Eur J Health Econ. 2026 Jul 21. doi: 10.1007/s10198-026-01956-x. Online ahead of print.
ABSTRACT
OBJECTIVES: This study provides a comprehensive overview of the Italian framework for the appraisal of drug innovativeness, covering all 294 appraisals conducted by AIFA between 2017 and June 2025. It aims to describe the evolution and application of the 2017 criteria, explore the determinants of innovativeness recognition, and discuss the implications of the 2025 revision for value assessment and market access.
METHODS: All AIFA innovativeness reports available up to June 2025 were systematically reviewed and coded across the three appraisal domains – therapeutic need, added therapeutic value, and quality of evidence – together with contextual and regulatory variables (orphan designation, study design, EMA approval type, accelerated assessment, and PRIME designation). Descriptive and multinomial regression analysis were performed to identify predictors of appraisal outcomes and to explore associations with European and national timelines.
RESULTS: Among 294 appraisals, 27% were fully innovative, 29% conditionally innovative, and 44% non-innovative. ATV and quality of evidence were the strongest determinants, while therapeutic need played a secondary role. Orphan designation, pediatric/mixed populations, and the availability of RCT-based evidence increased the likelihood of a positive outcome. Medicines with EMA accelerated assessment were more often granted full innovativeness but did not achieve shorter national reimbursement timelines (median 432 days). Fully innovative medicines followed shorter EMA pathways (median 330 vs. 448 days), though not necessarily implying faster national access.
CONCLUSIONS: AIFA’s framework proved internal coherence, with ATV as the main driver. The 2025 criteria may enhance methodological rigor but reduce flexibility, requiring continuous monitoring to ensure timely and equitable access.
PMID:42479353 | DOI:10.1007/s10198-026-01956-x
