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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
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For more information about the Institute of Medicine, visit the IOM home page
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Copyright 2012 by the National Academy of Sciences. All rights reserved.
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The serpent has been a symbol of long life, healing, and knowledge among almost
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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
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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
