关于中国罕见病治疗药物的发展建议

发布时间：2018-02-25 08:50

本文关键词： 罕见病孤儿药综合措施政策建议　出处：《国际药学研究杂志》2017年02期 　论文类型：期刊论文

【摘要】：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。
[Abstract]:The issue of access to orphan drugs has received a high degree of attention from all countries, Since 1980s, many countries and regions have enacted special laws and regulations to promote their development. At present, orphan drug research and development has become an important strategy for major pharmaceutical companies to develop their products. China has not yet defined rare diseases. Orphan drugs are almost completely dependent on imports. The lack of policy support in research, development, introduction, production and marketing has become a bottleneck restricting the development of related industries in the country. This paper will review the development status of rare diseases and their therapeutic drugs at home and abroad. Some suggestions on the development of orphan drugs in China are put forward in order to provide some references for the relevant government departments to formulate policies.
【作者单位】：军事医学科学院毒物药物研究所;国家卫生计生委医疗管理服务指导中心;
【基金】：国家重点研发计划精准医学研究重点专项“罕见病临床队列研究”资助项目(2016YFC0901500)
【分类号】：R95

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