EU-PEARL Simulation Framework

EU-PEARL Simulation Framework supports designing and simulating platform clinical trials through an iterative, simulation-guided approach that enables optimization from basic to complex trial designs.

Key Features:

• Staggered treatment entry simulation: Simulates treatments entering and leaving the trial over time throughout the trial duration.
• Control data sharing options: Supports choosing different options for sharing control data across treatment arms.
• Platform stopping rules: Allows specifying different stopping rules at the platform level.
• Platform-level operating characteristics: Enables specification and evaluation of operating characteristics at the platform level.
• Open-source R implementation: Provides open-source software based on the R programming language for access and modification of code.
• Independent verification: Separate use of software packages by independent teams implementing the same platform design obtained identical results.

Scientific Applications:

• Platform clinical trial design: Supports design of efficient platform trials for diseases requiring evaluation of multiple investigational treatments.
• Paediatric neurofibromatosis-1 trials: Addresses specific application to designing platform trials for paediatric neurofibromatosis-1.
• Schwannomatosis trials: Addresses specific application to designing platform trials for schwannomatosis.
• Iterative design optimization: Enables iterative, simulation-guided optimization from a basic "vanilla" design to a more complex "sprinkles" design.

Methodology:

The framework employs an iterative "vanilla and sprinkles" approach progressing from basic to more complex platform trial designs, with simulation studies guiding the design process. The software is implemented in the R programming language.