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EFFECTIVENESS AND OUTCOMES IN HEALTH CARE Proceedings of an Invitational Conference by the INSTITUTE OF MEDICINE Division of Health Care Services Kim A. Heithoff and Kathleen N. Lohr, editors National Academy Press Washington, D.C. 1990

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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. The members of the committee responsible for this report were chosen for their special competencies and with regard for appropriate balance. The Institute of Medicine was chartered in 1970 by the National Academy of Sciences to enlist distinguished members of the appropriate professions in the examination of policy matters pertaining to the health of the public. In this, the Institute acts under both the Academy's 1863 congressional charter responsibility to be an adviser to the federal government and its own initiative in identifying issues of medical care, research, and education. This conference was supported by the Health Care Financing Administration, U.S. Department of Health and Human Services, under Basic Ordering Agreement Contract No. 500-89-0008. Library of Congress Catalog Card No. 90-63194 International Standard Book Number 0-309-04342-5 Publication 90-003 Additional copies of this report are available from: National Academy Press 2101 Constitution Avenue, NW Washington, DC 20418 S225 Printed in the United States of America

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INSTITUTE OF MEDICINE Division of Health Care Services HCFA Effectiveness Initiative CORE COMMITTEE KENNETH I. SHINE, Chair, Dean, School of Medicine, University of California, Los Angeles MAUREEN M. HENDERSON, Head, Cancer Prevention Research Center, Fred Hutchinson Cancer Research Center, Seattle, Washington EMMETT B. KEELER, Senior Mathematician, Economics Department, The RAND Corporation, Santa Monica, California BARBARA J. MCNEIL, Head, Department of Health Care Policy, Harvard Medical School, Boston, Massachusetts DAVID G. MURRAY, Professor of Orthopedic Surgery, Health Services Center, State University of New York, Syracuse ALAN R. NELSON, Associate, Memorial Medical Center, Salt Lake City, Utah J. SANFORD SCHWARTZ, Professor, General Internal Medicine, Hospital of the University of Pennsylvania, Philadelphia G. RICHARD SMITH, Associate Professor of Psychiatry, Department of Psychiatry, University of Arkansas for the Medical Sciences, Little Rock HAROLD G. SOX, Professor and Chairman, Department of Medicine, Dartmouth Medical School, Hanover, New Hampshire BREAST CANCER COMMITTEE MARTIN D. ABELOFF, Professor and Associate Director, Johns Hopkins Oncology Center, Baltimore, Maryland BARBARA DANOFF FOWBLE, Professor, Department of Radiologic Oncology, Hospital of the University of Pennsylvania, Philadelphia SHELDON GREENFIELD, Senior Scientist, Institute for the Improvement of Medical Care and Health, New England Medical Center, Boston, Massachusetts VALERIE P. JACKSON, Associate Professor, Department of Radiology, Wishard Memorial Hospital, Indiana University School of Medicine, Indianapolis LUELLA KLEIN, Professor and Chair, Department of Obstetrics and Gynecology, Emory University, Atlanta, Georgia MARY K. KNOBS, Section on Medical Oncology, Yale Medical Center, New Haven, Connecticut . . .

