An overview of platform trials with a checklist for clinical readers

Abstract

Objectives: The objective of the study was to outline key considerations for general clinical readers when critically evaluating publications on platform trials and for researchers when designing these types of clinical trials.

Study design and setting: In this review, we describe key concepts of platform trials with case study discussion of two hallmark platform trials in STAMPEDE and I-SPY2. We provide reader's guide to platform trials with a critical appraisal checklist.

Results: Platform trials offer flexibilities of dropping ineffective arms early based on interim data and introducing new arms into the trial. For platform trials, it is important to consider how interventions are compared and evaluated throughout and how new interventions are introduced. For intervention comparisons, it is important to consider what the primary analysis is, what and how many interventions are active simultaneously, and allocation between different arms. Interim evaluation considerations should include the number and timing of interim evaluations and outcomes and statistical rules used to drop interventions. New interventions are usually introduced based on scientific merits, so consideration of these merits is important, together with the timing and mechanisms in which new interventions are added.

Conclusion: More efforts are needed to improve the scientific literacy of platform trials. Our review provides an overview of the important concepts of platform trials.

Keywords: Clinical trial design; Master protocols; Platform trials; Precision medicine; Primer.