FINANCIAL INCENTIVES TO ENCOURAGE
DEVELOPMENT OF THERAPIES THAT
ADDRESS UNMET MEDICAL NEEDS
FOR NERVOUS SYSTEM DISORDERS
WORKSHOP SUMMARY
Sheena M. Posey Norris, Evelyn Strauss, Christopher DeFeo, and
Clare Stroud, Rapporteurs
Forum on Neuroscience and
Nervous System Disorders
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.
THE NATIONAL ACADEMIES PRESS • 500 Fifth Street, NW • Washington, DC 20001
NOTICE: The workshop that is the subject of this workshop summary 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 project was supported by contracts between the National Academy of Sciences and AbbVie; the Alzheimer’s Association; American Diabetes Association; American Society of Microbiology; Amgen Inc. (Contract No. GHCCOPS-CSARF-63987); Association of American Medical Colleges; AstraZeneca; Baxter BioScience; Brain Canada Foundation; Burroughs Wellcome Fund (Contract No. 1015149); Critical Path Institute; the Department of Health and Human Services’ Food and Drug Administration (Contract No. 1R13FD005154-01) and National Institutes of Health (NIH, Contract Nos. HHSN26300026 and HHSN263201200074I, Task Order HHSN26300023 [Under Master Base #DHHS-10001292]) through the National Cancer Institute, National Center for Advancing Translational Sciences, National Center for Complementary and Integrative Health, National Eye Institute, National Institute of Allergy and Infectious Disease, National Institute of Mental Health, National Institute of Neurological Disorders and Stroke, National Institute on Aging, National Institute on Alcohol Abuse and Alcoholism, National Institute on Drug Abuse, and NIH Blueprint for Neuroscience Research; Department of Veterans Affairs (VA240-14-C-0057); Doris Duke Charitable Foundation (Contract No. 2015103); Eli Lilly and Company; FasterCures; Foundation for the National Institutes of Health; Friends of Cancer Research; the Gatsby Charitable Foundation; GlaxoSmithKline, Inc. (Contract No. 005319); Johnson & Johnson Pharmaceutical Research and Development, LLC; Lundbeck Research USA; Merck & Co., Inc. (Contract No. CMO-141224-000649); The Michael J. Fox Foundation for Parkinson’s Research; the National Multiple Sclerosis Society; the National Science Foundation (Contract No. BCS-1064270); New England Journal of Medicine; One Mind for Research; Orion Bionetworks; Pfizer Inc.; Pharmaceutical Product Development, LLC; Sanofi; the Society for Neuroscience; Takeda Pharmaceutical Company Limited (Contract No. 53108); and Wellcome Trust. The views presented in this publication do not necessarily reflect the views of the organizations or agencies that provided support for this project.
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Suggested citation: IOM (Institute of Medicine). 2015. Financial incentives to encourage development of therapies that address unmet medical needs for nervous system disorders: Workshop summary. Washington, DC: The National Academies Press.
“Knowing is not enough; we must apply.
Willing is not enough; we must do.”
—Goethe
INSTITUTE OF MEDICINE
OF THE NATIONAL ACADEMIES
Advising the Nation. Improving Health.
THE NATIONAL ACADEMIES
Advisers to the Nation on Science, Engineering, and Medicine
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 government 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 members, sharing with the National Academy of Sciences the responsibility for advising 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. C. D. Mote, Jr., 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. Victor J. Dzau 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 federal 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 engineering communities. The Council is administered jointly by both Academies and the Institute of Medicine. Dr. Ralph J. Cicerone and Dr. C. D. Mote, Jr., are chair and vice chair, respectively, of the National Research Council.
PLANNING COMMITTEE ON FINANCIAL INCENTIVES
TO SUPPORT UNMET MEDICAL NEEDS FOR NERVOUS
SYSTEM DISORDERS1
DENNIS CHOI (Co-Chair), Stony Brook University
TIMOTHY COETZEE (Co-Chair), National Multiple Sclerosis Society
MARGARET ANDERSON, FasterCures
ROBERT ARMITAGE, Eli Lilly and Company
MARC BOUTIN, National Health Council
LINDA BRADY, National Institute of Mental Health
WILLIAM FISHER, Harvard Law School
RICHARD HODES, National Institute on Aging
STEVEN HYMAN, The Broad Institute of Massachusetts Institute of Technology and Harvard University
JEFF KAHN, Johns Hopkins University
WALTER KOROSHETZ, National Institute of Neurological Disorders and Stroke
GARDINER LAPHAM, Citizens United for Research in Epilepsy
HUSSEINI MANJI, Johnson & Johnson Pharmaceutical Research and Development, LLC
RICHARD MOHS, Eli Lilly and Company
KIRAN REDDY, Biogen Idec
BENJAMIN ROIN, Harvard Law School
TODD SHERER, The Michael J. Fox Foundation for Parkinson’s Research
PHIL SKOLNICK, National Institute on Drug Abuse
GEORGE VRADENBURG, USAgainstAlzheimer’s
JANET WOODCOCK, Food and Drug Administration
STEVIN ZORN, Lundbeck Research USA, Inc.
