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Suggested Citation:"Front Matter." Institute of Medicine. 2014. Improving and Accelerating Therapeutic Development for Nervous System Disorders: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18494.
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IMPROVING AND ACCELERATING
THERAPEUTIC DEVELOPMENT FOR
NERVOUS SYSTEM DISORDERS

WORKSHOP SUMMARY

Forum on Neuroscience and
Nervous System Disorders

Board on Health Sciences Policy

Sheena M. Posey Norris, Diana E. Pankevich, Miriam Davis,
and Bruce M. Altevogt, Rapporteurs

INSTITUTE OF MEDICINE
              OF THE NATIONAL ACADEMIES

THE NATIONAL ACADEMIES PRESS

Washington, D.C.

www.nap.edu

Suggested Citation:"Front Matter." Institute of Medicine. 2014. Improving and Accelerating Therapeutic Development for Nervous System Disorders: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18494.
×

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 the Alzheimer’s Association; CeNeRx Biopharma; the Department of Health and Human Services’ National Institutes of Health (NIH, Contract No. HHSN26300026 [Under Master Base # DHHS-10001018]) through the National Eye Institute, 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 (101-D27015); Eli Lilly and Company; Fast Forward, LLC; Foundation for the National Institutes of Health; GE Healthcare, Inc. (2580261187); GlaxoSmithKline, Inc.; Johnson & Johnson Pharmaceutical Research and Development, LLC; Lundbeck Research USA; Merck Research Laboratories; The Michael J. Fox Foundation for Parkinson’s Research; the National Science Foundation (Contract No. OIA-0753701); One Mind for Research; Pfizer Inc.; the Society for Neuroscience; and Wellcome Trust. The views presented in this publication are those of the editors and attributing authors and do not necessarily reflect the view of the organizations or agencies that provided support for this project.

International Standard Book Number-13: 978-0-309-29246-7
International Standard Book Number-10: 0-309-29246-8

Additional copies of this report are available 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 2014 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.

Cover image courtesy of Dr. Lawrence Goldstein, University of California, San Diego.

Suggested citation: IOM (Institute of Medicine). 2014. Improving and accelerating therapeutic development for nervous system disorders: Workshop summary. Washington, DC: The National Academies Press.

Suggested Citation:"Front Matter." Institute of Medicine. 2014. Improving and Accelerating Therapeutic Development for Nervous System Disorders: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18494.
×

Knowing is not enough; we must apply.
Willing is not enough; we must do.
”      

                                                —Goethe

image

INSTITUTE OF MEDICINE
              OF THE NATIONAL ACADEMIES

Advising the Nation. Improving Health.

Suggested Citation:"Front Matter." Institute of Medicine. 2014. Improving and Accelerating Therapeutic Development for Nervous System Disorders: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18494.
×

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. 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 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.

www.national-academies.org

 

Suggested Citation:"Front Matter." Institute of Medicine. 2014. Improving and Accelerating Therapeutic Development for Nervous System Disorders: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18494.
×

PLANNING COMMITTEE ON ACCELERATING
THERAPEUTIC DEVELOPMENT FOR NERVOUS SYSTEM
DISORDERS TOWARD FIRST-IN-HUMAN TRIALS1

JOHN DUNLOP (Co-Chair), AstraZeneca

FRED GAGE (Co-Chair), Salk Institute

NEIL BUCKHOLTZ, National Institute on Aging

DANIEL BURCH, Pharmaceutical Product Development, LLC (PPDi)

ROBERT CONLEY, Eli Lilly and Company

DANIEL GESCHWIND, University of California, Los Angeles

MAGALI HAAS, Orion Bionetworks

STEVEN HYMAN, The Broad Institute of the Massachusetts Institute of Technology and Harvard University

NI KHIN, U.S. Food and Drug Administration

DAVID MICHELSON, Merck Research Laboratories

ALEXANDER PLOSS, Rockefeller University

WILLIAM POTTER, National Institute of Mental Health

RAJESH RANGANATHAN, National Institute of Neurological Disorders and Stroke

VICKI SEYFERT-MARGOLIS, Precision for Medicine, Inc.

STEVIN ZORN, Lundbeck Research USA

IOM Staff

BRUCE M. ALTEVOGT, Project Director

DIANA E. PANKEVICH, Program Officer

SHEENA M. POSEY NORRIS, Research Associate

RACHEL J. KIRKLAND, Senior Program Assistant

______________

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.

Suggested Citation:"Front Matter." Institute of Medicine. 2014. Improving and Accelerating Therapeutic Development for Nervous System Disorders: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18494.
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Suggested Citation:"Front Matter." Institute of Medicine. 2014. Improving and Accelerating Therapeutic Development for Nervous System Disorders: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18494.
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FORUM ON NEUROSCIENCE AND NERVOUS
SYSTEM DISORDERS
1

STEVEN HYMAN (Chair), The Broad Institute

SUSAN AMARA, Society for Neuroscience

MARC BARLOW, GE Healthcare, Inc.

