Committee on Large-Scale Science and Cancer Research Sharyl J. Nass and Bruce W. Stillman, Editors National Cancer Policy Board INSTITUTE OF MEDICINE OF THE NATIONAL ACADEMIES and Division on Earth and Life Studies THE NATIONAL ACADEMIES PRESS Washington, D.C. www.nap.edu Copyright © 2003 National Academy of Sciences. All rights reserved. Unless otherwise indicated, all materials in this PDF File provided by the National Academies Press (www.nap.edu) for research purposes are copyrighted by the National Academy of Sciences. Distribution, posting, or copying is strictly prohibited without written permission of the NAP. Generated for liuhan68@yahoo.com on Tue Jul 15 05:46:27 2003 http://books.nap.edu/catalog/.html 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. Support for this project was provided by The National Cancer Institute. The views pre- sented in this report are those of the Institute of Medicine and National Research Council Committee on Large-Scale Science and Cancer Research and are not necessarily those of the funding agencies. Library of Congress Cataloging-in-Publication Data Large-scale biomedical science : exploring strategies for future research / Sharyl J. Nass and Bruce W. Stillman, editors ; Committee on Large-scale Science and Cancer Research, National Cancer Policy Board and Division on Earth and Life Studies, National Research Council. p. ; cm. Includes bibliographical references. ISBN 0-309-08912-3 (pbk.) — ISBN 0-309-50698-0 (PDF) 1. Medicine—Research—Government policy—United States. 2. Cancer—Research—Government policy—United States. 3. Federal aid to medical research—United States. [DNLM: 1. Biomedical Research—United States. 2. Interinstitutional Relations—United States. 3. Research Design—United States. 4. Resource Allocation—United States. W 20.5 L322 2003] I. Nass, Sharyl J. II. Stillman, Bruce. III. National Cancer Policy Board (U.S.). Committee on Large-scale Science and Cancer Research. IV. National Research Council (U.S.). Division on Earth and Life Studies. R854.U5L37 2003 610'.7’2073—dc21 2003009162 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 2003 by the National Academy of Sciences. All rights reserved. Printed in the United States of America. Copyright © 2003 National Academy of Sciences. All rights reserved. Unless otherwise indicated, all materials in this PDF File provided by the National Academies Press (www.nap.edu) for research purposes are copyrighted by the National Academy of Sciences. Distribution, posting, or copying is strictly prohibited without written permission of the NAP. Generated for liuhan68@yahoo.com on Tue Jul 15 05:46:27 2003 http://books.nap.edu/catalog/.html The National Academy of Sciences is a private, nonprofit, self-perpetuating soci- ety of distinguished scholars engaged in scientific and engineering research, dedi- cated 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. Bruce M. Alberts 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 mem- bers, sharing with the National Academy of Sciences the responsibility for advis- ing 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 Sci- ences in 1916 to associate the broad community of science and technology with the Academy’s purposes of furthering knowledge and advising the federal gov- ernment. Functioning in accordance with general policies determined by the Acad- emy, 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 commu- nities. The Council is administered jointly by both Academies and the Institute of Medicine. Dr. Bruce M. Alberts and Dr. Wm. A. Wulf are chair and vice chair, respectively, of the National Research Council. www.national-academies.org Copyright © 2003 National Academy of Sciences. All rights reserved. Unless otherwise indicated, all materials in this PDF File provided by the National Academies Press (www.nap.edu) for research purposes are copyrighted by the National Academy of Sciences. Distribution, posting, or copying is strictly prohibited without written permission of the NAP. Generated for liuhan68@yahoo.com on Tue Jul 15 05:46:27 2003 