OBJECTIVES: To analyse the number of drugs successfully achieiving National Centre of Phamacoeconomics (NCPE) reimbursement in 2022 with non-orphan drug status compared to orphan drug status, and to identify any impacts achieving orphan drug status might have on reimbursement success.

METHODS: Rapid Review and full Health Technology Appraisal (HTA) outcomes were reviewed based on the decision date and whether full or partial reimbursement was achieved. Results were tabulated and descriptive statistics were compiled. The list of authorised pharmaceuticals with orphan designations was obtained from the European Medicines Agency (EMA) website, while information regarding reimbursement status was retrieved from the NCPE website.

RESULTS:

A total of 107 submissions (78 non-orphan and 29 orphan) were made in Ireland in 2022. The search was refined further by excluding any submissions still in development, which resulted in a final total of 14 orphan and 28 non-orphan submissions.

Of the 14 orphan submissions receiving a final decision, 3 (21%) achieved reimbursement and 11 (79%) did not. However, 2 of the 3 approved orphan submissions received a positive Rapid Review opinion and did not progress to full HTA. Of the 28 non-orphan submissions receiving a final decision, 9 (32%) achieved reimbursement and 19 (68%) did not. 5 of the 9 approved non-orphan drugs (received a positive Rapid Review opinion.

CONCLUSIONS: In Ireland in 2022, orphan drugs were approved for reimbursement at a lower rate than non-Orphan drugs; the Rapid Review process had a negligible impact on whether an orphan drug was reimbursed. While there could be numerous causes for this, it should be noted that the Irish reimbursement framework does not contain a process specifically designed to improve access to orphan drugs. As such, patients suffering from rare diseases in Ireland may experience setbacks as a consequence of the adoption of a ‘one-size-fits-all’ strategy.