Evaluating Co-primary Endpoints Collectively in Clinical Trials
Evaluating Co-primary Endpoints Collectively in Clinical Trials
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DOI：
10.1002/bimj.200710497
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发表时间：
2009-02-01
影响因子：
1.7
通讯作者：
Li, Qian H.
中科院分区：
文献类型：
--
作者：
Li, Qian H.
通常通过共同主要终点来评估治疗，以便获得治疗效果的综合情况。共同主要终点可以是针对疾病不同方面的不同医学评估，因此，共同用于加强治疗效果的证据。如果一种治疗无效，那么在所有共同主要终点显示治疗有效的机会应该很小，这是常识。因此，可能没有必要要求所有共同主要终点在i侧0.025水平上具有统计显著性，以控制错误批准无效治疗的错误率。相反，允许p值在接近0.025的范围内发生一定的变化是合理的。本文发展了统计方法来推导决策规则，以集体评估共同主要终点。决策规则将错误接受无效治疗的错误率控制在0.025的水平上，并将错误率控制在略高的水平上，即对所有共同主要终点都有效的治疗（可能除了一个）。决策规则还控制各个端点的错误率。介绍了该方法在临床试验中的潜在应用。
Often a treatment is assessed by co-primary endpoints so that a comprehensive picture of the treatment effect can be obtained. Co-primary endpoints can be different medical assessments angled at different aspects of a disease, therefore, are used collectively to strengthen evidence for the treatment effect. It is common sense that if a treatment is ineffective, the chance to show that the treatment is effective in all co-primary endpoints should be small. Therefore, it may not be necessary to require all the co-primary endpoints to be statistically significant at the I-sided 0.025 level to control the error rate of wrongly approving an ineffective treatment. Rather it is reasonable to allow certain variation for the p-values within a range close to 0.025. In this paper, statistical methods are developed to derive decision rules to evaluate co-primary endpoints collectively. The decision rules control the error rate of wrongly accepting an ineffective treatment at the level of 0.025 for a study and the error rate at a slightly higher level for a treatment that works for all the co-primary endpoints except perhaps one. The decision rules also control the error rates for individual endpoints. Potential applications in clinical trials are presented.