http://books.nap.edu/catalog/.html iv COMMITTEE ON LARGE-SCALE SCIENCE AND CANCER RESEARCH *JOSEPH V. SIMONE, M.D. (Chair), Simone Consulting, Dunwoody, GA *BRUCE W. STILLMAN, Ph.D. (Vice Chair), Director, Cold Spring Harbor Laboratory, Cold Spring Harbor, NY *ELLEN STOVALL (Vice Chair), Executive Director, National Coalition for Cancer Survivorship, Silver Spring, MD *DIANA PETITTI, M.D. (Vice Chair), Director, Research and Evaluation, Kaiser Permanente of Southern California, Pasadena, CA *JILL BARGONETTI, Ph.D. Associate Professor, Hunter College, New York, NY BARRY BOZEMAN, Ph.D. Regents Professor of Public Policy, Director of the State Data and Research Center, Georgia Institute of Technology, Atlanta, GA *TIM BYERS, M.D., M.P.H. Professor of Epidemiology and Associate Director, University of Colorado Cancer Center, University of Colorado School of Medicine, Denver, CO TOM CURRAN, Ph.D. Chairman of the Department of Developmental Neurobiology, St. Jude’s Children’s Research Hospital, Memphis, TN *TIMOTHY EBERLEIN, M.D. Bixby Professor and Chairman, Washington University School of Medicine, Department of Surgery, St. Louis, MO DAVID GALAS, Ph.D. Chief Academic Officer and Norris Professor of Applied Life Sciences, Keck Graduate Institute of Applied Life Sciences, Claremont, CA *KAREN HERSEY, J.D. Senior Intellectual Property Counsel, Office of Intellectual Property Counsel, Massachusetts Institute of Technology, Cambridge, MA *DANIEL J. KEVLES, Ph.D. Professor, Yale University, Department of History, New Haven, CT LAUREN LINTON, Ph.D., M.B.A. President, Linton Consulting, Lincoln, MA *WILLIAM W. MCGUIRE, M.D. Chairman and Chief Executive Officer, UnitedHealth Group, Minnetonka, MN *JOHN MENDELSOHN, M.D. President, University of Texas, M.D. Anderson Cancer Center, Houston, TX Copyright © 2003 National Academy of Sciences. All rights reserved. Unless otherwise indicated, all materials in this PDF File provided by the National Academies Press (www.nap.edu) for research purposes are copyrighted by the National Academy of Sciences. Distribution, posting, or copying is strictly prohibited without written permission of the NAP. Generated for liuhan68@yahoo.com on Tue Jul 15 05:46:27 2003 http://books.nap.edu/catalog/.html v *KATHLEEN H. MOONEY, Ph.D. Professor and Peery Presidential Endowed Chair in Nursing Research, University of Utah College of Nursing, Salt Lake City, UT *NANCY MUELLER, Sc.D. Professor of Epidemiology, Harvard School of Public Health, Department of Epidemiology, Boston, MA *PATRICIA A. NOLAN, M.D., M.P.H. Director, Rhode Island Department of Health, Providence, RI *CECIL B. PICKETT, Ph.D. Executive Vice President, Discovery Research, Schering Plough Institute, Kenilworth, NJ STEPHEN PRESCOTT, M.D. Executive Director H.A. and Edna Benning Presidential Chair in Human Molecular Biology and Genetics, Huntsman Cancer Institute, University of Utah, Salt Lake City, UT *LOUISE B. RUSSELL, Ph.D. Research Professor of Economics, Institute for Health, Rutgers University, New Brunswick, NJ *THOMAS J. SMITH, M.D., F.A.C.P. Professor, Medical College of Virginia at Virginia Commonwealth University, Division of Hematology, Richmond, VA *SUSAN WEINER, Ph.D. President, The Children’s Cause, Silver Spring, MD *ROBERT C. YOUNG, M.D. President, American Cancer Society and the Fox Chase Cancer Center, Philadelphia, PA STUDY STAFF SHARYL J. NASS, Ph.D. Study Director ROGER HERDMAN, M.D. Director, National Cancer Policy Board MARYJOY BALLANTYNE Research Associate NICCI DOWD Administrative Assistant (through January 2003) NAKIA JOHNSON Project Assistant (from February 2003) *Members of the National Cancer Policy Board, Institute of Medicine, The National Academies. Copyright © 2003 National Academy of Sciences. All rights reserved. Unless otherwise indicated, all materials in this PDF File provided by the National Academies Press (www.nap.edu) for research purposes are copyrighted by the National Academy of Sciences. Distribution, posting, or copying is strictly prohibited without written permission of the NAP. Generated for