Adaptive platform trials: definition, design, conduct and reporting considerations

Area of Science:

• Clinical Trials Methodology
• Biostatistics
• Drug Development

Background:

• Traditional randomized controlled trials (RCTs) face challenges in evaluating multiple interventions, distinct patient subgroups, or minimizing trial downtime.
• Growing interest from researchers, clinicians, policymakers, and patients necessitates more efficient and flexible clinical trial designs.

Purpose of the Study:

• To review common features and issues associated with adaptive platform trials (APTs).
• To provide recommendations for best practices in the design, conduct, oversight, and reporting of APTs.
• To facilitate the wider adoption and effective implementation of APTs in therapeutic intervention research.

Main Methods:

• Review of existing literature and methodologies concerning adaptive platform trials.
• Analysis of common features, challenges, and innovative aspects of APTs.
• Development of recommendations based on identified best practices.

Main Results:

• APTs allow for the continuous evaluation of multiple interventions within a single, perpetual trial framework.
• Interventions can enter and exit the platform based on predefined decision algorithms, enhancing efficiency.
• APTs offer a flexible approach to address complex research questions that are difficult for traditional RCTs.

Conclusions:

• Adaptive platform trials represent a significant innovation with the potential to reshape clinical trial landscapes.
• Adherence to best practices in design, conduct, oversight, and reporting is crucial for the successful implementation of APTs.
• This review aims to support researchers and stakeholders in leveraging APTs for more effective therapeutic intervention studies.