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OCR for page R1
Developing Biomarker-Based Tools for Cancer Screening, Diagnosis, and Treatment: The State of the Science, Evaluation, Implementation, and Economics
DEVELOPING BIOMARKER-BASED TOOLS FOR Cancer Screening, Diagnosis, and Treatment
The State of the Science, Evaluation, Implementation, and Economics
WORKSHOP SUMMARY
National Cancer Policy Forum
Margie Patlak and Sharyl Nass, Rapporteurs
INSTITUTE OF MEDICINE OF THE NATIONAL ACADMIES
THE NATIONAL ACADEMIES PRESS
Washington, D.C.
www.nap.edu
OCR for page R2
Developing Biomarker-Based Tools for Cancer Screening, Diagnosis, and Treatment: The State of the Science, Evaluation, Implementation, and Economics
THE NATIONAL ACADEMIES PRESS
500 Fifth Street, N.W. 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. HHSH25056133, HHSN261200611002C, 200-2005-13434, HHSM-500-2005-00179P, HHSP23320042509XI, and 223-01-2460 between the National Academy of Sciences and the Department of Health and Human Services. 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-10 0-309-10134-4
International Standard Book Number-13 978-0-309-10134-9
Additional copies of this report are available from the
National Academies Press,
500 Fifth Street, N.W., Lockbox 285, Washington, DC 20055; (800) 624-6242 or (202) 334-3313 (in the Washington metropolitan area); Internet, http://www.nap.edu.
For more information about the Institute of Medicine, visit the IOM home page at: www.iom.edu.
Copyright 2006 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.
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Developing Biomarker-Based Tools for Cancer Screening, Diagnosis, and Treatment: The State of the Science, Evaluation, Implementation, and Economics
“Knowing is not enough; we we must apply.
Willing is not enough; we must do.”
—Geothe
INSTITUTE OF MEDICINE OF THE NATIONAL ACADEMIES
Advising the Nation. Improving Health.
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Developing Biomarker-Based Tools for Cancer Screening, Diagnosis, and Treatment: The State of the Science, Evaluation, Implementation, and Economics
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. Wm. A. Wulf 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. Wm. A. Wulf are chair and vice chair, respectively, of the National Research Council.
www.national-academies.org
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Developing Biomarker-Based Tools for Cancer Screening, Diagnosis, and Treatment: The State of the Science, Evaluation, Implementation, and Economics
NATIONAL CANCER POLICY FORUM
HAROLD L. MOSES, MD (Chair), Professor of Cancer Biology, Medicine, and Pathology, Director Emeritus,
Vanderbilt-Ingram Comprehensive Cancer Center, Vanderbilt University Medical Center
PETER BACH, MD, MAPP, Senior Advisor,
Office of the Administrator, Centers for Medicare & Medicaid Services
EDWARD BENZ, JR., MD, President,
Dana-Farber Cancer Center
THOMAS BURISH, PhD, Chair,
ACS Board and Provost, Notre Dame University
MARK CLANTON, MD, MPH, Deputy Director,
Cancer Delivery Systems, National Cancer Institute
BETTY FERRELL, PhD, RN, FAAN, Research Scientist,
City of Hope National Medical Center
JOSEPH FRAUMENI, JR., MD, Director,
Division of Cancer Epidemiology and Genetics, National Cancer Institute
STEPHEN FRIEND, MD, PhD, Executive Vice President,
Oncology, Merck
PATRICIA GANZ, MD, ASCO Board Member and Professor of Medicine,
University of California, Los Angeles
THOMAS KEAN, MPH, Executive Director,
C-Change
WILLIAM LAWRENCE, MD, MS, Director,
Center for Outcomes and Evidence, Agency for Healthcare Research and Quality
LYNN MATRISIAN, PhD, Chair,
Division of Cancer Biology, Vanderbilt University
DAVID R. PARKINSON, MD, Senior Vice President,
Oncology Research and Development, Biogen Idec
EDITH PEREZ, MD, Director,
Cancer Clinical Study Unit, Mayo Clinic
SCOTT RAMSEY, MD, PhD, Full Member,
Fred Hutchinson Cancer Research Center
EDDIE REED, MD, Director,
Division of Cancer Prevention and Control, Centers for Disease Control and Prevention
