Addressing Legislative and Financial Hurdles in Rare Disease Medicines Accessibility: Insight From Czechia With a Focus on the Pediatric Population

OBJECTIVES: Advanced therapy medicinal products (ATMP) and orphan drugs offer crucial treatment options for rare diseases, particularly pediatric patients. However, the current system for financing and reimbursing these medicines in Czechia is often unsustainable and needs more transparency. This study maps the legislative framework for rare disease medicines reimbursement, highlights legislative grey zones, and addresses the challenges, advocating for necessary reforms.

METHODS: A qualitative analysis of Czech legislative documents related to ATMP and orphan drugs reimbursement was conducted, focusing on critical section §16 (Act No 48/1997 Coll.: On Public Health Insurance), which pertains to reimbursement in exceptional cases. Data were gathered from University Hospital Brno on the number of applications submitted under this section, approval rates, and financial expenditures on rare disease treatments. Legislative differences were also examined for mass-produced and individually prepared medicinal products applying for reimbursement conditions.

RESULTS: The legislative framework has significant gaps, particularly regarding the impractical requirement for standard cost-effectiveness analysis (CEA) for ATMPs in outpatient care. Data from University Hospital Brno show a marked increase in applications and approvals for rare disease medicines under §16, bypassing the standard reimbursement system. Positive reimbursement decisions for adult patients increased from 1 in 2017 to 105 in 2021, for pediatric patients from 19 to 98, and specifically for pediatric oncology patients from 1 to 22 cases. Over 98 % of all applications, numbering in the thousands, are approved, with 99 % approval for pediatric cases. The overuse of §16 reduces pressure on manufacturers to advocate for legislative changes to ensure predictability and transparency in reimbursement decisions.

CONCLUSIONS: The current rare disease medicines reimbursement system in Czechia is inadequate, especially for pediatric patients. Legislative reforms are needed to create a sustainable and transparent system that ensures equitable access to ATMPs and orphans and promotes fair medicine practices.