Economic analysis of enzyme replacement therapy for Fabry disease

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Fabry disease (FD) is included into the list of life-threatening and chronic progressive rare (orphan) diseases that reduce life expectancy and induce disability of patients. Russian government provides treatment for patients with the diseases included in this list. In Russia, agalsidase beta and agalsidase alpha are approved for treatment of patients with FD.


To evaluate economic impact of agalsidase beta and agalsidase alpha administration on the Russian healthcare system.

Material and methods

We conducted cost-minimization and budget impact analyses. Only the costs of enzyme replacement therapy of FD were taken into account.


Costs of enzyme replacement therapy with agalsidase beta per patient per year were 1.85 million Rubles (13.5%) lower compared with agalsidase alpha. The expansion of agalsidase beta into the entire population of patients with FD older than 8 years will result in regional healthcare budget savings of 112.2 million Rubles (8.9%). The centralized procurement of agalsidase beta will result in an additional reduction in the budget burden by 121.4 million Rubles (10.6%).


The use of agalsidase beta compared to agalsidase alfa in patients with FD is economically feasible and will be associated with saving for the budget.

Key words

Fabry disease, agalsidase beta, agalsidase alfa, cost-minimization analysis, budget impact analysis.