中国罕见病用药可负担性评价

Evaluation of the Affordability of Orphan Medical Products in China

目的评价国内已上市且有明确适应证的罕见病用药的可负担性,比较不同罕见疾病、城乡不同水平的可负担性差异,为完善我国罕见病用药保障提供参考。方法基于中国罕见病信息网和《罕见病诊疗指南(2019年版)》收集罕见病发病率等信息,基于药智网(https://www.gaozh.com/)收集罕见病用药的中标价格等信息,对基于数据可得性纳入研究的16种罕见病和34种罕见病用药,分别采用世界卫生组织/国际健康行动组织(WHO/HAI)标准调查法、致贫性评价法和灾难性支出评价法,分析城镇居民和农村居民的可负担性情况。结果按WHO/HAI标准调查法,34种药品中的16种药品使城镇居民负担较重,19种药品使农村居民负担较重,其中治疗戈谢病的伊米苷酶年度药品支出是城镇居民平均收入的114.67倍,是农村居民平均收入的303.17倍;按致贫性评价法,罕见病用药的支出会导致全国超过268万居民(农村居民131万,城镇居民137万)陷入贫困,其中治疗帕金森病(青年型、早发型)的屈昔多巴会造成全国约31.2万居民致贫;按灾难性支出评价法,34种罕见病用药会导致全国超过302万的居民发生灾难性支出,其中治疗帕金森病(青年型、早发型)的屈昔多巴、恩他卡朋双多巴、雷沙吉兰会导致65.61万城镇居民和49.65万农村居民发生灾难性支出。结论目前,我国居民罕见病用药可负担性因不同疾病类型有差异,但总体可负担性较差,不同收入水平的居民均有因病致贫、药品支付困难的现象,建议针对不同罕见病实施多层次医疗保障,提高罕见病用药可负担性。

Objective To evaluate the affordability of orphan medical products(OMPs)that have been listed in China and have clear indications,and to compare the affordability differences that vary in urban and rural areas and different rare diseases,so as to provide a reference for improving the protection of rare diseases in China.Methods The information of rare disease incidence was collected based on Domestic Rare Disease Information Network and Rare Diseases Diagnosis and Treatment Guideline(2019 edition),and the drug prices of OMPs were collected based on Yaozhi of net(https://www.yaozh.com/).Totally 16 rare diseases and 34 OMPs were included in the study based on data availability.World Health Organization/Health Action International(WHO/HAI)standard survey method,poverty assessment method and catastrophic expenditure evaluation method were used to analyze the affordability of urban residents and rural residents.Results According to the WHO/HAI standard survey method,16 kinds of 34 OMPs had a heavy burden on urban residents and 19 OMPs had a heavy burden on rural residents.Among them,the annual drug expenditure of imiglucerase for the treatment of Gauche’s disease was 114.67 times of urban residents’average income and 303.17 times of rural residents’average income.According to the poverty assessment method,the spending on OMPs would push more than 2.68 million people into poverty(1.31 million rural residents and 1.37 million urban residents),and the treatment of Parkinson’s disease(young and early-onset)with Droxidopa would push about 312000 people into poverty.According to the catastrophic expenditure evaluation method,34 OMPs would cause catastrophic expenditures for more than 3.02 million residents nationwide,and the treatment of Parkinson’s disease(youth,early-onset)with droxidopa,entacapone,levodopa and carbidopa,and rasagiline would cause catastrophic expenditures for 656100 urban residents and 496500 rural residents.Conclusion At present,the affordability of rare diseases in China varies according to the types of diseases,but the overall affordability is poor.Residents with different income levels suffer from poverty due to illness and difficulties in drug payment.It is suggested to implement multi-level medical insurance for different rare diseases to improve the affordability of OMPs.