研究者发起的儿童罕见病临床研究合规风险管理——医疗机构视角  

Compliance Risk Management of Investigator Initiated Trials on Children Rare Diseases:Medical Institution Perspective

作　　者：张婧绮 左连东 高雪琪 斯文越 罗睿 吴强 周文浩 ZHANG Jingqi;ZUO Liandong;GAO Xueqi;SI Wenyue;LUO Rui;WU Qiang;ZHOU Wenhao(Guangzhou Women and Children′s Medical Center,Guangzhou 510623,China)

机构地区：[1]广州医科大学附属妇女儿童医疗中心,广州510623

出　　处：《罕见病研究》2025年第1期132-138,共7页Journal of Rare Diseases

基　　金：广州市科技计划项目(2024A03J1127)。

摘　　要：儿童罕见病领域存在巨大的未被满足的治疗需求,研究者发起的临床研究(IIT)为新药或新治疗策略的检验和发展提供了重要途径。但由于其受试者群体的特殊性、创新技术的应用以及组织形式的复杂性,这类研究在实施过程中面临立项审批、合同管理、数据保护、利益冲突管理等方面的诸多挑战和合规风险。本研究旨在分析儿童罕见病IIT的特殊性和挑战,梳理相关的法规和监管要求,为医疗机构提供合规风险管理的参考框架,以最大化IIT的益处。通过文献查阅和法律法规分析,结合实际工作经验及其他机构的实践框架,总结了儿童罕见病IIT在受试者特点、创新技术和组织形式方面的特殊性。在此基础上,提出了针对性的合规管理建议,包括建立风险评级和全周期风险监察机制、符合儿童受试者特点的知情同意和伦理审查机制、稳健的合同管理机制、全流程数据安全管理机制以及多学科团队和多渠道补偿机制。研究结论强调,医疗卫生机构、资助方及其他合作主体应结合IIT的特点,实施合规性管理,以保障研究的安全性和有效性,推动儿童罕见病治疗领域的创新与发展。There is a substantial unmet need for treatments in the field of pediatric rare diseases,and investigator initiated trial(IIT)provide a critical pathway for testing and developing new drugs or treatment strategies.However,healthcare institutions,when conducting such research,must address compliance risks related to project approval,contract management,data protection,and conflict of interest management.This study aims to analyze the particularities and challenges of IIT in pediatric rare diseases,review relevant regulations and regulatory requirements,and provide healthcare institutions with a reference framework for compliance risk management to maximize the benefits of IIT.Based on literature review,analysis of laws and regulations,practical work experience,and frameworks from other institutions,we summarize the unique aspects of pediatric rare disease IIT in terms of participant characteristics,innovative technologies,and organizational structures.On this basis,targeted compliance management recommendations are proposed,which include establishing a risk rating and full-cycle risk monitoring mechanism,a consent and ethical review mechanism tailored to pediatric participants,a robust contract management mechanism,a comprehensive data security management mechanism,and a multidisciplinary team and multi-channel compensation mechanism.The study concludes that healthcare institutions,funders,and other collaborating entities should implement compliance management in line with the characteristics of IIT to ensure the safety and effectiveness of research and facilitate innovation and development in the treatment of pediatric rare diseases.

关 键 词：研究者发起的临床研究 儿童 罕见病 合规风险 

分 类 号：R-052[医药卫生]