Why and how should we simulate platform trials? Learnings from EU-PEARL

Area of Science:

• Clinical Trials Methodology
• Biostatistics
• Pharmaceutical Research

Background:

• Platform trials offer a flexible approach to evaluating multiple treatments within a master protocol.
• Increasing interest in platform trials highlights the need for adaptable and efficient design methodologies.
• Existing simulation software lacks the flexibility to accommodate unique platform trial features like staggered treatment entry.

Purpose of the Study:

• To address the complexities in designing platform trials.
• To develop a simulation-guided framework for platform trial design.
• To create open-source software for simulating platform trials.

Main Methods:

• Proposed an iterative, simulation-guided framework for platform trial design.
• Developed a suite of open-source software using the R programming language for simulation.
• Software features include simulating staggered treatment entry, control data sharing options, and platform stopping rules.

Main Results:

• The developed open-source software effectively simulates key platform trial design features.
• Independent validation confirmed the reliability and consistency of the simulation software.
• The software addresses functionality limitations and coding unavailability in existing tools.

Conclusions:

• A comprehensive framework and open-source software are provided for platform trial design and simulation.
• The software tools offer the necessary flexibility to model the intricate nature of platform trials.
• This facilitates more efficient and robust platform trial development.