我国罕用药可及性现状及其市场准入制度分析

Analysis of orphan drug accessibility and marketing authorization system in China

根据我国罕见药市场可及性现状,构建罕用药评价指标体系,为我国建立完善准入制度提供参考依据。研究表明,我国罕用药市场准入制度尚未完善,罕用药的可获得性、可供应性和可负担性指标均不及发达国家或地区,严重影响罕见病患者用药,阻碍罕用药产业发展。建议借鉴其他国家或地区的经验,完善我国罕用药市场准入制度,以期激励我国罕用药研发创新,提高罕见病患者的治疗水平。

According to the status-quo of orphan drug access in China, this paper constructedan index evaluation system so as to provide referable foundationfor establishing and improving orphan drug authorization system. Study results indicated that the less-improved orphan drug marketing authorization system, poorer orphan drug availability, supply capacity and affordability compared with developed countries and regions severely affected patients′ timely medication and hindered orphan drug industry development. With reference to foreign countries, it is urgent to improve orphan drugs marketing authorization system for the purpose of encouraging orphan drug R＆D and improving the health benefits of patients.