Clinical and economic evaluation of treatment for lysosomal acid lipase deficiency

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Clinical and economic evaluation of sebelipase alfa in the Russian patients with lysosomal acid lipase deficiency (LALD)

Material and methods

We conducted the cost of illness analysis and cost-effectiveness analysis for patients with cholesteryl ester storage disease (CESD) and Wolman disease (WD), respectively. Treatment with sebelipase alpha was compared with the standard symptomatic treatment (SST). The analysis included direct and indirect costs.


Without pathogenetic treatment, the cost of treatment of one patient with CESD per year is 5,239,277 rubles, and the total cost of treatment of all patients with LALD registered in Russia (n = 38) – 199 092 526) is 199,092,526 rubles per year. Over a 1-year modeling horizon for WD, the total costs of treatment with sebelipase alpha (9,040,8) – 199 092 52679 rubles) were significantly higher than those of SST (4,625,8) – 199 092 52610 rubles). In the sebelipase alpha group, the indirect costs and costs in the terminal stage of the disease were lower than in the SST group. Compared to SST strategy, treatment with sebelipase alpha was both more effective and more expensive strategy. The cost-effectiveness ratio was lower for the sebelipase strategy.


Annual expenditures from the health budget for the management of patients with LALD using SST may reach 199 million rubles per year. The use of sebelipase alpha was more cost-effective strategy than SST.

Key words

Lysosomal acid lipase deficiency, Wolman disease, cholesterol ester storage disease, sebelipase alpha, cost-effectiveness analysis, cost of illness analysis.