Division of Epidemiology, Biostatistics, and Preventive Medicine University of New Mexico, Albuquerque, New Mexico, USA.
Department of Biostatistics, St. Jude Children's Research Hospital, Memphis, Tennessee, USA.
Stat Med. 2023 May 10;42(10):1480-1491. doi: 10.1002/sim.9682. Epub 2023 Feb 20.
A multi-arm trial allows simultaneous comparison of multiple experimental treatments with a common control and provides a substantial efficiency advantage compared to the traditional randomized controlled trial. Many novel multi-arm multi-stage (MAMS) clinical trial designs have been proposed. However, a major hurdle to adopting the group sequential MAMS routinely is the computational effort of obtaining total sample size and sequential stopping boundaries. In this paper, we develop a group sequential MAMS trial design based on the sequential conditional probability ratio test. The proposed method provides analytical solutions for futility and efficacy boundaries to an arbitrary number of stages and arms. Thus, it avoids complicated computational effort for the methods proposed by Magirr et al. Simulation results showed that the proposed method has several advantages compared to the methods implemented in R package MAMS by Magirr et al.
多臂试验允许同时将多种实验治疗方法与一个共同的对照进行比较，与传统的随机对照试验相比具有显著的效率优势。已经提出了许多新的多臂多阶段（MAMS）临床试验设计。然而，采用常规分组序贯 MAMS 的一个主要障碍是获得总样本量和序贯停止边界的计算工作量。在本文中，我们基于序贯条件概率比检验开发了一种分组序贯 MAMS 试验设计。该方法为任意数量的阶段和臂提供了无效性和疗效边界的解析解。因此，它避免了 Magirr 等人提出的方法的复杂计算工作量。模拟结果表明，与 Magirr 等人在 R 包 MAMS 中实现的方法相比，该方法具有几个优势。
