美国发布指南明确电子健康记录和医疗索赔中的真实世界数据可支持药物和生物制品的监管决策  

FDA issued guidance clarifying the role of real-world data from electronic health records and claims data in drug and biological product regulatory decision-making

作　　者：姚晨[1,2,3] 王斌 阎小妍[2] Yao Chen;Wang Bin;Yan Xiaoyan(Department of Medical Statistics,Peking University First Hospital,Beijing 100034,China;Peking University Clinical Research Institute,Beijing 100191,China;Hainan Institute of Real world Data,Qionghai 571437,China;School of Clinical Medicine,Tsinghua University,Beijing 102218,China)

机构地区：[1]北京大学第一医院医学统计室,北京100034 [2]北京大学临床研究所,北京100191 [3]海南省真实世界数据研究院,琼海571437 [4]清华大学临床医学院,北京102218

出　　处：《数字医学与健康》2025年第1期12-19,共8页DIGITAL MEDICINE AND HEALTH

摘　　要：2024年7月,美国食品药品监督管理局发布指南,明确电子健康记录和医疗索赔数据作为真实世界数据在药物和生物制品监管决策中的应用。该指南为数据源选择、研究设计、数据质量控制和验证方法提供了框架。其发布符合美国《21世纪治愈法案》和《处方药用户付费修正案》的要求,并修订了2021年草案。指南强调数据来源的相关性和完整性,并提供评估数据质量的具体建议。这一举措为我国药物监管的实践提供了重要参考,尤其在数字化转型、药物研发、药物经济学评价、中医药融合创新及监管科技发展等方面。同时,指南还指出加强数据驱动监管体系和升级风险管理模式的必要性,为我国在全球药物市场中提升竞争力提供了参考。In July 2024,the U.S.Food and Drug Administration(FDA)released final guidance clarifying the use of electronic health records(EHR)and claims data as real-world data(RWD)in drug and biological product regulation.The guidance outlines frameworks for data source selection,study design,data quality control,and validation methods.Aligning with the 21st Century Cures Act and the Prescription Drug User Fee Amendments,it is also a revision of the 2021 draft guidance.The guidance emphasizes the relevance and completeness of data sources and offers specific recommendations for data quality assessment.This initiative provides valuable insights for Chinese regulatory practices,particularly in digital transformation,drug development,pharmacoeconomic evaluation,integration of traditional Chinese medicine,and regulatory technology.It also highlights the need for enhanced data-driven regulatory systems and risk management,offering new directions for improving China′s competitiveness in the global pharmaceutical market.

关 键 词：电子病历 医疗索赔数据 真实世界数据 监管决策 指南 

分 类 号：R95[医药卫生—药学]