Development and validation of a framework using multiple sources of real-world data to assess the benefit-risk balance of advanced therapies in inflammatory bowel disease

Assessing the benefit-risk balance of treatment is crucial in medicine to select the most appropriate treatment for patients.

This process faces major challenges, how to implement evidence in real time to guide treatment choice and how to summarize efficacy and safety outcomes to patients and clinicians, while incorporating patient preferences. Inflammatory bowel diseases (IBD) affect 3 million residents in Europe.

New advanced therapies have been recently approved, but in the absence of head-to-head randomized controlled trials (RCT), data are lacking to position each drug in the therapeutic management.

Multiple types of real-world data (RWD) sources are available to assess the effect of treatment, but each type has its proper strengths and limitations. Linking multiple RWD sources can overcome limitations and leverage the strengths of each data source.

We recently emulated clinical trials supporting the use of RWD to assess treatment effectiveness and safety in IBD.Based on the linkage of nationwide healthcare databases, electronic health records, and a prospective cohort of IBD patients, the aims of the current proposal are (i) to validate algorithms identifying IBD characteristics and outcomes in nationwide healthcare databases, (ii) to emulate head-to-head clinical trials comparing advanced therapies in IBD, (iii) to incorporate trials findings and patient preferences in a clinical decision support system for IBD patients and clinicians, whose impact will be assessed by a pragmatic RCT nested in nationwide healthcare databases.This challenging project will involve multi-disciplinary aspects, from qualitative approach to artificial intelligence, to clarify the position of each treatment in IBD and disseminate findings to patients and clinicians.

Besides scientific importance, it will have societal impact by setting the stage for a novel post-marketing drug surveillance framework applicable not only to future IBD treatments but also to other chronic diseases.