CDSS Centre for Drug Safety Science CERSI Center for Excellence in Regulatory Science and Innovation CF cystic fibrosis CFP Commissioner’s Fellowship Program CRO contract research organization CTP Center for Tobacco Products CTSA Clinical and Translational Science Awards ECPM European Center of Pharmaceutical Medicine EMA European Medicines Agency EU European Union FDA U.S. Food and Drug Administration HRA Health Research Alliance IMI Innovative Medicines Initiative IND Investigational New Drug IOM Institute of Medicine IOTF Interagency Oncology Task Force IRB Institutional Review Board xv

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xvi ACRONYMS MCM medical countermeasure MIT Massachusetts Institute of Technology NCATS National Center for Advancing Translational Sciences NCI National Cancer Institute NDA new drug application NEWDIGS New Drug Development Paradigms NIAID National Institute of Allergy and Infectious Diseases NIH National Institutes of Health NINDS National Institute of Neurological Disorders and Stroke PDP product development partnership PharmaTrain Pharmaceutical Medicine Training Programme RAPS Regulatory Affairs Professional Society RFA Request for Application TB tuberculosis UCSF University of California, San Francisco UNC University of North Carolina at Chapel Hill USC University of Southern California