National Academies Press: OpenBook
Suggested Citation:"Front Matter." Institute of Medicine. 2012. Genome-Based Therapeutics: Targeted Drug Discovery and Development: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13436.
×

GENOME-BASED THERAPEUTICS

Targeted Drug Discovery and Development

WORKSHOP SUMMARY

Adam C. Berger and Steve Olson, Rapporteurs

Roundtable on Translating Genomic-Based Research for Health

Board on Health Sciences Policy

INSTITUTE OF MEDICINE

OF THE NATIONAL ACADEMIES

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

Suggested Citation:"Front Matter." Institute of Medicine. 2012. Genome-Based Therapeutics: Targeted Drug Discovery and Development: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13436.
×

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 project was supported by contracts between the National Academy of Sciences and the American College of Medical Genetics and Genomics (unnumbered contract); American Heart Association (unnumbered contract); American Medical Association (unnumbered contract); American Society of Human Genetics (unnumbered contract); Blue Cross and Blue Shield Association (unnumbered contract); Centers for Disease Control and Prevention (Contract No. 200-2011-38807); College of American Pathologists (unnumbered contract); Department of the Air Force (Contract No. FA7014-10-P-0072); Department of Veterans Affairs (Contract No. V101(93) P-2238); Eli Lilly and Company (Contract No. LRL-0028-07); Genetic Alliance (unnumbered contract); Health Resources and Services Administration (Contract No. HHSH250201100119P); Johnson & Johnson (unnumbered contract); Kaiser Permanente Program Offices Community Benefit II at the East Bay Community Foundation (Contract No. 20121257); Life Technologies (unnumbered contract); National Cancer Institute (Contract No. N01-OD-4-2139, TO#189); National Coalition for Health Professional Education in Genetics (unnumbered contract); National Heart, Lung, and Blood Institute (Contract No. N01-OD-4-2139, TO#275); National Human Genome Research Institute (Contract No. N01-OD-4-2139, TO#264); National Institute of Mental Health (Contract No. N01-OD-4-2139, TO#275); National Institute on Aging (Contract No. N01-OD-4-2139, TO#275); National Society of Genetic Counselors (unnumbered contract); Office of Rare Diseases Research (Contract No. N01-OD-4-2139, TO#275); and Pfizer Inc. (Contract No. 140-N-1818071). Any opinions, findings, conclusions, or recommendations expressed in this publication are those of the authors and do not necessarily reflect the views of the organizations or agencies that provided support for the project.

International Standard Book Number-13: 978-0-309-26024-4
International Standard Book Number-10: 0-309-26024-8

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. Genome-based therapeutics: Targeted drug discovery and development: Workshop summary. Washington, DC: The National Academies Press.

Suggested Citation:"Front Matter." Institute of Medicine. 2012. Genome-Based Therapeutics: Targeted Drug Discovery and Development: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13436.
×

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

                                          —Goethe

001.jpg

INSTITUTE OF MEDICINE

OF THE NATIONAL ACADEMIES

Advising the Nation. Improving Health.

Suggested Citation:"Front Matter." Institute of Medicine. 2012. Genome-Based Therapeutics: Targeted Drug Discovery and Development: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13436.
×

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. Charles 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 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. Charles Vest are chair and vice chair, respectively, of the National Research Council.

www.national-academies.org

Suggested Citation:"Front Matter." Institute of Medicine. 2012. Genome-Based Therapeutics: Targeted Drug Discovery and Development: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13436.
×

PLANNING COMMITTEE1

GEOFFREY GINSBURG (Chair), Director, Center for Genomic Medicine, Institute for Genomic Sciences and Policy, Duke University, Durham, NC

THOMAS LEHNER, Director, Office of Genomics Research Coordination, National Institute of Mental Health, Bethesda, MD

GARRY NEIL, Corporate Vice President, Corporate Office of Science and Technology, Johnson & Johnson, New Brunswick, NJ

MICHELLE A. PENNY, Senior Director, Translational Medicine Group, Eli Lilly and Company, Indianapolis, IN

AIDAN POWER, Vice President and Global Head of Molecular Medicine, Pfizer Inc., Groton, CT

