PDG71 Comparative Analysis of the Transparency Committee Opinions Concerning the Medical Benefit of Drugs Available in EARLY Access Programs VS Other Drugs in France: 2020 Update and TREND Assesment

Abstract

The aim of this research was to update the analysis published last year about the comparison of 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. All the TC opinions concerning a first inscription to the reimbursement adopted between January 2016 and March 3rd, 2020 were analyzed. In the selected time period, 228 TC opinions (vs 188 last year) met the inclusion criteria, concerning 225 drugs: for the 75 medicines available through ATU (vs 67 last year) with 92 “Medical benefit” (vs 83 last year) evaluated (one per indication) vs 170 (vs 134 last year) for the 150 medicines without early access (vs 118 last year). “Medical benefit” determines if the medicine is reimbursed or not as well as the rate of reimbursement by the national health insurance (important: 65%; moderate: 30%; low:15%). Ninety-six percent (72/75) (vs 96% last year) of medicines available in the early access program had a favorable reimbursement decision with a sufficient medical benefit for at least one of its indications (68 important, 5 moderate, 7 low) vs 83% (124/150) vs (83% last year) of medicines not available through early access with a sufficient medical benefit (104 important, 8 moderate, 16 low). Medicines available through the ATU program received 12 insufficient medical benefit (13%) vs 42 for medicines not available through ATU (25%). This extended analysis confirms the trend already observed last year. 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 others. High unmet medical need (no alternative), common criterion to obtain an ATU and a sufficient medical benefit, could explained the difference observed between the two groups.