20 June 2024: Thomas Jaki (MRC-BSU / Regensburg)

Title: Mastering Master protocols: Some solutions and open questions
Abstract: Master protocols are a new class of clinical trial designs that allow treatment arms to enter and leave the trial and/or (sub)-populations to be added over time. They include platform, Basket and Umbrella trials and have proven to be particularly popular during the COVID-19 pandemic where no fewer than 58 trials have been registered as a platform trial. A common feature of these designs is the desire to answer several research questions within a single protocol. One of the questions arising from this feature that has generated a lot of discussion in the literature is the need for (or lack of need to) control error rates as well as the most appropriate type of error control. Another core feature of Master protocols is the desire to maximise the utility of information in the study by using the same information to answer multiple research questions (e.g. shared control group in platform trials or information borrowing in Basket trials).
In this talk I will begin by reflecting on the discussion around error rates and introduce different possible testing strategies that could be considered for Master protocols and platform trials in particular. I will then introduce possible strategies to borrow information across different research questions and discuss implications of such strategies on the sample size requirements of the study. Throughout this presentation I will highlight areas where further research is necessary to enable Master protocols to unleash their full potential.