Rabeneck L, Viscoli C M, Horwitz R I
Department of Internal Medicine, Yale University School of Medicine, New Haven, CT 06510.
Arch Intern Med. 1992 Mar;152(3):507-12.
To achieve the goal of validity, the randomized clinical trial has emerged as the scientific "gold standard" for evaluating therapies in clinical medicine. Regardless of how well randomized clinical trials are designed, however, problems often occur during the conduct of the trials that give rise to methodologic challenges in the analysis of results. Primarily two types of problems, changes in intended treatment and the failure to ascertain the study outcomes, occur during the conduct of randomized clinical trials. We studied the current analytic strategies that are used to deal with these problems and how the use of these analytic strategies can change the focus of the research so that the trial no longer answers the relevant question. To ensure that the right question is answered, new methods of design and analysis are required that balance the goals of validity and clinical pertinence.
为实现有效性目标，随机临床试验已成为临床医学中评估治疗方法的科学“金标准”。然而，无论随机临床试验设计得多么完善，在试验实施过程中常常会出现问题，这些问题给结果分析带来了方法学上的挑战。在随机临床试验实施过程中主要出现两种类型的问题，即预期治疗的改变和研究结果未得到确定。我们研究了当前用于处理这些问题的分析策略，以及这些分析策略的使用如何能够改变研究重点，从而使试验不再回答相关问题。为确保回答正确的问题，需要新的设计和分析方法，以平衡有效性和临床相关性的目标。
