姚峥嵘 阮未艾 王艳翚 张彧

摘 要 目的：為完善我国拓展性临床试验制度建设提供参考。方法：从制度建立、适用范围与原则、申请审批情况等3个方面介绍国际拓展性临床试验制度的实施经验，进一步分析我国拓展性临床试验制度的沿革及面临的挑战，并提出相关建议。结果与结论：美国、英国、澳大利亚以及欧盟等国家/地区拓展性临床试验制度的发展已较为成熟。而当前我国尚未正式出台拓展性临床试验制度的具体政策，在实施中也面临诸多挑战，如用药风险等带来的伦理问题，发起申请的阻力与收益间难以平衡而产生的发起者困境，以及风险评估责任方未明、审批时间紧急等导致的审查难点较多等。建议我国可通过完善现有法律法规，加强信息公开与风险预案工作，明确制度运行过程中的责任划分等措施予以应对;同时可借鉴国际现有模式，搭建我国拓展性临床试验管理体系，针对“单个患者紧急情况”“单个患者非紧急情况”和“两人以上群组患者”等不同申请类型设置相应的申请路径并发动各方力量合力监管，以促进拓展性临床试验制度的完善和落实。

关键词 拓展性临床试验;同情用药;现状;挑战;启示

International Status of the Extended Clinical Trial System and Its Enlightenment to China

YAO Zhengrong，RUAN Weiai，WANG Yanhui，ZHANG Yu（School of Health Economics and Management， Nanjing University of Chinese Medcine， Nanjing 210023， China）

ABSTRACT   OBJECTIVE： To provide reference for improving extended clinical trial system in China. METHODS： The implementation experience of the international extended clinical trial system was introduced from three aspects： system development， application scope and principles， application and approval; the evolution and challenges of the system in China were further analyzed， and relative suggestions were put forward. RESULTS & CONCLUSIONS： The development of international extended clinical trial system in the United States， the United Kingdom， Australia and the European Union had been relatively mature. The specific system had not formally been established in China， and the implementation of the system faced many challenges， such as the ethical problems caused by drug use risk， the sponsor dilemma caused by the difficult balance between the resistance of initiating application and the benefits， the difficulty of review caused by the unknown responsible party of risk assessment and the urgent time of approval. It can be dealt with by the following measures： improving the existing laws and regulations， strengthen information disclosure and risk prevention work， make clear the division of responsibilities in the process of system operation. Meanwhile， based on the existing international model， the management system of the expanded clinical trials in China is established. Corresponding application paths are set up for different application types such as “single patient emergency”， “single patient non emergency” and “two or more patients group”， and all parties should be mobilized to supervise so as to promote the improvement and implementation of the extended clinical trial system.

KEYWORDS   Extended clinical trial; Compassionate use; Status; Challenge; Enlightenment

2020年初，美国医务人员将仍处于临床试验阶段的核苷类抗病毒药物瑞德西韦（remdesivir）用于治疗新型冠状病毒肺炎。这一举措将拓展性临床试验制度带入了大众的视野[1]。美国对拓展性临床试验制度的最新定义是“患有立即威胁生命的疾病或严重疾病的患者在没有其他替代疗法时，获取研究性药物以进行临床试验之外治疗的措施”[2]。……