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JOHN S. MEYER, Pathology Department, St. Luke Hospital, Chesterfield, Missouri MONICA MORROW, Associate Professor of Surgery, University of Chicago, Chicago, Illinois WILLIAM C. WOOD, Medical Director, Cancer Center, Massachusetts General Hospital, Boston ACUTE MYOCARDIAL INFARCTION COMMITTEE HOOSHANG BOLOOKI, Jackson Memorial Hospital, Miami, Florida WILLIAM H. CARTER, The Charleston Cardiology Group, Charleston, West Virginia KATHLEEN DRACUP, Professor of Nursing, School of Nursing, University of California, Los Angeles KENNETH M. KENT, Director, Cardiac Catheterization Laboratory, Georgetown University School of Medicine, Washington, D.C. BRUCE C. PATON, Arapahoe Cardiovascular Surgeons, Denver, Colorado GERALD M. POHOST, Director, Division of Cardiovascular Disease, University of Alabama, Birmingham JOHN V. RUSSO, John Russo & Shellee Nolan, Washington, D.C. THOMAS J. RYAN, Chief of Cardiology, Boston University School of Medicine, Boston, Massachusetts HARRY P. SELKER, Director of Health Services Research Unit, New England Medical Center Hospital, Boston, Massachusetts GEORGE T. THIBAULT, Chief, Medical Service, VA Medical Center, West Roxbury, Massachusetts W. DOUGLAS WEAVER, Director, Division of Cardiology, University of Washington, Seattle MYRON L. WEISFELDT, Director of Cardiology, Johns Hopkins Hospital, Baltimore, Maryland NANETTE K. WENGER, Director, Cardiac Clinic at Grady Memorial Hospital, Emory University, Atlanta, Georgia HIP FRACTURE COMMITTEE CHRISTINE K. CASSEL, Chief, Section of General Internal Medicine, University of Chicago, Chicago, Illinois JOHN F. FITZGERALD, Assistant Professor of Medicine, Indiana University School of Medicine, Indianapolis HOWARD S. FRAZIER, Professor of Medicine, Harvard Medical School, Boston, Massachusetts JOHN J. GARTLAND, Director, Center for Research, Medical Education and Health Care, Jefferson Medical College, Philadelphia, Pennsylvania IV

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CAROL CLARKE HOGUE, Associate Professor, Center for Study of Aging and Human Development, University of North Carolina, Chapel Hill C. CONRAD JOHNSON, Chief of Endocrinology, Indiana University School of Medicine, Indianapolis ROSALIE A. KANE, Professor, School of Public Health, University of Minnesota, Minneapolis ROBERT B. KELLER, Executive Director, Maine Medical Assessment Foundation, Belfast ROBERT J. LLOYD, Arthritis Rehabilitation Center, Washington, D.C. JOHN L. MELVIN, Professor and Chair, Department of Physical Medicine and Rehabilitation, Medical College of Wisconsin, Milwaukee JANA M. MOSSEY, Professor, Department of Psychiatry, Medical College of Pennsylvania, Philadelphia RAYMOND J. RABIDOUX, President, Henry Ford Continuing Care Corporation, Detroit, Michigan WAYNE A. RAY, Associate Professor of Biostatistics and Director, Division of Pharmacoepidemiology, Vanderbilt University School of Medicine, Nashville, Tennessee STUDY STAFF Division of Health Care Services KARL D. YORDY, Director KATHLEEN N. LOHR, Deputy Director RICHARD A. RETTIG, Senior Staff Officer KIM A. HEITHOFF, Research Assistant PATRICK A. MATTINGLY, Consultant H. DONALD TILLER, Administrative Assistant THELMA L. COX, Senior Secretary THERESA H. NALLY, Senior Secretary Division of Health Promotion and Disease Prevention MARIA ELENA LARA, Program Officer v

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Acknowledgments The contributions of several members of the Institute of Medicine staff deserve special mention. Among them are H. Donald Tiller, administrative assistant, and Theresa Nally and Thelma Cox, senior secretaries. Kim Heithoff contributed greatly to the smooth logistics of the committee's workshop meetings and review of the first drafts of these proceedings. Richard Rettig, Kathleen Lohr, and Karl Yordy provided steady support and leadership throughout the entire project. The committee is particularly indebted to Blair Potter for the first draft edit of this report. Support for this study was provided by the U.S. Department of Health and Human Services, Health Care Financing Administration. We particularly wish to acknowledge the unflagging assistance and guidance of the government's project officer, John Spiegel, of the Health Standards and Quality Bureau. . . V11

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Preface This conference represented an important step in the Institute of Medicine's (IOM) effort to provide consultation to the Health Care Financing Administration (HCFA) in regard to its research on effectiveness. It provided an opportunity for the IOM's core committee to communicate its initial thoughts about the opportunities and challenges in this type of inquiry. It brought together many of the most knowledgeable individuals in the country whose insights were useful to government and to the research community in understanding the issues raised by effectiveness research. The IOM convened its committee of experts from clinical medicine, health services research, epidemiology, nursing, and a number of other areas in order to identify clinical conditions for effectiveness research. The deci- sion to identify conditions rather than procedures was based on the committee's desire to focus on the practice of medicine in a clinical setting, rather than upon technology assessment alone. At the same time, it was understood that all of the conditions which we identified can and will be included as part of the inquiries, procedures, and technology. Initially, five conditions hip fracture, breast cancer, angina pectoris, congestive heart failure, and acute myocardial infarction were identified by the committee for possible study. HCFA identified three of these condi- tions breast cancer, hip fracture, and acute myocardial infarction as its priorities. Three workshops were conducted to examine each at greater length. The membership of each workshop was drawn approximately equally from the committee and from individuals who are experts on the particular condition to be studied. Each workshop framed questions that might be asked in regard to the particular condition being explored, and attempted to identify the research strategies, approaches, and methodologies that might be used. In doing so, to

