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1
Introduction
The need to set national priorities for the assessment of medical prac-
tice is becoming undeniable. The increasing interest of both the private
and public sectors in evaluating the effectiveness of medical practice is
coupled with finite economic and human resources for this purpose. A
national prionty-setting process is needed to identify assessment areas in
which investments in medical practice evaluation would make the most
impact. Pr~onties must be set among He clinical conditions Cat affect Be
U.S. population, and among the medical technologies and practices used
to prevent, diagnose, and treat those conditions. The goal of this project is
to demonstrate a pilot approach for setting national priorities for the as-
sessment of clinical conditions and medical technologies.
A growing national consensus among patients, provided, payers, health
care administrators, manufacturers, and legislators to find out "what works
in the practice of medicine" (Roper et al., 1988) characterizes this "era of
assessment and accountability" in health care (Relman, 1988~. Patients
are better informed consumers of heath care and are, appropriately,
taking increased responsibility for decisions about their health. Heath
care providers strive to integrate advances in medical technology in order
to offer the most effective diagnostic and therapeutic options to Heir
~ In this report the term electiveness is used as it is formally defined in the assessment
literature (see the Glossary). However, Me term has been used to refer to "what works in
the practice of medicine" and broadened to include the efficacy, effectiveness, and appro-
priateness of health services and their relationship to health care costs (Roper et al., 1988~.
9
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10
NATIONAL ASSESSMENT PRIORITIES
patients. As Me demand for health care sentences continues to increase,
reports of variations and possible overudlization are frequently cited
(Wennberg, 1984; Chassin et al., 1986; Roos et al., 1988~. As health care
costs continue to rise—fimm 6.0 percent of the U.S. gross national product
in 1966, to 8.6 percent in 1976, and to 10.9 percent in 1986 Rational
Center for Health Statistics, 1989b~both private and public payers of
health care become Increasingly interested In the cost-effec~veness of
medical practices and technologies.
The Department of Health and Human Services, responding to this
growing need for the assessment of medical practice, embarked on a
national Medical Treatment Effectiveness Program in fiscal year 1990.
This national program incorporated two preceding federal programs, the
Patient Outcome Assessment Research Program of He National Center
for Health Services Research2 and the Effectiveness Initiative of He
Health Care Financing Administration (HCFA)3 (Clinton, 19891. These
activities have been further expanded and reorganized under a new Agency
for Health Care Policy and Research (U.S. Congress, House, 1989~.
These recent expansions in federal research programs and resources
have focused national attention on medical effectiveness and patient
outcomes research The broad domain of"patient outcomes'$4 and "effec-
tiveness research' builds upon He work of related fields of He clinical
2 Ckigmating m 1986 legislation, the Patient Outcome Assessment Research Program
was expended in 1989 to sponsor four assessment teams that will investigate the sources of
emanation m medical care that result m adverse outcomes or inappropriate resource utiliza-
tion. Lee ultunate goal of the research activities is to feed back the infonnation about
outcomes and the costs of alternative practice patterns to practicing physicians (National
Cent" for Health Services Research. 1988~.
3 ~ 1988 the Health Care Financing Administration proposed an Effectiveness Initiative
that would include monitonug the outcomes associated with alternative treatments using
Urge data bases, icing variations in the outcomes and patterns of care, assessing
attentive interventions to reduce variations, and feeding this infonnation back to provid-
e" and patients to educate them about the results of these effectiveness studies (Roper et
aL, 1988; Instate of Medicme, 1989).
4 The focus of patient outcomes research is on the evaluation of medical interventions
hom the patia~t's perspective to consider, systematically, all the outcomes Mat maw to
patients, such as mortality, morbidity, complications of treatments, induction of symptoms,
improvement of fimcdonal status, and quality of life (Wennberg, 1990~.
~ Effectivaless research ~dwsses He evaluation of patient care delivered in the every-
day practice of medians. This is in contrast to studying He efficacy of medical technolo-
gies and practices under controlled experimental conditions such as randomized clinical
As.
