This article presents the results of a retrospective analysis of data from the Ukrainian Patient Registry of the “Children with spinal muscular atrophy” Foundation, based on an active sample of 339 individuals as of 2025. The study revealed profound therapeutic inequality: the high coverage rate for patients with Type I spinal muscular atrophy (89.2 %) contrasts sharply with the critically low coverage of the adult cohort (58.7 %). Through the lens of the international “4A” model (Availability, Accessibility, Affordability, Acceptability) and the principles of ValueBased Healthcare, systemic barriers to access were identified, caused by outdated regulatory restrictions regarding genotype, age, and the number of copies of the SMN2 gene.

It was found that 44.6 % of treated patients are in a state of “therapeutic migration,” receiving care abroad due to the national system’s inability to ensure domestic access to medications (Health System Failure). The authors justify the strategic importance of real-world evidence as a tool for data-driven policy making to transition from restrictive protocols to innovative financing models, particularly risk-sharing agreements. It has been demonstrated that eliminating discriminatory barriers and ensuring access to disease-modifying therapy for all categories of patients, including adults and individuals with “Walkers” status, is critically necessary for preserving Ukraine’s human and intellectual capital in the context of the military crisis, as well as for patient care in general.