EVOLUTION OF TRANSLATIONAL
Lessons Learned and the Path Forward
Committee on the Review of Omics-Based Tests for
Predicting Patient Outcomes in Clinical Trials
Board on Health Care Services
Board on Health Sciences Policy
Christine M. Micheel, Sharyl J. Nass, and Gilbert S. Omenn, Editors
NATIONAL RESEARCH COUNCIL
OF THE NATIONAL ACADEMIES
THE NATIONAL ACADEMIES PRESS
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. The members of the committee responsible for the report were chosen for their special competences and with regard for appropriate balance.
This study was supported by Contract Nos. HHSN261200900003C (National Cancer Institute); HHSF223201010692P and HHSF22301018T (Food and Drug Administration); and 200-2011-38807 and 200-2005-13434 (Centers for Disease Control and Prevention). This study was also supported by the U.S. Department of Veterans Affairs, the American Society for Clinical Pathology, and the College of American Pathologists. Any opinions, findings, conclusions, or recommendations expressed in this publication are those of the author(s) and do not necessarily reflect the view of the organizations or agencies that provided support for this project.
International Standard Book Number 13: 978-0-309-22418-5
International Standard Book Number 10: 0-309-22418-7
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 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. Evolution of Translational Omics: Lessons Learned and the Path Forward. Washington, DC: The National Academies Press.
“Knowing is not enough; we must apply.
Willing is not enough; we must do.”
INSTITUTE OF MEDICINE
OF THE NATIONAL ACADEMIES
GoetheAdvising 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. 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 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 M. Vest are chair and vice chair, respectively, of the National Research Council.
COMMITTEE ON THE REVIEW OF OMICS-BASED TESTS FOR PREDICTING PATIENT OUTCOMES IN CLINICAL TRIALS
GILBERT S. OMENN (Chair), Professor of Internal Medicine, Human Genetics and Public Health; Director, University of Michigan Center for Computational Medicine and Bioinformatics, University of Michigan Medical School, Ann Arbor
CATHERINE D. DEANGELIS, Professor of Pediatrics, Johns Hopkins School of Medicine, Professor of Health Policy and Management, Johns Hopkins School of Public Health, and Editor-in-Chief Emerita, Journal of the American Medical Association, Baltimore, MD
DAVID L. DEMETS, Professor of Statistics and Biostatistics, University of Wisconsin, Madison
THOMAS R. FLEMING, Professor of Biostatistics, Statistics, University of Washington, Seattle
GAIL GELLER, Professor of Medicine, Berman Institute of Bioethics, Johns Hopkins University, Baltimore, MD
JOE GRAY, Gordon Moore Endowed Chair, Department of Biomedical Engineering, Center for Spatial Systems Biomedicine, Oregon Health & Science University Knight Cancer Institute, Portland
DANIEL F. HAYES, Clinical Director of the Breast Oncology Program, and Stuart B. Padnos Professor of Breast Cancer Research, University of Michigan Comprehensive Cancer Center, Ann Arbor
I. CRAIG HENDERSON, Adjunct Professor of Medicine, University of California, San Francisco and Helen Diller Family Comprehensive Cancer Center
LARRY KESSLER, Professor and Chair, Department of Health Services, University of Washington School of Public Health, Seattle
STANLEY LAPIDUS, Founder, President, and CEO, SynapDx Corporation, Southborough, MA
DEBRA G. B. LEONARD, Professor and Vice Chair for Laboratory Medicine, and Director of the Clinical Laboratories, Weill Medical College of Cornell University, New York, NY
HAROLD L. MOSES, Director Emeritus, Hortense B. Ingram Professor of Molecular Oncology, and Professor of Cancer Biology, Medicine, and Pathology, Vanderbilt-Ingram Cancer Center, Nashville, TN
WILLIAM PAO, Associate Professor of Medicine, Cancer Biology, and Pathology, Microbiology, Immunology, Ingram Associate Professor of Cancer Research, Director of the Division of Hematology/Oncology, and Director of Personalized Cancer Medicine, Vanderbilt University School of Medicine, Nashville, TN
REBECCA D. PENTZ, Professor of Hematology and Oncology in Research Ethics, Emory School of Medicine, Atlanta, GA
NATHAN D. PRICE, Associate Professor, Institute for Systems Biology, Seattle, WA
JOHN QUACKENBUSH, Professor of Computational Biology and Bioinformatics, Dana-Farber Cancer Institute, Boston, MA
ELDA RAILEY, Cofounder, Research Advocacy Network, Plano, TX
DAVID RANSOHOFF, Professor of Medicine, Clinical Professor of Epidemiology, University of North Carolina, School of Medicine and Gillings School of Global Public Health, Chapel Hill
E. ALBERT REECE, Vice President for Medical Affairs, University of Maryland, and John Z. and Akiko K. Bowers Distinguished Professor and Dean, University of Maryland School of Medicine, Baltimore
DANIELA M. WITTEN, Assistant Professor of Biostatistics, University of Washington, Seattle
CHRISTINE M. MICHEEL, Study Director (through November 2011)
SHARYL J. NASS, Director, National Cancer Policy Forum; Study Director (from December 2011)
LAURA LEVIT, Program Officer (from June 2011)
ERIN BALOGH, Associate Program Officer
SARAH DOMNITZ, Christine Mirzayan Science and Technology Policy Graduate Fellow (from August to December 2011)
JULIA E. DOOHER, Christine Mirzayan Science and Technology Policy Graduate Fellow (from January to May 2011)
NIHARIKA SATHE, Research Assistant (from June 2011)
MICHAEL PARK, Senior Program Assistant
PATRICK BURKE, Financial Associate
ROGER HERDMAN, Director, Board on Health Care Services
ANDREW POPE, Director, Board on Health Sciences Policy
JOHN BAILAR, Scholar in Residence
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 deliberative process. We wish to thank the following individuals for their review of this report:
Wylie Burke, Professor and Chair, Department of Bioethics and Humanities, University of Washington
Adam M. Clarke, Founder, MedTran Health Strategies, LLC
Susan S. Ellenberg, Professor of Biostatistics, Associate Dean for Clinical Research, Perelman School of Medicine, University of Pennsylvania
Charis Eng, ACS Clinical Research Professor, Chair, and Director, Genomic Medicine Institute, Cleveland Clinic Foundation, Case Western Reserve University School of Medicine
Marcus Feldman, Professor of Biology, Stanford University
David B. Flannery, Chief of Medical Genetics, Professor of Pediatrics, Vice Chair for Administration of the Department of Pediatrics, Georgia Health Sciences University
Stephen Friend, President and CEO, Sage Bionetworks
Larry Gold, Chairman and Chief Executive Officer, SomaLogic, Inc.
