OBJECTIVES: To estimate effectiveness of enzyme replacement therapy (ERT) in the treatment of symptomatic Gaucher's disease in the Ural's cohort  in Russia. METHODS: Information sources were the databases of regional registries in 3 big towns of Ural administration District of Russian Federation: Ekaterinburg, Perm, Chelyabinsk, updated January 2009. Most of the parameters were derived from the published literature. Doses of Cerezymeare varies from 1200un. till 2000un. ERT was assumed to restore patients to full health in the base case. Regional cohort include 21 patients, middle age 23 years old. Quality of life was tested by Short Form 36 (SF-36).  RESULTS:  Seventy percent of the patients were diagnosed in childhood  and have 1 type Gaucher Disease. The clinic characteristics were typical. Bone abnormalities were the most serious. Data on this topic were obtained from the primary medical sources.  The mean cost of the disease calculated about €120,000. The cost per patient varied considerably by dose.  Nonetheless , quality of life indicate that patients treated with ERT continue to have reduced health-related quality of life 0.5 compared with the general population 0.75. And as much more  younger seriously sick patients started ERT-quality of life index could be higher. The social efficacy of ERT equivalent extra number of life years, in this cohort it’s about 13.7 years.Economic evaluations were found, all of which calculated a very high cost per quality-adjusted life-year (QALY). Incremental cost per QALY incremental cost-effectiveness ratio (ICER) in the base case €210,000. CONCLUSIONS:  Although ERT for treating the 'average' Gaucher's disease patient supported by the national program for orphan diseases, despite of it great expense. However, although doing so will be of clinical interest, it is questionable whether, within the current pricing environment, such research would have any substantive impact on policy decisions.