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PREPUBLICATION COPY, UNCORRECTED PROOFS Improving Health Research on Small Populations PROCEEDINGS OF A WORKSHOP Nancy Kirkendall and Jordyn White, Rapporteurs Committee on National Statistics Division of Behavioral and Social Sciences and Education Board on Health Care Services Health and Medicine Division ADVANCE COPY NOT FOR PUBLIC RELEASE BEFORE Friday, May 4, 2018 9:00 am. EDT
PREPUBLICATION COPY, UNCORRECTED PROOFS THE NATIONAL ACADEMIES PRESS 500 Fifth Street, NW Washington, DC 20001 This activity was supported by contracts between the National Academy of Sciences, Engineering and Medicine and Contract No. 10003551/ Sponsor Award No. HHSN26300116 from the United States National Institutes of Health and Contract No. 10003839/Sponsor Award #75001 from the Robert Wood Johnson Foundation. Any opinions, findings, conclusions, or recommendations expressed in this publication do not necessarily reflect the views of any organization or agency that provided support for the project. International Standard Book Number-13: International Standard Book Number-10: Digital Object Identifier: https://doi.org/10.17226/2512 Additional copies of this publication 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. Copyright 2018 by the National Academy of Sciences. All rights reserved. Printed in the United States of America Suggested citation: The National Academies of Sciences, Engineering, and Medicine. (2018). Improving Health Research on Small Population: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: https://doi.org/10.17226/25112.
PREPUBLICATION COPY, UNCORRECTED PROOFS The National Academy of Sciences was established in 1863 by an Act of Congress, signed by President Lincoln, as a private, nongovernmental institution to advise the nation on issues related to science and technology. Members are elected by their peers for outstanding contributions to research. Dr. Marcia McNutt is president. The National Academy of Engineering was established in 1964 under the charter of the National Academy of Sciences to bring the practices of engineering to advising the nation. Members are elected by their peers for extraordinary contributions to engineering. Dr. C. D. Mote, Jr., is president. The National Academy of Medicine (formerly the Institute of Medicine) was established in 1970 under the charter of the National Academy of Sciences to advise the nation on medical and health issues. Members are elected by their peers for distinguished contributions to medicine and health. Dr. Victor J. Dzau is president. The three Academies work together as the National Academies of Sciences, Engineering, and Medicine to provide independent, objective analysis and advice to the nation and conduct other activities to solve complex problems and inform public policy decisions. The National Academies also encourage education and research, recognize outstanding contributions to knowledge, and increase public understanding in matters of science, engineering, and medicine. Learn more about the National Academies of Sciences, Engineering, and Medicine at www.national- academies.org.
PREPUBLICATION COPY, UNCORRECTED PROOFS Consensus Study Reports published by the National Academies of Sciences, Engineering, and Medicine document the evidence-based consensus on the studyâs statement of task by an authoring committee of experts. Reports typically include findings, conclusions, and recommendations based on information gathered by the committee and the committeeâs deliberations. Each report has been subjected to a rigorous and independent peer-review process and it represents the position of the National Academies on the statement of task. Proceedings published by the National Academies of Sciences, Engineering, and Medicine chronicle the presentations and discussions at a workshop, symposium, or other event convened by the National Academies. The statements and opinions contained in proceedings are those of the participants and are not endorsed by other participants, the planning committee, or the National Academies. For information about other products and activities of the National Academies, please visit www.nationalacademies.org/about/whatwedo.
PREPUBLICATION COPY, UNCORRECTED PROOFS STEERING COMMITTEE FOR IMPROVING HEALTH RESEARCH ON SMALL POPULATIONS: A WORKSHOP GRAHAM A. COLDITZ (Chair), Washington University School of Medicine JAMES ALLEN, University of Minnesota Medical School G. GRAHAM KALTON, Westat JANICE C. PROBST, University of South Carolina, Arnold School of Public Health LANCE A. WALLER, Emory University, Rollins School of Public Health NANCY J. KIRKENDALL, Project Director JORDYN WHITE, Senior Program Officer ANTHONY S. MANN, Program Associate v
