Disease landscape
SMA
Minor restriction in access to SMA diagnosis and treatment has been identified in the Visegrád Group (V4) countries as measured in the GAP score. Results vary however from country to country with the difference between the highest and the lowest scoring country of 21 points. The main reasons for adversely affecting the outcome in some states are diagnostic constraints. The low percentage of patients treated with gene therapy in 2023 in Slovakia is noteworthy.
Cross-Country
General GAP for SMA
Compliance with guidelines
Positive recommendations for 3 SMA drugs were found in clinical practice guidelines. All of them are available to SMA patients in all V4 countries.
From registration to reimbursement
Population burden
DALY* - Rate per 100k
Estimated values.
There is no data for the EU and Western Europe average.
*disability adjusted life-years
Target population
Gene therapy
Drug access
Number of patients treated (2023)
Drug access
Population treated (%)
Main issues
- The average time to reimbursement for SMA drugs in the V4 countries is 1.6 years. The difference between the shortest and the longest mean time to reimbursement among the countries is 2.8 years.
- Gene therapy’s reimbursement was implemented in 2020-2022 in all V4 states with varying levels of population coverage in 2023. Gene therapy was administered to all or almost all eligible patients in Poland, the Czech Republic and Hungary, while in Slovakia only less than 20% of the estimated target population received the treatment.
Project
We measured the gap in innovative drug access and diagnostics in selected therapeutic areas in the Visegrád (V4) countries (Poland, Hungary, Czechia and Slovakia).