liuhan68@yahoo.com on Tue Jul 15 05:46:27 2003 http://books.nap.edu/catalog/.html vi REVIEWERS This report has been reviewed in draft form by individuals chosen for their diverse perspectives and technical expertise, in accordance with pro- cedures approved by the 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 integ- rity of the deliberative process. We wish to thank the following individu- als for their review of this report: Mina J. Bissell, Ph.D. Distinguished Scientist, Life Sciences Division, Lawrence Berkeley National Laboratory Marvin Cassman, Ph.D. Director, QB3 at University of California, San Francisco Mildred Cho, Ph.D. Senior Research Scholar and Acting Co-director, Stanford Center for Biomedical Ethics Carol Dahl, Ph.D. Biospect, Inc. Chi Dang, M.D., Ph.D. Professor, Division of Hematology, Johns Hopkins University Department of Medicine Alfred G. Gilman, M.D., Ph.D. Regental Professor and Chairman, Department of Pharmocology, University of Texas Southwestern Medical Center Allen S. Lichter, M.D. Newman Family Professor of Radiation Oncology, Dean, University of Michigan Medical School Candace Swimmer, Ph.D. Research Fellow, Department of Genome Biochemistry, Exelixis, Inc. Shirley M. Tilghman, Ph.D. President, Princeton University Although the reviewers listed above have provided many construc- tive comments and suggestions, they were not asked to endorse the con- clusions or recommendations nor did they see the final draft of the report before its release. The review of this report was overseen by Enriqueta C. Bond, Ph.D., President, Burroughs Wellcome Fund and Charles E. Phelps, Ph.D., Provost University of Rochester. Appointed by the Na- tional Research Council and Institute of Medicine, they were responsible for making certain that an independent examination of this report was carried out in accordance with institutional procedures and that all re- view comments were carefully considered. Responsibility for the final content of this report rests entirely with the authoring committee and the institution. Copyright © 2003 National Academy of Sciences. All rights reserved. Unless otherwise indicated, all materials in this PDF File provided by the National Academies Press (www.nap.edu) for research purposes are copyrighted by the National Academy of Sciences. Distribution, posting, or copying is strictly prohibited without written permission of the NAP. Generated for liuhan68@yahoo.com on Tue Jul 15 05:46:27 2003 http://books.nap.edu/catalog/.html vii The committee gratefully acknowledges the contributions of many individuals who provided invaluable information and data for the study, either through formal presentations or through informal contacts with the study staff: Herman Alvarado, Bi Ade, Lee Babiss, Wendy Baldwin, John Carney, Robert Cook-Deegan, Carol Dahl, James Deatherage, Joseph DeRisi, Marie Freire, Jack Gibbons, John Gohagan, Eric Green, Judith Greenberg, Ed- ward Hackett, Edward Harlow, Nathaniel Heintz, David Hirsh, Nancy Hopkins, James Jensen, Marvin Kalt, Richard Klausner, William Koster, Rolph Leming, Joan Leonard, Arnold Levine, David Livingston, Rochelle Long, David Longfellow, Michael Lorenz, Richard Lyttle, Pamela Marino, Richard Nelson, Emanuel Petricoin, Michael Rogers, Jacques Rossouw, Walter Schaefer, William Schraeder, Stuart Schreiber, Edward Scolnick, Scott Somers, Paula Stephan, Marcus Stoffel, Robert Strausberg, Daniel Sullivan, Roy Vagelos, Craig Venter, LeRoy Walters, Barbara Weber, Michael Wigler, Robert Wittes. Acknowledgments Copyright © 2003 National Academy of Sciences. All rights reserved. Unless otherwise indicated, all materials in this PDF File provided by the National Academies Press (www.nap.edu) for research purposes are copyrighted by the National Academy of Sciences. Distribution, posting, or copying is strictly prohibited without written permission of the NAP. Generated for liuhan68@yahoo.com on Tue Jul 15 05:46:27 2003 http://books.nap.edu/catalog/.html Copyright © 2003 National Academy of Sciences. All rights reserved. Unless otherwise indicated, all materials