Department of Biostatistics and Data Sciences, Boehringer-Ingelheim Pharmaceutical Inc., Ridgefield, Connecticut, USA.
J Biopharm Stat. 2020 Nov 1;30(6):1077-1090. doi: 10.1080/10543406.2020.1821703. Epub 2020 Sep 29.
This paper provides in-depth discussion about different types of error generated in platform trials with a common control arm, and how they compare to the ones arisen from standard independent trials. We provide our views on some of the popular "myths" associated with such design, under the frequentist framework. It is found that platform trial generally performs quite well in terms of type I error rate, false discovery rate, and power. In most cases, these operating characteristics of a platform trial are comparable to or even better than running individual trials.
本文深入探讨了具有共同对照臂的平台试验中产生的不同类型的误差，以及它们与标准独立试验中产生的误差有何不同。我们在经典框架下对与这种设计相关的一些流行“误区”提出了看法。研究发现，平台试验在Ⅰ类错误率、假阳性率和功效方面表现相当出色。在大多数情况下，平台试验的这些运行特性与单独进行试验相当，甚至更好。
