我国罕见病界定标准初探

Preliminary study on Chinese standards for defining rare diseases

目的:为我国罕见病界定标准的确立提供参考。方法:比较欧美日等国家和地区罕见病定义并提炼出界定标准,分析影响各标准制定的关键因素,进而提出确立我国罕见病界定标准的建议。结果与结论:一个国家罕见病界定标准的制定受社会经济发展水平、医学发展水平、社会保障水平等因素影响,建议我国采用患病数、疾病严重程度、罕用药经济学标准来界定罕见病。

Objective：To suggest approaches for the establishment of Chinese standards for defining rare diseases and advance corresponding Chinese legislation. Methods：By comparing the standards for defining rare diseases in Europe, the United States, and other countries and studying key influencing factors of standards, this paper proposes suggestions for the establishment of proper Chinese standards for defining rare. Results and Conclusion：Social and economic development levels, medical development levels and social security levels influence the establishment of standards. It is recommended that the national conditions should be taken into consideration and the number of patients, severity of the diseases and economic indicators of orphan drugs be used to define rare diseases in China.