OBJECTIVES: To understand current and future scenario on ATMPs access in the EU5.

METHODS: ATMPs access and reimbursement status has been tracked up to July 2020 across the top 5 European Countries (EU5). Details were searched on institutional websites of each Country. Upcoming ATMPs were searched on published minutes of the Committee for Advanced Therapies (CAT).

RESULTS: At the time of the analysis (July 2020), 15 ATMPs were granted European marketing authorization (MA) and 4 were under CAT assessment. Regarding the authorised ATMPs, 5 were withdrawn from the market due to commercial reasons (the most recent case was Zalmoxis). ATMPs access and reimbursement was quite heterogeneous across EU5, and only CAR-Ts were reimbursed in all EU5. In Italy, of the 10 ATMPs with active MA, 4 were granted reimbursement (2 CAR-Ts), 2 were under the Italian Medicines Agency (AIFA) assessment, 1 was not reimbursed and the others had not yet submitted the price and reimbursement request. For 1 ATMP under evaluation, AIFA granted early access for single patients, while for the other the EAP request was rejected. Interestingly, an innovative approach was adopted for CAR-Ts, with the definition of a new payment agreement scheme and, for the first time, regional power regarding Centers of Expertise selection was limited by minimum criteria set at national level.

CONCLUSIONS: The arrival of CAR-Ts has initiated a profound change in ATMPs approach, confirming the great clinical expectations on these technologies. Indeed, all EU5 granted CAR-Ts access and reimbursement, despite sustainability issues related to price and organizational impact. In Italy these issues were overcome by introducing novel and ad hoc agreements and strategies. These results suggest the development of a new scenario in which ATMPs will become a well-established therapeutic option for the treatment of high unmet need diseases.