孤儿药获批适应证和儿童用药的国内外差异分析
Analysis of differences in orphan drug indications and pediatric use between domestic and international approvals
刘清扬 1王少红 1左玮 1刘鑫 1唐彦 1张波1
作者信息
- 1. 中国医学科学院北京协和医学院北京协和医院 药剂科,北京 100730
- 折叠
摘要
目的 分析孤儿药在FDA/EMA与我国药品说明书获批适应证和儿童用药差异,为国内罕见病的药物治疗、临床试验开展和超说明书用药管理等提供参考.方法 检索FDA或欧洲药品管理局(EMA)批准上市,并在我国国家药品监督管理局(NMPA)获得批准文号的孤儿药,比较国内外获批适应证和儿童用药的差异,并梳理国外获批而我国未获批药品的解剖学、治疗学、化学(ATC)分类.结果 截至2023年12月,在FDA和EMA上市的孤儿药分别有319种和123种在我国获得批准文号,其中分别有134种和69种可用于我国第一、第二批《罕见病目录》内疾病的治疗.FDA和EMA上市的的孤儿药分别有117种和38种在我国未获批适应证,其中分别有47种和13种涉及第一、第二批《罕见病目录》内疾病的治疗;另外,分别各有22种在我国未获批儿童用药,其中分别有12种和14种涉及《罕见病目录》内疾病的治疗;相较FDA和EMA,在我国用于《罕见病目录》外疾病的药物未获批适应证的比例更高,用于《罕见病目录》内疾病的药物未获批儿童用药的比例也高,抗肿瘤药和免疫机能调节药是FDA或EMA上市的孤儿药在国内获得批准文号比例最高的药物分类,也是国内未获批适应证或儿童用药出现频次最高的药物分类.结论 相较FDA和EMA,我国罕见病药物在获批适应证和儿童用药方面存在一定滞后.《罕见病目录》的发布对于我国罕见病药物适应证获批起到了一定的推动作用,但用于儿童等特殊人群的超说明书用药现象需进一步关注.
Abstract
Objective To summarize the differences in the approved indications and pediatric use of orphan drugs between the US Food and Drug Administration(FDA)/European Medicines Agency(EMA)and China's National Medical Products Administration(NMPA),providing reference for drug treatment,clinical trials and off-label drug use management of rare diseases.Methods Orphan drugs approved by the FDA or EMA and granted marketing authorization in China were analyzed to compare differences in indications and pediatric use.The anatomical,therapeutic,and chemical(ATC)classifications of drugs approved abroad but not in China were also reviewed.Results As of December 31,2023,319 and 123 orphan drugs approved by the FDA and EMA,respectively,had marketing authorizations in China,of which 134 and 69 were approved for the treatment of diseases listed in the first and second batches of China's Rare Disease Catalog.There were 117 orphan drugs of FDA and 38 orphan drugs of EMA that were not approved in domestic,of which 47 and 13 were related to the treatment of diseases in the Rare Disease Catalog,respectively.Additionally,22 drugs approved by both the FDA and EMA had unapproved pediatric use in China,with 12 and 14 targeting diseases in the Rare Disease Catalog.Compared to the FDA and EMA,China exhibited a higher proportion of unapproved indications for drugs used in non-Rare Disease Catalog conditions and unapproved pediatric use for drugs in the Rare Disease Catalog.Anti-cancer and immunomodulatory drugs were the most frequently proportion of orphan drugs of approved and unapproved categories in terms of indications and pediatric use.Conclusions:Compared with the FDA and EMA,there is a certain lag in the approval of orphan drug indications and pediatric use in China.The publication of the Rare Disease Catalog has played a role in promoting the approval of drug indications,but further attention should be paid to the phenomenon of off-label use of rare disease drugs for children.
关键词
罕见病/孤儿药/可及性/超说明书用药Key words
rare disease/orphan drug/accessibility/off-label use引用本文复制引用
出版年
2024
NETL
NSTL
万方数据