AccScience Publishing / TD / Volume 2 / Issue 2 / DOI: 10.36922/td.342
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REVIEW

Practice and consideration of master protocol in clinical trials

Jiali Song1† Zhiwei Rong1† Xinwen Zhong1 Yuhong Lu1 Jike Huang1 Yipei Yu1 Zhilin Liu1 Xuyuan Quan1 Nana Chen1 Kang Li2 Fengyu Sun3* Yan Hou1,4,5*
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1 Department of Biostatistics, School of Public Health, Peking University, Beijing 100191, China
2 Department of Epidemiology and Biostatistics, School of Public Health, Harbin Medical University, Harbin, 150086, China
3 National Medical Products Administration, Beijing 100022, China
4 Peking University Cancer Hospital and Institute, Beijing 100142, China
5 Clinical Research Center, Peking University, Beijing 100191, China
Tumor Discovery 2023, 2(2), 342 https://doi.org/10.36922/td.342
Submitted: 30 January 2023 | Accepted: 18 April 2023 | Published: 28 April 2023
© 2023 by the Author(s). This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution 4.0 International License ( https://creativecommons.org/licenses/by/4.0/ )
Abstract

There is considerable interest in expediting late-stage therapy development by efficiently conducting trial designs that encompass multiple therapies or multiple subpopulations simultaneously within a unified protocol. Such trial designs are referred to as master protocols, with specific designs characterized by the terms umbrella, basket, or platform. These designs, in contrast to the traditional trial designs, are full of complexity. What factors should be considered in designing a trial ensuring the safety of human subjects and demonstrating the efficacy of new therapy? This paper overviews the master protocol framework, comprehensively unifies the definitions, and illustrates essential design elements of representative example trials conducted in drugs and medical devices. Besides, to understand the master protocols deeply, it is also a need to summarize the commonly-used types of master protocols in various disease and treatment fields, along with the reasons for these phenomena by analyzing the characteristics of the diseases, the mechanism of therapeutic products, and the principles of various types of master protocols. Finally, we also propose practical considerations, including the design, ethical, statistics, and funding considerations that arise from implementing complex master protocols. This information serves to guide practitioners in designing more effective trials and identifying potentially valuable therapies.

Keywords
Design
Master protocol
Principle
Consideration
Clinical practice
Funding
National Natural Science Foundation
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Conflict of interest
The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper.
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Tumor Discovery, Electronic ISSN: 2810-9775 Published by AccScience Publishing