Master Protocol Designs

Overview

Traditionally we have designed separate, standalone trials to evaluate an intervention, even if there may be commonalities to ongoing and related studies. Master protocols serve as a flexible solution to this sometimes redundant approach to trials. In this module we discuss basket trials that allow multiple indications for a common intervention, umbrella trials that attempt to target eligible disease states for personalized therapy, and platform trials that allow for the evaluation of multiple therapies and populations.

Slide Deck

 

You can also download the original PowerPoint file.

Code Examples in R

While master protocols themselves can be analyzed with general software, some special packages that incorporate other elements include:

  • basket: implements asymmetric multi-source exchangeability models (MEMs) for basket trial designs where we wish to evaluate if pooling subgroups is possible based on exchangeability, restricted to binary outcomes
  • NCC: provides functions to implement and evaluate platform trials with non-concurrent controls
  • PlatformDesign: design parameters for two-period multiarm platform designs

References

Below are some references to highlight based on the slides and code: