OBJECTIVES : Since 2013, manufacturers are requested to submit to the French economic and public health assessment committee (CEESP) a pharmacoeconomic evaluation of all innovative products claiming an added clinical benefit (ASMR) level ≤III with a substantial impact on healthcare expenditures. The objective of this study was to review the CEESP decisions and provide a summary of the methodological concerns (MCs) reported in cardiology/diabetes indications.

METHODS : All available CEESP opinions for cardiology/diabetology indications published until May 2019 were reviewed. Specific indications, nature of the request and MCs were extracted.

RESULTS : Four opinions in cardiology being intended for a first inscription, and one in diabetology (for reevaluation) were reviewed and extracted. A total of 69 MCs were raised (2 major, 30 important and 37 minor concerns). Clinical data was the most frequent dimension of concern (20/69), and with the highest impact (2 major, 14 important and 4 minor concerns). The two major MCs were related to the lack of robustness of the clinical data as the populations simulated differed from the RCT ones. Other important concerns were related to 1- the estimation method (4/14), 2- uncertainty surrounding the data (4/14), 3- the use of surrogate outcomes to estimate treatment effect (2/14) and 4- the extrapolation method (2/14), with a probable impact on the external validity of the results. Another problematic dimension was the population simulated (13/61), with most of concerns questioning the representativeness to the French population (9/13).

CONCLUSIONS : The review of opinions emphasised the impact of use of surrogate endpoints, being particularly frequent in cardiology (biologic outcomes used in RCTs vs cardiovascular events, of interest from a HTA perspective). External validity was also raised as a key aspect for France, especially when it comes to patient characteristics.