Developing an adaptive platform trial for evaluation of medical treatments for Crohn’s disease

Abstract

There are a growing number of medical treatments demanding evaluation by patients and clinicians in Crohn’s disease. However conventional clinical trial designs may take several decades to provide the answers being sought particularly regarding sequencing of treatments. Adaptive platform trials using a master protocol approach offer an opportunity for more efficient trials i.e. provide answers from fewer patients and in a shorter timeframe. But there are multiple challenges to overcome prior to their implementation. A workshop was convened to consider the opportunities and challenges of developing a platform trial in Crohn’s disease. The workshop and reporting was contributed to by eight clinical experts, four experts in clinical trial methodology and statistics, with further input from two professional representatives from Crohn’s and Colitis UK, and two patient representatives.

Key disease-specific and trial design-specific considerations were discussed and recommendations made based on these discussions. Platform trials enable the evaluation of multiple interventions simultaneously, allowing seamless recruitment of patients, with addition of promising new interventions to an ongoing trial protocol. Crucially platform trials enable halting recruitment to interventions not demonstrating sufficient benefit (or indeed evidence of harm) – allowing resources to be focused on more promising and important questions for patients. Multiple benefits and potential challenges were identified in order to bring adaptive platform trials to Crohn’s disease. Successful delivery and implementation of such trials was considered achievable through commitment and partnership between patients, clinicians, trial methodologists, regulatory authorities, funding agencies and industry partners.