OBJECTIVES: Health technology assessment (HTA) bodies increasingly acknowledge the value of real-world evidence (RWE) for decision-making, with the UK’s National Institute for Health and Care Excellence (NICE) and the French Haute Autorité de Santé (HAS) having formalised their requirements. Alignment of HTA body RWE guidelines is key to designing studies that are transferable between countries in specific cases, such as rare diseases. This study reviewed RWE guidelines from NICE and HAS to assess the feasibility of conducting a single RWE study acceptable to both HTA bodies.

METHODS: The NICE RWE guidance published on 23^rd June 2022 and the HAS RWE guidance published on 11^th June 2021 were reviewed regarding requirements for database selection, analytical methodology and reporting.

RESULTS: Both NICE and HAS guidelines specify that the RWE database must be representative of the target patient population, but neither offers clear instructions on how the appropriateness of data from different countries should be assessed. Examples in each guideline focus on country-specific databases; nevertheless, the use of international data is not explicitly rejected. For providing consistent and structured information on data suitability NICE specifies the Data Suitability Assessment Tool, and HAS the Registry Evaluation and Quality Standards Tool. Regarding the methodology to be used, including confounding, causal inference, and assessment bias, NICE guideline provides links to existing NICE technical support documents; HAS guideline refers to international references and the EUnetHTA collaboration.

CONCLUSIONS: This review suggests it is possible to design an RWE study for HTA purposes that fulfils the requirements from both NICE and HAS and potentially other HTA bodies. However, the guidance available remains insufficiently clear on the criteria for transferability of international datasets. In the meantime, sponsors are urged to seek early scientific advice on this topic to ensure their RWE study design is accepted by NICE and HAS.