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How to Use and Interpret the Results of a Platform Trial


For our first Winter Weekend Read, Cytel presents How to Use and Interpret the Results of a Platform Trial, a JAMA Users Guide to the Medical Literature by Cytel Principal Scientist Dr. Edward Mills and colleagues. A complimentary version of this publication can be found on the JAMA website (click below).

Many in the clinical trials industry are familiar with classic platform trials like the UK Cancer Research Unit’s STAMPEDE Trial, or ISPY2 that have revolutionized breast cancer research. What might go unrecognized is that the number and variety of platform trials are rapidly expanding. According to one Cytel publication, there are over five times as many platform trials currently being implemented than there were in 2013 [1].

Their growing popularity led Cytel to offer the webinar series, the Expanding Application of Master Protocols, in autumn 2021 where Cytel scientists reviewed the contexts and paradigms of newer applications of platform trials.

Dr. Edward Mills, Principal Scientist at Cytel, who earlier presented on master protocols in a global health context, has recently released a JAMA Users Guide to the Medical Literature on How to Use and Interpret the Results of a Platform Trial.

Abstract to the Users Guide

Platform trials are a type of randomized clinical trial that allow simultaneous comparison of multiple intervention groups against a single control group that serves as a common control based on a prespecified interim analysis plan. The platform trial design enables introduction of new interventions after the trial is initiated to evaluate multiple interventions in an ongoing manner using a single overarching protocol called a master (or core) protocol. When multiple treatment candidates are available, rapid scientific therapeutic discoveries may be made. Platform trials have important potential advantages in creating an efficient trial infrastructure that can help address critical clinical questions as the evidence evolves. Platform trials have recently been used in investigations of evolving therapies for patients with COVID-19. The purpose of this Users’ Guide to the Medical Literature is to describe fundamental concepts of platform trials and master protocols and review issues in the conduct and interpretation of these studies. This Users’ Guide is intended to help clinicians and readers understand articles reporting on interventions evaluated using platform trial designs.

Click to Read


[1] Park, J.J., et. al (2019) Systematic review of basket trials, umbrella trials and platform trials: a landscape analysis of master protocols.


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