真实世界证据用于罕见病监管决策的思考  

Considerations on use of real-world evidence in regulatory decision-making for rare diseases

作　　者：张晓敏 刘晓溪[1] 黄哲[1,2] ZHANG Xiao-min;LIU Xiao-xi;HUANG Zhe(College of Business Administration,Shenyang Pharmaceutical University;Research Institute of Drug Regulatory Science,Shenyang Pharmaceutical University,Shenyang 110016,China)

机构地区：[1]沈阳药科大学工商管理学院 [2]沈阳药科大学药品监管科学研究院,沈阳110016

出　　处：《中国新药杂志》2024年第22期2314-2317,共4页Chinese Journal of New Drugs

摘　　要：真实世界证据在支持药品监管决策中的应用正受到广泛而热烈的讨论。全球多国药品监管机构纷纷发布真实世界证据相关政策和指南,积极研究和探索真实世界证据如何更好地用于支持监管决策。对于罕见病而言,因其发病率低、患者数量少、病情复杂且严重危及生命等疾病特点,导致传统的随机对照临床试验较难开展。而来源于真实临床环境中的真实世界证据为罕见病研究提供了一条新路径。本文对真实世界证据用于罕见病监管决策的现状进行综述,对应用过程中遇到的问题进行分析,并且提出合理的解决建议,以期为真实世界证据支持罕见病研发提供参考。Real-world evidence(RWE)has been actively discussed in terms of utilization for regulatory decision-making on the benefit/risk assessment of a drug.Regulatory agencies from multiple countries around the world have released some specific policies and guidelines which are related to RWE,to actively evaluate and explore ways to optimize the utility of RWE to support regulatory decision-making.For rare diseases,it may not always be feasible to conduct traditional randomized control clinical trials because of the characteristics of rare diseases,such as low morbidity,small number of patients,and complex and serious life-threatening conditions.Thus,the evidence derived from actual clinical practices could provide a new approach to rare disease research.This paper reviewed the current situation on the use of RWE in regulatory decision-making for rare diseases,analyzed the issues of RWE in regulatory decision-making,and proposed some reasonable suggestions to provide references for the development of drugs for rare diseases.

关 键 词：真实世界证据 罕见病 孤儿药 监管决策 

分 类 号：R95[医药卫生—药学]