Sauer Lukas D, Ritz Alexander, Kieser Meinhard
Institute of Medical Biometry, Heidelberg University, Heidelberg, Germany.
Institute of Mathematics, Clausthal University of Technology, Clausthal-Zellerfeld, Germany.
PLoS One. 2025 May 28;20(5):e0323097. doi: 10.1371/journal.pone.0323097. eCollection 2025.
Basket trial designs are a type of master protocol in which the same therapy is tested in several strata of the patient cohort. Many basket trial designs implement borrowing mechanisms. These allow sharing information between similar strata with the goal of increasing power in responsive strata while at the same time constraining type-I error inflation to a bearable threshold. These borrowing mechanisms can be tuned using numerical tuning parameters. The optimal choice of these tuning parameters is subject to research. In a comparison study using simulations and numerical calculations, we are planning to investigate the use of utility functions for quantifying the compromise between power and type-I error inflation and the use of numerical optimization algorithms for optimizing these functions. The present document is the protocol of this comparison study, defining each step of the study in accordance with the ADEMP scheme for pre-specification of simulation studies.
篮子试验设计是一种主方案，其中在患者队列的几个层次中对相同的治疗方法进行测试。许多篮子试验设计采用了信息借用机制。这些机制允许在相似层次之间共享信息，目的是在反应性层次中提高检验效能，同时将I型错误膨胀限制在可承受的阈值内。这些信息借用机制可以使用数值调整参数进行调整。这些调整参数的最佳选择有待研究。在一项使用模拟和数值计算的比较研究中，我们计划研究效用函数在量化检验效能和I型错误膨胀之间的折衷方面的应用，以及数值优化算法在优化这些函数方面的应用。本文件是这项比较研究的方案，根据模拟研究预规范的ADEMP方案定义了研究的每个步骤。
