ACCELERATING THE DEVELOPMENT
OF NEW DRUGS AND DIAGNOSTICS

MAXIMIZING THE IMPACT OF THE
CURES ACCELERATION NETWORK

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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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, NW Washington, DC 20001 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. 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 and HHSF223001003T), Abbott Pharmaceuticals, American Diabetes Association, 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, Janssen Research & Development, LLC, March of Dimes Foundation, Merck & Co., Inc., Novartis Pharmaceuticals Corporation, and Pfizer Inc. The views presented in this publication do not necessarily reflect the views of the organizations or agencies that provided support for the project. International Standard Book Number-13: 978-0-309-26116-6 International Standard Book Number-10: 0-309-26116-3 Additional copies of this report are available for sale from the National Academies Press, 500 Fifth Street, NW, Keck 360, Washington, DC 20001; (800) 624-6242 or (202) 334-3313; 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. Accelerating the Develop- ment of New Drugs and Diagnostics: Maximizing the Impact of the Cures Acceleration Network: 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 MAXIMIZING THE GOALS OF THE CURES ACCELERATION NETWORK TO ACCELERATE THE DEVELOPMENT OF NEW DRUGS AND DIAGNOSTICS1 CAROLYN COMPTON (Co-Chair), Critical Path Institute, Tucson, AZ LOUIS J. DEGENNARO (Co-Chair), the Leukemia & Lymphoma Society, White Plains, NY MARGARET ANDERSON, FasterCures, Washington, DC ANN BONHAM, Association of American Medical Colleges, Washington, DC C. THOMAS CASKEY, Baylor College of Medicine, Houston, TX WILLIAM W. CHIN, Harvard Medical School, Boston, MA PETER B. CORR, Celtic Therapeutics, LLLP, New York, NY PAUL R. EISENBERG, Amgen Inc., Thousand Oaks, CA KATHY L. HUDSON, National Institutes of Health, Bethesda, MD ANKIT MAHADEVIA, Atlas Venture, Cambridge, MA ROBERT T. O'NEILL, Food and Drug Administration, Chestnut Hill, MA BARRY PALLOTTA, Defense Advanced Research Projects Agency, Arlington, VA MARY K. PENDERGAST, Pendergast Consulting, Washington, DC ELLEN V. SIGAL, Friends of Cancer Research, Washington, DC WILLIAM L. WARREN, Sanofi Pasteur, Orlando, FL IOM Staff ANNE B. CLAIBORNE, Forum Director ADRIENNE STITH BUTLER, Senior Program Officer REBECCA A. ENGLISH, Associate Program Officer ELIZABETH F. C. TYSON, Research Associate ROBIN GUYSE, Senior Program Assistant ANDREW M. POPE, Director, Board on Health Sciences Policy 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 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 & Co., 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 Pharmaceuticals, Rockville, MD HARRY B. GREENBERG, Stanford University School of Medicine, CA STEPHEN GROFT, National Center for Advancing Translational Sciences, Bethesda, MD LYNN HUDSON, Critical Path Institute, Tucson, 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 MARK B. McCLELLAN, The Brookings Institution, Washington, DC CAROL MIMURA, University of California, Berkeley 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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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, Janssen Research & Development, LLC, Raritan, NJ JANET WOODCOCK, Food and Drug Administration, White Oak, MD IOM Staff ANNE B. CLAIBORNE, Forum Director RITA S. GUENTHER, Program Officer REBECCA A. ENGLISH, Associate Program Officer ELIZABETH F. C. TYSON, Research Associate ROBIN GUYSE, Senior Program Assistant ANDREW M. POPE, Director, Board on Health Sciences Policy 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: Barry S. Coller, The Rockefeller University Garry A. Neil, Johnson & Johnson Mary K. Pendergast, Pendergast Consulting Heather M. Snyder, Alzheimer's Association Although the reviewers listed above have provided many construc- tive comments and suggestions, they did not see the final draft of the report before its release. The review of this report was overseen by Brian L. Strom, University of Pennsylvania Perelman School of Medicine. 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 ACRONYMSxv 1INTRODUCTION 1 The History and Goals of CAN, 2 Overview of the Themes of the Workshop, 11 2 APPROACHES TO ACCELERATING TRANSLATIONAL SCIENCE 15 Cystic Fibrosis and the Need for Partnerships, 16 Alzheimer's Disease and the Drug Development Ecosystem, 18 NUT Midline Carcinoma and Open Innovation, 21 Sickle Cell Anemia and the Need for Hedgehogs, 23 Leukemia and Lymphoma and the Need for Partnerships, 25 Cross-Cutting Issues, 27 3 APPLICATION OF MATCHING AUTHORITY 29 The Small Business Innovation Research Program at the National Cancer Institute, 30 The Matching Requirement at the Cancer Prevention and Research Institute of Texas, 32 The Matching Requirement at the California Institute for Regenerative Medicine, 33 Perspectives of Matchers, 36 xi

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xiiCONTENTS 4 APPLICATION OF FLEXIBLE RESEARCH AUTHORITY 39 OTA at DARPA, 40 ARPA-E at the Department of Energy, 43 Promoting an Effective Medical Countermeasures Enterprise at BARDA, 45 OTA at DTRA, 47 Collaboration at the CHDI Foundation, 48 5 SITUATING CAN WITHIN THE DRUG DEVELOPMENT ECOSYSTEM 51 FDA, 52 The Office of Science and Technology Policy, 57 The Pharmaceutical Industry, 58 PublicPrivate Partnerships, 60 Tools and Cures, 63 6 FINAL REFLECTIONS ON WAYS TO MAXIMIZE THE GOALS OF CAN 65 Responding to Frustration, 66 A Rallying Cry, 67 Mastering the Details, 67 Beyond Rocket Science, 68 Changing the Culture and Showing Deliverables, 68 "The Status Quo Is Not Acceptable," 69 REFERENCES71 APPENDIXES A WORKSHOP AGENDA 73 B PUBLIC HEALTH SERVICE ACT, TITLE IV-- NATIONAL INSTITUTES OF HEALTH 87 C PARTICIPANT BIOGRAPHIES95

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Boxes 1-1 Statement of Task for the Workshop, 3 1-2 The Need for Translational Science, 6 1-3 Functions of CAN in Authorizing Legislation, 7 xiii

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Acronyms AHRQ Agency for Healthcare Research and Quality ARPA-E Advanced Research Projects AgencyEnergy BARDA Biomedical Advanced Research and Development Authority CAN Cures Acceleration Network CBRN chemical, biological, radiological, and nuclear CDC U.S. Centers for Disease Control and Prevention CDER Center for Drug Evaluation and Research CDRH Center for Devices and Radiological Health CIRM California Institute for Regenerative Medicine C-Path Critical Path Institute CPRIT Cancer Prevention and Research Institute of Texas CTSA Clinical and Translational Science Award DARPA Defense Advanced Research Projects Agency DoD Department of Defense DTRA Defense Threat Reduction Agency EUA emergency use authorization FAR Federal Acquisition Regulation FDA U.S. Food and Drug Administration FNIH Foundation for the NIH xv

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xviACRONYMS IND Investigational New Drug IOM Institute of Medicine LLS Leukemia & Lymphoma Society NCATS National Center for Advancing Translational Sciences NCI National Cancer Institute NIH National Institutes of Health OTA other transaction authority PCORI Patient-Centered Outcomes Research Institute SBIR Small Business Innovation Research STTR Small Business Technology Transfer TAP Therapy Acceleration Program TIA Technology Investment Agreement