Time to market access in Italy: duration of the P&R process for rare disease drugs

Objective: This paper aims to investigate the duration of the pricing & reimbursement (P&R) procedures submitted in Italy by pharmaceutical marketing authorization holders (MAH) for drugs indicated for rare diseases. Methods: All the data used in this analysis were publicly available on different sources of the Italian Ministry of Health, the Italian Medicines Agency (AIFA) and other official websites. The information was systematically collected to investigate the timeline (days) needed to complete the P&R process. The process was divided into 6 simplified steps and the median and range of days needed for each phase were estimated based on data reported in official/published documents. The analysis was stratified considering every single step of the assessment phase and included segmentation of drugs into indications for rare diseases, Orphan designation, Innovation assessment and Managed entry agreements (MEAs). Results: Overall, 181 first indication procedures were submitted to AIFA in the period considered and, of these, 167 (92.3%) were completed and 129 procedures were considered for the final analysis and the median duration of the entire process (MAH submission to final Gazette publication) was 434 days (range 176.0-918.0). The duration of procedures for rare diseases (n = 53) was longer than those for non-rare-disease procedures (n = 76) (463.0 days vs 407.5 days respectively). Among rare disease procedures, orphan designation and MEAs represent predictors for time prolongation while innovation is associated with a shorter assessment time. Conclusion: The study describes the time spent in each phase of the assessment and the appraisal process and demonstrates that uncertainty represents the main driver for the increment in the overall time.