发达国家通过立法鼓励用于罕见病的孤儿药的研发和生产,按照WHO的标准罕见病种高达6000~8000种,如血友病、本丙酮症等等,为了促进孤儿药的研发生产,很多国家地区都制订了激励政策。但我国孤儿药极度缺乏,罕见病患者未得到有效救治,目前我国罕见病患者治疗药物基本依赖进口,国家对罕见病的治疗费用尚无保证政策,一般治疗家庭无力承担治疗费用。从获得性 Developed countries through legislation to encourage the development and production of orphan drugs for rare diseases, according to the WHO standard rare disease up to 6,000 to 8,000 species, such as hemophilia, the acetonosis and so on, in order to promote the development of orphan drug production, many Incentives have been formulated in all countries. However, there is an extremely lack of orphan drugs in our country and rare diseases are not effectively treated. At present, the treatment of rare patients in our country is basically dependent on imports. There is no guarantee policy for the treatment costs of rare diseases in China. Generally, the treatment families can not afford the treatment costs. Acquired from