我国优先审评审批上市儿童用药的特征分析  

Characteristics analysis of pediatric medicines with priority review and approval for marketing in China

作　　者：杨浩宇 田侃[2] 游雪 淡宏伟 王乾[5] 喻小勇[2,6] YANG Haoyu;TIAN Kan;YOU Xue;DAN Hongwei;WANG Qian;YU Xiaoyong(School of Health Economics and Management,Nanjing University of Chinese Medicine,Nanjing 210023,China;Institute of Elderly Care Services and Management,Nanjing University of Chinese Medicine,Nanjing 210029,China;Dept.of ICU,Nanjing First Hospital,Nanjing 210006,China;Dept.of Anesthesiology and Pain Management,Zhongda Hospital,Southeast University,Nanjing 210009,China;Dept.of Development and Planning,the First Affiliated Hospital of Shihezi University,Xinjiang Shihezi 832008,China;School of Elderly Care Services and Management,Nanjing University of Chinese Medicine,Nanjing 210023,China)

机构地区：[1]南京中医药大学卫生经济管理学院,南京210023 [2]南京中医药大学养老服务与管理研究院,南京210029 [3]南京市第一医院重症医学科,南京210006 [4]东南大学附属中大医院麻醉手术与疼痛管理科,南京210009 [5]石河子大学第一附属医院发展规划科,新疆石河子832008 [6]南京中医药大学养老服务与管理学院,南京210023

出　　处：《中国药房》2025年第5期519-523,共5页China Pharmacy

基　　金：江苏省社会科学基金后期资助项目(No.20HQ019);江苏省研究生科研创新计划项目(No.KYCX24_2114);南京中医药大学养老服务与管理学院(养老产业学院)专项研究立项项目(No.2024YLFWYGL002)。

摘　　要：目的分析我国优先审评审批上市儿童用药的特征,为促进企业研发生产和完善儿童用药供应保障机制提供参考。方法基于《优先审评审批的儿童用药批准信息清单》、药融云生物医药数据库、《国家医保药品目录》等公开数据源,对优先审评审批上市儿童用药的主要特征进行梳理分析。结果截至2024年6月30日,共有68个优先审评审批上市的儿童用药,涉及12个治疗领域,其中口服剂型的占比达64.71%。从申请到纳入优先审评的中位数时间为35.50 d,平均时间为41.69 d;从纳入优先审评至批准上市的中位数时间为1.24年,平均时间为1.42年。68个优先审评审批上市儿童用药中包括12个国产新药、21个国产仿制药、35个进口药品,共涉及29个儿童专用药和21个罕见病用药。其中,有31个药品被纳入医保目录,占比为45.59%。结论国产与进口儿童用药之间显示出差异化竞争趋势,儿童用药的治疗领域不断丰富且剂型更贴近儿童需求,但仍存在新药研发进展缓慢、审评审批程序内部稳定性不足、医保纳入比例有待提高等问题。OBJECTIVE To analyze the characteristics of pediatric medicines with priority review and approval for marketing in China,providing a reference for promoting enterprise R&D and production,as well as improving the supply guarantee mechanism for pediatric medicines.METHODS Based on publicly available data sources such as List of Approved Information for Pediatric Medications Subject to Priority Review and Approval,Pharnexcloud biomedical database,and National Medical Insurance Drug Directory,this study conducted a comprehensive analysis of the main characteristics of pediatric medicines with priority review and approval for marketing.RESULTS As of June 30,2024,a total of 68 pediatric medicines had been approved through the priority review and approval process,covering 12 therapeutic areas,with oral dosage forms accounting for 64.71%.The median time from application to inclusion in priority review was 35.50 days,with an average of 41.69 days.The median time from inclusion in priority review to market approval was 1.24 years,with an average of 1.42 years.This included 12 domestic new medicines,21 domestic generic medicines,35 imported medicines,as well as 29 pediatric-specific medicines and 21 orphan medicines.Additionally,31 of these medicines had been included in the medical insurance catalog,representing a proportion of 45.59%.CONCLUSIONS Currently,a trend of differentiated competition is emerging between domestic and imported pediatric medicines.The therapeutic areas for pediatric medicines are continuously expanding,and the dosage forms are becoming more tailored to children’s needs.However,there are still issues such as slow progress in new medicine development,insufficient stability in the medicine review and approval process,and a need to increase the proportion of medicines included in medical insurance.

关 键 词：优先审评审批 儿童用药 儿童专用药 罕见病用药 

分 类 号：R95[医药卫生—药学] R985