Presentation (CTI)

OBJECTIVES: To compare real‑world data (RWD) and real‑world evidence (RWE) requirements across EU Joint Clinical Assessment (JCA), NICE, HAS, and IQWiG, and assess implications strategy and use.
METHODS: A review of RWE guidance across JCA, NICE, HAS, and IQWiG was conducted. Evidence requirements were mapped across two dimensions: (1) policy (data access, role, and value of RWE) and (2) methodology (causal inference frameworks and analytical standards). Structured expert elicitation with country market access stakeholders was undertaken to validate findings and capture implications for JCA PICO scoping, evidence generation, and dossier usability.
RESULTS: All frameworks require RWD and position RWE as complementary to randomized evidence, mainly to address evidence gaps. However, divergence exists between EU‑level emphasis on cross‑country transferability and national preference for locally relevant data. France and Germany strongly priorities national datasets, while NICE applies a more pragmatic approach and JCA remains descriptive to preserve national decision-making. Expert elicitation confirmed that this misalignment limits direct reuse of JCA evidence and necessitates early identification of EU‑core versus country‑specific data requirements.
All agencies align on the need for robust causal inference, with strong reliance on IPD for confounding adjustment and consensus on Target Trial Emulation as the preferred framework. Differences arise in implementation: NICE emphasizes transparency and bias analysis, HAS focuses on protocol validation and real‑world applicability, while JCA and IQWiG apply more conservative approaches, including shifted null hypothesis testing. Experts highlighted that these differences impact feasibility of RWE strategies and require early planning to ensure alignment across jurisdictions.
CONCLUSIONS: While aligned on principles, policy and methodological divergences limit RWE portability across jurisdictions. Integrating expert‑informed strategies early is critical to optimize JCA relevance and national HTA usability. It would be beneficial to develop JCA specific RWE guidelines to direct evidence generation preferences.