Analysis of the use of imiglucerase and it's revelation on the establishment of rare disease medical security system / 中国药学杂志

ABSTRACT

OBJECTIVE: To explore the burden for treating patients with Gaucher disease and provide a reference for the establishment of rare diseases medical securitysystem in China. METHODS: The basic information of the patients enrolled in this study and the prescriptions of the imiglucerase from 2009-2015 in our hospital were analyzed. The total number of patients diagnosed with Gaucher disease, the total cost of Imiglucerase and the average Imiglucerase cost per patient per year were focused by us. RESULTS: A total of 35 patients received imiglucerase from our hospital between 2009 and 2015. The enrolled patients increased from 25 to 35 in the past seven years. Annual growth rate is about 5.7% (14 patient per year). The total cost for imiglucerase is 130.065 million RMB. The average annual drug cost per patient is 918.6 thousands RMB from 2010 to 2015. CONCLUSION: In the past seven years, the number of patients with Gaucher disease in our hospital increased slightly. Due to the high drug costs of Gaucher disease, it is hardly for patients to pay for the drug themselves, and it is suggested to establish a multiple payment mechanism for rare disease.