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Introduction EFFECTIVENESS INITIATIVE In 1988, the Health Care Financing Administration (HCFA) of the U.S. Department of Health and Human Services (DHHS) proposed an Effectiveness Initiative, the purpose of which was to bring the resources of Medicare to bear on the question of what works in the practice of medicine. HCFA outlined two purposes for the Effectiveness Initiative: (1) assess the overall merit of competing health care interventions and (2) provide infor- mation that will help clinicians in the management of their patients, assist and improve He peer review process (e.g., of the Medicare Peer Review Organizations [PROs]), and aid policymakers in allocating Medicare resources. HCFA also identified a specific set of activities for the Effectiveness Initiative: (1) monitor time trends in the use of services by the Medicare population; (2) analyze geographic (population-based) variations in the use of services and in outcomes of care; (3) assess interventions through clinical demonstrations, observational studies, and randomized con- trolled trials (RCTs); and (4) conduct feedback and education activities. Other purposes are integral to this program as well. The progress in plan- ning for effectiveness and related research throughout DHHS since 198S, and the transfer of responsibility for effectiveness research to the Public Health Service as part of the Medical Treatment Effectiveness Program, make this fact clear. Among these over purposes are improving patient out- comes, providing information useful in the development of practice guide- lines, and identifying critical issues for further research. Thus, although this monograph discusses work conducted in 1989 for HCFA in the context of the original Effectiveness Initiative, it should be seen as pertinent to the full s

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6 EFFECTIVENESS INITIATIVE range of activities anticipated for effectiveness research supported by DHHS in 1990 and beyond. THE IOM CLINICAL WORKSHOP For guidance on this new program initiative, HCFA had consulted widely with individuals and organizations in the medical, health financing, and health services and policy research communities. It then requested the Institute of Medicine (IOM), National Academy of Sciences (NAS), to make recommendations about clinical conditions that should receive priori- ty attention at the outset of the agency's proposed Effectiveness Initiative. Conceptually, this emphasis on the clinical condition reflected a decision to choose this unit of analysis rather than to focus on specific procedures or technologies. To respond to HCFA's initial request for assistance in planning the Effectiveness Initiative, the Institute appointed a study committee and con- vened a "clinical workshop" in October 198X. The clinical workshop com- mittee recommended five clinical problem areas: stable and unstable angi- na, acute myocardial infarction (AM0, breast cancer, congestive heart fail- ure, and hip fracture. These conditions were given priority because they met several key selection criteria including high prevalence, burden of the illness on elderly persons, appreciable variations in the use of services and in outcomes, high costs, and alternative ways to manage patient care that reflect professional and clinical disagreement or uncertainty.! In addition to these general points, the 1988 workshop committee also noted that AMI is important in the elderly for several specific reasons. Outcomes for survivors of the acute event are highly variable and unpre- dictable, with different responses to treatment and variable long-term dis- ability. Practice patterns are changing rapidly because of new therapies and data. The committee also commented on other important dimensions of AMI, including (1) prevention, prognosis, and the role of exercise; (2) aspects of prehospital care, such as speed of diagnosis, resuscitation, initia- tion of treatment, and transport to sites of definitive care; (3) special man- agement issues relating to the elderly; (4) pharmacologic agents (e.g., thrombolytic, antiarrhythmic, and antiplatelet agents); (5) locus of care; (6) rehabilitation; (7) disability and quality of life, including return to work or daily functioning; and (8) psychological aspects of diagnosis, treatment, and prognosis (e.g., anxiety and depression). 1 The 1988 clinical workshop committee also Amended a second tier of clinical conditions that could receive later attention: cataracts' depressive disorders' prostatic hypeltraphy' and tran- slant ischemic attacks with or without occlusion. The report of this committee was published as Effectiveness Initiative: Setting Priorities for Clinical Conditions in April 1989; it is available from the National Academy Press (Report No. IOM-89 04).

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ACUTE HYOCARDIAL INFARCTION COND1TION-SPECIlIC RESEARCH WORKSHOPS 7 Purpose Following the clinical workshop, HCFA asked the IOM to conduct research and methods workshops for the three clinical areas on which the agency decided to focus first: breast cancer, AMI, and hip fracture. The research workshops had three objectives: (1) to examine each clinical condi- tion in greater detail; (2) to identify central topics within each condition deserting further investigation in terms of "effectiveness" as contrasted with "efficacy"; and (3) to propose appropriate research strategies and methods.2 The distinction between effectiveness and efficacy is especially important in this context. Efficacy typically refers to the outcome of an intervention when it is applied in "ideal," well-controlled circumstances, such as those inherent in prospective randomized controlled trials (RCTs). Gnat is, effica- cy is concerned with whether the intervention works at all. Outcomes of interest may be quite technical and oriented to physiologic variables and survival. By contrast, effectiveness is understood to mean the outcome of that intervention when it is applied in the "everyday" or "average" circum- stances of medical practice. These situations may involve patient subgroups that differ considerably from those studied in the RCIs. In addition, out- comes here may extend more broadly into quality-of-life concerns, such as physical and social functioning and emotional well-being. Recent multi-center clinical trials have established the efficacy of various diagnostic and therapeutic interventions in AMI (e.g., thrombolysis). These trials, however, do not always include the older Medicare-age patients, par- ticularly those over 75, and they typically do not consider outcomes beyond short-term mortality and improvement in physiologic functioning. Therefore, the effectiveness of some interventions in older elderly patients, particularly with respect to long-term physical and emotional functioning, remains unclear.3 Other questions not likely to be resolved by efficacy studies center on differences in medical interventions and outcomes in groups of patients who differ on demographic or clinical grounds. 2The reports an the other two workshops to identify patient management topics for breast cancer and hip fracture were published in 1990 (respectively, Breast Cancer: Setting Priorities for Effectiveness Research and Hip Fracture: Setting Priorities for Effectiveness Research). Both are available from the National Academy Press. 3Several recent clinical trials have studied the effectiveness of thrambolyiic agents (and in one study thrombolytics and percutaneous transluminal coronary angioplasty) in reducing in-hospital mortality from AMI. Some of these studies have included patients up to age 75 and have de~nonstrat- ed improved in-hospital survival in the over-70 age group comparable to the survival of younger patients (albeit with a sigr~ficantly greater occurrence of bleeding complications in the over-70 age group). However, these studies include few patients over age 75. Studies of other treatment modali- ties such as coronary artery bypass surgery have not generally included sufficient numbers of patients over 65 to enable definitive conclusions.

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8 EFFECTIVENESS INITIATIVE Research Workshop Committee To discharge the research workshop tasks, the IOM appointed a "core committee" of clinicians and researchers according to NAS procedures; it was chaired by Kenneth I. Shine, M.D., Dean of the School of Medicine, University of California, Los Angeles. For each condition-specific research workshop, the core committee was augmented with additional clinicians and researchers with recognized expertise in the condition of interest. The members of the AMI committee are listed at the beginning of this report. The committee was charged with two responsibilities: (1) to recommend to He HCFA administrator a small number (five to eight) of AMI patient management issues in the elderly that should receive priority in the Effectiveness Initiative and (2) after reaching some consensus on the patient management issues for initial study, to suggest specific research strategies or approaches that might be implemented to address those issues. An underlying premise was the need to understand what aspects of the present or proposed Medicare data bases might be used in this research, what should be added to the current data bases, and what studies must be con- ducted independently of the HCFA data bases. The remainder of this report summarizes the background information on He clinical aspects of AMI and conveys the committee's deliberations, find- ings, and recommendations. The appendix describes the study and work- . . . S. :lOp actlvltles.