论文标题
block响应自适应随机化的familywise错误率控制
Familywise error rate control for block response-adaptive randomization
论文作者
论文摘要
反应自适应随机化允许将患者分配给临床试验中的治疗方法，以根据先前观察到的响应数据进行更改，以实现不同的实验目标。在实践中使用此类设计的一个问题，尤其是从监管角度来看，是控制I类错误率。为了解决这个问题，Robertson and Wason（Biometrics，2019）提出了方法，该方法可以保证大量反应自适应设计的家庭错误率控制。在本文中，我们提出了对他们的提议的改进，在概念上，在构图的试验的特定情况下，对控制臂进行了固定的分配。我们显示了修改后的方法保证了数据块永远不会有负权重，并且还可以在实践中提供实质性的功率优势。
Response-adaptive randomization allows the probabilities of allocating patients to treatments in a clinical trial to change based on the previously observed response data, in order to achieve different experimental goals. One concern over the use of such designs in practice, particularly from a regulatory viewpoint, is controlling the type I error rate. To address this, Robertson and Wason (Biometrics, 2019) proposed methodology that guarantees familywise error rate control for a large class of response-adaptive designs. In this paper, we propose an improvement of their proposal that is conceptually simpler, in the specific context of block-randomised trials with a fixed allocation to the control arm. We show the modified method guarantees that there will never be negative weights for blocks of data, and can also provide a substantial power advantage in practice.