in this PDF File provided by the National Academies Press (www.nap.edu) for research purposes are copyrighted by the National Academy of Sciences. Distribution, posting, or copying is strictly prohibited without written permission of the NAP. Generated for liuhan68@yahoo.com on Tue Jul 15 05:46:27 2003 http://books.nap.edu/catalog/.html ix AAAS – American Association for the Advancement of Science AEC – Atomic Energy Commission (forerunner of DOE) AFCS – Alliance for Cellular Signaling AIP – American Institute of Physics AUTM – Association of University Technology Managers BAA – Broad Agency Announcement CDC – Centers for Disease Control and Prevention CEPH – Centre d’Etude du Polymorphisme Humaine CERN – Conseil European Pour La Rechierche Nucleaire CES – Cooperative Extension Services CGAP – Cancer Genome Anatomy Project COSEPUP – Committee on Science, Engineering, and Public Policy CRADA – Cooperative Research and Development Agreement CSR – Center for Scientific Review DARPA – The Defense Advanced Research Projects Agency DHHS – Department of Health and Human Services DOD – Department of Defense DOE – Department of Energy DTP – Developmental Therapeutics Program EDRN – The Early Detection Research Network EPA – Environmental Protection Agency EST – Expressed Sequence Tag Acronyms Copyright © 2003 National Academy of Sciences. All rights reserved. Unless otherwise indicated, all materials in this PDF File provided by the National Academies Press (www.nap.edu) for research purposes are copyrighted by the National Academy of Sciences. Distribution, posting, or copying is strictly prohibited without written permission of the NAP. Generated for liuhan68@yahoo.com on Tue Jul 15 05:46:27 2003 http://books.nap.edu/catalog/.html x ACRONYMS FDA – Food and Drug Administration GPRA – Government Performance and Results Act HGP – Human Genome Project HHMI – Howard Hughes Medical Institute HRT – Hormone Replacement Therapy HUGO - Human Genome Organization HUPO – Human Proteome Organization INS – Immigration and Naturalization Service IRG – Integrated Review Groups IUPAP – International Union of Pure and Applied Physics JCSG – Joint Center for Structure Genomics MBL – Marine Biology Laboratory MMHCC – Mouse Models of Human Cancers Consortium MOU – Memoranda of Understanding NACA – National Advisory Committee for Aeronautics NAS – National Academy of Sciences NASA – National Aeronautics and Space Administration NCAB – National Cancer Advisory Board NCI – National Cancer Institute NDRC – National Defense Research Committee NHGRI – National Human Genome Research Institute NHLBI – National Heart Lung and Blood Institute NIAID – National Institute of Allergy and Infectious Diseases NIEHS – National Institute of Environmental Health Science NIGMS – National Institute of General Medical Sciences NIH – National Institutes of Health NOAA – National Oceanic and Atmospheric Administration NOARL – Naval Oceanographic and Atmospheric Research Laboratory NRAC – Naval Research Advisory Committee NRC – National Research Council NRSA – National Research Service Awards NSF – National Science Foundation NTP – National Toxicology Program OES – Office of Experiment Stations OMB – Office of Management and Budget ONR – Office of Naval Research Copyright © 2003 National Academy of Sciences. All rights reserved. Unless otherwise indicated, all materials in this PDF File provided by the National Academies Press (www.nap.edu) for research purposes are copyrighted by the National Academy of Sciences. Distribution, posting, or copying is strictly prohibited without written permission of the NAP. Generated for liuhan68@yahoo.com on Tue Jul 15 05:46:27 2003 http://books.nap.edu/catalog/.html [...]... 