罕见病药物临床试验的伦理审查要点  

Elements for Ethic Review of Clinical Trials for Rare Disease Drugs

作　　者：白桦[1] BAI Hua(Department of Scientific Research,Peking Union Medical College Hospital,Chinese Academy of Medical Sciences&Peking Union Medical College,Beijing 100730,China)

机构地区：[1]中国医学科学院北京协和医院科研处,北京100730

出　　处：《罕见病研究》2024年第2期181-186,共6页Journal of Rare Diseases

摘　　要：近10年来,随着促进罕见病药物研发的政策落地,罕见病药物临床试验数量增加,给伦理审查带来新的挑战。罕见病药物临床试验涉及弱势人群,患者数量少、居住分散、相对集中于部分疾病领域,试验以早期为主,往往需要采用小样本、单臂设计,并且更多采用细胞、基因治疗等新技术手段。伦理审查除了满足常见病临床试验的一般要求外,还应当在风险获益评估、研究设计、知情同意、组织实施等方面根据罕见病的特殊性给予特殊的关注,切实促进罕见病新药研发和保护受试者的权益。本文通过分析罕见病药物临床试验的特点,提出伦理审查委员会在审查时需要特别关注的审查要点,旨在提高罕见病相关药物临床试验的伦理审查水平。In the past decade,with the implementation of policies to promote the research and development of rare disease drugs,the increase in the number of clinical trials for rare disease drugs has posed new challenges to ethical review.Clinical trials of rare disease drugs involve vulnerable populations with a small number of patients,scattered populations,and relative concentration in certain disease areas.The trials are mainly conducted in the early stages,often requiring small sample sizes,single arm designs,and more new technologies such as cellular and gene therapy.In addition to meeting the general requirements of clinical trials for common diseases,ethical review should also give special attention to the special characteristics of rare diseases in risk benefit assessment,research design,informed consent,trial operation,etc.Ethical review of clinical trials for rare diseases should effectively promote the development of new drugs for rare diseases and protect the rights and interests of participants.By analyzing the characteristics of clinical trials for rare diseases drugs,this paper presents the points of special concern during the review,aiming to improve the level of ethical review for clinical trials of drugs related to rare diseases.

关 键 词：罕见病 孤儿药 临床试验 伦理审查 

分 类 号：R-052[医药卫生]