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PART II Overview
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Research on the Effectiveness of Medical Treatment: New Challenges and Opportunities ]. Jarrett Clinton In this chapter I present the perspective of the Department of Health and Human Services (DHHS) on today's environment for a new component of health services research: medical treatment effectiveness research. In a November 1988 issue of the New England Journal of Medicine, William Roper, then Administrator of the Health Care Financing Adminis- tration (HCFA), presented a bold plan to evaluate and improve medical practice in the United States. True, others had also called for and were engaged in measuring the outcomes of medical care, but Dr. Roper's article jolted many into realizing the considerable potential of these measures (particularly as they apply to quality of life) for improving the quality of medical care by using population-based data to indicate which practices are most effective. Dr. Roper made these activities essential components of HCFA's Effec- tiveness Initiative. He invited substantive collaborative efforts between the public and private sectors to: · share data bases among public and private payers, · create greater uniformity in the collection of information to determine and measure medical outcomes, · establish a critical role for practicing medical professionals in plan- ning and carrying out research on medical treatment outcomes, · accelerate the training of health professionals in evaluation sciences such as decision analysis and clinical epidemiology, and · refine medical practice guidelines (parameters and standards of medi- cal practice created by practitioners and their professional organizations). Arnold Relman, editor of the New England Journal of Medicine, stated in an accompanying editorial that ". . . no one should underestimate the size or difficulty of the task. However, the logical necessity of this seems clear. 21
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22 EFFECTIVENESS AND O UTCOMES IN HEALTH CARE We can no longer afford to provide health care without knowing more about its successes and failings. The Era of Assessment and Accountability is dawning at last." I expect that most of us endorse and enthusiastically support the posi- tions taken by Dr. Roper and Dr. Relman. For some, this thesis is already a guiding principle; for others, it is still a dream. For some, it may be a source of considerable anxiety. The concept, indeed, is challenging, pro- vocative, and fraught with difficulties. Yet it gives us extraordinary poten- tial for advancing the practice of health care. ~., . .. an. ... MEDICAL TREATMENT EFFECTIVENESS PROGRAM In fiscal year (FY) 1990 DHHS will expand the original Effectiveness Initiative enunciated by Dr. Roper and others into a more formal Medical Treatment Effectiveness Program. The increased visibility of effectiveness research, and professional assimilation of that research, reflects the DHHS belief that years of careful scientific studies in this area have produced strong and credible results. We intend to use these advances in knowledge, and the further questions they raise, to catalyze DHHS support for and participation in this dramatic effort. Secretary of Health and Human Services Louis Sullivan has assigned primary responsibility for this new program to the Public Health Service (PHS). Consistent with the Dr. Sullivan's desire that the Medical Treat- ment Effectiveness Program be a cohesive, department-wide effort, PHS is collaborating closely with HCFA to develop sound, fresh, and forward- thinking strategies. The long-term goal of the program is to change the assessment of health care services, research and financing from a focus on processes, that is, procedures and interventions, to a focus on patient outcomes of these pro- cesses. The central questions thus become: Has the patient improved? Has the quality of his or her life improved? By how much? The specific purpose of the Medical Treatment Effectiveness Program is to improve the effectiveness and appropriateness of health care services by enhancing our understanding of which health care practices are most effec- tive what works best. Four components, or sets of activities, form the basis of the program. Collection and development of data This will be undertaken to ex- pand the data bases available for analysis and to improve the ability to link Medicare files and other data bases on additional populations. 2. Research on patient outcomes and clinical effectiveness Specific treat- ment will be assessed through studies such as small area analysis and multidisciplinary epidemiological research. For example, in FY 1989 the
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POUCY AND RESEARCH ENVIRONMENTS 23 National Center for Health Services Research (NCHSR)~ of the PHS awarded four major research grants to assess alternative means of managing of myo- cardial infarction, different procedures for treatment of cataracts, management of prostatic hyperplasia, and nonsurgical interventions for lower back pain. In addition, NCHSR awarded planning grants for assessments in several areas, including total hip replacement, colon polyps, peripheral vascular disease, and ischemic heart disease. 3. Dissemination and assimilation offindings As outcomes research is completed, results will be widely disseminated through journal articles, in- formation networks, and conferences sponsored by HCFA and NCHSR. We will also make use of the resources and expertise of the National Library of Medicine and the Health Resources and Services Administration. As a component of the latter agency, the Bureau of Health Professions will con- vey appropriate information to geriatric education centers, family medicine departments, general internal medicine departments, and the network of area health education centers which, in some states, are powerful continuing education networks. We also intend to explore new approaches to medical education to ensure that research findings are incorporated in academic curricula, continuing education, and other professional education programs. 4. Practice guidelines The fourth and most challenging component of the Medical Treatment Effectiveness Program is the development of prac- tice guidelines, that is, parameters and standards of care. These guidelines must be created by practicing physicians, be based on science, and be practical, explicit, and subject to revisions as needed. The research findings gener- ated by this program and by others will facilitate the development of these guidelines. We expect this process to involve the full participation of the following: · professional organizations, such as the American Medical Associa- t~on, and specialty organizations, such as the American College of Physicians; · scientific bodies, including the Institute of Medicine (IOM); · academic medical centers; · standard-setting organizations, for example, the Joint Commission on Accreditation of Healthcare Organizations; · quality measurement organizations, such as Peer Review Organiza- tions (PROs); research-based organizations, for example, the American Medical Re- view Research Center and the Association of Health Services Research. . iAs of December 1989, NCHSR became the Agency for Health Care Policy and Research. The agency is the main source of federal support for research on problems related to the quality, delivery, and costs of health services.
