发达国家及地区罕见病/罕用药界定策略分析  

Analysis of Definition of Rare Diseases and Orphan Drugs in Developed Countries

作　　者：李春潇[1,2] 谢婧[1,2] 胡欣[2] 

机构地区：[1]沈阳药科大学工商管理学院,辽宁沈阳110016 [2]卫生部北京医院临床药理室,北京100730

出　　处：《中国执业药师》2013年第3期51-56,共6页China Licensed Pharmacist

基　　金：卫生行业科研专项基金(BJ-2010-1)

摘　　要：目的:通过广泛研究发达国家及地区罕见病/罕用药政策,为我国相关政策制定提供参考。方法:应用文献研究法对发达国家及地区罕见病政策进行检索,发达国家及地区的筛选标准为联合国开发计划署发布的人文发展指数。结果和结论:多数发达国家及地区对罕见病制定了专门政策,各国罕见病界定策略大体分成三类:依照患病人数确定;依照发病率确定;无定量界定,接受民间自发呈报罕见病。罕见病/罕用药制定过程需综合考虑其自身社会发展程度、罕用药可及性、人口因素。人口较多的国家倾向采用患病人数作为定量标准界定罕见病,建议我国适当借鉴并采纳。Objective： To study the policies about rare diseases or orphan drugs in developed countries so as to provide a reference for the establishment of relative policies in China. Methods： Literature studies were conducted through retrieving the policies about rare diseases or orphan drugs in developed countries. The Human Development Index issued by the UN Development Programme was used as the screening standard for the definition of developed countries and regions. Results ＆ Conclusion： Specific policies were made by most developed countries on rare diseases. The strategy for definition of rare diseases was set up mainly according to three factors such as sum of patients, incidence and spontaneously reported diseases without qualitative requirements. The level of social development, availability of orphan drugs, demographic characteristics were the key elements for each country or region to define rare disease. The countries with large populations preferred defining the rare diseases by sum of patients, which can be considered and learned by China.

关 键 词：罕见病 罕用药 人口 人文发展指数 

分 类 号：R442.9[医药卫生—诊断学]