Cost of Enzyme Replacement Therapy for Mucopolysaccharidoses in Iran

Background The mucopolysaccharidoses are a group of inherited disorders caused by lack or insufficient activity of lysosomal enzymes, Enzyme replacement therapy (ERT) has proven to reduce symptoms of these disorders. ERT has become a challenge for health systems due to their expensive medicines. In this study, we aimed to examine the average annual cost of medicines used for ERT per patient. MethodsMedical consumption data was consolidated from different sources. After cleaning data, MPS patients were categorized into five age groups. The number of vials was extracted from Iran FDA database. The weighted average of per capita consumption and cost for MPS patients was calculated and compared against the Gross Domestic Production (GDP) per capita.Results The number of patients with MPS I, IV and VI was 50, 75 and 60. The average annual expenditure on ERT medications per patient was estimated at 206,070 USD. MPS IV patients had the highest rate of medicine consumption (An average of 333.15 vials). MPS VI medications despite having a lower share in medicine consumption, are responsible for over 240 thousand USD annually per capita. The average annual cost has shown to be about 38 times the current GDP per capita.ConclusionThe cost of ERT for MPS disease is substantial to the health system. In order to ensure the best clinical effectiveness and efficient use of financial resources, it is highly recommended that health care policymakers use an evidence-based clinical practice guideline as well as improve the data quality in rare diseases registry.