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.
Abstract
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.
基金项目
国家中医药局青年岐黄学者支持项目(国中医药人教函[2020]218号)
国家中医药局青年岐黄学者支持项目(YL2202020414)
组分中药国家重点实验室青苗基金(YL230102013723)
国家药监局药品监管科学体系建设重点项目(RS2024Z004)
万方数据