[关键词]
[摘要]
罕见病用药指用于治疗、诊断和预防罕见病或罕见症状的药物。近年来罕见病用药的研发逐渐成为一个可获利的研发策略,受到高度关注和重视。从目前国外罕见病药品市场情况、参与罕见病药物研发的公司和重点品种、美国和欧盟等发达国家和地区罕见病药物的指定和批准情况等方面对罕见病用药的现状及发展趋势进行简述。通过借鉴这些国家和地区罕见病药物发展的成功经验,为制定中国罕见病药物开发的刺激措施提出建议,为相关研发人员提供参考。
[Key word]
[Abstract]
The orphan drug is a pharmaceutical product aimed at curing, diagnosing, and preventing rare diseases or disorders. The research and development of orphan drugs has been a profitable development strategy gradually, and has drawn people's special attentions in recent years. This article highlights the trends in prescription sales for orphan drugs, orphan products and company performance, and the designation and approval analysis in US and Europe. The successful implementation experiences of developed countries in this field have provided suggestions for the establishment of incentive measures for facilitating orphan drugs in China.
[中图分类号]
[基金项目]