Problem Statement: This case study assessed the quality of real-world data (RWD) incorporated in economic evaluation (EE) appraisals of innovative health products (IHP) performed by HAS from 2016 to 2022, as well as identified limitations related to their use.

Description: The EE appraisals of IHP are performed by HAS for price negotiation between the French Economic Committee of Health Products (CEPS) and pharmaceutical companies. There is a need to evaluate the extent to which these data are adequately used to meet the HAS requirements and the decision makers’ expectations. An in-depth analysis of RWD examples was performed using a RWD reporting guidance and a grid analysis; 138 appraisals of EE identified and assessed the main sources of RWD used to populate the EE (e.g., cohorts used in the external validation of survival outcomes of the standard of care).

Lessons Learned: The use of RWD in EE appraisals of IHP were subject to important limitations and uncertainties (e.g., a lack of comparability of used registries or cohorts describing the disease or the standard of care with the French population of the drug indication, insufficient documentation of the external validation of the long-term effectiveness of the comparator(s)) in almost half of appraisals. EEs were invalidated in 5% of the appraisals due to insufficient Real-World Evidence (RWE) related to the benefit of the studied IHP in terms of effectiveness or quality of life.

Future update of the HAS doctrine may include recommendations improving the assessment of the quality of RWD used in EE (e.g. type of RWD, requirements in terms of specific documentation of the drug adherence and external validation of the long-term survival outcomes and health-state utilities).

Stakeholder perspective: HTA agency