OBJECTIVES: To understand the temporal trends in Conditional Marketing Authorizations (CMAs) granted by the European Medicines Agency (EMA) over the years and their correlation with Health Technology Assessment (HTA) outcomes in France.

METHODS: The compilation of Conditional Marketing Authorizations (CMAs) from January 1, 2014, to December 31, 2023, was sourced from the EMA's annual reports. The outcomes of the French HTA body, Haute Autorité de Santé (HAS), were cross-referenced with the information available on their website. Key insights were then extracted and analyzed.

RESULTS: The findings of this study suggest that the number of CMAs granted by the EMA increased during the period from 2019 to 2023 compared to 2014-2018 (12% vs. 4%). Overall, the top three therapeutic areas with the most CMAs granted were oncology (57%), vaccines (13%), and hematology (10%). Among these CMAs, more than half received a positive HAS recommendation (54%), followed by 16% that were not recommended. Meanwhile, 24% were not assessed, and for the remaining 6%, the marketing authorization was withdrawn. However, it is worth noting that the rate of positive HAS recommendations for CMA-granted drugs decreased from the 2014-2018 period to the 2019-2023 period (71% vs. 48%).

CONCLUSIONS: CMAs are becoming increasingly common, with the potential for regulatory approval during the initial phases of clinical trials. While most CMAs within the study timeframe secured positive HTA outcomes in France, there has been a noticeable decline in the trend toward positive reimbursement decisions. For manufacturers, it is crucial to thoroughly assess their approaches to evidence generation, considering not just the accelerated regulatory routes such as CMAs but also the specific evidence requirements of HTA bodies.