OBJECTIVES:

As of January 2022, a new unique pathway for reimbursement of orphan drugs was opened in the Czech Republic. After one year, an evaluation of its effects is in order. We therefore set out to:

• Determine the influence of inclusion of governmental and societal perspectives on the results of health technology assessment of orphan drugs in the Czech Republic.
• Estimate the total number of patients who are expected to be treated with the orphan drugs.
• Estimate the expected cost of the orphan drugs in the context of the total expenditure of the Czech healthcare system on drugs.

METHODS:

Public documentation of 100% administrative proceedings for the reimbursement of orphan drugs initiated in 2022 was reviewed. Most up-to-date information was collected for each intervention and indication, where available:

• ICER and net budget impact from healthcare, governmental and societal perspectives,
• estimated costs of the orphan drugs,
• number of patients expected to be treated

Data was described statistically and contrasted with public information on total drug-related expenditures of the Czech healthcare system.

RESULTS:

Across all evaluable proceedings (N=19), ICER from governmental perspective was lower than ICER from the payers' perspective by 3.1% and ICER from societal perspective was lower by 22.8%.

Total of 1,600 - 2,300 patients are expected to be treated by these orphan drugs each year.

Total expenditures for these orphan drugs represent 10% of the projected drug expenditures of the whole Czech healthcare system five years from now.

CONCLUSIONS:

The new pathway towards reimbursement for orphan drugs in the Czech Republic was utilized in 19 cases in the first year. Generally, inclusion of governmental and societal perspective resulted in lower ICER, and budget impact thus increased the value of these drugs. Expenditures for these orphan drugs are expected to account for 10% of the overall spending of the Czech healthcare system on drugs.