Use of RWD in the assessment of economic evaluations of innovative health products: lessons learned from the French National Authority for health (HAS)

OBJECTIVES: Our objective was to identify key patterns and discuss the lessons learned from the use of real-world data (RWD) in the cost-effectiveness analyses (CEAs) of innovative health products (IHPs) as assessed by the French National Authority for Health from January 2016 to May 2023. METHODS: A retrospective analysis was conducted on the use of RWD in the CEAs of IHPs. Our material included HAS assessments of CEAs and manufacturers' technical reports. The RWD studies were classified into eight categories, and a specific template was constructed to report and discuss their use in terms of predefined methodological aspects. RESULTS: In all, 88 percent (129/147) of the CEAs integrated RWD studies. Retrospective cohorts were the most frequently used kind of study in the CEAs, while prospective cohorts were mainly used to identify the analyzed population and to externally validate models. We identified opposing temporal trends in the use of cohort studies versus registries. Approximately 8 percent (10/129) of the CEAs could be adjudged as invalidated due to major limitations regarding RWD use (e.g., lack of relative effectiveness). CONCLUSIONS: We learned several lessons from the use of RWD in the HAS assessments of the CEAs of IHPs. Retrospective cohort studies were the most commonly used RWD source to populate CEA parameters of the CEAs regardless of the type of IHP, and their use has increased over time. The implementation of good practices for the use of RWD studies should improve the role of RWD in economic modeling and address uncertainties surrounding CEAs.