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美国FDA加速批准和随后的撤销:对日本临床瘤学实践指南的影响

Hayase Hakariya^1,2, Akihiko Ozaki^3,4, Tetsuya Tanimoto^4,5

¹Interfaculty Institute of Biochemistry, University of Tuebingen, 72076, Tuebingen, Germany. hayase.hakariya@uni-tuebingen.de.

Investigational new drugs

|April 3, 2025

概括

一些在美国被撤销的癌症药物在日本仍然得到批准. 日本的指导方针在七种癌症药物中推了四种,尽管美国因未经证实的益处而退出,这突出了全球监管差异.

科学领域:

瘤学药物监管药物监管
全球卫生保健政策全球卫生保健政策
临床试验证据评估

背景情况:

美国FDA的加速批准途径允许基于代用终点的癌症药物的早期获取,需要营销后试验.
在监管决策中存在显著的全球差异,一些美国撤回的瘤药物仍在日本获得批准.

研究的目的:

分析日本专业社会对美国市场撤销但在日本保留的七种瘤药物的指导方针.
在日本指南中评估这些药物的证据水平和治疗偏好.

主要方法:

对日本职业社会指导方针的横截面分析.
对七种特定的瘤药物加速批准建议的评估.
评估证据水平和治疗偏好等级.

主要成果:

在七种药物中,有四种药物 (57%) 在日本指南中获得了高或中等偏好:gemtuzumab ozogamicin,gefitinib,bevacizumab和atezolizumab.
在美国和日本监管机构的利益风险评估中发现了差异.
像gemtuzumab ozogamicin和gefitinib这样的药物具有有争议的益处数据,但仍被推.

结论:

日本的指导方针建议因未经证实的益处而在美国撤回瘤药物,这表明区域监管和评估差异.
日本的监管决策和指导方针建议需要更加透明.
与美国评估相比,批准具有可疑临床益处的药物需要更明确的理由.

关键词:

加快审批的方法是加速审批.准则建议准则建议日本日本日本日本日本.目标响应率目标响应率制药和医疗器械机构制药和医疗器械机构没有进展的生存率.监管机构监管机构美国食品和药物管理局.

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