OBJECTIVES: Real-world evidence (RWE) obtained from an analysis of real-world data (RWD) can bridge gaps in evidence not addressed by interventional clinical trial-based research and is thus valuable to payers for decision-making. The use of RWE to support pivotal trial data is rapidly increasing, and is particularly prevalent in oncology. However, less focus has been placed on its utility in non-oncology submissions.

METHODS: HTA submissions to NICE (UK), HAS (France), and G-BA (Germany) between January and December 2023 were reviewed to identify non-oncology submissions that leveraged RWE; trends in the use and acceptability of such evidence were analyzed.

RESULTS: 41 non-oncology appraisals were found for NICE, 13 non-oncology appraisals were found for HAS, and 61 non-oncology appraisals were found for G-BA. The majority of submissions to NICE (75%) included some form of RWE, with 35% of submissions including multiple forms of RWE. Just under half (46%) of submissions to HAS included RWE – two-thirds of which leveraged RWE to support the tolerance and safety of the assessed drug. The market with the least use of RWE was Germany, where only 9% of non-oncology appraisals included RWE. This data was only accepted and used in decision‑making in one instance.

CONCLUSIONS: The use of RWE in non-oncology HTA submissions varied across scope markets, with the UK being the most welcoming market, and Germany being the least. RWE was used in multiple ways including supporting clinical effectiveness, tolerance, and cost-effectiveness. Where RWE wasn’t accepted, the main criticisms were levied at the quality and completeness of data. With the advent of AI tools that facilitate data collection and quality control, these critiques may be more easily addressed by manufacturers in the future.