OBJECTIVES: The reimbursement level of drugs in France is based on their medical assessment by the Transparency Committee. The aim of this study is to understand the rationale behind the assessment of drugs in all therapeutic areas except oncology by determining the criteria that influence the Actual Benefit (AB) levels.

METHODS: We performed a retrospective analysis between January 2014 and March 2017 of the new products and the new indications but oncology drugs were excluded. We searched relevant criteria of the Actual Benefit assessment carried out in each indication pertaining to the drug evaluation from the opinion of the Transparency Committee.

RESULTS: In total, 88 drugs in 111 indications were evaluated including 43 moderate AB, 12 mild AB and 56 insufficient AB. Among the criteria taken into account in the AB, we found the following relations: a better clinical efficacy/effectiveness and safety ratio of the medicine led to a better AB (80% of the moderate benefit-risk ratio obtained a moderate AB, 50% of the low benefit-risk ratios obtained a mild AB and 100% insufficient benefit-risk ratio obtained an insufficient AB). Treatments of 3^rd or last intention or that have no place in the therapeutic strategy led to mild and insufficient AB (95% of the cases). As to the methodology, most moderate AB was obtained with superiority comparative (88% of cases) and phase III (93% of cases) studies. A non-randomized and non-double-blind study leads to insufficient AB (except for an open case) and finally, we found that moderate AB could be obtained with placebo-controlled studies despite the presence of therapeutic alternatives (42% of cases).

CONCLUSIONS: The factors influencing the moderate, mild and insufficient AB levels were the clinical efficacy/effectiveness and safety of the medicine, the therapeutic strategy as regards to therapeutic alternatives and the methodology of the study (randomization, blinding and kind of study).