关于中国罕见病治疗药物的发展建议

Suggestions on the development of rare disease drugs in China

孤儿药可及性问题已得到各国的高度关注,很多国家和地区从20世纪80年代开始就制定了专门的法律法规以促进其开发。目前孤儿药研发已成为各大制药公司进行产品开发的重要策略。中国目前尚未定义罕见病,孤儿药几乎完全依赖进口,其研发、引进、生产及销售等缺乏政策支持,已成为制约国家相关产业发展的瓶颈。本文拟对国内外罕见病及其治疗药物的发展现状作一综述,并对中国的孤儿药研发提出相应的建议,以期为国家相关部门制定政策提供一定参考。

The accessibility of orphan drugs has been the focus of many countries. Many countries and regions have estab- lished special laws and regulations to promote the development of orphan drugs since 1980＇ s. The development of orphan drugs has now become to be an important strategy for the drug development of many large pharmaceutical companies. At present, China has not yet defined the rare diseases, and the orphan drugs used in China are almost dependent on imports. The domestic situation that the re- search and development, introduction, production and sales of orphan drugs lack the policy support has been the bottleneck restricting the development of related industries in China. This paper reviews the present status of worldwide development of the rare diseases and their therapeutic drugs, and put forward related suggestions for the development of orphan drugs in China.