PMU47 ORPHAN DRUG DESIGNATION: NO IMPACT ON P&R PROCESS TIMING IN ITALY

Abstract

To assess the orphan designation influence on Italian Medicines Agency (AIFA) P&R process timings. Approval process steps of new medicinal products (first reimbursement process) were tracked in internal database, with data coming from EMA website, AIFA committees meeting outcomes and Italian Official Gazette; drugs with completed P&R process from January 2015 til May 2019 were analysed; 32 reimbursed drugs out of 121 had orphan designation. From database analysis, the average (n=121; 100%) timing of P&R process resulted in 271 days from CTS opening to Official Gazette publication. Orphan drugs (32, 26%) resulted in a similar time to reimbursement compared to non-orphan drugs (89, 74%): 286 vs 265 days respectively. Categorization by therapeutic area yielded a 312 days P&R process for orphan-oncological drugs (16, 13%) compared to 260 days of orphan-non-oncological drugs (16, 13%). When AIFA granted innovative status to an orphan drug (14, 12%) the process took 248 days on average. The presence of a monitoring registry also impacts on the approval timing: 23 orphan drugs (19%) with monitoring registry have a longer process compared to 9 (7%) other orphan drugs (278 vs 213 days). The P&R process of orphan drugs is mostly impacted by committees assessment timing, while post assessment (bureaucratic) phase remained comparable (about 87 days). Data analysis shows a similar approval timing for orphan drugs compared to non-orphan drugs: even if unmet medical need is often recognised in rare disease area, limited data availability and knowledge on the specific disease may need further research by AIFA. Therapeutic area has a significant impact on timing of P&R process, probably due to the need of more detailed assessment for oncological drugs compared to non-oncological drugs. The combination of orphan and innovative designation accelerates the approval process, highlighting the key role of innovative status to facilitate access in Italy.