Presentation (CTI)

OBJECTIVES: Italy’s Law 648-96 enables early access (EA) to medicines not yet authorised nationally but that are either approved abroad, under investigation in clinical trials, or used off-label based on supporting phase II clinical data. France’s Early Access Authorisation (AAP) programme similarly allows temporary access to products with or without a marketing authorisation (MA) for patients lacking therapeutic alternatives, until a reimbursement decision is made. This study compares the two frameworks to assess their effectiveness in delivering EA and implications for routine pricing and reimbursement.
METHODS: We analysed EA requests submitted between January 2023 and December 2024 in Italy (n=126) and between January 2023 and June 2024 in France (n=56), using AIFA and HAS databases, respectively. For each request, we assessed product types, access timing (pre- or post-MA), timelines, success rates, and the relationship between EA outcomes and routine reimbursement.
RESULTS: Out of 126 applications in Italy, 30 were on-label and 96 off-label, the latter not being comparable with the approach of AAP. Among on-label requests, 73% were for new active substances and 27% were for indication expansions in both countries. Orphan drugs represented 50% of requests in Italy and 41% in France. The majority of requests were submitted pre-MA (57% in Italy; 59% in France). Average application processing time was longer in Italy than France (163 vs 111 days). EA-rejection rate was higher in Italy than France (67% vs 54%). Products with EA-approved decision reached routine reimbursement more quickly in France compared to Italy (312 vs 498 days). Reimbursement followed for 60% versus 25% of EA-approved, and 67% versus 42% of EA-rejected requests, respectively in Italy and France.
CONCLUSIONS: Italy’s 648-96 framework shows higher rejection rates and longer timelines but more transitions to routine access compared to France’s AAP, highlighting key differences between the two EA programmes.