OBJECTIVES: Efficacy of interventions from randomised controlled trials (RCTs) forms the backbone of reimbursement submissions. However, RCTs have limited external validity, providing insufficient insights for payers to make resource-allocation decisions for their populations. Real-world evidence (RWE) provides a more accurate representation of drug utilisation and healthcare expenditure in clinical practice, and can inform on comparative effectiveness in the absence of head-to-head trials. We present examples of vedolizumab (VDZ) outcomes in inflammatory bowel disease (IBD) to demonstrate how RWE can support value-based decision-making by payers.

METHODS: MEDLINE-, Cochrane-, Embase-indexed publications and conference abstracts were systematically searched from 1/5/2014–10/1/2017 for studies reporting real-world VDZ effectiveness, safety, healthcare resource utilisation, and treatment patterns. Reports for patients <18 years or off-label VDZ use were excluded.

RESULTS: The review identified