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x PREFACE the committee learned a great deal about the currently available HCFA databases and about other issues that must be confronted if effectiveness research is to fulfill its promise. The committee learned about the anxieties that conscientious clinicians and scientists have about effectiveness research. After each of the workshops, a report was generated for that particular condition. Subsequently, the committee drafted a statement about recurrent concerns which it believed required attention in order that the overall effort in effectiveness research be pursued successfully. That summary document was distributed prior to this conference. EFFECTIVENESS VERSUS EFFICACY Among the most important principles to be understood by clinicians and scientists is the difference between effectiveness and efficacy. The randomized clinical trial provides important information with regard to therapeutic al- ternatives. However, the vast majority of such trials have traditionally excluded the elderly. Without further evidence for each condition, the extrapolation of the results of such trials to the elderly may or may not be justified. Moreover, there is evidence that physicians themselves have made judg- ments about the nature of disease and about the treatment of elderly patients that are sometimes inconsistent with the results of clinical trials. For example, some physicians behave as if breast cancer in an elderly woman is a different disease than in a younger patient. As a result, the same range of therapy may not be offered. We must develop data that will allow judgments to be made rationally. This requires an understanding not only of what may be efficacious, but also of what is actually done and what the outcomes are in the real world of medical care. METHODOLOGIES Some have expressed anxiety that effectiveness studies may be performed to the exclusion of other kinds of scientific inquiry. This is both unrealistic and undesirable. It is essential that initial observations regardless of their source whether the HCFA databases or an investigator's imagination-be addressed by the most appropriate kinds of inquiry. This may require more detailed epidemiological study, demonstration projects, or randomized clinical trials. A few of these studies may be satisfactorily conducted by intramural programs of the federal government, but the vast majority are likely to require funding of research activities conducted by extramural investigators who work in a variety of settings.

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PREFACE Xl IMPACT UPON COSTS In general, the participants at this conference agreed that effectiveness research is an important endeavor that should be undertaken. There was a general assumption that the results of such research could help physicians make clinical decisions, but there was considerable uncertainty as to the best way of altering physician behavior. Results of effectiveness research might be expected to influence the management and organization of our health care system, but there was general consensus that the results of such research, in and of themselves, are not likely to alter the rate of growth of health care expenditures. However, it was strongly felt that information is absolutely essential if policymakers are to make rational decisions about management, organization, and reimbursement. RISK ADJUSTMENTS AND OUTCOMES While HCFA has regularly reported mortality data in a variety of formats, there was a strong consensus that such data were of limited value unless issues of morbidity, disability, function, and cost were also more satisfactorily considered. Comorbidity, risk stratification, and function assessment are major challenges. Recent studies of transurethral versus transabdominal prostatectomy, for example, suggest that small differences in a mortality rate associated with each procedure, which might range from 1 to 1.5 percent, could be invisible to an active urologist. However, the impact on the Medicare population as a whole might be several thousand deaths a year. At the same time, in the absence of a randomized clinical trial, we are limited in our ability to stratify for risk satisfactorily. Although significant progress is being made in this area, it is not uncommon to find that only 50 to 65 percent of outcomes can be predicted by currently available risk stratification. Clini- cians will continue to be skeptical of retrospective results when only a relatively small proportion of risk can be accurately adjusted. A particular highlight of the discussions was the potential role of the patient in providing assessments of morbidity, disability, and function. Patients' assessment may be of comparable accuracy with that of physicians, and, in some cases, much more easily obtainable. CONCLUSIONS As an educator, I am concerned that medical students and house staff understand issues of effectiveness and appropriateness. This will become more important in the future as health maintenance organizations, indepen

