基于5种罕见病可负担性评价的我国罕见病保障机制研究

目的:为我国建立完善罕见病保障机制提出政策建议,以解决因其所致的"因病致贫、因病返贫"问题。方法:分别通过世界卫生组织/国际健康行动组织(WHO/HAI)标准调查法、灾难性支出评价法和致贫作用评价法3种方法对我国城镇和农村居民多种羧化酶缺乏病、苯丙酮尿症等5种罕见病的可负担性进行分析。结果:按WHO/HAI标准调查法,5种罕见病年治疗费用都很高,费用最低的疾病也相当于城镇居民0.28年的收入,农村居民比城镇居民疾病负担更重;按灾难性支出评价法,5种罕见病在全国范围内造成灾难性支出的人口比例均不超过0.060 6‰,但对部分罕见病,一旦居民患病并采用药物治疗,即陷入灾难性卫生支出;按致贫作用评价法,5种罕见病在城镇、农村的致贫率均很低,但仅这5种罕见病在全国范围内会导致超过20万人陷入贫困。结论:我国罕见病及罕用药的可负担性较差,不同收入水平的居民均有一定的支付困难,因病致贫、因病返贫现象普遍。因此应完善罕见病患者保障机制,建立专门针对罕用药的费用负担方式。

Research on Rare Disease Security Mechanism in China Based on the Affordability Evaluation of 5 Rare Diseases

OBJECTIVE： To give suggestions to the establishment of rare disease security mechanism in China so as to solve the problems of ＂illness-caused poverty＂. METHODS： The 5 rare diseases （as carboxylase deficiency, phenylketonuria） affordability of urban and rural residents were analyzed according to WHO/HAI standardized approach method, the catastrophic expenditure evaluation method and the impoverishment evaluation method. RESULTS： Based on WHO/HAl standardized approach method, the cost of 5 rare diseases was very high and as 0.28 times as the urban income; the rural burden is heavier than the urban. Based on catastrophic expenditure evaluation method, the population of catastrophic expenditure caused by 5 rare diseases occupied less than 0.060 6%e. However, the residents who suffered from some rare diseases and received drug treatment would fall into catastrophic expenditure. Based on the impoverishment evaluation method, the rate of poverty caused by 5 rare disease was very low in urban and rural areas but the only 5 rare diseases would cause over 200 000 people into poverty in nationwide. CONCLUSIONS： The affordability of rare diseases and orphan drugs is poor in China. There is certain payment difficulty for residents with different levels of income. Due to the common phenomenon of illness-caused poverty, we should improve the security mechanism of the rare disease and set up specialized cost affording ways for orphan drugs.