摘要
目的 汇总并分析2023年中国、美国、欧盟及日本的罕见病用药上市概况,为中国医药企业、医药管理和新药研发部门提供实证参考.方法 通过中国、美国、欧盟、日本的药品监督管理官方机构发布的2023年药品上市数据,梳理罕见病用药上市概况,对其适应证、上市日期、在中国研发状态、治疗领域、特殊审评审批途径等进行分析.结果 2023年美国上市28种孤儿药,抗肿瘤领域占比最高,为32.1％(9/28);欧盟上市17种孤儿药,抗肿瘤领域占比最高,为47.0％(8/17);日本上市22种孤儿药;中国上市45种罕见病用药.2023年欧盟及日本上市的孤儿药已在中国进入临床试验/上市申请中/批准临床/临床申请中的比例均已超过70％(日本86.4％,欧盟70.6％).结论 中国目前尚无孤儿药资格认定体系,但2023年中国在4个国家和国际组织中上市罕见病用药数量最多,体现了中国罕见病目录对药物研发充分的引导作用;同时中国药品审批审评改革和罕见病目录发布时间尚短,罕见病用药审批和研发仍处于追赶阶段.
Abstract
Objective To summarize and analyze what the listing in market of orphan drugs in China,the United States,the European Union,and Japan in 2023,and to provide empirical reference to Chinese phar-maceutical enterprises,pharmaceutical management,and new drug research and development departments.Methods Collect the 2023 drug market data released by official drug regulatory agencies in four countries and international organization-China,the United States,the European Union,and Japan categorize the market of rare disease drugs and and their indications,approval dates,R&D status in China,component therapeutic are-as,and special review and approval pathways were analyzed.Results Findings in 2023,28 orphan drugs were listed in the United States,mostly anti-tumor related,accounting for 32.1％(9 out of 28);17 orphan drugs were listed in the European Union,anti-tumor related accounting for 47.0％(8 of 17);22 listed in Japan;and 45 listed in China.In 2023,over 70％orphan drugs listed in the European Union and Japan entered the phase of clinical trials/marketing and applications/approved clinical applications in China(86.4％from Japan and 70.6％from European Union).Conclusions Currently,there is no qualification accreditation mechanism for orphan drugs in China.However,in 2023,the number of rare disease drugs listed in China was the greatest among the four countries/international organization.It reflects that China's Catalog for Rare Diseases played a guiding role in drug research and development.Meanwhile,the reform of drug approval and review in China and the issue of rare disease catalogs took place in a short period of time,so the review and approval,as well as re-search and development of rare disease drugs are still in the catching up stage.
基金项目
中央高水平医院临床科研业务费项目(2022-PUMCH-B-059)
中国医科院医学与健康科技创新工程项目(2021-I2M-1-003)
维普
万方数据