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Review
. 2021 Nov;55(6):1145-1154.
doi: 10.1007/s43441-021-00315-7. Epub 2021 Jun 23.

Practical Considerations and Recommendations for Master Protocol Framework: Basket, Umbrella and Platform Trials

Chengxing Cindy Lu #  1 , Xiaoyun Nicole Li #  2 , Kristine Broglio  3 , Paul Bycott  4 , Qi Jiang  5 , Xiaoming Li  6 , Anna McGlothlin  7 , Hong Tian  8 , Jingjing Ye  9
Affiliations
Review

Practical Considerations and Recommendations for Master Protocol Framework: Basket, Umbrella and Platform Trials

Chengxing Cindy Lu et al. Ther Innov Regul Sci. 2021 Nov.

Abstract

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.

Keywords: Adaptive design; Basket trial; Complex clinical trial; Master protocol; Platform trial; Umbrella trial.

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Conflict of interest statement

The authors acknowledge that they have no conflicts of interest.

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