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The Evolution of Master Protocol Clinical Trial Designs: A Systematic Literature Review
Elias Laurin Meyer1, Peter Mesenbrink2, Cornelia Dunger-Baldauf3
1Center for Medical Statistics, Informatics, and Intelligent Systems, Medical University of Vienna, Vienna, Austria.
Clinical Therapeutics
|July 6, 2020
Summary
Master protocols offer flexible clinical trial designs, increasing efficiency in drug development. These innovative approaches, particularly platform trials, show great promise despite ongoing challenges.
Area of Science:
- Clinical Trials
- Drug Development
- Biostatistics
Background:
- Traditional clinical trial designs are evolving.
- Master protocols enable flexible investigation of multiple substudies within a single trial structure.
- These designs offer advantages over traditional adaptive and group sequential trials.
Purpose of the Study:
- To identify and characterize existing master protocol studies.
- To summarize relevant literature on the design and methods of master protocol trials.
- To review platform, basket, and umbrella trial designs.
Main Methods:
- A comprehensive systematic literature search was conducted.
- Articles were included regardless of statistical complexity.
- Literature was classified into reviews of planned/conducted trials, trial designs, or statistical methods.
Main Results:
- Most master protocol trials are single-arm, Phase II oncology trials using binary endpoints and frequentist rules.
- There has been an exponential increase in publications related to master protocols.
- Master protocols offer significant advantages in efficiency and flexibility for clinical drug development.
Conclusions:
- Master protocol trials, especially platform trials, have the potential to revolutionize drug development.
- Overcoming methodological and operational challenges is key to realizing the full potential of master protocols.
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