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Tumor Discovery

REVIEW ARTICLE
Practice and consideration of master protocol in

clinical trials

1
1†
Jiali Song , Zhiwei Rong , Xinwen Zhong , Yuhong Lu , Jike Huang , Yipei Yu ,
1†
1
1
1
Zhilin Liu , Xuyuan Quan , Nana Chen , Kang Li , Fengyu Sun *, and Yan Hou 1,4,5 *
1
1
2
3
1
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
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
† These authors contributed equally therapy? This paper overviews the master protocol framework, comprehensively
to this work. unifies the definitions, and illustrates essential design elements of representative
*Corresponding authors: example trials conducted in drugs and medical devices. Besides, to understand the
Fengyu Sun master protocols deeply, it is also a need to summarize the commonly-used types of
(sun.129.com@163.com)
Yan Hou master protocols in various disease and treatment fields, along with the reasons for
(houyan@bjmu.edu.cn) these phenomena by analyzing the characteristics of the diseases, the mechanism of
Citation: Song J, Rong Z, therapeutic products, and the principles of various types of master protocols. Finally,
Zhong X, et al., 2023, Practice and we also propose practical considerations, including the design, ethical, statistics, and
consideration of master protocol in funding considerations that arise from implementing complex master protocols.
clinical trials. Tumor Discov,
2(2): 342. This information serves to guide practitioners in designing more effective trials and
https://doi.org/10.36922/td.342 identifying potentially valuable therapies.
Received: January 30, 2023
Accepted: April 18, 2023 Keywords: Design; Master protocol; Principle; Consideration; Clinical practice
Published Online: April 28, 2023
Copyright: © 2023 Author(s).
This is an Open Access article
distributed under the terms of the
Creative Commons Attribution 1. Introduction
License, permitting distribution,
and reproduction in any medium, Conventionally, the development of new therapeutic products has relied on a series
provided the original work is of clinical trials, each testing one or two therapies for a single disease. This process is
properly cited. both time-consuming and expensive. Therefore, there is growing interest in accelerating
Publisher’s Note: AccScience therapy development through performing trials that simultaneously test multiple
Publishing remains neutral with therapies or multiple subpopulations under a single protocol. Such trials, known
regard to jurisdictional claims in
published maps and institutional as master protocols, have gained popularity and have been extensively researched in
affiliations. the fields of drugs and medical devices, especially in oncology. The Food and Drug

Volume 2 Issue 2 (2023) 1 https://doi.org/10.36922/td.342