OP28ECCO Young Researcher Award - Improving IBD patient outcomes through clinical trial innovations

Abstract Background Randomised controlled trials have been pivotal in generating an evidence-base to support clinical practice, healthcare policy and clinical guidelines. Despite the importance of trials and the undoubted improvements in patient care over the intervening decades, it is clear that there are now multiple problems with the current clinical trial enterprise in IBD. With a demand from patients, clinicians and other key stakeholders for faster answers to the most pressing clinical questions. Key challenges and solutions are considered on how to improve treatment strategies in IBD, particularly through the adoption of clinical trial innovations. Methods Three major challenges are considered, including potential solutions to overcome them. First, there is a focus on use of master protocols including platform trial designs to answer multiple questions in parallel. Second, adaptive trial designs are evaluated including use of Bayesian approaches to address unanswered questions for less common phenotypes of IBD. Third, is evaluation of investigator-initiated (academic) trials and how these can be used to answer treatment strategy questions of most importance to patients, clinicians and the wider field. Results The first international workshop on development and delivery of an academic multi-arm, multi-stage platform trial has identified multiple clinical, methodological and regulatory challenges – but also opportunities for change and the ability to speed up the evaluation of promising interventions and treatment strategies. Moreover, the first Bayesian prior elicitation project in IBD has provided a framework for investigators to take forward and address areas/phenotypes of unmet need. The generated priors and implementation into an upcoming clinical trial provides a template that could be adopted across a range of phenotypes in IBD. Additionally, despite there being only a limited number of academic, investigator-initiated trials in IBD, the benefits of such trials will be demonstrated through generation of answers to some of the most important clinical questions in the field including the benefits of early, effective intervention in Crohn’s disease. Delivery and inclusivity of pragmatic trial designs will be demonstrated including how they fit within the regulatory landscape. Conclusion Despite their importance, clinical trials in IBD face many challenges to overcome. Adaptive and platform trial approaches, adoption of Bayesian methods and increased incorporation of biomarkers within clinical trials are likely to be key. Embracing such innovations will allow delivery of more efficient trials and speed up the rate at which answers to key clinical questions can be provided for patients living with IBD. References: 1.Noor NM, Davies N, Tahir W, et al. Anti-TNF treatment from diagnosis is more effective and less costly than conventional ‘step-up’ care for patients with active Crohn’s disease: a cost-effectiveness analysis from the PROFILE trial. Journal of Crohns and Colitis. 2025. PMID 41125135. 2.Noor NM, Choodari-Ooskei B, Radford S, Gordon M, Hart AL, Hepburn T, Juszczak E, Lindsay JO, Kennedy NA, Parmar MKB, Jairath V, Moran G. Bringing an adaptive platform trial to Crohn’s disease. Nature Reviews Gastroenterology and Hepatology. 2025. PMID 40045038. 3.Noor NM, Estevinho MM, Solitano V, Yuan Y, Kahan B, Jairath V. Applying the estimand framework to IBD clinical trials. Lancet Gastroenterology & Hepatology. 2025. PMID 39914434. 4.Noor NM, Sebastian S, Parkes M, Raine T. The need for affordable, pragmatic, investigator-led clinical trials of treatment strategies in inflammatory bowel disease. Lancet Gastroenterology & Hepatology. 2024. PMID 39094586. 5.Noor NM, Lee JC, Bond S, et al. A biomarker-stratified comparison of top-down versus accelerated step-up treatment strategies for patients with newly-diagnosed Crohn’s disease (PROFILE): a multicentre, open-label randomised controlled trial. Lancet Gastroenterology & Hepatology. 2024. PMID 38402895. 6.Noor NM, Raine T. Innovations to improve the efficiency of phase II IBD clinical trials. Nature Reviews Gastroenterology and Hepatology. 2023. PMID 37308780. 7.Noor NM, Parkes M, Raine T. Moving towards patient-centred clinical trials in IBD. Nature Reviews Gastroenterology & Hepatology. 2021. PMID 34312526. Conflict of interest: Noor, Nurulamin: Grants: NMN reports grants from Celltrion, Dr Falk, Pfizer, Pharmacosmos, Tillotts. Personal Fees: NMN reports personal fees from AbbVie, BMS, Celltrion, Ferring, J&J, Lilly, Pfizer, Pharmacosmos, Takeda. Advisory board fees: NMN reports advisory board fees from AbbVie, BMS, Pfizer, Takeda.