Presentation (CTI)

OBJECTIVES: To assess if access to orphan-designated therapies has improved in the Czech Republic as a result of the dedicated orphan drug reimbursement mechanism introduced under Section 39da of the Public Health Insurance Act in 2022, which includes rare disease patients’ organizations in the decision-making process.
METHODS: A comprehensive survey of the reimbursement procedures conducted under Section 39da between January 1, 2022, and June 25, 2025 was carried out, as well as a survey of all the available information in the public domain on the orphan-drug reimbursement mechanism.
RESULTS: The new mechanism has led to a significant increase in the number of applications for reimbursement for orphan-designated therapies in the Czech Republic. While in 2019-2021 there were just 36 applications, in 2022-2024, there were 66 applications, the vast majority for Section 39da procedures. Out of 35 completed Section 39da procedures, 24 (68.6%) ended in positive reimbursement decisions and 22 of the assessed orphan-designated therapies (62.9%) have been included in the reimbursement list; two therapies obtained reimbursement through other pathways. In the case of 31 out of the 35 Section 39da procedures (88.6%), the Advisory Body (AB) and the MoH were aligned in their recommendations and decisions - in 24 cases (68.6%) both made positive recommendations and decisions, and in seven cases (20%), both made negative ones. For four procedures (11.4%), their opinions diverged, with the AB giving a positive recommendation and the MoH giving a negative decision.
CONCLUSIONS: The introduction of the orphan-drug specific reimbursement mechanism under Section 39da has had an overall positive impact on access to new orphan-designated therapies, as well as to orphan-designated therapies that have been available for some years already, but only on the basis of Section 19 exceptions, through individual requests to health insurance companies.