Characteristics, design, and statistical methods in platform trials: a systematic review.
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BACKGROUND AND OBJECTIVE: Platform trials (PTs) are gaining popularity in clinical research due to their innovative and flexible methodologies. The objective was to determine the characteristics, methodological, and statistical practices in PTs. METHODS: We identified PTs from trial registries and bibliographic databases up to August 2024. Eligible PTs were randomized controlled trials studying multiple interventions within a single population, with flexibility to add or drop arms. Data were extracted on trial status, design, statistical methods, and reporting practices. RESULTS: We identified 189 PTs. Most focused on infectious diseases (77, including 57 for COVID-19) and oncology (68). PT initiation peaked during the COVID-19 pandemic but has since stabilized at 84 active PTs, with 25 in planning. A complete master protocol was available for 47% (89/189) of PTs. Bayesian designs featured in 58/189 PTs vs. 56/189 frequentist trials, 20/189 trials utilizing both (unclear in 55/189 PTs). Overall, 25/111 trials (23%) were designed without a predetermined target sample size, all of which were Bayesian. Among these, 16 were explicitly reported as "perpetual" trials. The number of interim analyses was predetermined in 18% (10/57) of Bayesian trials vs. 58% (28/48) of frequentist trials. Simulations to evaluate operating characteristics were used in 93% (39/42) of Bayesian trials. Simulation reports were available in 67% (26/39) of cases, and the procedures were detailed for 62% (24/39) of trials. Only two trials shared the simulation code. CONCLUSION: PTs remain popular and increasingly diverse. Efforts to enhance transparency and reporting, especially in complex Bayesian PTs, are essential to ensure reliability and broader acceptance.