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I~RODUCrlON
11
evaluative sciences, such as clinical research, epidemiology, health serv-
ices research, health status and functional assessment, medical decision-
analysis, medical technology assessment, and quality assessment. As
activities In the assessment of medical practice continue to develop at the
national level, Be emphasis on patient outcomes and effectiveness re-
search is bringing together the complementary perspectives and me~od-
olog~es of these fields.
In this report, the Institute of Medicine and its Council on Health Care
Technology demonstrate a pilot approach to setting national priorities for
the evaluation of medical practice that addresses the diverse assessment
interests that arise In a changing research, health care delivery, and policy
environment. This approach to setting national pnor~ties is based on the
broad-based participation of diverse parties and on a conceptual frame-
wow that integrates their perspectives. Although individual patients, pro-
viders, payers, and others continue to set priorities based on their respec-
tive needs and resources, a national agenda may stimulate evaluations of
broader interest or capture assessment topics of national importance Cat
are not being addressed. The pnonty-seuing group that chose the 20
national assessment priority areas identified in this pilot study represents
major health care constituencies, including academia, govemment, health
care providers, industry, and third-parLy payers. In addition, the concep-
tual framework for sewing priorities was developed after considenng
background information provided by 14 major heath care organizations
which span He field of health care delivery (see Appendix C).
During this pilot project, He council reviewed previous priority-sethng
approaches and identified two major types of pnonty-setting methods—
consensus and modeling. The consensus approach relies on the synthesis
of the expert opinion of a group of individuals. Consensus processes for
setting priorities may vary with respect to He formality of their approach
to identifying potential assessment topics, their reliance upon quantitative
information, and their use of implicit as well as explicit criteria to rank me
topics. These consensus approaches reflect the judgment of the individu-
als who form the prionty-setting group. The priorities identified by this
approach are influenced by the individuals' areas of expertise and their
role within broader hemp care systems. In spite of these limitadons,
consensus processes that involve wet/-balanced committees of experts
may be He only way to address problems when primary data are not
available or readily accessible. This consensus approach was used, for
instance, in 1988 by the Institute of Medicine when it appointed a com-
mittee of clinicians to provide advice to HCFA on initial pr~onties for He
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NATIONAL ASSESSMENT PRIORITIES
agency's Effectiveness Initiative. The priorities identified in that study
were limited to clinical conditions affecting the Medicare population.6
Modeling approaches involving quantitative methods may be used to
identify priority assessments or to generate estimates of their expected
value. To explore the feasibility of this approach, the council commis-
sioned two investigators to develop priority-setting models. Charles
Phelps of the University of Rochester developed an econometric model,
using a large hospice discharge data base, that estimates the annual wel-
fare loss nationally fin dollars) that may result from variations in medical
practice associated with the use of inpatient procedures for some cI=cal
conditions (Phelps and Parente, 1989~. The pnority-setting mode] of
David Eddy of Duke University uses evidence or, if necessary, expert
opinion to generate quantitative estimates of the expected value of an
assessment according to a defined set of health and economic outcomes
fly, in press). These modeling approaches add rigor to the pnonty-
setting process by defining objective cr~tena that facilitate quantitative
ranking of potential assessment topics. However, quantitative models are
necessanly limited by the degree to which assumptions are explicit and
reasonable, the availability and quality of data, and the difficulty of
accounting for important, but difficult to quantify, environmental and
other factors.
