Recent innovations in clinical trial design for inflammatory bowel disease

Abstract

Clinical trial design in inflammatory bowel disease (IBD) is evolving to address challenges in drug development and approvals. For clinical development, notable innovations include Bayesian designs, adaptive designs, integrated-phase trials and master protocols (such as umbrella, basket, and platform trials). The inclusion of biomarker-driven strategies and precision medicine (PM) trials bring aim to enable patient stratification based on prognostic or predictive markers, leveraging molecular signatures to customize therapy. However, recent studies highlight both the promise and complexity of this approach. Patient-reported outcomes (PROs) have gained prominence as key endpoints, aligning trials with patient-centric measures and regulatory guidance that emphasize symptoms and quality-of-life metrics. Digital health tools and artificial intelligence (AI) are being integrated to streamline trial conduct, from remote monitoring and telemedicine visits to AI-assisted recruitment and data analysis. Pragmatic trials and the integration of real-world evidence (RWE) aim to complement traditional efficacy trials by evaluating treatments in routine care settings. Together, these innovations mark a new era in IBD clinical trial design, aiming to expedite therapeutic development and enhance the relevance of trials to patient care.