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. . Xl! PREFACE dent practice associations, insurance companies, government, corporations, employers, and employees seek to restrain health care costs. Without reli- able data, decisions about the provision of health care, the role of prevention, and rehabilitation will continue to be unduly influenced by economics and politics rather than by reason. Although the IOM core committee has iden- tified many concerns, I believe it is important to move forward in effectiveness research, not only with HCFA, but also though linkages with state programs such as Medi-Cal and the private insurance sector. Developments in government and industry will make this possible and imperative. Kenneth I. Shine Chair, Committee on the HCFA Effectiveness Initiative

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Contents ACKNOWLEDGMENTS PREFACE ..... ......... vii . ix Kenneth I. Shine PART I: INTRODUCTION 1. Genesis of the Effectiveness Initiative and IOM's Role 3 Kim A. Heithoff, Kathleen N. Lohr, and Richard A. Rettig 2. Promise and Limitations of Effectiveness and Outcomes Research ~ e ~ Summary Statement of the IOM Core Committee PART II: OVERVIEW Policy and Research Environments 3. Research on the Effectiveness of Medical Treatment: New Challenges and Opportunities ...................... J. Jarrett Clinton 4. The Health Care Financing Administration and the Effectiveness Initiative................................. Louis B. Hays 5. The Effectiveness Initiative: Retrospective and Prospects William L. Roper Perspectives on Effectiveness and Outcomes Research 6. The Social Perspective... Uwe E. Reinhardt 7. The Clinical Perspective .......... Paul F. Griner 8. The Legislative Perspective. . John D. Rockefeller, IV . . x`~t 21 27 31 34 38 44

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xiv CONTENTS PART III: THE IOM CLINICAL CONDITION WORKSHOPS Introduction ~51 Kenneth I. Shine 9. Breast Cancer ...................... Valerie P. Jackson 10. Hip Fracture ......................................... David G. Murray 11. Claims Data and Effectiveness: Acute Myocardial Infarction and Other Examples ................................... Barbara J. McNeil PART IV. METHODOLOGICAL ISSUES AND WORK IN PROGRESS Use of Large Data Bases Introduction ......... Emmett B. Keeler 12. The Role of Large Data Bases in Effectiveness Research Janet B. Mitchell 13. Administrative Data in Effectiveness Studies: The Prostatectomy Assessment...................... Elliott S. Fisher and John E. Wenaberg 14. Issues in the Use of Large Data Bases for Effectiveness Research .................... Stephen F. Jencks Collection of Primary Data Introduction ..................... Harold C. Sox 15. Measuring Patient Function and Well-Being: Some Lessons from the Medical Outcomes Study. . John E. Ware, Jr. 16. The Uniform Clinical Data Set........ Henry Krakauer Development and Use of Outcomes Measures Introduction ......... G. Richard Smith 17. Assessing Health-Related Quality of Life Outcomes. Donald L. Patrick 18. Using Patient Reports of Outcomes to Assess Effectiveness of Medical Care ................... Paul D. Cleary . . 53 - 61 65 73 ... 74 80 105 107 120 135 ...... 137 .152 19. Studying Outcomes for Patients with Depression: Initial Findings from the Medical Outcomes Study 160 M. Audrey Burnam

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CONTENTS Application to Clinical Practice Introduction . . J. Sanford Schwartz 20. Effectiveness Research and Changing Physician Practice Patterns ................................ Harold C. Sox 21. Applying Effectiveness and Outcomes Research to Clinical Practice ..................................... Albert G. Mulley 22. An Attempt to Manage Variation in Obstetrical Practice Stephen C. Schoenbaum 23. Using Outcome Measures to Improve Care Delivered by Physicians and Hospitals........................... Eugene C. Nelson PART V. WHERE DO WE GO FROM HERE? 24. The Need for Reasonable Expectations Henry J. Aaron 25. Use of Effectiveness Research in Managed Care Plans Howard L. Bailit 26. Gaining Acceptance for Effectiveness and Outcomes Research............................................ John D. Stobo List of Authors...... xv .171 ..... 173 179 ... 190 ... 201 ...... 215 .... 218 224 ...... 227

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