OBJECTIVES: Policymakers in different countries consider various aspects of orphan drug reimbursement. In Central and Eastern European (CEE) countries, some common characteristics can be seen when it comes to strategies for drug policy. We decided to investigate which aspects of the clinical data from orphan drugs might affect drug reimbursement in selected CEE countries.

METHODS: Necessary data were collected within a questionnaire study in a group of experts from nine EU CEE countries between September 2021 and January 2022. Data were categorized, and statistical analysis was performed

RESULTS: The survey was conducted for: Bulgaria, Croatia, Czechia, Estonia, Hungary, Lithuania, Poland, Romania, and Slovakia. Safety and efficacy (effectiveness) of orphan drugs was considered in the reimbursement process in 8 countries and partially considered in 1 country. The impact of orphan drug safety and effectiveness on reimbursement decisions was rated: strong in 56%, moderate in 22%, and low in 22% of countries. Furthermore, the chances of reimbursement of an orphan drug appeared to be reduced by 38% (OR = 0.62) in countries where the level of impact of safety and effectiveness was rated strong compared to other countries (p<0.05). The acceptable safety profile of an orphan drug was a mandatory reimbursement criterion in 44% of countries, but in 56% of countries no separate safety evaluation was required. However, the evaluation of the efficacy of orphan drugs was mandatory in the reimbursement process among 78% of countries, partially mandatory in 11%, and not required in 11%. Additional clinical aspects influenced reimbursement decision in 44% of countries.

CONCLUSIONS: Preliminary findings indicate differences between countries in orphan drug reimbursement policies in the area of clinical aspects. This reveals the complexity and difficulty in evaluating orphan drugs.