Coverage and Pricing Recommendations of the French National Health Authority for Innovative Drugs: A Retrospective Analysis From 2014 to 2020

This study provides a retrospective analysis of the recommendations of the French National Health Authority on the reimbursement and pricing of innovative drugs. The analysis includes drugs subjected to both economic and clinical evaluations in France from 2014 to 2020. Ordered logistic and quantile regressions are used to estimate the factors associated with the clinical value (SMR), the clinical added value (ASMR), and the incremental cost-utility ratio (ICUR) of innovative drugs. All variables used in the regression analyses are extracted from the Clinical and Economic Opinions for the 146 observations. Regression analyses indicate that 2 of the 5 official criteria, the efficacy-adverse events balance of the drug and its function, are significantly associated with the SMR rating. The ASMR is positively associated with the disease severity, the quality-adjusted life-year (QALY) gain provided by the drug, and the validation of the ICUR in the Economic Opinion. At the first quartile of the ICUR distribution (approximately €50 000/QALY), higher ICUR levels are observed for drugs with a smaller target population and for drugs claimed as more innovative. Higher ICUR levels are also observed for pediatric drugs and for drugs with no therapeutic alternative at the third quartile of the distribution (approximately €240 000/QALY). Not all official criteria of the SMR are associated with actual ratings obtained. Regarding the ASMR, the results support the idea of a convergence between the 2 independent clinical and economic appraisal processes. Finally, the factors influencing the ICUR level vary across the distribution of ICUR. •The analysis of the recommendations of the French National Health Authority for innovative drugs entering the market between 2014 and 2020 shows that not all official criteria are considered in the reimbursement and pricing decisions, which questions the transparency and predictability of the French health technology assessment process.•Although the clinical and economic assessments are independent in the French health technology assessment process, results show consistency between the clinical and economic evaluations regarding the added value of new drugs.•We document the incremental cost-utility ratio (ICUR) levels of innovative drugs in France, and we find that drug and disease characteristics associated with the ICURs vary across the ICUR distribution.