Kopp-Schneider Annette, Wiesenfarth Manuel, Witt Ruth, Edelmann Dominic, Witt Olaf, Abel Ulrich
Division of Biostatistics, German Cancer Research Center (DKFZ), Heidelberg, Germany.
Clinical Trial Center, National Center for Tumor Diseases, Heidelberg, Germany.
Biom J. 2019 May;61(3):488-502. doi: 10.1002/bimj.201700209. Epub 2018 Sep 2.
A multistage single arm phase II trial with binary endpoint is considered. Bayesian posterior probabilities are used to monitor futility in interim analyses and efficacy in the final analysis. For a beta-binomial model, decision rules based on Bayesian posterior probabilities are converted to "traditional" decision rules in terms of number of responders among patients observed so far. Analytical derivations are given for the probability of stopping for futility and for the probability to declare efficacy. A workflow is presented on how to select the parameters specifying the Bayesian design, and the operating characteristics of the design are investigated. It is outlined how the presented approach can be transferred to statistical models other than the beta-binomial model.
考虑进行一项具有二元终点的多阶段单臂II期试验。在中期分析中使用贝叶斯后验概率来监测无效性，在最终分析中监测有效性。对于贝塔 - 二项式模型，基于贝叶斯后验概率的决策规则根据迄今为止观察到的患者中的反应者数量转换为“传统”决策规则。给出了因无效性而停止试验的概率以及宣布有效性的概率的分析推导。提出了一个关于如何选择指定贝叶斯设计的参数的工作流程，并研究了该设计的操作特征。概述了所提出的方法如何能够转移到除贝塔 - 二项式模型之外的统计模型。