ALLEN D. ROSES, President and Chief Operating Officer, Cabernet, Shiraz and Zinfandel Pharmaceuticals; and Jefferson–Pilot Professor of Neurobiology and Genetics, Professor of Medicine (Neurology); Director, Deane Drug Discovery Institute; Senior Scholar, Fuqua School of Business, R. David Thomas Executive Training Center, Duke University, Durham, NC

SHARON TERRY, President and Chief Executive Officer, Genetic Alliance, Washington, DC

IOM Staff

ADAM C. BERGER, Project Director

SEAN P. DAVID, James C. Puffer, M.D./American Board of Family Medicine Fellow

SARAH H. BEACHY, Christine Mirzayan Science and Technology Policy Graduate Fellow (August to November 2011)

CLAIRE F. GIAMMARIA, Research Associate

TONIA E. DICKERSON, Senior Program Assistant

image

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.

Suggested Citation:"Front Matter." Institute of Medicine. 2012. Genome-Based Therapeutics: Targeted Drug Discovery and Development: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13436.
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Suggested Citation:"Front Matter." Institute of Medicine. 2012. Genome-Based Therapeutics: Targeted Drug Discovery and Development: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13436.
×

ROUNDTABLE ON TRANSLATING GENOMIC-BASED RESEARCH FOR HEALTH1

WYLIE BURKE (Co-Chair), Professor and Chair, Department of Bioethics and Humanities, University of Washington, Seattle

SHARON TERRY (Co-Chair), President and Chief Executive Officer, Genetic Alliance, Washington, DC

NAOMI ARONSON, Executive Director, Technology Evaluation Center, Blue Cross and Blue Shield Association, Chicago, IL

EUAN ANGUS ASHLEY, Representative of the American Heart Association; Director, Center for Inherited Cardiovascular Disease, Stanford University School of Medicine, Palo Alto, CA

PAUL R. BILLINGS, Chief Medical Officer, Life Technologies, Carlsbad, CA

BRUCE BLUMBERG, Institutional Director of Graduate Medical Education, Northern California Kaiser Permanente, The Permanente Medical Group, Oakland, CA

DENISE E. BONDS, Medical Officer, Division of Prevention and Population Sciences, National Heart, Lung, and Blood Institute, Bethesda, MD

JOANN A. BOUGHMAN, Executive Vice President, American Society of Human Genetics, Bethesda, MD

C. THOMAS CASKEY, Professor, Baylor College of Medicine, Houston, TX

SARA COPELAND, Acting Chief, Genetic Services Branch, Health Resources and Services Administration, Rockville, MD

VICTOR DZAU, President and Chief Executive Officer, Duke University Health System; Chancellor for Health Affairs, Duke University, Durham, NC

W. GREGORY FEERO, Special Advisor to the Director for Genomic Medicine, National Human Genome Research Institute, Bethesda, MD

ANDREW N. FREEDMAN, Branch Chief, Clinical and Translational Epidemiology Branch, Epidemiology and Genetics Research Program, Division of Cancer Control and Population Sciences, National Cancer Institute, Rockville, MD

GEOFFREY GINSBURG, Director, Center for Genomic Medicine, Institute for Genomic Sciences and Policy, Duke University, Durham, NC

RICHARD J. HODES, Director, National Institute on Aging, Bethesda, MD

image

1 Institute 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.

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×

SHARON KARDIA, Professor and Chair of Epidemiology; Director, Public Health Genetics Program; Director, Life Science and Society Program; Co-Director, Center for Public Health and Community Genomics, University of Michigan School of Public Health, Ann Arbor

MOHAMED KHAN, Representative of the American Medical Association; Leader of Radiation Oncology, Vancouver Cancer Centre, BC Cancer Agency, Vancouver, BC, Canada

MUIN KHOURY, Director, National Office of Public Health Genomics, Centers for Disease Control and Prevention, Atlanta, GA

THOMAS LEHNER, Director, Office of Genomics Research Coordination, National Institute of Mental Health, Bethesda, MD

DEBRA LEONARD, Representative of the College of American Pathologists; Professor and Vice Chair for Laboratory Medicine; Director of the Clinical Laboratories; Director of the Pathology Residency Training Program, Weill Cornell Medical Center of Cornell University, New York, NY

