Establishing Stakeholder Priorities for Development of a Multi‐Arm Multi‐Stage Platform Trial in Crohn's Disease
Abstract
ABSTRACT Background and Aims The expanding range of advanced therapies for Crohn's disease (CD) has increased complexity in treatment selection, particularly in refractory disease. Conventional randomised controlled trials are limited in evaluating multiple treatment strategies efficiently. We aimed to establish stakeholder consensus regarding the key clinical priorities required to inform subsequent development of a multi‐arm multi‐stage adaptive platform trial for Crohn's disease. Methods A two‐stage modified Delphi process was conducted with international stakeholders, including clinicians, trialists, allied health professionals and patients with CD. Delphi 1 identified priority populations, interventions and comparators; Delphi 2 focused on outcomes. Consensus was predefined as ≥ 70% agreement. Results A total of 112 participants were included in Delphi 1 and 240 in Delphi 2. Patients with exposure to ≥ 2 advanced therapy classes were prioritised as the target population (83%). Multiple advanced therapies across mechanistic classes were selected, with support for inclusion of dual advanced therapy strategies (82%). Standard‐of‐care comparator strategies reflecting real‐world practice were endorsed (83%). Clinical remission was identified as the preferred primary outcome (100%), with support for composite endpoints including endoscopic response at 48 weeks (70%). Objective measures, including endoscopy, imaging and biomarkers, were prioritised, alongside central reading and standardised assessment timepoints. Conclusions This study established stakeholder consensus on key priorities relating to population selection, interventions, comparator strategies and outcome measures to inform development of a future multi‐arm multi‐stage adaptive platform trial in Crohn's disease. These findings provide an evidence‐informed foundation for subsequent protocol development rather than defining the final trial architecture.
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Authors: Shellie Jean Radford, Nurulamin Noor, Daniel Lightowler, Jonathan Segal, Patricia Kelly, Shahida Din, Klaartje Bel Kok, Shaji Sebastian, Peter Irving, Charles Maxwell-Armstrong, Stuart Taylor, Rachel Cooney, James Lindsay, Bruce Sands, Remo Panaccione, Gordon W Moran
Institutions: Icahn School of Medicine at Mount Sinai, The University of Melbourne, Nottingham University Hospitals NHS Trust, Nottingham Biomedical Research Centre, University of Calgary, University College London, The London College, University of Edinburgh, University of Cambridge, Guy's and St Thomas' NHS Foundation Trust, Thomas Foundation, The University of Western Australia, University of Birmingham, Queen Mary University of London, The Royal Melbourne Hospital, Cambridge University Hospitals NHS Foundation Trust, Bridge University, Royal Prince Alfred Hospital, University College Birmingham, Barts Health NHS Trust, University Hospitals Birmingham NHS Foundation Trust, Institute of Genetics and Cancer, Crohn's and Colitis UK, Western General Hospital