《肿瘤临床试验运用核心患者报告结局行业指南(草案)》解读

Chinese Joumal of New Drugs 2023,32(7)药物临床试验规范与进展专栏·《肿瘤临床试验运用核心患者报告结局行业指南（草案）》解读刘铭睿，姚弥23，周慧，高翔，元刚56(1中山大学药学院，广州510006：2北京大学第一医院全科医学科，北京100034：3北京大学医学部全科医学学系，北京100191：4中山大学附属第一医院药学部，广州510080：5中山大学附属第一医院老年医学科，广州510080：6中山大学附属第一医院I期临床试验中心，广州510080)[摘要]以患者为中心的药物研发理念愈发受到国内外研究者的重视，患者报告结局(patient-reportedoutcome,PRO)已逐渐成为临床试验的终点观察指标之一。PR0提供直接来自患者的健康信息，在医生的临床决策中起到重要作用。但肿瘤临床试验中PR0评估方法的运用和数据解释还有待改进。核心PR0指标的选用对PR0评估标准的一致性至关重要。2021年美国FDA发布的《肿瘤临床试验运用核心患者报告结局行业指南（草案）》针对肿瘤临床试验的药物研发，为申办方核心PRO收集、PR0评估工具选择和规范化试验设计提供指导意见。本文对该指南核心PR0的系统评估及运用进行解读，帮助新药临床试验从业者进一步了解PRO,对PRO在新药临床试验中的运用起到良好的促进作用。[关键词]肿瘤临床试验：患者报告结局：试验设计[中图分类号]R969.4[文献标志码]A[文章编号]1003-3734(2023)07-0719-05Interpretation of the "Core Patient-reported Outcomes in Cancer ClinicalTrials Guidance for Industry draft)"LIU Ming-rui',YAO Mi23,ZHOU Hui,GAO Xiang',YUAN Gang3.6(1 School of Pharmaceutical Sciences,Sun Yat-Sen University,Guangzhou 510006,China:2 Department ofGeneral Practice,Peking University First Hospital,Beijing 100034,China;3 Department of General Practice,Peking University Health Science Center,Beijing 100191,China;4 Department of Pharmacy,the First AffiliatedHospital,Sun Yat-Sen University,Guangzhou 510080,China;5 Department of Geriatrics,the First AffiliatedHospital,Sun Yat-Sen University,Guangzhou 510080,China;6 Phase I Clinical Trial Center,the FirstAffiliated Hospital,Sun Yat-Sen University,Guangzhou 510080,China)Abstract]The concept of patient-focused drug development has been paid increasing attention by researchersin China and abroad.Patient-reported outcome PRO)has gradually become one of the endpoints in clinical trials.PRO provides health information directly from the patient and plays an important role in the clinical decision makingby physicians.However,the use of PRO assessment and data interpretation in oncology clinical trials are still waitingfor further improvement.The selection of core PRO indicators is critical to the consistency of PRO assessment criteria.In 2021,U.S.FDA issued the "Core Patient-Reported Outcomes in Cancer Clinical Trials Guidance for Industrydraft)"for drug development in cancer clinical trials,providing guidance for sponsors on core PRO collection,PRO assessment tool selection,and standardized trial design.This paper interprets the systematic assessment andapplication of core PRO in the guideline,helps practitioners of new drug clinical trials to further understand PRO[作者简介]刘铭春，女，项士研究生，主要从事药理学研究。E-mail:liun6@mmi卫.sys.ed加.cn[通讯作者]元刚，男，酬主任医师，主要从事药物早期格床试验研究、药物怡床试验管理工作。E-mail:yuangang(@mail.ss.e山.cn。719中国新药杂志2023年第32卷第7期(C)1994-2023 China Academic Journal Electronic Publishing House.All rights reserved.http://www.cnki.net