MARK BEAR, Massachusetts Institute of Technology

KATJA BROSE, Neuron

DANIEL BURCH, Pharmaceutical Product Development, LLC (PPDi)

MARIA CARRILLO, Alzheimer’s Association

C. THOMAS CASKEY, Baylor College of Medicine

TIMOTHY COETZEE, Fast Forward, LLC

EMMELINE EDWARDS, National Center for Complementary and Alternative Medicine

MARTHA FARAH, University of Pennsylvania

RICHARD FRANK, GE Healthcare, Inc.

DANIEL GESCHWIND, University of California, Los Angeles

HANK GREELY, Stanford University

MYRON GUTMANN, National Science Foundation

MAGALI HAAS, Orion Bionetworks

STUART HOFFMAN, U.S. Department of Veterans Affairs

RICHARD HODES, National Institute on Aging

THOMAS INSEL, National Institute of Mental Health

PHILLIP IREDALE, Pfizer Global Research and Development

DANIEL JAVITT, Nathan S. Kline Institute for Psychiatric Research

FRANCES JENSEN, University of Pennsylvania School of Medicine

STORY LANDIS, National Institute of Neurological Disorders and Stroke

ALAN LESHNER, American Association for the Advancement of Science

HUSSEINI MANJI, Johnson & Johnson Pharmaceutical Research and Development, LLC

DAVID MICHELSON, Merck Research Laboratories

RICHARD MOHS, Lilly Research Laboratories

JONATHAN MORENO, University of Pennsylvania

ATUL PANDE, GlaxoSmithKline, 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.

Page viii Cite
Suggested Citation:"Front Matter." Institute of Medicine. 2014. Improving and Accelerating Therapeutic Development for Nervous System Disorders: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18494.
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STEVEN PAUL, Weill Cornell Medical College

TODD SHERER, The Michael J. Fox Foundation for Parkinson’s Research

PAUL SIEVING, National Eye Institute

MARC TESSIER-LEVIGNE, Rockefeller University

WILLIAM THIES, Alzheimer’s Association

JOANNE TORNOW, National Science Foundation

NORA VOLKOW, National Institute on Drug Abuse

KENNETH WARREN, National Institute on Alcohol Abuse and Alcoholism

DAVID WHOLLEY, Foundation for the National Institutes of Health

JOHN WILLIAMS, Wellcome Trust

STEVIN ZORN, Lundbeck Research USA

CHARLES ZORUMSKI, Washington University School of Medicine

IOM Staff

BRUCE M. ALTEVOGT, Forum Director

DIANA E. PANKEVICH, Program Officer

SHEENA M. POSEY NORRIS, Research Associate

RACHEL J. KIRKLAND, Senior Program Assistant

ANDREW M. POPE, Director, Board on Health Sciences Policy

Suggested Citation:"Front Matter." Institute of Medicine. 2014. Improving and Accelerating Therapeutic Development for Nervous System Disorders: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18494.
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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:

Bruce Baron, Sanofi

Bill Deakin, University of Manchester

Martien Kas, University Medical Center Utrecht

Bruce Psaty, University of Washington School of Medicine

Scott Small, Columbia University

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 Eric B. Larson, Group Health Research 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.

Suggested Citation:"Front Matter." Institute of Medicine. 2014. Improving and Accelerating Therapeutic Development for Nervous System Disorders: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18494.
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Improving and Accelerating Therapeutic Development for Nervous System Disorders is the summary of a workshop convened by the IOM Forum on Neuroscience and Nervous System Disorders to examine opportunities to accelerate early phases of drug development for nervous system drug discovery. Workshop participants discussed challenges in neuroscience research for enabling faster entry of potential treatments into first-in-human trials, explored how new and emerging tools and technologies may improve the efficiency of research, and considered mechanisms to facilitate a more effective and efficient development pipeline.

There are several challenges to the current drug development pipeline for nervous system disorders. The fundamental etiology and pathophysiology of many nervous system disorders are unknown and the brain is inaccessible to study, making it difficult to develop accurate models. Patient heterogeneity is high, disease pathology can occur years to decades before becoming clinically apparent, and diagnostic and treatment biomarkers are lacking. In addition, the lack of validated targets, limitations related to the predictive validity of animal models - the extent to which the model predicts clinical efficacy - and regulatory barriers can also impede translation and drug development for nervous system disorders. Improving and Accelerating Therapeutic Development for Nervous System Disorders identifies avenues for moving directly from cellular models to human trials, minimizing the need for animal models to test efficacy, and discusses the potential benefits and risks of such an approach. This report is a timely discussion of opportunities to improve early drug development with a focus toward preclinical trials.

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