An ideal pnonty-setting methodology would account explicitly and
quantitatively for aU factors significantly influencing choices among po-
tential assessment topics. Such a pnonty-seuing process would be sub-
mitted to periodic review and modification so that developments In emerg-
ing technologies, the management of clinical conditions, or other factors
that affect decision-makers' perspectives may be taken into account. The
complexity of pnority-setting decision-making processes makes this ideal
difficult to attain. The judgment of decision-makers with different roles
in health care delivery may differ concerning which topics are of greater
pnonty, what standards or criteria to use for selecting them, and what
reladve importance to give these cntena. The pnma~y data for making
these decisions are often not available or readily accessible. As a result,
pnonty-se~ng decisions may involve implicit perceptions and judgments
6 The report of the Institute of Medicine Committee on the Effectiveness Initiative of He
Heals Care Financing Adrninistraiion was published as Effectiveness Initiative: Setting
Priorities for Clinical Conditions in April 1989 (Institute of Medicine, 1989). Although
the recommendations of that committee were among die sources for the pilot review of
priority-setting approaches for this report (see Appendix C), the process of determining the
national assessment priorities for this report was undertaken separately.
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I~RODUCHON
13
that may dominate these decisions (Weinstein and Statson, 1977~. To the
extent that such approaches for setting assessment priorities are Convex by
intuition or chance, they may not lead to the optimal use of limited
resources.
Recognizing the difficulties inherent in establishing national medical
assessment pnonties, the council can provide a national focus for pnonty-
seeting through its ability to convene experts from Me pertinent health
care sectors. The pilot study and proposal for future efforts described in
this report may evolve into national pnonty-seuing mechanisms that are
better and more efficient than the current ones.
By combining consensus and group judgment methods with a model-
ing approach, the national pnority-setting methodology proposed here
takes into account both objective and subjective factors Mat may be
important in pnority-setting decisions. The nationally applicable criteria
defined herein provide explicit standards by which different pnondes
may be ranked. During the pilot effort, the pnonty-sefflng group of the
council used the criteria outlined in Chapter 2 to choose the 20 high-
pnonty clinical condition and technology assessment areas described in
Chapter 3. In selecting this preliminary set of priorities from a larger list
of candidates (see Appendix A), the group considered seconder, data pro-
vided by a selection of health care organizations (see Appendix C). The
two-round modified Delphi process used to choose the pnondes is dis-
cussed in more detail In Chapter 2. For a foBow-up effort of this pilot
project, the council would like to develop and apply a decision-analysis
model to estimate, In a more quantitative way, the potential impact of
candidate assessment topics by all Me criteria outlined. This mode} would
draw on state-of-the-art information about Me potential assessment topics
and on a fuller review of the preferences that representatives of different
sectors, including patients and the public, may have for certain pnorities.
The national pnonty-sefflng approach descnbed here, therefore, is
distinct from previous approaches in two import ways. First, the
approach attempts to recognize the assessment needs of venous pardes
interested in the evaluation of medical practice and consolidate them unto
a set of criteria that will be nationally applicable to many patient popula-
tions. Second, Me conceptual framework used here accommodates Me
different manners in which assessment needs may arise. For instance, a
comparative evaluation of the available altemative medical technologies
or practices may be needed to determine the best or most appropn ate
approach for managing a clinical condition such as myocardial infarction
or hip fracture. On Me other hand, Me in~ducdon of a promising new
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14
NATIONAL A-SSE5SME2VTPRIORmES
technology, such as a genetic ally en~nee~d clot-l ysing agent, a compu -
tenzed radiolo~c system, a fonn of laser surgery, or a home diagnostic
test kit raises questions about safer, cost, or over effects of an individual
technology. Thus, the national priority-setting approach proposed here
uses bow a clerical condition approach and a technology approach In
considering national assessment pnonties.
The set of 20 priority areas listed in Chapter 3 provides a starting point
for the evaluation of medical practice. The hnstitute of Medicine and He
Council on Health Care Technology look forward to continued coopera-
tion with rep~senmives of pnv ate and public sector interests, in efforts ~
furler define He specific questions to be addressed about these general
priority areas and to improve the methodology for sewing pnondes. This
cooperation would help ~ ensure that this and filture sets of priority topics
can be established and addressed at He national level.
Representative terms from entire chapter:
assessment topics
