Mind the Gap: Efficacy Versus Effectiveness and Pivotal Prostate Cancer Clinical Trial Demographics

摘要

The results of clinical trials are critical to informing the care of patients with cancer. These results are used as a basis for the regulatory approval of new interventions, to construct treatment guidelines or pathways, and to facilitate shared medical decisions. However, despite being considered among the highest levels of evidence in the evidence-based medicine paradigm, clinical trials are not without limitations. Trials are expensive to conduct, typically generate results slowly, may not be published, may not be published in a transparent manner, or may be discontinued without completing enrollment.1"3 Perhaps the most often cited limitation of clinical trials, however, is the potential lack of generalizability of the results. Restrictive eligibility criteria and socioeconomic and geographic barriers to participation often result in study populations that do not resemble the general population of patients with the disease being studied, leading to a gap between the "efficacy" and "effectiveness" of new treatments.