目的 探讨戈谢病患者用药负担性,为我国罕见病用药保障体系提供参考。方法 对我院2009~2015年戈谢病患者基本情况和伊米苷酶使用情况进行分析,重点关注患者总人数、每年新增或递减人数、患者用药时间、伊米苷酶总药费和平均药费。结果 2009~2015年共有35人在我院领取伊米苷酶,我院戈谢病患者患者从2009年的25人增加至2015年的35人,戈谢病患者年增长率约5.7%(约1.4名·年-1);35名患者伊米苷酶总药品费用13 006.5万元,患者年平均用药金额91.86万元(2010~2015年)。结论 近7年,我院就诊的戈谢病人数相对固定;戈谢病用药非常昂贵,个人几乎无法承担,亟须建议建立个人、政府和社会多方付费的罕见病用药保障体系。
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% (1.4 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.
关键词
思而赞 /
伊米苷酶 /
罕见病 /
孤儿药
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Key words
cerezyme /
imiglucerase /
rare disease /
orphan drug
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中图分类号:
R951
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参考文献
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脚注
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基金
国家临床重点专科建设项目
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