Pharmacoeconomic analysis of taliglucerase alfa used in the russian federation for gaucher’s disease

Objective. To determine the optimal enzyme replacement therapy (ERT) regarding clinical and economic analysis and to evaluate the possible budget savings after widespread introduction of taliglucerase alfa into clinical practice for the treatment of Gaucher’s disease. Material and methods. Pharmacoeconomic analysis was carried out using cost minimization, since taliglucerase alfa, imiglucerase and velaglucerase alfa are characterized by similar efficacy. In analysis of the impact on the budget, current clinical practice (taliglucerase is not used) was compared with simulated clinical practice implying administration of taliglucerase in 40% of patients in the first year, 60% in the second year and 80% in the third year. Initial budget of high-cost nosology program, intended to ensure patients with Gaucher’s disease with ERT, was estimated considering the volume of drugs purchased in 2019. Only drug costs were considered. Analysis implied 2 variants of prices for the compared drugs (maximum selling prices of manufacturers including VAT 10% and weighted average prices according to state tenders in 2019). At the final stage, we assessed sensitivity regarding price changes for the compared drugs, single dose of drugs and drug consumption volume. Results. In case of government procurement price, annual cost of therapy with taliglucerase alfa in adults weighing 70 kg is lower by 23-28% compared to other drugs, in children weighing 35 kg-lower by 26-32%. Thus, fixed budget of the modern high-cost nosology program, sufficient to ensure 7 patients with ERT, may be applied for the therapy with taliglucerase alfa in 10 patients in an equivalent dose. In case of maximum retail price with VAT 10%, cost reduction achieved 23-41% in adults and 26-43% in children. Administration of taliglucerase alfa (40% of patients in the first year, 60% in the second year and 80% in the third year) will result total budget savings near 770.6 million rubles (12.26%) over 3 years in case of state procurement prices and 932.0 million rubles (13.82%) in case of maximum selling price with VAT 10%. Analysis of sensitivity has demonstrated stability of these results against changes in input parameters over a wide range. Conclusion. Administration of taliglucerase alfa for ERT in patients with Gaucher’s disease instead of the modern drugs is expedient. Taliglucerase alfa is characterized by similar efficacy and safety and significantly reduce ERT cost. © I.N. DYAKOV, S.K. ZYRYANOV.