美国临床试验用药物扩大使用制度沿革与发展

Evolution and development of the expanded access of investigational new drugs in the United States of America

通过查阅美国法律法规、指南文件、国内外文献,深入分析美国扩大使用临床试验用药物的立法背景、制度要素以及实施现状,为我国临床试验用药物扩大使用相关法规建立提供建议。美国扩大使用制度经历了雏形、初建、发展完善3个阶段。美国临床试验用药物扩大使用法律定义明确且具有灵活性;扩大使用监管对象按患者数量和风险控制划分3类;扩大使用申请要求与批准程序根据申请类型、申请主体简化安排;风险保障措施完备,包括知情同意、伦理审查、安全报告。建议我国从保护和促进公众健康出发,引入建立适合我国国情的扩大使用制度,并明确扩大使用的类型和适用情况;建立扩大使用申请和简化程序;采取必要的患者保障和风险控制措施。

In this paper, we reviewed FDA laws and regulations, guidance documents, domestic and foreign literature, and analyzed the expanded access in the areas of legislative background, institutional elements and the status quo, for providing suggestions for the establishment of relevant laws and regulations in China. The system of expanded access in the United States has undergone three stages： prototype, initial construction, and development and perfection. With clear and flexible legal definition and applicability, the expanded access divided into three categories and has adequate risk control, including informed consent, ethical review, and safety report. It is suggested that China should establish an expanded access system suitable for the situation of our country from the point of protecting and promoting public health, and make clear the type and application of expanded access. Also, the submission and review system as well as necessary measures of patient protection and risk control should be established.