| الوصف: |
Background & Aim Advance therapy medicinal products (ATMPs) represent promising therapy for many untreatable or life-threatening diseases. To guaranty rapid accessibility to these therapies, Health agencies from United State (FDA), Europa (EMA) and Japan (MHLW) have established accelerated procedures through, respectively, breakthrough (BT) or regenerative medicine advance therapies (RMAT), Priority medicines (PRIME) and Sakigake (ST) designations. These designations, granted on the base of preliminary data of safety and effectiveness in unmet or life-threatening indications, permit to access to priority and accelerated review of clinical trial and marketing access submission. Methods, Results & Conclusion Based on informations from health agency and industrial websites, we listed 97 designations granted. Within 26 were multi-granted products (4 BT/RMAT, 14 BT/PRIME, 7 RMAT/PRIME, 1 BT/ST, 2 RMAT/ST and 1 PRIME/ST) leading to 71 accelerated investigational (65) or approved (6) ATMPs. We also listed 25 approved ATMPs without designation. By comparing the approval times after submission between accelerated and classic ATMPs, we observed that designations really improved this delay by dividing it by more than twice (only 41.5% of a classic approval time for a BT approval, 40.8% and 30.3% for PRIME and ST approvals, respectively). This difference is less important when comparing accelerated and classic approval time of submission after 2014 only (respectively 51.8%, 53.7% and 50.2%). Indeed, regulatory approval times have been reduced since the first ATMP approvals, to goal today to 10, for FDA and EMA, to 12 months, for MHLW, for classic approval and to 5 months for an accelerated one. In practice, if the mean approval times have decreased by more than twice, with a mean time of 13.2, 19.5 and 12.2 month respectively for classic approval submit after 2014, the goal times have not been reached except for Japan. The same observation can be done for accelerated ATMPs, with a mean approval time of 6.8, 10.4 and 6.1 months. We can conclude from these data that designation for accelerated procedure is indeed the fastest way to get market access approval for a product, but stay a selective pathway, with only 38.3% and 24.2% of the RMAT and PRIME granted, respectively. However, the problematic of the difference of approval time for classic and accelerated pathway between EMA and FDA or MHLW need to be solved by the European regulatory and review committee to permit a faster patient access to innovative therapies. |
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