Seamless Clinical Trials with Doubly Adaptive Biased Coin Designs
Seamless Clinical Trials with Doubly Adaptive Biased Coin Designs
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DOI：
10.51387/23-nejsds25
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发表时间：
2023
期刊：
影响因子：
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通讯作者：
Hongjian Zhu;Jun Yu;D. Lai;Li Wang
中科院分区：
文献类型：
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作者：
Hongjian Zhu;Jun Yu;D. Lai;Li Wang
除了科学问题，临床试验人员经常探索或要求其他设计特征，如在控制I型错误率的同时增加功率，最大限度地减少不必要的劣质治疗，以及在一项临床试验中比较多种治疗方法。我们建议采用自适应无缝设计(ASD)和响应自适应随机化(RAR)来满足不同临床试验的设计目标。然而，ASD和RAR的组合在控制类型I误码率方面提出了挑战。在本文中，我们研究了如何利用这两种自适应方法的优点来控制I类误码率。我们为这一过程提供了理论基础。数值研究表明，我们的方法可以在控制第一类错误率的同时达到有效和道德的目标。
In addition to scientific questions, clinical trialists often explore or require other design features, such as increasing the power while controlling the type I error rate, minimizing unnecessary exposure to inferior treatments, and comparing multiple treatments in one clinical trial. We propose implementing adaptive seamless design (ASD) with response adaptive randomization (RAR) to satisfy various clinical trials’ design objectives. However, the combination of ASD and RAR poses a challenge in controlling the type I error rate. In this paper, we investigated how to utilize the advantages of the two adaptive methods and control the type I error rate. We offered the theoretical foundation for this procedure. Numerical studies demonstrated that our methods could achieve efficient and ethical objectives while controlling the type I error rate.