OBJECTIVES

: According to the framework agreement, the price of the product is driven by the Clinical Added Value. However, we explored whether other criteria, such as disease severity and quality of the submitted clinical data, could be used by Economic Committee to fix the price in France. The aim of this study was to define whether the annual treatment cost, based on the list price in France and assessment criteria are correlated.

METHODS

: Two descriptive analysis respectively focused on orphan drugs (OD) and blood cancers treatments (BCD), were retrospectively conducted from a MyPB^® database query. Products assessed by the Transparency committee with at least their first registration published between 01/01/2015 and 31/03/2018 for OD and between 01/01/2013, and 31/03/2018 for BCD were selected. For each product, the disease severity and quality of clinical data have been quantified according to a prespecified rating scale (from 1 to 5 ranking). The annual treatment cost was calculated in line with the posology notified in the Market Authorization and the list price, which had to be published in the Official Journal.

RESULTS

: During the two defined periods, 15 drugs have been evaluated in 17 indications, which were analyzed one by one. BCD are indicated in severe diseases (grade ≥4). However, the quality of their clinical data is disparate. The analysis shows a positive correlation between the quality of the clinical data and the price of BCD; disease severity being similar. Among OD, the results are more heterogeneous, which do not enable to distinguish a consistency in the setting of their treatment cost.

CONCLUSIONS

: It seems not relevant to compare products on the basis of this regulatory criteria. A pricing benchmark is more appropriate for products within the same therapeutic class, keeping in mind that other criteria than those mentioned are taken into account in the price setting.