Beyond Reimbursement Status: Availability of Advanced Therapy Medicinal Products Across the European Union.

Abstract

Background: Advanced Therapy Medicinal Products (ATMPs) represent an innovative therapeutic approach with the potential to impact the treatment of rare diseases significantly. Although authorised centrally in the European Union, their market launch differs across Member States (MS). This study aimed to describe the ATMP market availability in MS and explore potential influencing factors, providing insights into specific barriers beyond pricing and reimbursement policies.

Methods: ATMP availability was defined as the product launch in each MS. Data was collected through open governmental sources, databases, and communication with national competent authorities. Spearman's correlation coefficients were calculated to examine the relationship between ATMP availability and their characteristics (time since granting marketing authorisation, target patient population size, and cost).

Results: We collected the availability data on 18 ATMPs from 23 EU MS. Market uptake varied significantly, with Germany (89%), France and Italy (61%) leading. Estonia and Latvia confirmed that no ATMP has been launched on their markets yet. Six ATMPs were available in more than one-third of the analysed MS. No significant correlation was observed between ATMP availability and analysed product characteristics except for time dependency for CAR T-cell therapies.

Conclusion: Beyond pricing and reimbursement processes, the ATMP commercialisation in particular MS is influenced by the marketing authorisation holder's decision and capacity. ATMPs face product-specific challenges in achieving EU-wide availability, including complex manufacturing, distribution, and administration processes. To increase the accessibility of innovative ATMP-based treatments, implementing the cross-border access framework or individual ATMP production under the hospital exemption is essential, especially in underserved MS.