OBJECTIVES

The aim of this research was to compare the distribution of the medical benefit levels (SMR) issued by the HAS transparency committee (TC) depending on the early access status (ATU or not) in France.

METHODS

All the TC opinions concerning a first inscription to the reimbursement adopted by French Health Authority 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

In the selected time period, 188 TC opinions met the inclusion criteria, concerning 185 drugs: for the 67 medicines available through ATU with 83 “Medical benefit” (SMR) evaluated (one per indication) vs 134 for the 118 medicines without early access. “Medical benefit” determines if the medicine is reimbursed (important, moderate, low) or not (insufficient medical benefit) and the rate of reimbursement by the national health insurance (Important: 65%; moderate: 30%; low:15 %). Ninety-seven percent (64/67) of medicines available in early access program had a favourable reimbursement decision with sufficient medical benefit (62 important, 4 moderate, 6 low) vs 83% (98/118) of medicines not available through early access with sufficient medical benefit (82 important, 7 moderate, 12 low). Medicines available through ATU program received 11 insufficient medical benefit (13.2 % of all SMR granted in this group) vs 33 for medicines not available through ATU (27.9 %).

CONCLUSIONS

Medicines which benefited from an early access program were more likely to obtain a favorable reimbursement decision and a better “rating” for medical benefit than the products which were not available through early access. High unmet medical need (no alternative) which is a common criterion to obtain an early temporary authorization and a sufficient medical benefit, could explained the difference observed between the two groups.