OBJECTIVES : In Ireland, the National Centre for Pharmacoeconomics (NCPE) examines the cost effectiveness of all new drugs for which reimbursement by the health payer is sought. Applicant companies make submissions to the NCPE for evaluation. We aim to determine the proportion of all drugs with European Medicines Agency (EMA) designated orphan status and marketing authorization (MA) that are submitted to the NCPE. We aim to evaluate the time from MA to submission to the NCPE.

METHODS : This was a retrospective review of publicly available websites. The number of drugs that had received EMA orphan designation (January 2009 to February 2019 inclusive) and subsequent MA was established. The percentage of these drugs that were subsequently subject to a NCPE submission was determined. Timelines for submission to the NCPE were examined. Outcomes were analyzed as a function of year of orphan designation and indication (‘cancer’ vs. ‘non-cancer’). Analyses were performed on Microsoft Excel and IBM SPSS Statistics (version 24.0).

RESULTS : In total, 83 eligible drugs (i.e. held both EMA orphan designation and MA) were identified. Of these, 51 (61.4%) had been submitted to the NCPE. The mean time from MA to NCPE submission for all drugs was 21.1 months. The mean time ranged from 1 month to 22.1 months depending on the year of orphan designation. Those drugs for cancer were more likely to be submitted to the NCPE (vs. drugs for other indications); p=0.008; Chi-squared test.

CONCLUSIONS : Not all drugs with EMA designated orphan status and MA are subject to a NCPE submission. As such, reimbursement is not sought in all instances. Timelines for submission to the NCPE vary depending on year of orphan designation. Drugs for cancer are more likely to be submitted to the NCPE (versus drugs for other indications).