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PART I
Intro flu c t i on
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1
Genesis of the Effectiveness
Initiative and lOM's Role
Kim A. Heithoff, Kathieen N. Lohr, and
Richard A. Rettig
In 1988, the Health Care Financing Administration (HCFA) of the U.S.
Department of Health and Human Services (DHHS) proposed a research
program called the Effectiveness Initiative to bring the resources of Medi-
care to bear on the question of what works in the practice of medicine. This
initiative, in HCFA's view, was intended to help it fulfill its responsibilities
for ensuring the quality of care of some 30 million Medicare beneficiaries.
The initial objectives of the Effectiveness Initiative were (a) to assess the
merits of alternative health care interventions; (b) to provide information
that would help clinicians in the management of their patients; (c) to assist
and improve the Medicare program's quality assurance efforts; and (d) to
aid policymakers in allocating Medicare resources. The subsequent evolu-
tion of the DHHS effectiveness and outcomes research programs has made
it clear that improving patient outcomes is a unifying, primary objective and
that identifying additional issues for further research is also important.
HCFA originally identified the following activities as elements of the
Effectiveness Initiative: (a) monitoring time trends in the use of services by
the Medicare population; (b) analyzing geographic (population-based) variations
in the use of services and in outcomes of care; (c) assessing interventions
by clinical demonstrations, observational studies, and randomized controlled
trials (RCTs) in addition to monitoring and analyses of variations; and (d)
feeding information back to clinicians.
THE BROADER CONTEXT
The Effectiveness Initiative did not occur in a vacuum. Within HCFA, it
represented another step in the evolution of its responsibilities in quality
assurance. Its other responsibilities include the Peer Review Organizations
3
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4
EFFECTIVENESS AND OUTCOMES IN HEALTH CARE
(PROs), which followed the Professional Standards Review Organizations
(PSROs), and the periodic release of hospital mortality data, a highly con-
troversial step that has provided a powerful stimulus for clarifying the use-
fulness of mortality data as a measure of quality. In a different vein, HCFA
has acted, through its Bureau of Data Management and Strategy, to facilitate
the research community's access to its major data bases. In general terms,
then, the Effectiveness Initiative both extended these earlier efforts and brought
them into a more coherent framework.
Elsewhere in DHHS an Outcomes Research Program had been authorized
by Congress in 1987 and was being administered by the National Center for
Health Services Research (NCHSR). This program, inspired largely by the
work of John E. Wennberg and associates in small-area variations in utiliza-
tion and outcomes of medical interventions, invited research proposals in
late 1988 and announced the first four awards in September 1989, a few
weeks before the conference held by the Institute of Medicine (IOM). The
program intends to make a number of additional awards on a regular cycle.
Conceptually, outcomes and effectiveness research are very similar; dif-
ferences lie in legislative, administrative, and funding histories. Consequently,
when the DHHS, through Secretary Louis Sullivan, announced in mid-1989
that the department was including the HCFA Effectiveness Initiative in a
comprehensive outcomes and effectiveness research program, the announcement
was greeted with approval by many in the health services research commu-
nity.
Another strong general influence in the evolution of DHHS efforts has
been the emphasis on appropriateness of care. The issue here deals with
whether effective medical interventions-effective, that is, in the context of
normal practice situations-are being used appropriately or inappropriately,
given the indications for use and the characteristics of the particular patient.
Robert Brook and his colleagues have been strongly associated with this
research emphasis and have published several important papers in the past
few years. Appropriateness research, then, constitutes one more converging
stream of influence in the broader developments leading to the DHHS effort
in effectiveness and outcomes research.
Ideas in good currency influence Congress, as well as the executive branch.
Various legislative proposals in 1988 and 1989, therefore, culminated in the
Omnibus Budget Reconciliation Act of 1989 and a reorganization of part of
the Public Health Service (PHS). The NCHSR was disestablished, and the
Agency for Health Care Policy and Research was authorized in its place.
The new agency absorbed from the prior organization the functions of health
services research (including outcomes research) and technology assessment,
especially the PHS advisory function to Medicare.
In addition, a new function was added, namely, responsibility for devel-
oping medical practice guidelines. This responsibility represents congres
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INTRODUCTION
signal thinking that such guidelines represent the practical application of
outcomes and effectiveness research to the practice of medicine.
This set of developments evolution of the HCFA responsibility in qual-
ity assurance, emergence of outcomes research and its incorporation into
federal research programs, articulation of the general concern for appropri-
ateness and of specific research approaches in this area, and creation of a
new federal agency with responsibility for the development of practice
guidelines provides the nutrient bath in which the IOM contribution has
grown.
