目的 为探索FDA在药品审评中促进替代终点开发和使用的监管模式和理念。方法 笔者参考了FDA的相关法规、指导性文件和指南以及国内外文献,经过详细梳理,通过对替代终点药品开发以及监管接受历史进行追溯, FDA制定的促进替代终点科学开发模式进行解读,并结合替代终点在癌症、艾滋病、糖尿病等严重疾病领域中广泛应用的现状。结果与结论 归纳得出FDA对于促进替代终点监管与使用的经验主要有3点,包括制定多种指南促进替代终点理解;多种程序结合推动替代终点发展;替代终点使用基于风险分类监管利于资源配置。
Abstract
OBJECTIVE To figure out the regulatory models and concepts of FDA in drug review process that promotes surrogate endpoints development. METHODS This paper made a thorough research to the FDA regulations, guidance documents and guidelines and literatures, and through historical retrospective of surrogate endpoints used in clinical drug trials and regulatory acceptance, interpretation of FDA's established development model in scientific use of surrogates, combined with the status quo surrogate endpoints in the field of serious diseases as cancer, AIDS, diabetes. RESULTS AND CONCLUSION Then conclusion of FDA's experience in promoting regulation and utility of surrogate endpoints is summed up, including the development of guidelines to promote common understanding of specific use of surrogates; combining a variety of programs promote the development of surrogate endpoints; and using a risk-based classification in favor of the allocation of resources.
关键词
美国食品药品监督管理局 /
生物标记物 /
替代终点 /
艾滋病 /
肿瘤
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Key words
FDA /
biomarkers /
surrogate endpoint /
AIDS /
cancer
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中图分类号：
R95
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参考文献
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