Summary

As part of its planning for an “Effectiveness Initiative,” the Health Care Financing Administration (HCFA) of the U.S. Department of Health and Human Services asked the Institute of Medicine (IOM) in late 1988 to conduct workshops on research topics and methods for three high-priority clinical conditions: breast cancer, acute myocardial infarction, and hip fracture. The research workshops had three objectives: (1) to examine each clinical condition in detail; (2) to identify key patient management topics for each condition that deserve further investigation in terms of “effectiveness” as contrasted with “efficacy”; and (3) to propose appropriate research strategies and methods. To conduct these workshops, the IOM appointed special study committees, and this report documents the IOM committee’s recommendations from the workshop on breast cancer.

The committee recommended that effectiveness research give separate attention to four nonclinical or methods topics. First was continued improvement and expansion of the database to serve the goals of effectiveness research. HCFA data sets, as presently constituted, can best be utilized to describe, track, and compare broad patterns of care for breast cancer according to subgroups of providers, practitioners, and patients. The committee emphasized the need to have adequate data on the ambulatory management of breast cancer, because analysis of the effectiveness of alternative therapies (e.g., chemotherapy, radiation, or tamoxifen) using only hospital records will have major limitations. These data may need to be collected separately.

Second was patterns of care and variations in those patterns. With respect to breast cancer, three generic sets of variables were thought to be important to comprehensive analyses of patterns of care and variations: geographic region; certain characteristics of institutional providers (e.g., types of hospitals; membership in “cancer center” programs); and profes-



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Breast Cancer: Setting Priorities for Effectiveness Research Summary As part of its planning for an “Effectiveness Initiative,” the Health Care Financing Administration (HCFA) of the U.S. Department of Health and Human Services asked the Institute of Medicine (IOM) in late 1988 to conduct workshops on research topics and methods for three high-priority clinical conditions: breast cancer, acute myocardial infarction, and hip fracture. The research workshops had three objectives: (1) to examine each clinical condition in detail; (2) to identify key patient management topics for each condition that deserve further investigation in terms of “effectiveness” as contrasted with “efficacy”; and (3) to propose appropriate research strategies and methods. To conduct these workshops, the IOM appointed special study committees, and this report documents the IOM committee’s recommendations from the workshop on breast cancer. The committee recommended that effectiveness research give separate attention to four nonclinical or methods topics. First was continued improvement and expansion of the database to serve the goals of effectiveness research. HCFA data sets, as presently constituted, can best be utilized to describe, track, and compare broad patterns of care for breast cancer according to subgroups of providers, practitioners, and patients. The committee emphasized the need to have adequate data on the ambulatory management of breast cancer, because analysis of the effectiveness of alternative therapies (e.g., chemotherapy, radiation, or tamoxifen) using only hospital records will have major limitations. These data may need to be collected separately. Second was patterns of care and variations in those patterns. With respect to breast cancer, three generic sets of variables were thought to be important to comprehensive analyses of patterns of care and variations: geographic region; certain characteristics of institutional providers (e.g., types of hospitals; membership in “cancer center” programs); and profes-

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Breast Cancer: Setting Priorities for Effectiveness Research sional characteristics of physicians (explicitly including physician specialty, such as “specialist” versus “generalist” and type of specialist). Third, definitions and patterns of outcomes were judged to need considerable attention. Outcomes should be more comprehensively defined in line with recent developments in the field of health status and quality-of-life measurement. Use of reliable and valid “generic” measures of health status coupled with selected “disease-specific” measures is an appropriate, desirable, and practical research strategy. For all patients, measures of morbidity (including pain and other symptoms), functional status (including physical capacity and ability to function in daily life), psychological and emotional well-being, social functioning and support networks, and general outlook on health are important. Patient values and preferences need to be explicitly taken into account, so it is important to differentiate between outcomes and patient preferences for outcomes and to encourage the acquisition and use of information on patient preferences, given different outcomes. The committee also recommended that agencies involved in effectiveness research solicit outside expert opinion to define and select an adequate, appropriate set of outcome measures (other than mortality) and instruments to be considered for use in this research effort generally and for use explicitly for breast cancer. The committee identified three other topics relating to outcomes. One was complications of surgery, especially in the Medicare population, and whether those complications vary by geographic region, by provider, or by type of surgery and whether the use of chemotherapy or tamoxifen is related to complications in any way. Another was toxicity of standard therapies (radiation and chemotherapy) in elderly women. The last was quality-of-life outcomes for women with recurrent breast disease. Fourth among the nonclinical or methods topics was that other general issues of methodology, including indices of severity, case mix, and staging, were felt to warrant separate attention. This included methods for gathering a minimum set of appropriate clinical data and for developing a standard severity-of-illness or staging index. Among the elements in such an index might be comorbidity (e.g., coexisting diagnoses of the sort that can be derived from Medicare Part A files), pathological stage and type of disease, and hormone receptor status. The committee also recommended three clinical or patient management topics for initial attention in the effectiveness research program targeted on breast cancer: screening and mammography; therapeutic alternatives for primary cancer, particularly as a function of age and stage of disease and of provider characteristics; and approaches to follow-up monitoring and care (including therapy for recurrent disease). With respect to screening and mammography, the committee recommended that agencies responsible for effectiveness research begin preliminary analy-

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Breast Cancer: Setting Priorities for Effectiveness Research ses and simultaneously undertake a detailed planning for a comprehensive prospective, longitudinal research program. Key topics include evaluating the effectiveness of mammography for screening as well as diagnosis; examining community versus medical center practices; interpreting mammograms; and interpreting information indicating that a women had a mammogram (presumably reimbursed by Medicare) when she received no subsequent care related to breast cancer. Related topics are the role of breast self-examination and physician examination (for screening) and the utility of other imaging modalities as a substitute for biopsy following a positive mammogram. Critical studies of primary treatments would focus on patterns of use of services and as patient outcomes for at least the following. Mastectomy—simple (total), modified radical (extended simple), radical, and extended radical; Lumpectomy (wide excision) with or without axillary node dissection; Lumpectomy (wide excision) with or without axillary node dissection and radiation; Postmastectomy radiation; Adjuvant chemotherapy in node-positive or node-negative disease; Adjuvant hormonal therapy; and Primary treatment of intraductal (noninvasive) breast cancer. Appropriate ways to follow up and monitor women initially treated successfully for primary disease is a major area of concern. The main impetus for this topic is the clinical controversy over what tests and other interventions should be used in standard follow-up protocols, with what frequency, and over what period of time. Studies on this topic would contribute to better understanding of the diagnosis and treatment of women with recurrent breast cancer.

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