The Challenges of Running Platform Trials

29 May 2019

Our linked trio of papers on implementing platform protocols are now available in Trials.

The potential efficiencies of asking multiple questions in a single protocol are increasingly understood. This could be achieved using any or all of the following:

  • a multi-arm multi-stage (MAMS) design to ask multiple questions from the start
  • a platform (or “living”) protocol to later add in new questions in a structured way
  • a biomarker-stratified design to ask questions for multiple subsets of patients with a shared screening process.

Our new papers focus on the operational considerations in undertaking such designs, drawing particularly on MRC CTU at UCL’s extensive experience with the STAMPEDE and FOCUS4 trials.

The first paper by Schiavone et al focuses on issues that trial managers or trial coordinators might have in running the operational side of these trials.

The second paper by Hague et al focuses on issues that data managers, data scientists and programmers might have in running the operational side of these trials.

The third paper by Morrell et al draws out the experiences of central trials unit staff in running these trials.

Each paper clearly sets out the strengths of these designs and addresses frankly the challenges that any group choosing to take on these designs may face. so that they can prepare well.

These papers complement our recent papers by Parmar et al and Blenkinsop et al on statistical guidelines on how to implement efficacy stopping guidelines in MAMS trials, as well as further guidance on statistical considerations in adding arms will follow soon from Oskooei et al.

To help others implement these designs, we are currently developing a one-day course, which will be launched later this year.

Links to the three papers in Trials Journal

Further information