Ukraine

There is no national rare disease strategy and the national health reform agenda does not have a designated rare diseases section.

• There is no national RD strategy in Ukraine
• The unavailability of enough patient data has prevented the assessment of needed actions as well as the expenditure for a national strategy on RDs
• An update to Ukrainian legislation in 2015 defined the notion of a "rare (orphan) disease" within the law and a number of legal provisions aimed at the prevention of these diseases were included, e. g. clause 53 regarding coverage for orphan diseases but no specific national strategy exists

In February 2020, CSMA reported a total of 267 Ukrainian SMA patients in the national CSMA registry which records epidemiological and clinical history data.

Since 2004, CSMA has implemented a patient registry for children with SMA supported by the Institute of Neurology, Psychiatry, and Narcology of the National Academy of Medical Sciences in Ukraine. The MoH do not currently recognise or use the information in this registry despite its availability. Since 2018, an expanded core dataset is collected, this includes personal, diagnosis, treatment, and outcomes data including validated motor outcome measures and PROs. The registry has been harmonised with other European registries through INDRA and TREAT-NMD but is not publicly available with the last published status update in February 2020. Since 2004, there have been 429 patients registered from Ukraine and neighbouring countries without their own registries since 2004. In February 2020, there were a total of 267 alive Ukrainian SMA patients registered.

CSMA have a political mandate but policymakers do not always recognise or consider activities. They also support the maintenance of the national SMA registry.

Treatment guidelines are currently based on the 2017 TREAT-NMD international standards of care.

Studies of prevalence of SMA are outdated, based on a small sample size or focused on all neuromuscular diseases.

Early access programs are not available for reimbursement in Ukraine.

• There is non-reimbursed access on the basis of a compassionate use programme.
• There is no specialized pathway for orphan drugs.