PERSPECTIVES ON BIOMARKER AND SURROGATE ENDPOINT EVALUATION
Discussion Forum Summary
Alison Mack, Erin Balogh, and Christine M. Micheel, Rapporteurs
INSTITUTE OF MEDICINE
OF THE NATIONAL ACADEMIES
THE NATIONAL ACADEMIES PRESS
Washington, D.C.
www.nap.edu
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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 Contract No. HHSF223200810020I between the National Academy of Sciences and the Food and Drug Administration. 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.
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Suggested citation: IOM (Institute of Medicine). 2011. Perspectives on biomarker and surrogate endpoint evaluation: Discussion forum summary. Washington, DC: The National Academies Press.
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 QUALIFICATION OF BIOMARKERS AND SURROGATE ENDPOINTS IN CHRONIC DISEASE
JOHN R. BALL (Chair), Senior Advisor,
American Society for Clinical Pathology
MICHELLE A. ALBERT, Assistant Professor of Medicine, Associate Physician, and Director of Behavioral and Neurocardiovascular Cardiology,
Brigham and Women’s Hospital, Harvard Medical School
FRED APPLE, Medical Director,
Clinical Laboratories, Hennepin County Medical Center, and
Professor,
Laboratory Medicine and Pathology, University of Minnesota School of Medicine
ROBERT M. CALIFF, Vice Chancellor for Clinical Research and Professor of Medicine,
Cardiology, Duke University School of Medicine
VICTOR DE GRUTTOLA, Professor and Chair,
Biostatistics, Harvard School of Public Health
DAVID DEMETS, Professor,
Biostatistics & Medical Informatics, University of Wisconsin–Madison
ROBERT GERSZTEN, Research Director and Faculty Member,
Massachusetts General Hospital, and
Associate Professor of Medicine,
Harvard Medical School
WILLIAM HARLAN, JR., Consultant
ALLAN JAFFE, Professor of Medicine,
Mayo Clinic
RONALD KRAUSS, Director,
Atherosclerosis Research and
Senior Scientist,
Children’s Hospital Oakland Research Institute
HARLAN M. KRUMHOLZ, Harold H. Hines, Jr., Professor of Medicine
and Epidemiology and Public Health, Yale University School of Medicine
MARIA LOPES-VIRELLA, Professor,
Bioengineering, Medical University of South Carolina
ROBERTA NESS, Dean,
University of Texas Health Science Center, School of Public Health
JENNIFER VAN EYK, Professor,
Departments of Medicine and Biological Chemistry, Johns Hopkins University
JOHN A. WAGNER, Vice President,
Clinical Pharmacology, Merck & Co., Inc.
Consultant
ELIZABETH YETLEY, Consultant,
National Institutes of Health, Office of Dietary Supplements
Study Staff
CHRISTINE M. MICHEEL, Study Director
SHARYL NASS, Senior Program Officer
ERIN BALOGH, Associate Program Officer
CAIRA WOODS, Christine Mirzayan Science and Technology Policy Graduate Fellow (January to April 2010), Research Associate (April to July 2010)
ASHLEY MCWILLIAMS, Senior Program Assistant
PATRICK BURKE, Financial Associate
ROGER HERDMAN, Director,
Board on Health Care Services
LINDA MEYERS, Director,
Food and Nutrition Board
ANDREW POPE, Director,
Board on Health Sciences Policy
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:
Douglas Balentine, Unilever
Thomas Fleming, University of Washington
Philip Greenland, Northwestern University
James Mayne, Pfizer, Inc.
Rebecca Miksad, Harvard Medical School
Jack Zakowski, Beckman Coulter, Inc.
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 Sharon B. Murphy, scholar-in-residence, Institute of Medicine. Appointed by the Institute of Medicine, she was responsible for making certain that an