IOM Staff
BRUCE M. ALTEVOGT, Project Director (until February 2015)
CLARE STROUD, Senior Program Officer (as of October 2014)
ANNE B. CLAIBORNE, Senior Program Officer
DIANA PANKEVICH, Program Officer (until August 2014)
_______________
1Institute 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.
REBECCA ENGLISH, Program Officer
CHRISTOPHER DEFEO, Program Officer
SHEENA M. POSEY NORRIS, Associate Program Officer
KATHRYN HOWELL, Senior Program Assistant
SOPHIE YANG, Senior Program Assistant (from December 2014)
ANNALYN WELP, Senior Program Assistant (from November 2014)
ANDREW M. POPE, Director, Board on Health Sciences Policy
FORUM ON NEUROSCIENCE AND NERVOUS
SYSTEM DISORDERS1
STEVEN HYMAN (Chair), The Broad Institute of Massachusetts Institute of Technology and Harvard University
STORY LANDIS (Vice Chair), Former Director, National Institute of Neurological Disorders and Stroke
SUSAN AMARA, Society for Neuroscience
STEPHEN BRANNAN, Takeda Pharmaceuticals International, Inc.
KATJA BROSE, Cell Press
DANIEL BURCH, Pharmaceutical Product Development, LLC
SARAH CADDICK, Gatsby Charitable Foundation
ROSA CANET-AVILES, Foundation for the National Institutes of Health
MARIA CARRILLO, Alzheimer’s Association
C. THOMAS CASKEY, Baylor College of Medicine
KAREN CHANDROSS, Sanofi US
TIMOTHY COETZEE, National Multiple Sclerosis Society
FAY LOMAX COOK, National Science Foundation
WILLIAM DUNN, Food and Drug Administration
EMMELINE EDWARDS, National Center for Complementary and Integrative Medicine
MARTHA FARAH, University of Pennsylvania
DANIEL GESCHWIND, University of California, Los Angeles
HANK GREELY, Stanford University
MAGALI HAAS, Orion Bionetworks
RAMONA HICKS, One Mind for Research
RICHARD HODES, National Institute on Aging
STUART HOFFMAN, Department of Veterans Affairs
THOMAS INSEL, National Institute of Mental Health
PHILLIP IREDALE, Pfizer Global Research and Development
JOHN ISAAC, Wellcome Trust
INEZ JABALPURWALA, Brain Canada Foundation
FRANCES JENSEN, University of Pennsylvania School of Medicine
GEORGE KOOB, National Institute on Alcohol Abuse and Alcoholism
WALTER KOROSHETZ, National Institute of Neurological Disorders and Stroke
_______________
1Institute of Medicine forums and roundtables do not issue, review, or approve individual documents. The responsibility for the published workshop summary rests with the workshop rapporteurs and the institution.
ALAN LESHNER, American Association for the Advancement of Science (Emeritus)
HUSSEINI MANJI, Johnson & Johnson Pharmaceutical Research and Development, LLC
DAVID MICHELSON, Merck Research Laboratories
RICHARD MOHS, Eli Lilly and Company
JAMES OLDS, National Science Foundation
ATUL PANDE, Tal Medical
STEVEN PAUL, Weill Cornell Medical College, Voyager Therapeutics
TODD SHERER, The Michael J. Fox Foundation for Parkinson’s Research
DAVID SHURTLEFF, National Center for Complementary and Integrative Medicine
PAUL SIEVING, National Eye Institute
NORA VOLKOW, National Institute on Drug Abuse
STEVIN ZORN, Lundbeck Research USA, Inc.
IOM Staff
BRUCE M. ALTEVOGT, Forum Co-Director (until May 2015)
CLARE STROUD, Forum Co-Director
SHEENA M. POSEY NORRIS, Associate Program Officer
ANNALYN M. WELP, Senior Program Assistant
ANDREW M. POPE, Director, Board on Health Sciences Policy
FORUM ON DRUG DISCOVERY, DEVELOPMENT, AND
TRANSLATION1
JEFFREY M. DRAZEN (Co-Chair), New England Journal of Medicine
STEVEN K. GALSON (Co-Chair), Amgen Inc.
