OBJECTIVES

The aim of this research was to compare the distribution of the added medical benefit levels issued by the HAS transparency committee depending on the early access status (Temporary Use Authorization (ATU) or not). Early access program is granted in case of no alternative.

METHODS

All the TC opinions concerning a first inscription to the reimbursement adopted between January 2016 and April 17^th, 2019 were analysed. Simplified procedures and new applications following a previous withdrawal of application or a previous negative opinion have been excluded.

RESULTS

188 TC opinions met the inclusion criteria, concerning 185 drugs: 64 medicines available through ATU were evaluated for the “Added Medical benefit” (73 ASMR assessed, one per indication) vs 98 medicines not available in early access (100 ASMR assessed). The added medical benefit measures the clinical added value compared to existing therapies already reimbursed and is used to determine the price. Fourteen (22%) medicines available through ATU were granted with an ASMR considered as innovative (between I and III: 0 major ASMR I, 3 important ASMR II, 11 moderate ASMR III) vs 4 medicines without early access (1 important ASMR II and 3 moderate ASMR III). Twenty-two (34 %) drugs received a unique minor ASMR IV for medicines available in early access vs 15 (15 %) in the group without early access. Twenty-one (33 %) drugs received no added medical benefit in the group of medicines in ATU vs 79 (80,6 %) in the group without early access. Eleven drugs were granted with two ASMR: 9 in the ATU group vs 2 in the other group.

CONCLUSIONS

Medicines which benefited from an early access program were more likely to obtain an ASMR considered as innovative and a better “rating” for added medical benefit than the products which were not in early access.