评估转换为癌症药物常规批准后出现的结果 药物指示获得加速批准,1992-2021年
Ariadna Tibau1,2,3,4, Alejandra Romano3,4, Ian T T Liu1,2
1Program On Regulation, Therapeutics, And Law (PORTAL), Division Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women's Hospital, Boston, Massachusetts, USA.
Journal of the National Cancer Institute
|July 21, 2025
概括
在转换为普通批准后,加速瘤学批准的总体存活率或生活质量增长的证据仍然很少. 患者需要在批准时清楚地了解证据变异性的信息.
科学领域:
- 瘤学 药物批准 药物批准
- 临床试验的终点临床试验的终点
- 监管科学 监管科学
背景情况:
- 美国食品和药物管理局的加速批准途径允许基于代孕标记的早期癌症药物获取.
- 需要进行确认性试验来确认临床益处,但通常使用代用措施,而不是整体存活率 (OS) 或生活质量 (QoL).
研究的目的:
- 评估癌症药物的FDA加速批准转换为常规批准时证明临床益处 (OS,QoL,实质性临床益处) 的频率.
- 在转化后的随访中评估OS,QoL或实质性临床益处的出现.
主要方法:
- 对FDA加速批准转换为定期批准 (1992-2021) 的回顾性队列研究.
- 在转换和转换后评估确认性试验终点 (OS,QoL).
- 使用ESMO-MCBS (欧洲医学瘤学会 - 临床益处尺度的大小) 评估临床相关性.
主要成果:
- 只有32%的确认性试验在转换时显示了OS益处;68%使用了代用措施.
- 在转换后,OS益处仅在9%的额外迹象中出现.
- 在转换时,QoL的益处为12%,在转换后为5%.
- 47%的试验在转换时达到ESMO-MCBS评估的实质性临床益处值.
结论:
- 在瘤药物转换为常规批准后,获得OS或QoL增长的新证据是罕见的.
- 确保在转换时可获得可靠的临床益处数据至关重要.
- 建议向患者提供关于证据变异性和ESMO-MCBS的实用性的信息.
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