A Review of the Quality-of-Life Data Use in French Economic and Public Health Assessment Committee’S (CEESP) Efficiency Opinions for Immune-Checkpoint Inhibitors (ICIS) and Chimeric Antigen Receptor T (CAR-T CELL THERAPIES)

OBJECTIVES: Cost-utility analysis (CUAs) are used to inform Health Technology Assessment. In these CUAs, quality-adjusted life years are calculated based on utilities. The objective was to describe the use of quality-of-life (QoL) data in French efficiency opinions (EOs) and to identify CEESP reservations relating to this use.

METHODS: A systematic literature review of QoL data used in French EOs for ICIs and CAR-T cell therapies from 2015 to 2022 was performed. In each EO, data sources, valuation methods and CEESP reservations regarding QoL data use were reviewed.

RESULTS: 36 EOs were included. The main source of utilities were clinical trials (97%; n=35), including open-label trials in 74% (n=26/35) of EOs. Open-label trials informed treatment-related utilities in 19% (n=5/26) of EOs. EQ-5D was widely (89%; n=32) used for valuation. Three (8%) EOs used a non-French value set. A repeated measurement mixed model was used in 25% (n=9) of EOs. Same health states utilities were assigned to all comparators in 53% (n=19) of EOs. Utilities were adjusted for age in 11% (n=4) of EOs. Disutilities are included for 86% (n=31) of EOs and based on literature (exclusively non-French) in 58% (n=18/31) of EOs. Disutilities for adverse events were considered in all 31 EOs and disutilities for administration in 13% (n=4/31). Some other methodological information, such as questionnaire completion rates, were underreported. QoL was among the 5 most sensitive parameters in 54% of deterministic sensitivity analyses. Proportion of CEESP questions regarding this topic increased from 4% to 15% between 2015 and 2021. Methodological reservations regarding QoL was mentioned in 67% (n=24) of EOs with 1 major reservation in 2021.

CONCLUSIONS: While QoL valuation methods are becoming an important challenge, this study highlights heterogeneity in QoL data use in French EOs. Specific French guidelines sustained by an increasing industrial/CEESP experience could help harmonize approaches and limit CEESP reservations.