Steven Goodman, Associate Dean for Clinical and Translational Research, Professor of Medicine & Health Research and Policy, Stanford University School of Medicine
Robert Gray, Professor of Biostatistics, Dana Farber Cancer Institute
Stephen Grubbs, Principal Investigator, Delaware Christiana Care Community Clinical Oncology Program
David Korn, Vice Provost for Research, Professor of Pathology, Harvard University
Marc Ladanyi, William J. Ruane Chair in Molecular Oncology, Memorial Sloan-Kettering Cancer Center
Bernard Lo, Professor of Medicine and Director, Program in Medical Ethics, University of California, San Francisco
David Madigan, Professor of Statistics, Columbia University
Bettie Sue Siler Masters, The Robert A. Welch Distinguished Professor in Chemistry, University of Texas Health Science Center at San Antonio
Charles E. Phelps, Provost Emeritus, Professor of Political Science and of Economics, University of Rochester
Dan Roden, Professor of Medicine, William Stokes Chair in Experimental Therapeutics, Professor of Pharmacology, Vanderbilt University Medical Center
Larry Shapiro, Executive Vice Chancellor for Medical Affairs and Dean, Washington University School of Medicine
Peter Shields, Professor and Chief, Deputy Director, Comprehensive Cancer Center and Professor, College of Medicine, The Ohio State University Medical Center
Steve Teutsch, Chief Science Officer, Los Angeles County Public Health
David Wong, Professor of Bioengineering, Director of the Dental Research Institute, University of California, Los Angeles
Although the reviewers listed above have provided many constructive comments and suggestions, they were not asked to endorse the conclusions or recommendations, nor did they see the final draft of the report before its release. The review of this report was overseen by Kristine Gebbie, Adjunct Professor, Flinders University School of Nursing and Midwifery and Lawrence D. Brown, Professor, Department of Statistics, The Wharton School, University of Pennsylvania. Appointed by the Institute of Medicine and the National Research Council, they were 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 authoring committee and the institution.
The committee is grateful to many individuals who provided valuable input and information for the study, either through formal presentations or through informal communications with study staff and committee members:
Patrick Anquetil, SynapDx
Keith Baggerly, MD Anderson Cancer Center
John Bailar, Institute of Medicine
William Barry, Duke University
Robert Becker, Food and Drug Administration
Robert Califf, Duke University
Ned Calonge, The Colorado Trust
Michael Cuffe, Duke University
Jeffrey Drazen, New England Journal of Medicine
John Falletta, Duke University
Geoffrey Ginsburg, Duke University
Alberto Gutierrez, Food and Drug Administration
Scott Henderson, Vermillion
Michael Kelley, Duke University
Katrina Kelner, Translational Medicine and Science
Véronique Kiermer, Nature Publishing
Sally Kornbluth, Duke University
Sumithra Mandrekar, Mayo Clinic
Anne-Marie Mazza, The National Academies
Ross McKinney, Duke University
Lisa McShane, National Cancer Institute
Stephen A. Merrill, The National Academies
Mitch Nelles, XDx
Joseph Nevins, Duke University
Nicole Osmer, Nicole Osmer Healthcare Communications
Harold Paz, Pennsylvania State University
Charles Perou, The University of North Carolina at Chapel Hill
Peter Pronovost, Johns Hopkins University
William Sellers, Novartis Institutes for BioMedical Research
Steven Shak, Genomic Health
Richard Simon, National Cancer Institute
Ed Stevens, Pathwork Diagnostics
Susan Su, United States Patent and Trademark Office
Paul Uhlir, The National Academies
Laura van ‘t Veer, Helen Diller Family Comprehensive Cancer Center
Pablo Whaley, United States Patent and Trademark Office
Scott Zeger, Johns Hopkins University
We thank the National Cancer Institute, the Food and Drug Administration, the Centers for Disease Control and Prevention, the U.S. Department of Veterans Affairs, the American Society for Clinical Pathology, and the College of American Pathologists for supporting this study.