Integrating Real-World Evidence for Advanced Therapies in Inflammatory Bowel Disease Into Reimbursement Submissions: Insights and Considerations From the UK
Author(s)
Neil R. Brett, PhD1, Garthiga Manickam, PhD2, Roy Mootoosamy, PhD3, Daniela Castano, MD4, John S. Sampalis, MSc, PhD1.
1PPD™ Observational Studies, Thermo Fisher Scientific, Montreal, QC, Canada, 2PPD™ Evidera™ Health Economics and Market Access, Thermo Fischer Scientific, Montreal, QC, Canada, 3PPD™ Evidera™ Health Economics and Market Access, Thermo Fisher Scientific, London, United Kingdom, 4Thermo Fisher Scientific, San Diego, CA, USA.
1PPD™ Observational Studies, Thermo Fisher Scientific, Montreal, QC, Canada, 2PPD™ Evidera™ Health Economics and Market Access, Thermo Fischer Scientific, Montreal, QC, Canada, 3PPD™ Evidera™ Health Economics and Market Access, Thermo Fisher Scientific, London, United Kingdom, 4Thermo Fisher Scientific, San Diego, CA, USA.
OBJECTIVES: In inflammatory bowel diseases (IBD), recent studies show key differences between clinical trial and real-world (RW) IBD populations (age, comorbidities, etc.), supporting the UK’s National Institute for Health and Care Excellence (NICE) strategy to use RW data to fill reimbursement submissions knowledge gaps. The objective was to evaluate RW evidence (RWE) use in previous UK health technology assessment (HTA) submissions for IBD advanced therapies (biologic or JAK-inhibitor therapies) for adults and offer recommendations for future submissions.
METHODS: Review of NICE appraisals was performed for Crohn’s disease and ulcerative colitis to understand RWE expectations, integration and usage. Considerations are provided for future submissions.
RESULTS: 16 advanced therapy single technology appraisals from 2015-2025 were reviewed (5 for CD, 9 for UC, 2 for CD and UC). RWE was only included in one cost comparison submission (for a UC therapy). RWE was used for the comparator to inform proportions of dose escalation, but no RW data was available for the treatment being appraised. Network meta-analyses to evaluate comparative efficacy (indirect comparisons) also excluded RWE. In IBD, we suggest that RWE could inform health economics modelling (e.g. effectiveness, costs, dosing, resource use), provide insights into patient populations for future trials planning, and understanding patient profiles that respond better to certain treatment paradigms. Data landscaping and literature reviews could be used for comparative RWE if similar treatments are already reimbursed. Efficient patient-centered data collection post-product approval could allow for early insights into treatment patterns and shorter-term outcomes. RWE could also further support treatment positioning after product reimbursement. NICE guidelines can support RWE planning and conduct.
CONCLUSIONS: RWE is under-utilized in HTA submissions to NICE for advanced therapies for IBD. Future submissions should consider knowledge gaps that could most benefit from RWE integration, including modelling assumptions.
METHODS: Review of NICE appraisals was performed for Crohn’s disease and ulcerative colitis to understand RWE expectations, integration and usage. Considerations are provided for future submissions.
RESULTS: 16 advanced therapy single technology appraisals from 2015-2025 were reviewed (5 for CD, 9 for UC, 2 for CD and UC). RWE was only included in one cost comparison submission (for a UC therapy). RWE was used for the comparator to inform proportions of dose escalation, but no RW data was available for the treatment being appraised. Network meta-analyses to evaluate comparative efficacy (indirect comparisons) also excluded RWE. In IBD, we suggest that RWE could inform health economics modelling (e.g. effectiveness, costs, dosing, resource use), provide insights into patient populations for future trials planning, and understanding patient profiles that respond better to certain treatment paradigms. Data landscaping and literature reviews could be used for comparative RWE if similar treatments are already reimbursed. Efficient patient-centered data collection post-product approval could allow for early insights into treatment patterns and shorter-term outcomes. RWE could also further support treatment positioning after product reimbursement. NICE guidelines can support RWE planning and conduct.
CONCLUSIONS: RWE is under-utilized in HTA submissions to NICE for advanced therapies for IBD. Future submissions should consider knowledge gaps that could most benefit from RWE integration, including modelling assumptions.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA208
Topic
Economic Evaluation, Health Technology Assessment, Study Approaches
Topic Subcategory
Value Frameworks & Dossier Format
Disease
Biologics & Biosimilars, Gastrointestinal Disorders