OBJECTIVES: Real-world evidence (RWE) has gained increasing attention as a tool to supplement clinical trial data in technology appraisals, particularly in oncology, where single-arm trials are common. Despite publication of a framework on the appropriate use of RWE in appraisals, the extent to which the National Institute for Health and Care Excellence (NICE) is willing to accept RWE as a source of comparative evidence remains unclear. This research investigated the acceptance of RWE in this context in prior oncology appraisals.

METHODS: Oncology appraisals with published final guidance on the NICE website up to 14^th June 2023 were manually searched to identify the 10 most recent appraisals in which RWE was used as a source of comparative efficacy. Key information was systematically extracted for analysis relating to the use of RWE, external assessment group (EAG) and committee critique of the RWE, and the outcomes of the appraisal.

RESULTS: Of the 10 appraisals reviewed, 7 received positive recommendations. Overall, the EAG and committee acknowledged the need for RWE, and considered its use appropriate in most cases. However, the presented RWE formed part of the EAG’s key issues in 9 of the reviewed appraisals and represented an important source of committee uncertainty in 8. Opaque reporting of the provenance, identification and quality of RWE sources, and suitable reporting of methods used for statistical adjustments for differences between RWE and trial populations, were commonly reported issues by the EAG.

CONCLUSIONS: This review demonstrates NICE’s willingness to accept the use of RWE as a source of comparative evidence in oncology appraisals. However, clear concerns remain, particularly around generalisability to the relevant population in clinical practice. Whilst such uncertainty associated with RWE as a source of comparative evidence is challenging to fully resolve, practical but important steps to reduce uncertainty were lacking in company submissions.