PREPUBLICATION COPY, UNCORRECTED PROOFS BOARD ON HEALTH CARE SERVICES DAVID BLUMENTHAL (Chair), President, The Commonwealth Fund PETER BACH, Director, Center for Health Policy and Outcomes, Memorial Sloan Kettering Cancer Center OTIS BRAWLEY, Chief Medical Officer, American Cancer Society MELINDA J. BEEUWKES BUNTIN, Professor and Chair, Department of Health Policy, Vanderbilt University School of Medicine NIRANJAN BOSE, Deputy Chief of Staff, Gates Foundation, bgC3 NEIL S. CALMAN, President and CEO, The Institute for Family Health; Professor and System Chair, Family Medicine and Community Health, Icahn School of Medicine at Mount Sinai CAROLYN CLANCY, Executive in Charge, U.S. Veterans Health Administration PATRICIA M. DAVIDSON, Professor and Dean, School of Nursing, Department of Acute and Chronic Care, Johns Hopkins University JANE DELGADO, President and Chief Executive Officer, National Alliance for Hispanic Health JENNIFER E. DEVOE, Professor and Chair, Department of Family Medicine, Oregon Health & Science University R. ADAMS DUDLEY, Director, UCSF Center for Healthcare Value; Associate Director for Research, Philip R. Lee Institute for Health Policy Studies; Professor of Medicine and Health Policy, University of CaliforniaâSan Francisco RICHARD G. FRANK, Margaret T. Morris Professor of Health Economics, Department of Health Care Policy, Harvard Medical School TERRY FULMER, President, The John A. Hartford Foundation CINDY GILLESPIE, Executive Director, Arkansas Department of Human Services NADINE KASLOW, Professor, Emory University School of Medicine, Department of Psychiatry and Behavioral Science, Grady Health System CHRISTOPHER KOLLER, President, Milbank Memorial Fund DAVID B. PRYOR, Executive Vice President, Ascension, President and Chief Executive Officer, Ascension Clinical Holdings TRISH RILEY, Executive Director, National Academy for State Health Policy WILLIAM SAGE, James R. Dougherty Chair for Faculty Excellence, School of Law, Professor of Surgery and Perioperative Care, Dell Medical School, The University of Texas at Austin SHARYL NASS, Board Director vi
PREPUBLICATION COPY, UNCORRECTED PROOFS COMMITTEE ON NATIONAL STATISTICS ROBERT M. GROVES (Chair), Department of Mathematics and Statistics and Department of Sociology, Georgetown University FRANCINE BLAU, Department of Economics, Cornell University MARY ELLEN BOCK, Department of Statistics (emerita), Purdue University ANNE C. CASE, Woodrow Wilson School of Public and International Affairs, Princeton University MICHAEL E. CHERNEW, Department of Health Care Policy, Harvard Medical School JANET CURRIE, Woodrow Wilson School of Public and International Affairs, Princeton University DONALD A. DILLMAN, Department of Sociology, Washington State University CONSTANTINE GATSONIS, Center for Statistical Sciences, Brown University JAMES S. HOUSE, Survey Research Center, Institute for Social Research, University of Michigan THOMAS L. MESENBOURG, U.S. Census Bureau (retired) SUSAN A. MURPHY, Department of Statistics, University of Michigan SARAH M. NUSSER, Department of Statistics, Iowa State University COLM OâMUIRCHEARTAIGH, Harris School of Public Policy Studies, University of Chicago JEROME P. REITER, Department of Statistical Science, Duke University ROBERTO RIGOBON, Sloan School of Management, Massachusetts Institute of Technology JUDITH A. SELZTER, Department of Sociology, University of California, Los Angeles EDWARD H. SHORTLIFFE, Department of Biomedical Informatics, Columbia University and Arizona State University BRIAN HARRIS-KOJETIN, Director CONSTANCE F. CITRO, Senior Scholar vii
PREPUBLICATION COPY, UNCORRECTED PROOFS ACKNOWLEDGEMENTS This Proceedings of a Workshop was reviewed in draft form by individuals chosen for their diverse perspectives and technical expertise. The purpose of this independent review is to provide candid and critical comments that will assist the National Academies of Sciences, Engineering, and Medicine in making each published proceedings as sound as possible and to ensure that it meets the institutional standards for quality, objectivity, evidence, and responsiveness to the charge. The review comments and draft manuscript remain confidential to protect the integrity of the process. We thank the following individuals for their review of this proceedings: Graham A. Colditz, Prevention and Control, Siteman Cancer Center, Washington University, School of Medicine; Kelly J. Devers, Health Care, NORC at the University of Chicago; Peggye Dilworth- Anderson, Gillings School of Global Public Health, The University of North Carolina at Chapel Hill; Amy Kilbourne, Department of Psychiatry, University of Michigan, and Quality Enhancement Research Initiative, U.S. Department of Veterans Affairs. Although the reviewers listed above provided many constructive comments and suggestions, they were not asked to endorse the content of the proceedings nor did they see the final draft before its release. The review of this proceedings was overseen by Constantine Gatsonis, School of Public Health, Brown University. He was responsible for making certain that an independent examination of this proceedings was carried out in accordance with standards of the National Academies and that all review comments were carefully considered. Responsibility for the final content rests entirely with the rapporteurs and the National Academies. viii
PREPUBLICATION COPY, UNCORRECTED PROOFS CONTENTS 1. Introduction and Background 1 2. What Do We Mean by âSmall Populationsâ? 5 3. Challenges in Using Available Data for Small Population Health Research 13 4. Techniques Used in Survey Research to Identify and Find Small Populations 22 for Health Research 5. New and Emerging Designs for Intervention Studies 36 6. Recruitment, Retention, and Collection of Data 50 7. Analysis Techniques for Small Population Research 62 8. Closing Remarks 74 Appendixes A. Agenda and List of Participants 81 B. Biographical Sketches of Steering Committee and Speakers 92 ix
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