PRO30 ECONOMIC BURDEN OF POMPE DISEASE IN THE RUSSIAN FEDERATION

Abstract

Pompe disease (Glycogen storage disease type II, PD), is an autosomal recessive metabolic disorder which damages muscle and nerve cells throughout the body. In the Russian Federation above 480 patients have this diagnosis. Economic burden of PD has been calculated based on direct (DC) and non-direct costs (NDC). DCs are included: costs of medical aid for pathogenetic, symptomatic, supported therapy for patients with different age, including treatment of complications; costs of emergency room admission and costs of medical devices. NDCs are included: GDP and other social-economic losses due to temporary disability of one parent due to the care of a sick child, payment for disability patients in adult age etc. DC for all PD’s patients, including children and adults were $150 mln in 2017, and more than 50% of expenditures were in severe forms of PD. Standard symptomatic treatment average cost for one child was $300 thousand annually, and in the same time new approach with enzyme therapy reduced expenditures on 11,3%. NDC were on 40% less for enzyme therapy group. DC for one adult patient was $613 thousand annually, and trend for decrease of expenditures was in enzyme therapy group on 35,9% due to slowing the progression of complications, and NDC was on 42,2% less as well. Expenditures for medical devices were higher in standard symptomatic treatment group. Dynamic of NDC during 3 years was positive, and reached 9,7%. Sensitivity analysis has confirmed data received. PD has a social-economic burden in the Russian Federation and expenditures can be less in case of modern treatment technologies usage, which should prevent progressive of illness, and it’s severe complications.