Presentation (CTI)

OBJECTIVES: The introduction of a dedicated reimbursement pathway for orphan medicinal products (OMP) in January 2022 represented a major policy shift in the Czech Republic. This analysis examines how OMP submissions have been assessed since the legislative changes, with a focus on administrative timelines and pharmacoeconomic evidence.
METHODS: All applications assessed by the national HTA body under Section 39da of the Public Health Insurance Act between 1 January 2022 and 31 December 2025 were reviewed. Only cases with a published HTA assessment report and reimbursement decision report were included. Descriptive statistical methods were applied for data analysis.
RESULTS: A total of 39 proceedings were identified, of which 27 resulted in a positive decision. Neurological indications were the most common (23.1%). On average, 1,145 days elapsed between EMA authorisation and submission to the Czech payer. The mean time from submission to the first evaluation report and final decision was 141 days and 349 days, respectively. Among submissions with positive decisions, 61 CUAs and 1 CMA were identified. Comparators were primarily active treatments (79.0%) versus best supportive care (21.0%). Most analyses (93.5%) used a lifetime horizon. The mean incremental gain was 2.87 QALYs per patient; caregiver QALYs were included in 15 cases, with a mean gain of 3.08 QALYs. The base-case ICER averaged €110,085/QALY, while the CMA demonstrated cost savings. A societal perspective was applied in 45 CUAs. All positively decided submissions included budget impact analyses with a 5-year horizon. By year 5, in total 3,278 new patients were projected to be treated, with a mean net budget impact of €5 million per technology.
CONCLUSIONS: Overall, OMPs were associated with substantial improvements in quality of life for both patients and caregivers, typically evaluated over a lifetime horizon. Accounting for broader societal perspectives generally reduced both ICERs and net budget impact.