Analytics and Data Sciences, Biogen Inc, 300 Binney St, Cambridge, MA, 02142, USA.
Biostatistics and Research Decision Sciences, Merck & Co., Inc, Kenilworth, NJ, 07033, USA.
Ther Innov Regul Sci. 2021 Nov;55(6):1145-1154. doi: 10.1007/s43441-021-00315-7. Epub 2021 Jun 23.
Master protocol, categorized as basket trial, umbrella trial or platform trial, is an innovative clinical trial framework that aims to expedite clinical drug development, enhance trial efficiency, and eventually bring medicines to patients faster. Despite a clear uptake on the advantages in the concepts and designs, master protocols are still yet to be widely used. Part of that may be due to the fact that the master protocol framework comes with the need for new statistical designs and considerations for analyses and operational challenges. In this article, we provide an overview of the master protocol framework, unify the definitions with some examples, review the statistical methods for the designs and analyses, and focus our discussions on some practical considerations and recommendations of master protocols to help practitioners better design and implement such studies.
主方案（Master protocol），也被归类为篮子试验、伞式试验或平台试验，是一种创新的临床试验框架，旨在加速临床药物开发、提高试验效率，并最终更快地为患者带来药物。尽管在概念和设计方面优势明显，但主方案仍未得到广泛应用。部分原因可能是主方案框架需要新的统计设计和分析考虑，以及操作挑战。本文提供了主方案框架的概述，用一些例子统一了定义，回顾了设计和分析的统计方法，并重点讨论了主方案的一些实际考虑因素和建议，以帮助实践者更好地设计和实施此类研究。
