摘要
目的 分析我国孤儿药临床试验的现状和面临的挑战,从受试者入组情况和随访依从性2个方面提出可操作的建议或者意见。方法 通过检索2018年1月至2021年6月在“临床试验登记与信息公示平台”上登记的孤儿药临床试验,分析我国孤儿药临床试验的现状及面临的挑战。采用半结构化访谈的方式,对15位专家就如何提高受试者入组数量以及如何改善受试者随访依从性2个方面进行访谈。结果 共检索到68项孤儿药临床试验,其中近43.9%的孤儿药临床试验未达到目标入组人数,并且临床试验机构地区的选择主要分布在我国经济较发达的城市。通过对15位专家的访谈,针对受试者入组和受试者随访2个问题提出的解决措施,包括:申办方制定以患者为中心的研究设计,特别是在目标人群的考量上;研究者以及机构对罕见病受试者全病程管理;扩大各级转诊网络并建立罕见病信息网,以及推进罕见病联盟的工作;将多方支付的概念纳入临床试验,对受试者随访费用进行时报时销,以及实施远程随访以提高受试者随访的依从性。结论 通过专家访谈,初步提出了一些可操作的针对如何提高受试者入组数量和改善受试者随访依从性的思路和解决措施,为完善孤儿药临床试验的设计和管理提供了一定的指导和参考。
AIM To analyze the status of clinical trials of orphan drugs in China and provide the feasible suggestions or opinions from two aspects of the enrollment of subjects and the follow-up compliance of subjects.METHODS The status and challenges of clinical trials of orphan drugs in China were analyzed by searching for orphan drug clinical trials registered on the "Clinical Trial Registration and Information Disclosure Platform" from January 2018 to June 2021.Totally 15 experts were interviewed on the consideration of increasing the enrollment of subjects and improving the follow-up compliance of subjects.RESULTS A total of 68 orphan drug clinical trials were retrieved,and nearly 43.9% of the orphan drug clinical trials failed to reach the target enrollment number,and the sites selected were mainly located in economically developed cities in China.Through interviewing with 15 experts,the solutions proposed for the enrollment of subjects and follow-up of subjects included:The sponsor developed patient-centered study design,especially for the consideration of targeted population.Investigators and institutions developed the whole course disease management for rare disease subjects.The referral network at all levels was expanded and a rare disease information network was established,and the work of the Rare Disease Alliance was promoted.Multi-party payment practice was incorporated into clinical trial,timely payment procedures for follow-up expenses of subjects were carried out,and remote follow-up visit was implemented to improve subjects compliance.CONCLUSION This study has preliminarily explored some feasible solutions and strategies on how to improve subject enrollment and improve subject follow-up compliance and provides some guidance and reference for improving the design and management of orphan drug clinical trials in China.
作者
周姚
向瑾
李秀杰
于锋
张庆
ZHOU Yao;XIANG Jin;LI Xiujie;YU Feng;ZHANG Qing(School of Basic Medicine and Clinical Pharmacy,China Pharmaceutical University,Nanjing 210009,China;Clinical Trial Center,West China Hospital of Sichuan University,Chengdu 610041,China;Center of Clinical Operations Excellence,IQVIA,Shanghai 200124,China)
出处
《中国临床药学杂志》
CAS
2023年第2期81-87,共7页
Chinese Journal of Clinical Pharmacy
关键词
罕见病
孤儿药
临床试验
入组
随访
rare disease
orphan drug
clinical trial
enrollment
follow-up
