OBJECTIVES

In France, the Transparency Committee (TC) evaluates orphan drugs (OD) in the same way than others. In its opinions, the TC grants by indication one of the four levels of “medical benefit” (SMR) (important, moderate, low, insufficient) which determines the reimbursement and an “Added Medical benefit” (ASMR) regarding the existing therapies (five ratings: I, II, III, IV, V) used in price negotiations. The aim of this analysis was to identify the convergence in estimated value via the SMR and ASMR between companies’ requests and TC opinions and the hearing impacts on TC evaluation.

METHODS

SMR and ASMR levels were analyzed by indication of new OD evaluated on initial registration between January 2016 and December 2019. The success rate was defined by matching the levels of the SMR/ASMR couple and the SMR or ASMR independently.

RESULTS

Of the 55 OD evaluated in 62 indications, only 8 (12.9%) convergences on SMR/ASMR couples were observed. Concerning SMR levels, companies and TC agreed on most of the cases (75.8%). SMR was considered “insufficient” by the TC in 4 cases and the indication was restricted for 5 drugs. Concerning ASMR, a 17.74% convergence was observed. Six ASMR were not granted for lack of reimbursement. Out of 15 ASMR II requests, only 2 were finally granted with ASMR II, 6 ASMR III, 7 ASMR IV or V. Out of 33 hearings with a request for improvement of the SMR and/or ASMR, 11 (33%) received a better SMR level than that initially granted and 12 (36%) a better ASMR level.

CONCLUSIONS

In spite of the TC being attentive to companies’ requests in most cases, the value of new OD estimated by companies is not shared by the TC. This may be the consequence of the difficulty in conducting methodological studies within the framework of the TC.