HTA34 A Review of Real-World Evidence in Health Technology Assessment Decision-Making of Gene Therapies in the US, Canada, UK, EU4, and Japan

Abstract

Evidence submitted for health technology assessments (HTAs) of gene therapies (GTs) are limited and associated with uncertainty. Real-world evidence (RWE) may address these uncertainties, with many health authorities adopting new guidelines and increasingly requesting RWE. Our aim was to evaluate the role of RWE in the HTA decision-making of GTs.