Davis C E
Department of Biostatistics, University of North Carolina, School of Public Health, Chapel Hill 27599-7400.
Control Clin Trials. 1994 Feb;15(1):11-4. doi: 10.1016/0197-2456(94)90023-x.
Although randomized controlled clinical trials have become the "gold standard" for evaluating new treatments, only a small subset of the population considered for treatment participate in randomized clinical trials. To what extent is it reasonable to generalize beyond the boundaries of a specific clinical trial? This paper argues that several pieces of information are necessary to determine the extent of extrapolation or generalization warranted in a specific clinical trial. The necessary items of information are derived from basic science laboratory studies; animal studies; genetic studies (where applicable); observational, clinical, and epidemiological studies; and other randomized clinical trials in similar settings or with similar treatments. An example from the field of cholesterol reduction is presented.
尽管随机对照临床试验已成为评估新疗法的“金标准”，但考虑接受治疗的人群中只有一小部分参与随机临床试验。超出特定临床试验范围进行推广在多大程度上是合理的？本文认为，确定在特定临床试验中进行外推或推广的合理程度需要若干信息。这些必要的信息来源于基础科学实验室研究、动物研究、基因研究（如适用）、观察性、临床和流行病学研究，以及类似环境或采用类似治疗方法的其他随机临床试验。文中给出了胆固醇降低领域的一个例子。
