Azita Nabizadeh - Department of Pharmacoeconomics and Pharmaceutical Administration, Faculty of Pharmacy, Tehran University of Medical Sciences, Tehran, Iran
Mehrnaz Kheirandish - Department for Assessment and Control of Prescribing and Use of Medicines, Iran Food and Drug Administration
Zahra Gharibnaseri - National Institute for Health Research, Tehran University of Medical Sciences, Tehran, Iran
Naghmeh oloomi - pharmacoeconomic department, pharmacy faculty, Tehran University of medical science
Fatemeh Azizi - Islamic University
Najmeh Moradi - Health Management and Economics Research Center, Iran University of Medical Sciences, Tehran, Iran.

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 (۵-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 ۱۸۵ patients with MPS was analyzed. The frequency of patients with MPS I, IV, and VI was ۲۷, ۴۰.۶, and ۳۲.۴%, respectively. The average annual expenditure on ERT medications per patient was estimated at ۲۰۶.۰۷ United States Dollars. The average annual cost has shown to be about ۳۸ 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.

Cost of Care, Healthcare System Expenditure, Enzyme replacement therapy, Iran, MPS