The Italian assessment system for innovative medicines. An 8-year retrospective analysis of outcomes, criteria, timelines and funding dynamics

Background: The Italian assessment system for innovative medicines represents a distinctive European regulatory approach. Integrated with the Italian Medicines Agency (AIFA), since 2017 it has provided a formal evaluation based on three predefined criteria, Therapeutic Need (TN), Added Therapeutic Value (ATV) and Quality of Evidence (QoE), granting access to dedicated funds and simplified procedures. Objective: This study investigated the evolution of the Italian system from 2017 to the 2025 reform, focusing on outcomes, decision criteria, timelines, and financial implications. Methods: Publicly available AIFA data on 266 assessments were analysed and stratified by outcome, therapeutic area, orphan status and Advanced Therapy Medicinal Product (ATMP) designation. Logistic regression models were used to assess the internal consistency of appraisal criteria and to explore whether regulatory attributes modified their association with innovativeness outcomes. Time to recognition (TIR) and Decision-to-Implementation Interval (DII) were calculated for “Full Innovative” medicines. Annual spending was compared to fund availability to assess the financial impact of innovative medicines. Results: Among the 266 evaluations, approximately 29% were classified as “Full Innovative”, 29% “Conditionally Innovative” and 42% “Not Innovative”. Added Therapeutic Value and QoE showed the strongest associations with positive designations, while orphan and ATMP status showed no independent association. Access timelines were comparable across therapeutic areas, with slightly longer delays for ATMPs. Spending exceeded fund limits several times but fell below budget after fund unification in 2022. Conclusions: The Italian system has been applied consistently over time, with appraisal outcomes largely reflecting the formal decision criteria rather than regulatory status. The 2025 reform may represent a turning point, potentially changing designation patterns and their determinants. Ongoing monitoring is needed to ensure access does not compromise evidence standards or sustainability, offering lessons that may be relevant to other health systems considering similar mechanisms.