THE INSTITUTE OF MEDICINE'S CONTRIBUTION
In planning the Effectiveness Initiative, HCFA consulted widely in 1987
and 1988 with representatives of medicine, health financing, and health
services and policy research. It also coordinated its efforts closely with
other agencies of DHHS. In August 1988, William L. Roper' then Adminis-
trator of HCFA, asked Samuel O. Thier, president of the Institute of Medi-
cine, to convene a group of clinicians to advise the agency on the Effeci~ve-
ness Initiative. HCFA specifically asked the IOM for advice concerning
what clinical conditions ought to receive priority in the initial period of the
new program. Clinical conditions, rather than specific procedures or tech-
nologies, were chosen as the unit of analysis because they permitted exami-
nation of the full range of patient care opportunities, including prevention
and follow-up care.
THE IOM CLINICAL WORKSHOP
The IOM hosted a meeting of clinicians for the above purpose in October
1988. This "clinical workshop committee,' recommended that five condi-
tions receive highest priority: angina (stable and unstable); acute myocar-
dial infarction; carcinoma of the breast; congestive heart failure; and hip
fracture. These conditions were selected because of their high prevalence,
the substantial burden''they impose on elderly persons, appreciable varia-
tions in use of services and in outcomes, high costs, and the existence of
alternative ways of managing patient care that reflect professional and clinical
disagreement or uncertainty. The committee also recommended a second
tier of clinical conditions for later attention: cataracts, depressive disorders,
prostatic hypertrophy, and transient ischemic attacks with or without occlu-
sion.~
The report of this study was published as Effectiveness Initiative: Setting Priori-
ties for Clinical Conditions in April 1989; it is available from the National Academy
Press (Report No. IOM-89-04~.
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6
EFFECTIVENESS AND OUTCOMES IN HEALTH CARE
THE CONDITION-SPECIFIC WORKSHOPS
After the clinical workshop, HCFA asked the IOM to organize three
additional workshops, one on each of the three clinical conditions of highest
priority to the agency namely breast cancer, acute myocardial infarction,
and hip fracture. These condition-specific workshops were held in March,
May, and July 1989, respectively. Each had three objectives: (a) to identify
key research questions in more detail than had occulted at the October
workshop; (b) to identify critical patient care topics deserving further inves-
tigation; and (c) to propose appropriate research strategies and methods.2
The IOM appointed a core committee to oversee the entire series of
workshops. This core group included: Kenneth I. Shine (chair), Maureen
M. Henderson, Emmett B. Keeler, Barbara J. McNeil, David G. Murray,
Alan R. Nelson, J. Sanford Schwartz, G. Richard Smith, and Harold C. Sox.
(Drs. Shine, Murray, Nelson, Smith, and Sox had also been on the clinical
workshop committee.) For each meeting, IOM augmented the core group
with additional experts in the condition under consideration. The names of
all participants can be found in the committee rosters in the front of this
monograph.
EFFECTIVENESS AND OUTCOMES CONFERENCE
The September 1989 IOM conference, "Effectiveness and Outcomes in
Health Care," concluded this series of activities. It had four main objec-
tives: to explore the social, clinical, and legislative environment for research
on the topic of "what works in the practice of medicine"; to review the
conclusions and recommendations of the IOM workshops on breast cancer,
acute myocardial infarction, and hip fracture; to highlight four important
methodological issues In effectiveness studies, specifically, use of adminisaai~ve
data bases, collection of primary data, development and use of outcome
measures, and applications in clinical practice; and to examine the question
of where we go from here.
PROCEEDINGS
This book represents the proceedings of that conference and is divided
into five parts. The first is an introduction consisting of the foregoing
description of how the Effectiveness Initiative and IOM's role in it evolved
2The reports of the three research workshops have been or are being published as
a series: Breast Cancer; Hip Fracture; Acute Myocardial Infarction, with the subtitle
Setting Priorities for Electiveness Research. All are available from the National Academy
Press.
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INTRODUCTION
7
and the summary statement of the TOM core committee. Although most of
the issues in the summary are addressed in the individual workshop reports,
the committee believed it would be helpful to draw them together in a
single statement. That statement was distributed in advance to particpants
in the September conference to stimulate discussion and is published here
as Chapter 2.
The second, third, fourth, and fifth parts comprise the conference pro-
ceedings papers. Each section focuses on one of the four main objectives of
the conference described above.
