Incorporating data from multiple endpoints in the analysis of clinical trials: example from RSV vaccines.
Incorporating data from multiple endpoints in the analysis of clinical trials: example from RSV vaccines.
复制标题
将来自多个终点的数据纳入临床试验分析：例如 RSV 疫苗。
DOI：
10.1101/2023.02.07.23285596
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发表时间：
2023
期刊：
影响因子：
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通讯作者：
Weinberger,DanielM
中科院分区：
文献类型：
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作者：
Prunas,Ottavia;Willemsen,JoukjeE;Bont,Louis;Pitzer,VirginiaE;Warren,JoshuaL;Weinberger,DanielM
背景：为了满足监管部门的批准，干预措施必须在随机临床试验中证明对主要结果的有效性。然而，当有多个临床相关的结果时，选择单一的主要结果是具有挑战性的。合并来自多个结果的数据可能会增加临床试验的统计能力。受呼吸道合胞病毒(RSV)干预试验的启发，我们研究了分析多个终点的数据的方法。方法：我们开发了一种新的置换检验，代表个体结局检验统计量(WAVP)的加权平均值，以在多终点分析中评估干预效果。我们将这种方法的功率和I类错误率与Bonferroni校正(BonfT)和MINP置换测试进行了比较。我们使用来自三个不同干预效果、相关性和结果发生率的假想试验的模拟数据以及来自真实世界的RSV临床试验的数据来评估不同的方法。
Background:To meet regulatory approval, interventions must demonstrate efficacy against a primary outcome in randomized clinical trials. However, when there are multiple clinically relevant outcomes, selecting a single primary outcome is challenging. Incorporating data from multiple outcomes may increase statistical power in clinical trials. We examined methods for analyzing data on multiple endpoints, inspired by real-world trials of interventions against respiratory syncytial virus (RSV).Method:We developed a novel permutation test representing a weighted average of individual outcome test statistics (wavP) to evaluate intervention efficacy in a multiple endpoint analysis. We compared the power and type I error rate of this approach to the Bonferroni correction (bonfT) and the minP permutation test. We evaluated the different approaches using simulated data from three hypothetical trials varying the intervention efficacy, correlation, and incidence of the outcomes, and data from a real-world RSV clinical trial.Results: