我国罕见病药物研发现状与技术领域分析--以第二批罕见病目录为例  

Study on the current situation and technical fields of China's rare disease drug research and development

作　　者：焦杨 颜建周[1] 李树祥[1] JIAO Yang;YAN Jian-zhou;LI Shu-xiang(School of International Pharmaceutical Business,China Pharmaceutical University,Nanjing 211198,China)

机构地区：[1]中国药科大学国际医药商学院,南京211198

出　　处：《中国新药杂志》2024年第22期2305-2313,共9页Chinese Journal of New Drugs

基　　金：国家自然科学基金资助项目(72274217);江苏高校哲学社会科学研究项目“江苏省生物医药创新研究:专利视角”(2020SJA0086)。

摘　　要：随着罕见病受到越来越多的关注,我国各类药物研发机构也开始投入到罕见病治疗药物的研发。本文基于第二版罕见病目录病种检索相应的药物专利数据,对我国罕见病药物研发现状以及研发技术领域进行分析。研究结果显示,当前我国罕见病药物的研发主体中,高校和研究所占据了较为重要的位置,企业申请的专利较少,而且专利国际申请较少;在研发技术领域中,C12Q1和A61K31是2类最为常见的技术领域。虽然我国罕见病药物专利已取得了一些成绩,但是未来仍需要鼓励高校-研究所-企业之间的研发合作,激励企业更多参与罕见病药物的研发,才能更好地将研发专利转化为实际治疗药物,提高转化率。As rare diseases receive more and more attention,all kinds of drug research and development(R&D)organizations in China have also invested in the research and development of therapeutic drugs for rare diseases.Based on the second edition of the rare disease catalog,this paper searched the corresponding drug patent data,and analyzed the current situation of China's rare disease drug R&D and R&D technology fields.The results of the study showed that universities and research institutes occupied an important position in the main body of R&D of rare disease drugs in China,enterprises applied for fewer patents,and only a small number of international patents were applied;in the field of R&D technology,C12Q1 and A61K31 were the two most common technical fields.Although China's rare disease drug patents have made some achievements,but in the future,we still need to encourage R&D cooperation between universities and research institutes and enterprises and incentivize enterprises to participate more in the R&D of rare disease drugs,so that we can better transform the R&D patents into actual therapeutic drugs to improve the conversion rate.

关 键 词：罕见病 研发现状 技术领域 二模网络 第二版罕见病目录 

分 类 号：R95[医药卫生—药学]