PSY51 - CLINICAL AND ECONOMIC ASPECTS OF ENZYME REPLACEMENT THERAPY IN RUSSIAN PATIENTS WITH GAUCHER DISEASE TYPE 1

Abstract

To evaluate the pharmacoeconomic effectiveness of drugs for enzyme replacement therapy (ERT), i.e. imiglucerase, velaglucerase alpha and taliglucerase alfa, in adults with Gaucher disease Type 1 (GD1), who receive treatment in conditions of the Russian health care system. The analysis of the results of clinical trials revealed no statistically significant advantages of any of the considered drug for ERT. Therefore, cost-minimization analysis (CMA) was conducted. The budget impact model considered three scenarios of enzyme replacement therapy of Gaucher disease Type 1 in adults on the stage of stable disease development (imiglucerase, velaglucerase alpha and taliglucerase alfa). The costs of original ERT drugs were calculated from the maximum manufacturer's prices indicated in the list of vital and essential medicines of 1 March 2018. The accepted dosing regimen in ERT was 60 U/kg of body mass (4 200 U). The cost of one administration was 508,200 RUB for taliglucerase alfa, 1,017,014.44 RUB for velaglucerase alpha, and 1,025,530.71 RUB for imiglucerase. The considered drugs were administered once per two weeks, so the costs of one-year course of treatment were 12,196,800.00 RUB, 24,408,346.51 RUB and 24,612,737.00 RUB per patient, respectively. Therefore, the cost of ERT in patients with Gaucher disease in taliglucerase alfa group was two times lower compared to the alternative drugs. It is possible to say that taliglucerase alfa is efficient from the pharmacoeconomic point of view. The introduction of a treatment schedule with taliglucerase alfa can lead to 50% decrease of the cost of ERT in adult patients with Gaucher disease in conditions of the Russian health care system