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Users' Guides to the Medical Literature
January 4, 2022

How to Use and Interpret the Results of a Platform Trial: Users’ Guide to the Medical Literature

Author Affiliations
  • 1Division of Experimental Medicine, Department of Medicine, University of British Columbia, Vancouver, Canada
  • 2Department of Health Research Methods, Evidence, and Impact, McMaster University, Hamilton, Ontario, Canada
  • 3Berry Consultants LLC, Austin, Texas
  • 4Department of Pediatrics, Faculty of Medicine, University of British Columbia, Vancouver, Canada
  • 5Cytel Inc, Vancouver, British Columbia, Canada
JAMA. 2022;327(1):67-74. doi:10.1001/jama.2021.22507
Abstract

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.

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