在罕见病药物临床研发中应用去中心化临床试验的考虑  

Application of Decentralized Clinical Trials in the Research and Development of Drugs for Rare Diseases

作　　者：崔欢欢 唐凌 崔灿 姚珠星 杨志敏 王海学 CUI Huanhuan;TANG Ling;CUI Can;YAO Zhuxing;YANG Zhimin;WANG Haixue(Center for Drug Evaluation,National Medical Products Administration,Beijing 100076,China)

机构地区：[1]国家药品监督管理局药品审评中心,北京100076

出　　处：《罕见病研究》2024年第2期175-180,共6页Journal of Rare Diseases

摘　　要：罕见病药物临床试验面临患者人数少、招募困难、试验周期长、试验期间访视频次多等特殊挑战。在罕见病药物临床研发中,实施“以患者为中心”为研发理念的去中心化临床试验(DCT)模式,借助去中心化元素和数字健康技术,可打破地域限制,受试者可不局限于传统临床试验实施现场(医院/研究中心),能够显著减轻受试者负担、增加受试者的代表性、获得更加广泛科学的研究数据。为指导行业在罕见病药物研发中科学规范的应用DCT,国家药品监督管理局药品审评中心组织起草了《在罕见疾病药物研发中应用去中心化临床试验的技术指导原则》。指导原则为罕见病药物研发实施DCT提供了科学建议,旨在解决罕见病药物研发难点痛点、提高研发效率、优化患者体验。本文将结合该指导原则中的研发理念,阐述当前在罕见病药物研发中应用DCT的研发思考,以期为业界提供参考。Clinical trials of drugs for rare diseases face special challenges such as a limited number of patients,difficult recruitment,long trial period,and frequent video interviews during the trial.Therefore,in the clinical operation of rare diseases,a decentralized clinical trials(DCT)model based on the"patientcentred"research and development concept is implemented.With the help of decentralized elements and digital health technology,the barriers of geographical restrictions can be overcome and subjects do not have to be limited to traditional clinical trial sites(hospitals/research centers),which can significantly reduce the burden on subjects,increase their representation,and obtain a wider range of scientific research data.To guide the industry′s scientific and standardized application of DCT in the research and development of drugs for rare diseases,the Center for Drug Evaluation of the National Medical Products Administration(NMPA)organized the stake holders to draft the Technical Guideline for the Application of Decentralized Clinical Trials in the Research and Development of Drugs for Rare Diseases.This guideline provides scientific recommendations for the development and implementation of DCT for rare disease drugs.It aims to solve the difficult and key problems during rare disease drug research and development,improve the efficiency and optimize patient experience.This article,combining the research and development concepts in the guideline,explains the current research and development thinking on the application of DCT in the research and development of rare disease drugs,with a view of providing reference for the industry.

关 键 词：罕见病 去中心化临床试验 药物 临床研发 

分 类 号：R-33[医药卫生]