OBJECTIVES: Specific reimbursement policy for Orphan medicinal products (OMPs) was adopted in January 2022. The aim of this study was to assess OMPs reimbursement process after the legislation change with regards to administrative proceedings and pharmacoeconomic evaluations.

METHODS: Applications submitted and evaluated by the HTA authority according to Section 39da of the Act on Public Health Insurance between 1 January 2022 and 23 May 2024 were identified. Applications after publication of decision report from the HTA authority were included in the analysis. Descriptive statistics were used for data analysis.

RESULTS: 15 administrative proceedings were identified in the defined period. OMPs most frequently targeted neurological diseases (33.3%). Mean time since EMA registration until the submission was 1,189 days. Mean time until the first evaluation report and decision since the submission was 141 days and 330 days, respectively.

In total, 30 CUAs and 1 CMA were analysed. The comparators were BSC (51.6%) or active therapy (48.4%). Lifetime horizon was adopted in 90.3% of analyses. Mean patient increment of QALY was 3.07. Caregiver’s QALY was reported in 9 analyses, mean QALY increment was 2.15. Mean reported base case ICER was 279 853 EUR/QALY. CMA was cost-saving. Total of 25 CUAs from societal perspective were reported, with mean ICER change of ‑8.70%.

Overall, 15 BIAs were presented. Altogether, 1908 patients have been assumed to be treated in the 5th year after reimbursement decision. Considering the maximal patient potential, mean net BIA was 5.3 million EUR. BIA from societal perspective was reported in 9 cases, mean 5^th year net BIA change was -8.86%.

CONCLUSIONS: Treatment with OMPs generated considerable benefit in terms of patients’ and caregivers’ QoL, usually over a lifetime horizon. Applying societal perspective mostly led to reduced ICER and net BIA.