建立和实施罕用药制度的探讨

Establishment and operation of an orphan drug nmnagement system

实施罕用药制度可以显著提高罕见病患者治疗水平和生存质量，促进医药科技发展进步。作者参考国外的罕用药法规，汇集各国罕用药管理经验，包括罕用药认定、研制开发的扶持措施、市场保护、临床使用管理等，结合中国国情，讨论建立实施罕用药制度的方法策略和现实意义。

The orphan drug management system can significantly improve the quality of care and that of life of patients suffering from rare diseases, and accelerate the progress of medicinal science. With reference to laws and regulations on orphan drugs in other countries, the authors summarized the experiences of practicing the orphan drug management system in developed countries and regions, including certification of orphan drugs, supports for developing orphan drugs, market protection and clinic application management. Based on such studies, the authors discussed the feasibility and methodology of establishing an orphan drug management system in China, along with the significance of such a system.