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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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