Introduction Gaucher disease (GD) is an inherited recessive enzyme deficiency with a multisystem condition. The Iranian government covers the therapeutic expenditure of GD patients as it is not affordable for the patients. The aim of this study is to identify the main components of the cost of care in Gaucher patients (GPs) in Iran. Methods The Gaucher patients were identified from the Iran Food and Drug Administration (IFDA) national registry database. The direct medical costs, including medication, diagnostic services, and physician visits were considered. The prices of therapeutic and diagnostic services were extracted from Iranian medical tariff book 2014-15. Iran Food and Drug Administration determined the cost of medications. Results 164 Gaucher patients have been registered in Iran. A valid and reliable diagnostic tests are not used to identify the type of GD. The average health care cost per annum was 20,758 USD per patient, which is higher than 4 GDP per capita in Iran. Medication cost constitutes 95.2% of the total cost. The average cost of each GP was $1,473,818 in his/her total life. Conclusion GD is amongst the high-cost diseases and should be managed effectively. The application of oral medication for eligible GPs could improve allocative efficiency in GD management significantly. A sound, valid and reliable national clinical guideline could improve the efficiency of healthcare resources effectively. Selecting appropriate strategies for reducing the birth of a child with Gaucher, could support allocative efficiency of the limited resources effectively.

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Gaucher's disease (GD) is one of the most common lysosomal diseases in humans. It results from β-glucosidase deficiency and leads to necrosis, especially in macrophages with the accumulation of glucosylceramidase in cells. Most of the deleterious effects of the disease are seen in the liver, spleen, and bone marrow. The aim of this study was to compare the efficacy of Imiglucerase with Eliglustat in treating patients with GD. PubMed/Medline, Cochrane Library, Scopus, Web of Science, Embase, and Google Scholar were searched from inception to August, 2018. Predefined inclusion criteria for included studies were based on search methodology and are as follows: All randomized, quasi-randomized controlled, and cohort studies about patients with GD Type 1 that Imiglucerase was compared with Eliglustat were included. Two authors independently choose the papers based on the inclusion criteria. From 2979 recognized studies, three studies including two randomized clinical trials and one cohort study were recognized to meet the inclusion criteria. The primary outcomes were hemoglobin level, platelets count, liver, and spleen size, and the secondary outcomes were the immunological side effects of the medicines and bone complications. The results showed that there is no meaningful difference between the two medicines in terms of increasing blood hemoglobin, platelets count, and reducing the liver and spleen size. The findings of this review showed that both medicines are effective in the treatment of GD Type 1 and there is no statistically significant difference between their efficacies.

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