OBJECTIVES: Advanced therapies (ATMPs) are increasingly approaching the European markets. This analysis aims to have an early assessment of the Italian HTA experience evaluating the rate of approval, the time to reimbursement, the level of value recognition (innovative status) and payment and reimbursement methods.

METHODS: Different available sources as PubMed, EMA/AIFA website and public reports were investigated for data analysis.

RESULTS: At 30^th June 2020, 9 therapies were approved at EMA level and just 5 (Holoclar^®, Strimvelis^®, Alofisel^®, Kimriah^® and Yescarta^®) entered the Italian market (one tissue therapy, one gene therapy, 3 cell therapies). The EMA time approval of ATMPs is about 12,5 months (from 10 to 17 months) while time to reimbursement in Italy varies from 4 months to 2 years (mean: 1,5 year) among therapies. 3 therapies (Strimvelis^®, Kimriah^® and Yescarta^®) got the innovative status by AIFA (60%). 4 up to 5 were reimbursed via managed entry agreements (2 payment at results and 2 payment by results); treatment costs (ex-factory price) of reimbursed products (Holoclar^®, Strimvelis^®, Kimriah^® and Yescarta^®) are consistent with the european ones. Alofisel^® is in the C class, which means that it is approved but not reimbursed by the Italian NHS.

CONCLUSIONS: Despite few case studies are present in literature, this analysis shows preliminary interesting results about ATMP access in Italy so far. Rate of approvals and variability in time to reimbursement underline the complexity, both from a clinical and economic point of view, of assessing those therapies by AIFA; the uncertainty on efficacy in the long term is mainly managed through performance based agreements but is not impacting the overall value recognition once reimbursement is granted (60% innovative status). Treatment costs needs to be put in the context of rare disease and small eligible patient populations. A specific pathway for advanced therapies is required.