Presentation (CTI)

OBJECTIVES: In France, the Commission d'Evaluation Economique et de Santé Publique (CEESP) evaluates the cost-efficiency of health technologies eligible for reimbursement. Its reference case is the cost-utility analysis (CUA), measuring outcomes as quality-adjusted life years (QALYs); cost-effectiveness analysis using life-years gained (LYG) is used when health-related quality of life (HRQoL) is not a primary consequence, or when utility data are unavailable. We examined CEESP economic opinions to characterise how often and in what way HRQoL and clinical outcome assessment (COA) evidence was subject to methodological objections (reserves, graded minor, important, or major), and whether evidence gaps co-occurred with demands for post-inscription real-world studies
METHODS: All CEESP economic opinions adopted between 2021 and to-date were identified through the HAS registry. Each opinion was reviewed to extract characteristics of the economic evaluation, utility invalidation (where applicable), methodological critiques related to HRQoL strategy, and whether recommended post-listing real-world studies included HRQoL endpoints.
RESULTS: In a preliminary sample of 13 opinions (2025-2026), 12 submitted a QALY-based CUA (one invalidated). Among the 12 CUA-based opinions, 10 carried methodological reserves related to the QALY estimate: 5 concerned questionnaire missing data patterns and handling, incomplete collection, or inadequate measurement timepoints in the pivotal trial, and 5 concerned analytical and modelling choices applied to the utility data. A post-inscription real-world study was recommended in 12 opinions; of these, 9 explicitly requested HRQoL or QoL data collection.
CONCLUSIONS: In this preliminary sample, reserves related to HRQoL and utility evidence were near-universal, and the majority traced to choices made before the economic model. The high rate of real-world study recommendations including QoL endpoints suggests that evidence judged insufficient is frequently deferred to post-launch studies rather than collected prospectively. These findings underscore that HRQoL strategy gaps are often ones that could have been anticipated and addressed at the trial design stage.