and type of practitioner, and by type of breast cancer case (e.g., identified solely by screening or as presented clinically). Other topics were based on specific classes of patients, such as those patients with node-negative disease. Throughout the discussion were expressions of concern about the relative lack in the HCFA database of necessary clinical and therapeutic data and how those deficiencies might be overcome through effectiveness research.

Eighteen specific study topics were advanced by committee members and recorded during the early part of the discussion; they are shown, in somewhat condensed form, in Table 4. Some relate to clinical issues involving the use of diagnostic or therapeutic modalities (e.g., mammography, surgical options); others relate more to research methods (e.g., outcomes measurement) or cross-cutting topics (e.g., age, node-negative disease). After discussion, the committee members voted for five topics that should receive the highest priority attention in effectiveness research on breast cancer. These were tabulated and the highest ranking topics formed the basis for the remaining part of the discussion. The following section elaborates this discussion and our recommendations.


This workshop discussion did not propose testable hypotheses or specific research questions. Rather, it was meant to arrive at a broader set of issues, the exploration of which would depend critically on the data that might be available in the near term (e.g., through the Medicare files) or that could be collected relatively efficiently by independent effectiveness research projects. Precision about topics is limited by the information that is available or could reasonably be seen to be become available. Even with this caveat, it became clear that a large number of useful questions might be addressed, and we discuss here the major groups of questions that we concluded an effectiveness research agenda might include.

First, we recommend separate attention to four nonclinical or methods topics:

  1. continued improvement and expansion of the database to serve the goals of effectiveness research;

  2. patterns of care and variations in those patterns;

  3. definitions and patterns of outcomes, with particular attention to dimensions not addressed yet by HCFA data or analysis, such as psychosocial, functional, and quality-of-life parameters; and

  4. other general issues of methodology, including indices of severity, case mix, and staging.

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