Abstract

There is a general agreement that before a therapy can be accepted into widespread clinical use, demonstration of efficacy in randomized clinical trials (RCTs) is absolutely necessary. There are, however, a growing number of experts who believe that demonstration of RCT efficacy might not be a sufficient basis for widespread acceptance.1 RCTs may not enroll subjects representative of patients in community settings, who may differ from RCT subjects in age, gender distribution, disease severity, comorbidity burden, and intensity of ancillary therapy. Additionally, randomized trials may not enroll sufficient numbers of subjects to demonstrate safety from rare adverse events. As a result of these differences, some therapies with demonstrated efficacy may have a smaller effect or no effect at all in community patients. For example, spironolactone reduced mortality in a randomized trial,2 but in community-based studies, its effectiveness was limited by rates of adverse events significantly higher than in the trial.3,4 Article see p 7 Payers and consumers are increasingly concerned about unmeasured gaps between efficacy and effectiveness. As a result, calls are escalating for comparative effectiveness research to guide decisions regarding which new treatments to pay for. For example, the state of Washington has a health technology assessment committee that recently recommended limiting reimbursement for drug eluting stents to patients with diabetes mellitus, treatment of left main coronary disease, treatment of in stent restenosis, or situations in which stent diameter is 3 mm or less or stent length is 15 mm or more, on the grounds that effectiveness beyond these relatively narrow indications has not been demonstrated.5 These strong arguments for making effectiveness research customary, however, run headlong into the reality of a healthcare system poorly able to conduct such work. First, funding for routine collection of effectiveness data is scarce, and financially squeezed healthcare organizations …

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