真实世界数据用于监管与HTA决策：六个欧盟项目的经验与建议
Toward the Use of Real‐World Data for Regulatory and HTA Decision‐Making: Experiences and Recommendations from Six EU‐Funded Projects
作者：Martin Russek、Jonas Peltner、Kerstin Pfeifer、Theodoros N. Arvanitis、Dirk De Valck、Sieta T. de Vries、Michel Dumontier、Vera Ehrenstein、Omaima El Hakouni、Gregory Epiphaniou、Gokhan Ertaylan、Joana Fernandes、Scott Hansen、Stuart Harrison、Erzsébet Horváth‐Puhó、Saif Ul Islam、Pekka Kahri、Julia K. Mader、Carsten Maple、Nikolaos Matragkas
单位：Research Division, Federal Institute for Drugs and Medical Devices, Bonn, Germany.；Department of Electronic, Electrical and Systems Engineering, School of Engineering, University of Birmingham, Birmingham, UK.；European Organisation for Professionals and People with ALS (EUpALS), Leuven, Belgium.；Department of Clinical Pharmacy and Pharmacology, University of Groningen, University Medical Center Groningen, Groningen, the Netherlands.；Department of Advanced Computing Sciences, Maastricht University, Maastricht, the Netherlands.；Department of Clinical Epidemiology and Center for Population Medicine, Aarhus University, Aarhus N, Denmark.；Division of Endocrinology and Diabetology, Department of Internal Medicine, Medical University of Graz, Graz, Austria.；WMG, University of Warwick, Coventry, UK.
摘要
发表于《Clinical Pharmacology & Therapeutics》早期线上版（2026-10-02）。文章汇总了六个欧盟资助项目在将真实世界数据（RWD/RWE）应用于监管决策和卫生技术评估（HTA）方面的实践经验，提出了数据整合、证据质量评价和监管路径对接的方法学建议。
AI 主编 · 推荐理由推荐理由
EMA与各国HTA机构正在推进RWE应用落地，本文提供系统性方法论参考。拟申报RWE支持注册或HTA申报的申办方及药企注册团队，可借此了解欧盟层面证据要求与最佳实践，提前规划数据收集方案。