罕见病临床研究的伦理审查现况及问题研究  

Research on the current situation and issues of ethical review of clinical research on rare diseases

作　　者：朱乐兰 马磊[2] 阮栾绮 耿雯倩 ZHU Yuelan;MA Lei;RUAN Luanqi;GENG Wenqian(Department of Medical Affairs,Shanghai General Hospital,Shanghai 200080,China;Department of Scientific Research,Shanghai General Hospital,Shanghai 200080,China)

机构地区：[1]上海市第一人民医院医务处,上海200080 [2]上海市第一人民医院科研处,上海200080

出　　处：《中国医学伦理学》2025年第4期434-440,共7页Chinese Medical Ethics

基　　金：2023年度上海申康医院发展中心市级医院诊疗技术推广及优化管理项目“基于瞬感动态血糖监测系统的一体化全院血糖管理模式探讨”(SHDC12023634)。

摘　　要：罕见病临床研究的伦理审查是确保研究科学性和受试者权益的关键环节。从罕见病的定义出发,分析了国内外在罕见病临床研究方面的法规、伦理审查的现状,并从临床研究的科学性和伦理性两个维度,探讨包括研究目的、方法、风险与获益评估、研究人员资质、研究基础设施、知情同意流程、数据安全和隐私保护、儿童等弱势群体的保护等伦理审查的重点要素。针对罕见病临床研究伦理审查,可通过加强伦理审查人员培训、开展多中心协作审查、关注试验长期安全性等策略,来提升伦理审查质量,保护受试者安全,确保临床研究的效率和质量。The ethical review of clinical research on rare diseases is crucial in ensuring the scientific validity of the research and the rights and interests of the subjects.Starting from the definition of rare diseases,this paper analyzed the current situation of domestic and international regulations and ethical review in clinical research on rare diseases.It also explored the key elements of ethical review from the two dimensions of scientific and ethical aspects of clinical research,including research objectives,methods,risk and benefit assessment,researcher qualifications,research infrastructure,informed consent process,data security and privacy protection,and protection of vulnerable groups such as children.Regarding the ethical review of clinical research on rare diseases,strategies can be adopted such as strengthening the training of ethics review personnel,conducting multi-center collaborative reviews,and focusing on the long-term safety of trials,to improve the quality of ethical review,protect the safety of the subjects,and ensure the efficiency and quality of clinical research.

关 键 词：罕见病 临床研究 伦理审查 

分 类 号：R-052[医药卫生]