MICHELE A. LLOYD-PURYEAR, Representative of the Office of Rare Diseases Research; Senior Medical and Scientific Advisor, National Institute of Child Health and Human Development

ELIZABETH MANSFIELD, Director of the Personalized Medicine Staff, Office of In Vitro Diagnostic Device Evaluation and Safety, Center for Devices and Radiological Health, U.S. Food and Drug Administration, Silver Spring, MD

GARRY NEIL, Corporate Vice President, Corporate Office of Science and Technology, Johnson & Johnson, New Brunswick, NJ

ROBERT L. NUSSBAUM, Chief, Division of Medical Genetics, Department of Medicine and Institute of Human Genetics, University of California, San Francisco, School of Medicine

MICHELLE A. PENNY, Senior Director, Translational Medicine Group, Eli Lilly and Company, Indianapolis, IN

AIDAN POWER, Vice President and Global Head of Molecular Medicine, Pfizer Inc., Groton, CT

VICTORIA M. PRATT, Chief Director, Molecular Genetics, Quest Diagnostics Nichols Institute, Chantilly, VA

RONALD PRZYGODZKI, Associate Director for Genomic Medicine and Acting Director of Biomedical Laboratory Research and Development, Department of Veterans Affairs, Washington, DC

Suggested Citation:"Front Matter." Institute of Medicine. 2012. Genome-Based Therapeutics: Targeted Drug Discovery and Development: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13436.
×

ALLEN D. ROSES, President and Chief Operating Officer, Cabernet, Shiraz and Zinfandel Pharmaceuticals; and Jefferson–Pilot Professor of Neurobiology and Genetics, Professor of Medicine (Neurology); Director, Deane Drug Discovery Institute; Senior Scholar, Fuqua School of Business, R. David Thomas Executive Training Center, Duke University, Durham, NC

KEVIN A. SCHULMAN, Professor of Medicine and Business Administration; Director, Center for Clinical and Genetic Economics; Associate Director, Duke Clinical Research Institute, Duke University School of Medicine, Durham, NC

JOAN A. SCOTT, Executive Director, National Coalition for Health Professional Education in Genetics, Lutherville, MD

DAVID VEENSTRA, Professor, Pharmaceutical Outcomes Research and Policy Program, Department of Pharmacy, University of Washington, Seattle

MICHAEL S. WATSON, Executive Director, American College of Medical Genetics and Genomics, Bethesda, MD

DANIEL WATTENDORF, Deputy Chief, Medical Innovations, Department of the Air Force; Program Manager, DARPA/Defense Sciences Office, Arlington, VA

CATHERINE A. WICKLUND, Past President, National Society of Genetic Counselors; Director, Graduate Program in Genetic Counseling; Associate Professor, Department of Obstetrics and Gynecology, Northwestern University, Chicago, IL

IOM Staff

ADAM C. BERGER, Project Director

SEAN P. DAVID, James C. Puffer, M.D./American Board of Family Medicine Fellow

SARAH H. BEACHY, Christine Mirzayan Science and Technology Policy Graduate Fellow (August to November 2011)

CLAIRE F. GIAMMARIA, Research Associate

TONIA E. DICKERSON, Senior Program Assistant

ANDREW POPE, Director, Board on Health Sciences Policy

Suggested Citation:"Front Matter." Institute of Medicine. 2012. Genome-Based Therapeutics: Targeted Drug Discovery and Development: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13436.
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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 published 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:

Jeffrey A. Bluestone, Executive Vice Chancellor and Provost, University of California, San Francisco

Walter M. Capone, Chief Operating Officer, Multiple Myeloma Research Foundation, Norwalk, CT

Steven E. Hyman, Harvard Distinguished Service Professor; Professor of Stem Cell and Regenerative Biology, The Broad Institute, Cambridge, MA

Gary Palmer, Senior Vice President, Medical Affairs and Commercial Development, Foundation Medicine, Inc., Cambridge, MA

Although the reviewers listed above have provided many constructive comments and suggestions, they did not see the final draft of the report before its release. The review of this report was overseen by Melvin Worth.

Suggested Citation:"Front Matter." Institute of Medicine. 2012. Genome-Based Therapeutics: Targeted Drug Discovery and Development: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13436.
×

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 author and the institution.

