Multiplicity considerations in the design and analysis of clinical trials

Multiplicity considerations in the design and analysis of clinical trials
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DOI:
10.2307/2983471
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发表时间:
1996-01-01
影响因子:
2
通讯作者:
Farewell, VT
Farewell, VT
中科院分区:
数学4区
文献类型:
--
作者:
Cook, RJ;Farewell, VT

文献摘要

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在医学研究中有效利用现有资源的需要导致了基于多种反应、多种治疗手段和重复重要试验的临床试验设计的吸引力增加。近年来,与这些类型的多重比较有关的方法学工作相当多,共同的目标通常是控制实验I类错误率。在此,我们在各种情况下重新考虑这些目标的适当性,并建议经常不必要地采用多重比较程序。特别是,我们认为,如果在设计中指定了一些重要的明确定义的临床问题,则在某些情况下,可以在不控制实验I类错误率的情况下执行多个有意义的测试。这一点合理的主要限制是测试结果被边际解释。
The need for efficient use of available resources in medical research has led to the increased appeal of clinical trial designs based on multiple responses, multiple treatment arms and repeated tests of significance. In recent years there has been considerable methodological work pertaining to these types of multiple comparison, with the common objective typically being the control of the experimental type I error rate. Here we reconsider the appropriateness of these objectives iii a variety of contexts and suggest that multiple-comparison procedures are frequently adopted unnecessarily. In particular we argue that, provided that a select number of important well-defined clinical questions are specified at the design, there are situations in which multiple tests of significance can be performed without control of the experimental type I error rate. The primary restriction for this to be reasonable is that test results are interpreted marginally.