RUSS BIAGIO ALTMAN, Stanford University
MARGARET ANDERSON, FasterCures
HUGH AUCHINCLOSS, National Institute of Allergy and Infectious Diseases
CHRISTOPHER P. AUSTIN, National Center for Advancing Translational Sciences
ANN C. BONHAM, Association of American Medical Colleges
LINDA BRADY, National Institute of Mental Health
GAIL H. CASSELL, Harvard Medical School (Visiting)
ANDREW M. DAHLEM, Eli Lilly and Company
JAMES H. DOROSHOW, National Cancer Institute
GARY L. FILERMAN, Atlas Health Foundation
HARRY B. GREENBERG, Stanford University School of Medicine
KATHY L. HUDSON, National Institutes of Health
LYNN D. HUDSON, Critical Path Institute
S. CLAIBORNE JOHNSTON, Dell Medical School, University of Texas, Austin
MICHAEL KATZ, March of Dimes Foundation
JACK D. KEENE, Duke University Medical Center
RUSTY KELLEY, Burroughs Wellcome Fund
RONALD L. KRALL, University of Pittsburgh
FREDA C. LEWIS-HALL, Pfizer Inc.
CAROL MIMURA, University of California, Berkeley
BRIGGS W. MORRISON, AstraZeneca
BERNARD H. MUNOS, InnoThink Center for Research in Biomedical Innovation, FasterCures
ELIZABETH (BETSY) MYERS, Doris Duke Charitable Foundation
JOHN J. ORLOFF, Baxter BioScience
RAJESH RANGANATHAN, National Institute of Neurological Disorders and Stroke
ROBERT E. RATNER, American Diabetes Association
MICHAEL ROSENBLATT, Merck & Co., Inc.
_______________
1Institute of Medicine forums and roundtables do not issue, review, or approve individual documents. The responsibility for the published workshop summary rests with the workshop rapporteurs and the institution.
MICHAEL SEVERINO, AbbVie
JAMES S. SHANNON, GlaxoSmithKline
ELLEN V. SIGAL, Friends of Cancer Research
LANA R. SKIRBOLL, Sanofi
BRIAN L. STROM, Rutgers, the State University of New Jersey
JANET TOBIAS, Ikana Health
JOANNE WALDSTREICHER, Johnson & Johnson
JANET WOODCOCK, Food and Drug Administration
IOM Staff
ANNE B. CLAIBORNE, Forum Director
REBECCA A. ENGLISH, Program Officer
CHRISTOPHER J. DEFEO, Program Officer
EMILY BUSTA, Associate Program Officer
KATHRYN HOWELL, Senior Program Assistant
ANDREW M. POPE, Director, Board on Health Sciences Policy
___________________
Reviewers
This workshop summary 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 published workshop summary as sound as possible and to ensure that the workshop summary 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 workshop summary:
SUSAN AXELROD, Citizens United for Research in Epilepsy
RAJ LONG, The Bill & Melinda Gates Foundation
ARTI RAI, Duke School of Law
DAVID RIDLEY, Duke University
KATIE SALE, American Brain Coalition
ANDREW SPERLING, National Alliance on Mental Illness
PAUL SUMMERGRAD, Tufts University School of Medicine
LUC TRUYEN, Johnson & Johnson
Although the reviewers listed above have provided many constructive comments and suggestions, they did not see the final draft of the workshop summary before its release. The review of this workshop summary was overseen by BRADFORD GRAY, Urban Institute. Appointed by the Institute of Medicine, he was responsible for making certain that an independent examination of this workshop summary was
carried out in accordance with institutional procedures and that all review comments were carefully considered. Responsibility for the final content of this workshop summary rests entirely with the rapporteurs and the institution.
Contents
Unmet Medical Needs in Nervous System Disorders
Topics Highlighted During Presentations and Discussions
Business Implications of the Stagnant R&D Climate for CNS Drugs
Overview of the Current Intellectual Property (IP) Environment for the Private Sector
Legislation Establishing Market Protection Periods
Potential New Approaches to Extending Market Protection
Opportunities to Incentivize R&D Through Improved Market Protection
3 Strengthening the Regulatory Pathway
Drug Development: A Regulatory Viewpoint
Regulatory Challenges Specific to CNS Disorders
Potential Mechanisms to Address Regulatory Challenges
Clarity of Regulatory Processes and Decisions
Reimbursement: The Decision-Making Process
Opportunities to Incentivize R&D by Strengthening Regulatory Pathways