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Acknowledgments

The support of the sponsors of the Institute of Medicine Roundtable on Translating Genomic-Based Research for Health was crucial to the planning and conduct of the workshop New Paradigms in Drug Discovery: How Genomic Data Are Being Used to Revolutionize the Drug Discovery and Development Process and the development of the workshop summary report titled Genome-Based Therapeutics: Targeted Drug Discovery and Development. Federal sponsors are the Centers for Disease Control and Prevention; Department of the Air Force; Department of Veterans Affairs; Health Resources and Services Administration; National Cancer Institute; National Heart, Lung, and Blood Institute; National Human Genome Research Institute; National Institute of Mental Health; National Institute on Aging; and Office of Rare Diseases Research. Nonfederal sponsorship was provided by the American College of Medical Genetics and Genomics; the American Heart Association; the American Medical Association; the American Society of Human Genetics; the Blue Cross and Blue Shield Association; the College of American Pathologists; Eli Lilly and Company; the Genetic Alliance; Johnson & Johnson; the Kaiser Permanente Program Offices Community Benefit II at the East Bay Community Foundation; Life Technologies; the National Coalition for Health Professional Education in Genetics; the National Society of Genetic Counselors; and Pfizer Inc.

The Roundtable wishes to express its gratitude to the expert speakers whose presentations helped outline the challenges in as well as the opportunities for genomics-guided strategies for drug development. The Roundtable also wishes to thank the members of the planning committee for their work in developing an excellent workshop agenda. The project director would like to thank project staff who worked diligently to develop both the workshop and the resulting summary.

Suggested Citation:"Front Matter." Institute of Medicine. 2012. Genome-Based Therapeutics: Targeted Drug Discovery and Development: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13436.
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Abbreviations and Acronyms

ALK anaplastic lymphoma kinase
 
CDER Center for Drug Evaluation and Research
CDRH Center for Devices and Radiological Health
CFTR cystic fibrosis transmembrane conductance regulator
CLL chronic lymphocytic leukemia
CMS Centers for Medicare & Medicaid Services
 
DARPA Defense Advanced Research Projects Agency
 
FDA U.S. Food and Drug Administration
FEV forced expiratory volume
FFPE formalin-fixed paraffin-embedded
FISH fluorescence in situ hybridization
 
GWAS genome-wide association studies
 
IUO investigational use only
 
MMRC Multiple Myeloma Research Consortium
MMRF Multiple Myeloma Research Foundation
 
NCATS National Center for Advancing Translational Sciences
NIH National Institutes of Health
NPC NCATS Pharmaceutical Collection
Suggested Citation:"Front Matter." Institute of Medicine. 2012. Genome-Based Therapeutics: Targeted Drug Discovery and Development: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/13436.
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NSCLC non-small-cell lung cancer
 
PwC PricewaterhouseCoopers
 
SNP single nucleotide polymorphism
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The number of new drug approvals has remained reasonably steady for the past 50 years at around 20 to 30 per year, while at the same time the total spending on health-related research and development has tripled since 1990. There are many suspected causes for this trend, including increases in regulatory barriers, the rising costs of scientific inquiry, a decrease in research and development efficiency, the downstream effects of patient expirations on investment, and the lack of production models that have successfully incorporated new technology. Regardless, this trajectory is not economically sustainable for the businesses involved, and, in response, many companies are turning toward collaborative models of drug development, whether with other industrial firms, academia, or government. Introducing greater efficiency and knowledge into these new models and aligning incentives among participants may help to reverse the trends highlighted above, while producing more effective drugs in the process.

Genome-Based Therapeutics explains that new technologies have the potential to open up avenues of development and to identify new drug targets to pursue. Specifically, improved validation of gene-disease associations through genomics research has the potential to revolutionize drug production and lower development costs. Genetic information has helped developers by increasing their understanding of the mechanisms of disease as well as individual patients' reactions to their medications. There is a need to identify the success factors for the various models that are being developed, whether they are industry-led, academia-led, or collaborations between the two.

Genome-Based Therapeutics summarizes a workshop that was held on March 21, 2012, titled New Paradigms in Drug Discovery: How Genomic Data Are Being Used to Revolutionize the Drug Discovery and Development Process. At this workshop the goal was to examine the general approaches being used to apply successes achieved so far, and the challenges ahead.

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