摘要
为了在临床试验中充分考虑患者相关因素,美国食品和药品监督管理局制定了一系列“以患者为中心的药物研发”的指导原则。本文围绕指导原则3“选择与研发合适的临床结局评估”,对临床结局评估的内涵、研制流程、评分机制、干扰因素及敏感性五个方面进行概述,并介绍四种常见类型的临床结局评估,为以患者为中心的临床试验结局评价工作实施提供参考。
In order to better incorporate patient input in clinical trials,the US Food and Drug Administration has included"patient-focused drug development"in the selection and development of clinical outcome assessments,and formulated a series of guidelines.Based on the third guiding principle,"Selecting,Developing,or Modifying Fit-for-Purpose Clinical Outcome Assessments",this article summarizes the clinical outcome assessments from five aspects:concept,development process,scoring mechanism,interference factors and sensitivity,and introduces four different types of clinical outcome assessments,providing new ideas for"patient-focused drug development"efficacy evaluation in clinical trials.
作者
张晨瑶
吴晓蕾
史梦龙
马毓聪
庞稳泰
杜亮
刘炳林
曹璐佳
张俊华
ZHANG Chenyao;WU Xiaolei;SHI Menglong;MA Yucong;PANG Wentai;DU Liang;LIU Binglin;CAO Lujia;ZHANG Junhua(Evidence-based Medicine Center,Tianjin University of Traditional Chinese Medicine,Tianjin 301617,P.R.China;Chinese Evidence-based Medicine Center,West China Hospital of Sichuan University,Chengdu 610041,P.R.China;Center for Drug Evaluation,China Food and Drug Administration,Beijing 100022,P.R.China)
出处
《中国循证医学杂志》
CSCD
北大核心
2024年第4期472-477,共6页
Chinese Journal of Evidence-based Medicine
基金
国家中医药管理局青年岐黄学者支持项目(国中医药人教函〔2020〕218号)(编号:YL2202020414)
组分中药国家重点实验室青苗基金(编号:YL230102013723)
国家药监局药品监管科学体系建设重点项目(编号:RS2024Z004)。
关键词
以患者为中心
药物研发
临床结局评估
Patient-focused
Drug development
Clinical outcome assessments