Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2022 Dec;19(6):690-696.
doi: 10.1177/17407745221110199. Epub 2022 Sep 9.

Mobilizing the clinical trial ecosystem to drive adoption of master protocols

Abby Bronson  1   2 , Marianne K Chase  3 , Kimberly Fisher  4 , Daniel Millar  4 , Jane Perlmutter  5 , Nicholas Richardson  6
Affiliations

Mobilizing the clinical trial ecosystem to drive adoption of master protocols

Abby Bronson et al. Clin Trials. 2022 Dec.

Abstract

Master protocol studies typically use an overarching protocol to answer several questions by guiding a variety of sub-studies. These sub-studies can incorporate multiple diseases, therapies, or both. Although this innovative approach offers many benefits, including the ability to deliver clinical research that is more patient-centric and efficient, several common barriers curtail widespread adoption. The Clinical Trials Transformation Initiative (CTTI) convened industry representatives, regulatory agencies, patient groups, and academic institutions to identify emerging best practices and develop resources designed to help sponsors and other stakeholders overcome these challenges. We first identify some broad changes needed in the clinical trials ecosystem to facilitate mainstream adoption of master protocol studies, and we subsequently summarize CTTI's resources designed to support this effort.

Keywords: Master protocol studies; basket trials; clinical research funding; clinical trial design; collaboration; multiple targeted therapies; patient-centric clinical trial design; platform trials; umbrella trials.

PubMed Disclaimer

Conflict of interest statement

The author(s) declared the following potential conflicts of interest with respect to the research, authorship, and/or publication of this article: Daniel Millar and Kimberly Fisher report employment and shareholder interest in Johnson & Johnson. The other authors have no conflicts of interest to disclose.

Figures

Figure 1.
Figure 1.
Pre-planning, planning, and execution stages of master protocol studies.
See this image and copyright information in PMC

Similar articles

See all similar articles

Cited by

References

    1. Woodcock J, LaVange LM. Master protocols to study multiple therapies, multiple diseases, or both. N Engl J Med 2017; s377: 62–70. - PubMed
    1. U.S Food and Drug Administration. FDA in brief: FDA provides guidance on master protocols for evaluating prevention, treatment options for COVID-19, https://www.fda.gov/news-events/press-announcements/fda-brief-fda-provid... (2021, accessed 16 March 2022).
    1. Alexander BM, Ba S, Berger MS, et al.. Adaptive global innovative learning environment for glioblastoma: GBM AGILE. Clin Cancer Res 2018; 24(4): 737–743. - PubMed
    1. I-SPY. The I-SPY trials, https://www.ispytrials.org/ (accessed 16 March 2022).
    1. Paganoni S, Berry JD, Quintana M, et al.. Adaptive platform trials to transform amyotrophic lateral sclerosis therapy development. Ann Neurol 2022; 91(2): 165–175. - PubMed

Publication types

LinkOut - more resources