上海戈谢病患者特效药费用保障分析

Analysis of costs coverage for specialized drugs for patients with Gaucher disease in Shanghai

针对戈谢病这一需要高值用药的罕见病,上海较早建立了由基本医疗保险基金、上海市罕见病防治基金会溶酶体贮积症专项救助基金和上海市中小学生、婴幼儿住院医疗互助基金等构成的多元保障体系。该保障体系在当时处于全国领先水平,上海戈谢病患者也因此能真正"用得上药"。为了解该保障体系对戈谢病患者高值特效药的保障情况,该文分析2018至2020年戈谢病用药费用分担情况,结果显示6例患者共发生费用3 634.75万元,每年人均201.93万,基本医疗保险基金、上海市罕见病防治基金会溶酶体贮积症专项救助基金和个人自付比例分别为79.02%、16.28%和4.70%。基于上海保障经验得出,罕见病高值用药由于价格昂贵,需较高的保障水平才可保证患者用药需求,故需要多方共同建立保障机制。

For Gaucher Disease,a rare disease,which requires high-value medication for treatment,Shanghai has established a multi-tiered system consisting of the Basic Medical Insurance(BMI),the Shanghai Rare Disease Prevention and Control Foundation Lysosomal Storage Disease Special Aid Fund(SAF),and the Shanghai Primary and Secondary School Students,Infants and Children Hospitalization Medical Mutual Fund(CHMF) for insuring Gaucher Disease.This security system was the leading one in China at that time,and patients with Gaucher Disease in Shanghai could really have access to medicine.In order to understand the guarantee of high-value specific drugs for Gaucher Disease patients,this paper analyzed the cost-sharing situation of Gaucher Disease from 2018 to 2020,and found that the total cost for six patients was 36.35 millions Chinese Yuan(CNH),an average of 2.02 millions CNH for per person per year.Among the costs,the proportions of BMI,SAF and individual out-of-pocket rate were 79.02%,16.28% and 4.70%,respectively.From the Shanghai coverage experience,it can be found that high-value drugs for rare diseases are very expensive,a high level of coverage is needed to ensure that patients actually have access to the drugs,and this often requires multiple parties to work together to establish a coverage mechanism.