Use of Real-World Evidence in Health Technology Assessments of Non-Oncology Rare Disease Therapies

Background: Real-world evidence (RWE) can complement clinical trials to inform health technology assessments (HTAs). This study examined the extent to which RWE is considered in HTAs of non-oncology orphan medicinal products across six agencies globally. Methods: Published European Medicines Agency decisions were reviewed to identify approved non-oncology orphan medicinal products (2018–2023) that included RWE within their submission package, which was anticipated to align with the inclusion of RWE in HTA submissions. Data were extracted from the corresponding HTA reports published by six national agencies (Australia, Canada, France, Germany, Sweden, and the UK). Results: RWE was included in 105 regulatory submissions and 52.6% of the corresponding HTA reports (range: 29.9% [Germany] to 78.8% [Canada]), nearly 90% of which received a positive decision (range: 44.4% [Australia] to 100.0% [Germany]). RWE was derived from a variety of study designs and commonly supported clinical efficacy across many therapeutic areas. Conclusions: RWE commonly supports HTAs of recently approved non-oncology orphan medicinal products, strengthening the evidence base and contributing to demonstration of product value.