Gaucher disease is among the more costly of chronic diseases to treat in Iran, and efforts are needed to minimize the financial burden facing patients and health authorities in the country, researchers say.
Findings of their study, “Healthcare resource utilization and cost of care for Gaucher patients in Iran,” were published in the Journal of Diabetes & Metabolic Disorders.
Gaucher disease, one of the most common lysosomal disorders, is caused by an abnormal production of the enzyme beta-glucocerebrosidase, which degrades a fatty substance called glucocerebroside, due to a genetic mutation in the GBA gene.
Glucocerebroside accumulates inside immune cells called macrophages, which become Gaucher cells present in the liver, spleen, bone marrow, and nervous system. Patients usually develop anemia, an abnormally large liver and spleen, bone disease, and show delayed growth.
While no medication can prevent or cure the disease, therapies help patients manage their symptoms and have a better quality of life. These include enzyme replacement therapy (ERT) and substrate reduction therapy (SRT), but their cost is beyond the abilities of most people in Iran to meet.
“The Iranian government covers the therapeutic expenditure of GD [Gaucher disease] patients as it is not affordable for [most of] the patients,” the study noted.
Researchers at Tehran University of Medical Sciences evaluated the healthcare resource utilization and medical care costs associated with Gaucher disease in Iran.
They used the Iran Food and Drug Administration (IFDA) national registry database to identify people with Gaucher disease. All medical costs associated with the disease, including medication, diagnostic services, and clinical visits were taken into consideration.
Information about the costs of treatment and diagnostic services were obtained from the Iranian medical tariff book 2014–15, while the cost of medications was provided by the IFDA.
A total of 164 Gaucher patients (85 women and 79 men) were registered in the IFDA database.
According to estimations, each faces an annual healthcare cost of $20,758 (U.S.).
Currently, two types of ERTs are available to treat Gaucher disease in Iran: Sanofi Genzyme‘s Cerezyme (imiglucerase for injection) and ISU Abxis‘ Abcertin (imiglucerase for injection). Both have an average annual cost of about $19,763 per patient, corresponding to roughly 95.2% of their total yearly healthcare expenses.
“These figures show clearly that medication cost encompasses the vast majority of the resources used for GPs [Gaucher patients], and thus any program for improving the efficiency of GD management should target the medication therapy of GPs,” the researchers wrote.
According to the study, strategies that might be implemented to lower costs include prioritizing the use of the least expensive imiglucerase brand available in the country, and considering that eligible patients being treated with intravenous ERT switch to oral SRTs like Zavesca (miglustat, by Actelion) and Cerdelga (eliglustat, also by Sanofi Genzyme).
“The treatment of GPs starts mainly from childhood in Iran and their life expectancy are usually 4 years shorter than normal population. Since the life expectancy in Iran is 75 years, each GP [is estimated to face a healthcare cost of] $1,473,818 in his total life, which is terribly high. This may encourage health policy makers to think about methods for preventing the borne of such patients,” the investigators wrote.
A valid diagnostic test also needs to be identified and put into practice, as “our data showed that a considerable number of patients had been wrongly diagnosed as a GP and then had taken wrong medications for years,” the team added, and because the lack of a proper diagnosis does not distinguish among Gaucher’s types.
