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2 Objectives, Principles, and Methods
Pages 26-37

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From page 26...
... Under the labels of technology assessment and evidence-based medicine, researchers, caregivers, payers, policymakers, and others have been seeking agreement on criteria, procedures, and techniques for evaluating evidence and reaching valid and credible conclusions about what works and what does not work in medical care. This chapter reviews the committee's principles for reaching conclusions and its analytic strategy for assessing evidence and estimating coverage costs.
From page 27...
... While acknowledging their importance, the committee also did not examine the full range of ethical, economic, cultural, political, and other issues relevant to decisions about Medicare coverage policies or other options for achieving health goals. Criteria and Trade-Offs For each intervention examined, the committee found it helpful to consider a version of the "evidence pyramid" that Figure 2-1 depicts for a generic health care intervention.
From page 28...
... Consistent with the discussion in (Chapter 1, Chapters 3, 4, and 5 each discuss why the effectiveness~of coverage in achieving desired health goals cannot be assumed. In practice, the application of the criteria represented in the evidence pyramids involves other trade-c~ffs besides the weighing of benefits against harms.
From page 29...
... For example, the analysis of eliminating the three-yeax limit on Medicare coverage of immunosuppressive drugs for transplant patients does not compare the estimated cost per life year gained from eliminating the limit with a similar estimate for extending coverage to outpatient antihypertensive drugs. ANALYTIC STRATEGY With the assistance of its consultants, the committee employed an analytic strategy that included several steps: (1)
From page 30...
... The committee and its consultants often found various tabular and graphic tools useful in analyzing the quite different kinds of clinical problems and interventions examined here.2 These tools helped the committee and consultants to 2See, for example, Detsky et al., 1997; Eddy et al., 1992; Owens et al., 1997; and Scheinkopf, 1999. Although this report omits a detailed discussion of the varied graphic and tabular tools used at intermediate stages of the committee's work, some that proved helpful included probability trees, decision trees, evaporating clouds, influence diagrams, current and future reality trees, and Venn diagrams.
From page 31...
... Some studies employ stronger research designs that allow more confidence in their findings than studies using weaker designs. Ideally, analysts would locate evidence directly relating the intervention to the outcomes of interest, for example, multiyear, properly randomized, controlled trials that followed people over age 65 who had been screened or not screened for skin cancer and then reported consistent findings.
From page 32...
... , the text includes reviews of the evidence on the efficacy of tests such as blood cultures for infectious diseases and mammography for breast cancer screening. Appendixes, which were developed in collaboration with the Blue Cross and Blue Shield Association using a formal process of evidence assessment and expert judgment, provided recommendations on the use of these tests in clinical practice.
From page 33...
... As a result, the committee had to rely on its own experience and expertise in suggesting how people might value different outcomes for themselves or others. For example, the committee judged that the scarring produced by most biopsies for false negative results for skin cancer screening examinations was not likely to be viewed by most people as an important risk of screening, whereas the disfigurement that might result from late diagnosis and surgical treatment of squamous cell carcinoma was likely to be viewed as important.
From page 34...
... This decision reflected the committee's wish to provide Congress with estimates that were based on familiar procedures. Unlike cost-effectiveness analyses intended to inform broad public policy decisions, the CBO approach does not take a societal perspective, nor does it recognize costs to beneficiaries, families, or others affected by coverage policies.
From page 35...
... The committee was not asked to estimate costs to the federal-state Medicaid program that might be added or reduced if Medicare extended coverage to the services examined in this report. For example, if the three-year limit on Medicare coverage of immunosuppressive drugs were eliminated, federal and state Medicaid costs should decrease because that program would be spending less for these drugs for beneficiaries who were eligible for both Medicare and Medicaid.
From page 36...
... The diagonal row of question marks indicates the less clear-cut decision situations, for example, the not uncommon circumstance that an intervention produces better results but at a higher cost. Cost pressures can focus attention on options that might produce worse outcomes but reduce Medicare costs.
From page 37...
... Even if the committee had gone further, it would have encountered difficulties given the limited evidence of effectiveness and the lack of quality-of-life or patient preference data for the interventions examined. Studies have compared health-related quality of life for patients on renal dialysis with posttransplant patients taking immunosuppressive drugs, but the committee did not find comparable data on the other conditions considered here.


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