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Characteristics, design, and statistical methods in platform trials: a systematic review
Clément R Massonnaud1, Christof Manuel Schönenberger2, Malena Chiaborelli2
1Université Paris Cité, Inserm, IAME, Paris F-75018, France; AP-HP, Hôpital Bichat, Département d'Épidémiologie, Biostatistique et Recherche Clinique, Paris F-75018, France.
Journal of Clinical Epidemiology
|May 11, 2025
Summary
Platform trials (PTs) are increasingly popular in clinical research, featuring diverse methodologies. Enhancing transparency in complex Bayesian PTs is crucial for reliability and acceptance.
Area of Science:
- Clinical Research
- Biostatistics
- Epidemiology
Background:
- Platform trials (PTs) offer innovative and flexible methodologies gaining traction in clinical research.
- The study aimed to characterize the evolving landscape of PTs.
Purpose of the Study:
- To determine the characteristics, methodological approaches, and statistical practices employed in platform trials.
- To analyze trends in PT initiation and design.
Main Methods:
- A systematic search of trial registries and bibliographic databases identified 189 eligible platform trials.
- Data extraction focused on trial status, design features, statistical methods, and reporting quality.
- Eligible PTs were randomized controlled trials with multiple interventions in a single population, allowing arm flexibility.
Main Results:
- Infectious diseases and oncology were the primary focus areas for PTs, with a significant number related to COVID-19.
- Bayesian designs were prevalent, with 23% of trials lacking a predetermined sample size, often designated as perpetual trials.
- Frequentist PTs more commonly predetermined interim analyses, while Bayesian PTs extensively utilized simulations, though code sharing was minimal.
Conclusions:
- Platform trials remain a popular and diversifying research modality.
- Improved transparency and reporting, particularly for complex Bayesian designs, are vital for ensuring the reliability and acceptance of platform trials.
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