目的 为完善我国拓展性临床试验提供思路。方法 通过查阅国内外文献,从国内外发展、特许进口创新药械使用及超说明书用药的异同点3个方面对拓展性临床试验进行对比分析。结果 疾病谱日益演变的背景下,拓展性临床试验因其人文特性及治病需求而具有重要价值,但这一制度在我国尚无实践案例,其运行机制仍有待完善。结论 我国应借鉴国外经验,参考相关非常规用药行为规范,加强政策引导,实现制度的嫁接和运用。
Abstract
OBJECTIVE To provide ideas for the improvement of expanded access trials in China. METHODS By referring to domestic and foreign literatures, this study made a comparative analysis of expanded access trials from three aspects: domestic and foreign development, similarities and differences with special innovative medical device and off-label use. RESULTS Expanded access trials is of great value in the context of the increasingly evolving disease spectrum because of its humanism and treatment needs. However, there are still no practical cases in China and its operating mechanism still needs to be improved. CONCLUSION China should learn from foreign experience, refer to relevant norms of special drug use behavior, strengthen policy guidance, and realize the grafting and application of the system.
关键词
拓展性临床试验 /
特许创新药械 /
超说明书用药
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Key words
expanded access trial /
special innovative medical device /
off-label use
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中图分类号：
R95
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参考文献
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脚注
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基金
江苏省科学技术协会调研课题资助(JSKXKT 2021023)
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