OBJECTIVES: Since the Russian Federation has a complex federal structure, drug cover for patients with orphan diseases is handled by various institutional arrangements and is characterized by inequality. The aim of this research is to apprise the effectiveness of the public drug coverage for patients with MPS I, II and VI as one of the most socially important and severe orphan disease funded by regional budget in Russia, and evaluate the expediency of purchase centralization.

METHODS: To evaluate sufficiency of the patient drug supply we have analyzed the data from federal patients’ registry and report of public drug purchase in order to amount of patient receiving enzyme replacement therapy and total expenditure for MPS I, II and VI in 2017. The model (decision tree with the Markov cycle) was developed to evaluate thereal patient demand for this therapy based on the dosage regimen and specific patientcharacteristics. In addition, this model was used to estimate the cost effectiveness of the enzyme replacement therapy (ERT) as a cost for 1 additional life-year gained adjusted for centralization scenario.

RESULTS: Totally 308 669 135.62 € were contributed to ERT for patients with MPS I, II and VI from regional budget. However, the real rate of unmet drug therapy needs is higher than represented in registry – 30.7% versus 24.9% respectively. When centralizing ERT drug purchase, one life-year gained will be cost 203 619.82 €, 492 441.80 € and 866 488.42 € for MPS I, II and VI respectively.

CONCLUSIONS: This study provides feasibility of drug procurement centralization in case of MPS and other rare diseases. Despite of the low cost-effectiveness of ERT this measure will give an opportunity to provide equitable access to medicines for all patients suffering MPS and in addition to get under control purchasing price.