Disease landscape
SMA
Relatively 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 from country to country with the difference between the highest and the lowest scoring country of about 23 points. The main reasons for adversely affecting the outcome in some states are diagnostic constraints and relatively low proportion of patients treated with gene therapy in some states in 2022.
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 (2022)
*Overall 17 patients were treated with gene therapy, including those diagnosed in previous years
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 2022: 24% and 36% of target population was treated in Poland and Slovakia whereas 100% of target population received the drug in Hungary and Czechia.
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).