OBJECTIVES:

In France, the CEESP assesses the cost-effectiveness of products claiming a moderate to major therapeutic progress. A “major reservation” is issued by CEESP if the economic analysis doesn’t comply with the published methodological guidelines. The aim of this project is to assess which methodological items correlate with a risk of major reservation.

METHODS:

We extracted all pertinent variables (n=24) with less than 20% missing data from the base case analysis of 144 Economic Opinions published for new innovative drug between October 2013 and April 2022. A logistic regression, where the dependent variable corresponds to the dichotomic event “major reservation”, was fitted. Variables with a p-value below 0.20 in a univariate regression were included in a stepwise multivariate regression (p < 0.05).

RESULTS:

From the 24 variables tested, six were associated with a p-value below 0.20 indicating a possible correlation with a “major reservation” and their inclusion in the stepwise multivariate regression. The variables were the therapeutic area, the context of the submission (1^st evaluation, line extension, reassessment), the presence of an external competitors included in the dossier, utility estimation method (direct, indirect, mapping) and the tariff of the utility. Drugs submitted in virology, the presence of an external competitor, an ad-hoc study to estimate the utility and utility estimated in UK tariff were associated with a higher probability of “major reservation”.

CONCLUSIONS:

From all the information gathered in the 144 Economic Opinions published, it sems that the anticipation of a “major reservation” based on specific items isn’t straightforward. This result is consistent with the lack of predictability or standardization observed in the conclusions of CEESP opinions. Future work is needed to investigate the causality and identify clear items to predict the risk of “major reservation”.