摘要：Background: The mucopolysaccharidoses (MPSs) are a group of rare inherited metabolic disorders caused by lack or insufficient activity of lysosomal enzymes. Although no cure has been identified until now, Enzyme Replacement Therapy (ERT) can reduce symptoms of some types of MPSs. providing ERT for patients is a challenge for health systems due to expensivity of the medicines. In this study, we aimed to examine the average annual cost of medicines used for ERT per patient in Iran healthcare system.Methods: Data on patients were consolidated from different sources of Iran Food and Drug Administration’s (IFDA). After collecting data, patients with MPS I, IV, and VI were categorized into five age groups (5-years interval). The number of vials distributed monthly was used for the estimation of the annual medication cost for each patient and compared against the Gross Domestic Product (GDP) per capita.Results: Data on 185 patients with MPS was analyzed. The frequency of patients with MPS I, IV, and VI was 27, 40.6, and 32.4%, respectively. The average annual expenditure on ERT medications per patient was estimated at 206.07 United States Dollars. The average annual cost has shown to be about 38 times the current GDP per capita of the country.Conclusions: The medication cost of ERT for MPS disease is substantial to the health system. To ensure the best clinical effectiveness and efficient use of financial resources, it is highly recommended that healthcare policymakers use an evidence-based clinical practice guideline and improve the data quality in rare diseases registry.
