Breast Cancer: Setting Priorities for Effectiveness Research (1990)

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. The original objectives of the Effectiveness Initiative were, first, to assess the overall merit of competing health care interventions and, second, to provide information that would help clinicians in the management of their patients, assist and improve the peer review process, for instance, of the Medicare Peer Review Organizations (PROs), and aid policymakers in allocating Medicare resources. HCFA identified four activities for its Effectiveness Initiative: (1) monitoring time trends in the use of services by the Medicare population; (2) analyzing geographic (population-based) variations in the use of services and in outcomes of care; (3) assessing interventions including clinical demonstrations, observational studies, and randomized controlled trials (RCTs) in addition to monitoring and variations analyses; and (4) feeding back information to clinicians in an educational orientation.

The progress of planning for effectiveness and related research throughout DHHS since 1988, and the movement of responsibility for effectiveness research into the Public Health Service as part of the Medical Treatment Effectiveness Program, makes clear that additional purposes for this program are important as well. Among these are improving patient outcomes, providing information useful in the development of practice guidelines, and identifying critical issues for further research. Thus, although this report covers work conducted for HCFA in the context of the original Effectiveness Initiative, it should be seen as pertinent to the full range of activities anticipated for effectiveness research supported by DHHS.

HCFA had consulted widely with individuals and organizations in the medical, health financing, and health services and policy research communities for guidance on its program initiative. It then requested the Institute of Medicine (IOM), National Academy of Sciences, to make recommendations about clinical conditions that should receive priority attention. Conceptually, this emphasis on the clinical condition reflected a decision to choose this unit of analysis rather than focus on specific procedures or technologies.

To respond to HCFA’s initial request for assistance in this planning, the IOM formed a study committee and convened a “clinical workshop” in October 1988. The clinical workshop committee recommended five clinical problem priority areas: stable and unstable angina, acute myocardial infarction, breast cancer, congestive heart failure, and hip fracture.¹ 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.

Apart from these points, the 1988 clinical workshop committee concluded that breast cancer takes on added importance because its incidence rises with age, which has special implications for morbidity and mortality as the elderly population grows and becomes predominantly female. Breast cancer was seen as a model for studies of diagnosis and management strategies in other neoplastic disease. The workshop committee also highlighted several patient management topics that deserved investigation: screening issues (e.g., who should be screened, how often); alternative approaches to diagnosis (e.g., screening versus diagnostic use of mammography, physical breast examination, biopsy); staging of disease; therapeutic approaches, such as medical versus surgical interventions, alternative surgical options (e.g., how extensive surgery should be in elderly women), use of radiation therapy and adjunct chemotherapy; rehabilitation issues, including use and type of prostheses; and emotional health.

Following the clinical workshop, HCFA asked the IOM to conduct condition-specific workshops on research and methods for three of the five high-priority clinical areas—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 central topics within each condition deserving further investigation in terms of “effectiveness” as contrasted with “efficacy”; and (3) to propose appropriate research strategies and methods.

The point about effectiveness and efficacy is especially important in this context. Efficacy is typically taken to mean the outcome of an intervention when it is applied in “ideal,” well-controlled circumstances, such as those inherent in prospective randomized controlled trials (RCTs). 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 “everyday” or “average” circumstances, such as the daily practice of medicine broadly defined. These everyday situations may involve patient subgroups that differ marginally, or even considerably, from those studied in RCTs and have outcomes that extend more into quality-of-life concerns, such as physical and social functioning and emotional well-being.

For breast cancer, a great deal of data about the efficacy of certain diagnostic or therapeutic interventions is available, often on women 50 and older, but generally not in the Medicare age group (65 and older). By contrast, little information on effectiveness as defined above is available for any age group. Because including elderly women in efficacy studies and RCTs is likely to remain rare, and because quality of life looms very large for many women with breast cancer, the need for effectiveness studies becomes even more important.

For the research workshops, the IOM appointed a “core committee” of clinicians and researchers chaired by Kenneth I.Shine, M.D., Dean of the School of Medicine, University of California, Los Angeles.² For each condition-specific research workshop, the core committee was augmented by additional clinicians and researchers with recognized expertise in the condition of interest.

This reports covers the breast cancer workshop. The committee had two charges: (1) to recommend to the HCFA administrator a small number (five to eight) of breast cancer patient management issues in elderly women 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 databases might be used to accomplish the research, what should be added to the current databases, and what studies must be conducted independently of the HCFA databases.

The remainder of this report summarizes background information on clinical aspects of breast cancer and conveys the committee’s deliberations, findings, and recommendations. Appendix A documents the study and workshop activities.