Social-economic burden of Pompe disease with late onset in the Russian Federation

Abstract

Pompe disease (PD) — a rare disease with progressive muscles weakness, cardiac and pulmonary insufficiency and others vital life-threatening symptoms without enzyme replacement therapy (ERT). Treatment of PD is very expensive and data about social-economic burden of PD in the Russian Federation is absent yet. Analysis of direct costs (cost of ERT, laboratory and others tests, cost of medical devisees etc.) and non-direct costs (personal income losses due to temporary or permanent disability or necessity of care for a sick child, GDP losses etc.) have been calculated for PD with late onset. Direct medical cost in case of alglucosidase alfa use was less on 11.4 % vs standard therapy for PD onset in childhood as well as non-direct non-medical cost on above 40 % less for one year horizon of modelling. Alglucosidase alfa has high efficacy for PD treatment for patients with late onset treatment in adult age, but more costly vs standard therapy — 44.1 mln. RUR / patient / year. Expenditures for PD complications were in 2 times less in case of alglucosidase alfa usage. Cornerstone of expenditures in alglucosidase alfa group are cost of ERT, in the same time in standard treatment group — cost of PD complications, treatment of terminal phase and death. ERT could be extended to 16.4 % due to resource-saving effects of the use of alglucosidase alfa. Calculated socio-economic burden of PD has shown that, it seems appropriate to transfer funding for its treatment to the Federal level.