Background and Importance Advanced therapy medicinal products (ATMPs) represent the forefront of healthcare innovation. Despite the approval of the first ATMP in Italy in 2016, there is currently a lack of scientific evidence concerning the utilisation patterns of ATMPs.

Aim and Objectives Study aim was to evaluate the drug utilisation patterns among patients receiving ATMP treatments in Italy.

Material and Methods Retrospective study using data sourced from the Monitoring Registries of the Italian Medicine Agency, specifically the Drug Product Registry (DPR) containing information on dispensed treatments and clinical data for patients utilising ATMPs in Campania Region ( ~6 million, 10% of the national population) and residents treated in a different Italian Region. Final cohort included individuals who received at least one prescription for ATMP drugs in the Italian market between 2016 and 2023. We analysed prescription patterns focusing on the index treatment, diagnoses, treatment interruptions, mortality rates and adverse events.

Results In total, 92 patients initiated ATMP treatments between 1 January 2016 and 1 September 2023. 21.6% received voretigene neparvovec, 25% onasemnogene abeparvovec, 22.8% tisagenlecleucel and 21.7% axicabtagene ciloleucel. The overall occurrence of adverse events was low (1.1%), primarily associated with autologous human corneal epithelial cells treatments. The overall mortality rate was 12%, affecting only two drugs: 28.6% tisagenlecleucel and 25.0% axicabtagene ciloleucel. Notably, nearly 90% of subjects completed their treatment without experiencing adverse events or mortality.

Conclusion and Relevance This study highlights the low occurrence of adverse events and mortality associated with ATMPs, emphasising their potential as a promising frontier for treating severe diseases lacking therapeutic alternatives in real-world scenarios.

Conflict of Interest No conflict of interest.