Vertex Pharmaceuticals Incorporated, Boston, Massachusetts, USA.
Clin Transl Sci. 2021 Jul;14(4):1250-1258. doi: 10.1111/cts.12998. Epub 2021 May 1.
Dose-ranging studies are a crucial part of the phase II drug development process. They complement the understanding gained from exposure-response analyses. However, the statistical design issues related to dose-ranging studies are not always keenly understood and a poorly designed study can be costly for later development. In this tutorial, we review five key statistical principles in designing such a study. We also describe some popular statistical approaches, including pairwise comparison, modeling, and Multiple Comparison Procedure modeling in the context of principles.
剂量范围研究是药物开发二期临床试验的重要组成部分。它们补充了通过暴露反应分析获得的认识。但是，与剂量范围研究相关的统计设计问题并不总是被深刻理解，一个设计不佳的研究可能会给后期开发带来高昂的代价。在本教程中，我们回顾了设计此类研究的五个关键统计原则。我们还描述了一些流行的统计方法，包括在原理背景下的两两比较、建模和多重比较程序建模。