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24 EFFECTIVENESS AND OUTCOMES IN HEALTH CARE In the not-too-distant future, nursing professionals must also be engaged to develop nursing care guidelines. Patient advocacy groups must be incor- porated to ensure that the programs, processes, guidelines, and measures are relevant and understandable from the patient's perspective. IMPLEMENTATION OF THE PROGRAM To accomplish the goals and objectives of the Medical Treatment Effec- tiveness Program, Dr. Sullivan intends to implement it from a departmental perspective. Much of the research activity and budget notations will be assigned to NCHSR. Much of the data development work, however, will be done by HCFA. In addition, all components of the PHS, including the National Institutes of Health, the Health Resources and Services Adminis- tration, the Food and Drug Administration, the Centers for Disease Control, and the Alcohol, Drug Abuse, and Mental Health Administration, will participate in program development, implementation, and review. The President's FY 1990 budget request for the program is $52 million. We plan to use this money to support a broad array of activities in each of the four program components-data development, research, dissemination of information, and development of guidelines. Congress is in the process of making final decisions on the President's budget. Collectively, we must ensure that this program has adequate resources to accomplish its far-reach- ing goals. It has a preliminary House mark of $20 million and a Senate mark of $35 million. I do not need to explain how essential it is that this program be adequately funded right from the start. Certainly, we will implement our agenda, even with reduced funding, but our progress will be slower and our goals more elusive if smaller budgets are appropriated. I want to reemphasize that the Medical Treatment Effectiveness Program has been incorporated into key objectives enunciated by Dr. Sullivan as goals for his administration at DHHS. We therefore have the full support of the DHHS in facing the greatest of challenges in health care. PRIORITIES FOR RESEARCH There has been substantial debate as to who would establish research topic priorities and by what criteria. HCFA has obviously asked the IOM to assist in setting priorities, and this volume contains summary judgments regarding the three clinical conditions that have received substantial review (1,2,31. We expect to respond to these, as well as to recommendations from appropriate advisory councils, Institutes in the National Institutes of Health, and the Alcohol, Drug Abuse, and Mental Health Administration.
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POLICY AND RESEARCH ENVIRONMENTS 25 Congress, too, powerfully affects us. The Senate Appropriations Com- mittee suggested the following topics for full consideration: · effectiveness of prevention services, · effectiveness of alcohol, drug abuse, and mental health treatment pro- grams, · effectiveness of nonphysician health providers, such as nurse practi tioners and physician assistants. Final judgments on broad research topics will consider all of these per- spectives. Through an intradepartmental committee incorporating PHS, HCFA, and other policy offices of DHHS, we are certain that a research agenda can be agreed upon. NEW PATTERNS OF COLLABORATION The work planned cannot be accomplished within the traditional patterns of relatively distinct and separate research undertakings. New teams of investigators, across disciplines, from the different institutions, and tran- scending traditional academic and geographic barriers, are essential for program progress. We need new constellations of researchers and data base manag- ers. To facilitate the assimilation of findings, we need the practitioners and the specialty societies. Yet, collaboration based on mutual cooperation and trust does not occur spontaneously. It requires each person, organization, and institution to re- affirm that quality of care in America transcends the traditional precepts held by each of these entities. It requires strong and visionary leadership. Finally, as one might expect, we have heard criticisms of this program. These include arguments that: · There are not sufficient researchers to undertake a large research pro- gram of this nature; The health services research community is not well organized or com- fortable with collaborative efforts; Social scientists and physicians have yet to demonstrate large-scale collaborative efforts; · The data bases for population-based research are inadequate; · The PRO data bases and processes are too dissimilar across states to create a unified national approach; · Organized medicine resists guidelines and parameters; · "True science" is found only in randomized controlled trials. We believe these arguments are not based on fact and that they ignore potential. Each is a challenge to the private sector and to government-a
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26 EFFECTIVENESS AND OUTCOMES IN HEALTH CARE challenge to commit ourselves to cooperative efforts to strengthen the sci- entific foundation on which clinical judgments rest. I have outlined here an exciting new program reflecting DHHS's deter- mination to ensure that medical care is of the highest quality. We know that there are no quick or easy answers to many of the questions surround- ing effectiveness of medical treatment. Because there are none, we are positioning the Medical Treatment Effectiveness Program for the long haul. Uncertainties will give way to scientifically sound research, and answers will come. By going forward with each component of our program~ata development, outcomes research, dissemination and assimilation of findings, and development of practice guidelines we will add more knowledge to the physician's armamentarium. A higher quality of care will become the new standard. We ask that the private sector join us in this effort. The synergism of federal-private sector collaboration will be the force moving us closer to our mutual goals. REFERENCES 1. Jackson,V.P. Breast Cancer. Pp.53-60in Effectiveness and OutcomesinHealth Care. Heithoff, K.A. and Lohr, K.N., eds. Washington, D.C.: National Academy Press, 1990. 2. Murray, D.G. Hip Fracture. Pp. 61-64 in Effectiveness and Outcomes in Health Care. Heithoff, K.A. and Lohr, K.N., eds. Washington, D.C.: National Academy Press, 1990. 3. McNeil, B.J. Claims Data and Effectiveness: Acute Myocardial Infarction and Other Examples. Pp. 65-70 in Effectiveness and Outcomes in Health Care. Heithoff, K.A. and Lohr, K.N., eds. Washington, D.C.: National Academy Press, 1990.