美国制药企业罕见病药品的市场开发策略

Market Development Strategies of Orphan Drugs Used by the USA Pharmaceutical Enterprises

在美国,为了加快罕见病药品(罕用药)的上市速度,制药企业申请政府促进罕用药新药研发基金项目和使用加快罕用药新药临床试验进程等激励政策;为了加快收回投资回报,企业实施罕用药上市后的市场独占期政策,并采取了高定价和专业药房的销售模式,标签外用药等适应证扩展策略。美国的经验为我国罕用药市场激励机制的建立和其可及性产业政策的良性发展提供了有益借鉴。但罕用药的标签外用药的销售方式在我国还需要慎行。

In the USA,in order to speed up orphan drugs marketing,pharmaceutical enterprises apply for the grants of orphan drug research and adopt the accelerated procedure of the clinical trials for orphan drugs. And in order to increase the investment returns,the companies practice the market monopoly policy for orphan drugs,and use high pricing strategy and sale channel of specialty pharmacy and off-label use of orphan drugs,etc.The experience of the USA provides a useful reference for the establishment of the incentive mechanism and the positive development of orphan drug accessibility.However,the off-label sales of orphan drugs in China need to be cautiously conducted.