109 Nonfederal funding of large-scale biomedical research projects, 115 Industry Funding of Large-Scale Biomedical Research, 116 Nonprofit Funding of Large-Scale Biomedical Research, 123 Issues associated with international collaborations, 125 Summary, 126 5 ORGANIZATION AND MANAGEMENT OF LARGESCALE BIOMEDICAL RESEARCH PROJECTS Examples of management assessment for large-scale projects, 131 80 130 Copyright... as research tools may offer the greatest challenge in this regard because it is difficult to predict the future applications and value of a particular tool, and because a number of different tools may be needed for a single research project Since many large-scale projects in the biosciences aim to produce data and other tools for future research, this subject is especially salient for large-scale research. .. field of biomedical research is adapting to the inclusion of large-scale projects • Identify obstacles to the implementation of large-scale projects in cancer research • Provide policy recommendations for improving the process for conducting large-scale projects in cancer research should they be undertaken in the future This report is organized as follows Chapter 2 develops a working definition of large-scale. .. Associated with Large-Scale Projects Potential Paths to Solutions No systematic method for assessing large-scale biomedical research opportunities exists Develop an NIH-wide mechanism for soliciting and reviewing proposals for large-scale projects, with input from all relevant sectors of biomedical science Carefully planning and orchestrating the launch as well as the phase out of a large-scale project... staff for large-scale projects, and for recognizing and rewarding scientific collaborations and team-building efforts • Funding agencies should develop appropriate career paths for individuals who serve as program managers for the large-scale projects they fund • Academic institutions should develop appropriate career paths, including suitable criteria for performance evaluation and promotion, for those... Research Projects Agency, 74 Summary, 77 4 FUNDING FOR LARGE-SCALE SCIENCE History of federal support for scientific research, 82 Allocation of federal funds for scientific research, 83 NIH funding, 94 Congressional Appropriations to NIH, 95 NIH Peer Review of Funding Applications, 105 Funding Mechanisms for Extramural Research and Solicitation of NIH Grant Applications, 109 Nonfederal funding of large-scale. .. accomplished through a large-scale science venture in biology But is this the best or only way to take on future large-scale biomedical research? There are other strategies for funding and organizing such projects, some of which have never been used in biology but have worked well in other scientific fields Because the concept of large-scale science is relatively new to the field of biomedical research, and... large-scale science” with respect to cancer research; (2) identify examples of ongoing large-scale projects to determine the current state of the field; (3) identify obstacles to the implementation of largescale projects in biomedical research; and (4) make recommendations for improving the process for conducting large-scale biomedical science projects, should such projects be undertaken in the future. .. initiation and phase-out of a large-scale project Careful assessment of past and current large-scale projects to identify best practices and determine whether the large-scale approach adds value to the traditional models of research would also provide highly useful information for future endeavors Recommendation 1: NIH and other federal funding agencies that support large-scale biomedical science (including... research • Examine the current state of large-scale science in biomedical research (what is being done and how) • Examine other potential models of large-scale biomedical research Copyright © 2003 National Academy of Sciences All rights reserved Unless otherwise indicated, all materials in this PDF File provided by the National Academies Press (www.nap.edu) for research purposes are copyrighted by the . Cataloging-in-Publication Data Large-scale biomedical science : exploring strategies for future research / Sharyl J. Nass and Bruce W. Stillman, editors ; Committee on Large-scale. Policy Board, 15 2 DEFINING LARGE-SCALE SCIENCE” IN BIOMEDICAL RESEARCH 17 Examples of potential large-scale biomedical research projects, 20 Genomics,