WILLIAM ROBINSON, MD, MPH, Director,
Office of Minority Health and Health Disparities, Health Resources and Services Administration
CHARLES SAWYERS, MD, Chairman,
Human Oncology and Pathogenesis Program, Memorial Sloan-Kettering Cancer Center
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Developing Biomarker-Based Tools for Cancer Screening, Diagnosis, and Treatment: The State of the Science, Evaluation, Implementation, and Economics
MARGARET SPITZ, MD, Chair of Epidemiology,
M.D. Anderson Cancer Center
ELLEN STOVALL, President and CEO,
National Coalition for Cancer Survivorship
JANET WOODCOCK, MD, Deputy Commissioner for Operations,
Food and Drug Administration
Staff
SHARYL NASS, PhD, Study Director
ROGER HERDMAN, MD, Director,
ALIZA NORWOOD, Research Assistant
MARY ANN PRYOR, Senior Program Assistant
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Developing Biomarker-Based Tools for Cancer Screening, Diagnosis, and Treatment: The State of the Science, Evaluation, Implementation, and Economics
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 (NRC’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:
Margaret Pepe, PhD, University of Washington School of Public Health
G. Gregory Raab, PhD, Consultant, Raab & Associates, Inc.
David Ransohoff, MD, University of North Carolina, Chapel Hill
Howard Schulman, PhD, Pharmaceutical Product Development, Inc. (PPD), Biomarker Discovery Sciences
Janet Warrington, PhD, Affymetrix, 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 Melvin Worth, MD. Appointed by the NRC, he was responsible for making certain that an
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Developing Biomarker-Based Tools for Cancer Screening, Diagnosis, and Treatment: The State of the Science, Evaluation, Implementation, and Economics
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 rapporteurs and the institution.
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Developing Biomarker-Based Tools for Cancer Screening, Diagnosis, and Treatment: The State of the Science, Evaluation, Implementation, and Economics
Contents
Developing Biomarker-Based Tools for Cancer Screening, Diagnosis, and Treatment: The State of the Science, Evaluation, Implementation, and Economics
Introduction
1
Overview of Technologies Used to Discover Cancer Biomarkers
3
Genomics, Proteomics, and Metabolomics
3
Molecular Imaging
9
Meeting the Technical Challenges of Biomarker Validation and Qualification
11
Coordinating the Development of Biomarkers and Targeted Therapies
20
Therapeutics Industry Perspective
21
Diagnostics Industry Perspective
24
NCI Perspective
26
Clinical Investigator Perspective
28
Biomarker Development and Regulatory Oversight
29
FDA Critical Path Initiative
30
Oversight of Diagnostic Tests
32
Designing Clinical Studies of Biomarkers
36
Assessment and Adoption of Biomarker-Based Technologies
39
Federal Programs for Technology Assessment
40
Insurance Coverage Decisions and Practice Guidelines
43
CMS Coverage of Biomarkers
46
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Developing Biomarker-Based Tools for Cancer Screening, Diagnosis, and Treatment: The State of the Science, Evaluation, Implementation, and Economics
Economic Impact of Biomarkers
48
Cost-Effectiveness Analysis
48
The Value of Information and Research
53
Technology Assessment in the Private Sector
53
Clinical Development Strategies for Biomarker Utilization Discussion
57
Strategies for Implementing Standardized Biorepositories Discussion
61
Strategies for Determining Analytic Validity and Clinical Utility of Biomarkers Discussion
66
Strategies to Develop Biomarkers for Early Detection Discussion
69
Mechanisms for Developing an Evidence Base Discussion
73
Evaluation of Evidence in Decision Making Discussion
76
Incorporating Biomarker Evidence into Clinical Practice Discussion
80
Acronyms
85
Glossary
86
References
92
APPENDIXES
A
Workshop Agenda
93
B
Workshop Speakers, Moderators, Invited Discussants, and Participants
99
