Access to Orphan Medicines in Ireland

OBJECTIVES: To evaluate reimbursement access to orphan designated drugs in Ireland

METHODS: GlobalData collected information on all medicines with marketing authorization approval (MAA) that are listed as orphan drugs (OD) by the EMA. This information was cross-referenced with an analysis of the reimbursement status of ODs with MAA in Ireland, which was obtained through GlobalData’s proprietary pricing and reimbursement database POLI.

RESULTS: Only 39 (25.6%) of the 152 orphan-designated medicines with MAA in the EU-27 were approved for reimbursement in Ireland, as of May 2024. Of the152 identified OD medicines, 30 products (19.7%) held general reimbursement status; 9 (5.9%) were available through managed access protocols (MAPs), 3 (2.0%) were not yet priced/reimbursed in Ireland. Additionally, 3 products (2.0%) had been withdrawn having previously been reimbursed. Average time to reimbursement (TTR) for ODs in Ireland from the date of MAA was 1,370 days (minimum 62, maximum 4,365 days). In comparison, the average TTR for all originator drugs is currently 830 days according to POLI. For MAP ODs - involving a confidential price discount in exchange for limited prescription access on a named-patient basis - the average TTR was 1,968 days (n=8/9). Only 3 ODs gained a first reimbursement approval in Ireland during H1 2024, with an average TTR of 1,474 days (ranging from 478 to 2,018 days). Only six orphan drugs gained reimbursement in 2023 and 8 eight in 2022, with an average TTR of 1,326 days and 1,626 days, respectively.

CONCLUSIONS: Access to ODs in Ireland remains limited. Only a quarter of ODs are reimbursed, and patients wait for reimbursement for an average of 45 months after the date of MAA. A MAP adds an average of 598 days to the OD reimbursement timeline.