Disease landscape
SMA
Severe to moderate restriction in access to SMA diagnosis and treatment has been identified in the Visegrád (V4) countries as measured in the GAP score. Results vary greatly from country to country with the difference between the highest and the lowest scoring country of about 43 points. The main reasons for relatively low V4 performance in some states are no or limited access to SMA screening program, varied access to registered drugs due to lack of reimbursement or major reimbursement restrictions, and the very low proportion of patients treated with gene therapy in most states in 2021.
Cross-Country
General GAP for SMA
Compliance with guidelines
Positive recommendations for 3 SMA drugs were found in clinical practice guidelines. At least two of them are available to SMA patients in all V4 countries, the respective scores are then 67–100.
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 (2021)
*Overall 33 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 more than 1.7 years. The difference between the shortest and the longest mean time to reimbursement among the countries is 1.3 years.
- Gene therapy’s reimbursement was just implemented in 2021-2022 in all V4 states but Czechia and the percentage of target population treated with this therapy was very low: 0%-37%. Czechia however, which commenced reimbursement in 2020, achieved a score of 100% of target population patients treated with gene therapy in 2021.
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).