乳腺癌药物的HTA决策的替代终点:实用性和陷
Kristin Wright1, Abhenil Mittal2, Bishal Gyawali1,3,4
1Department of Oncology, Queen's University.
Current opinion in oncology
|August 25, 2023
概括
乳腺癌试验中的大多数代孕终点不能可靠地预测整体存活率或生活质量. 这对公共药物资助决策的健康技术评估 (HTA) 提出了挑战.
科学领域:
- 在瘤学瘤学.
- 卫生技术评估 卫生技术评估
- 临床试验设计 临床试验设计
背景情况:
- 卫生技术评估 (HTA) 对于确定癌症药物的公共资金至关重要.
- 临床试验越来越多地使用代用终点,特别是在乳腺癌研究中.
研究的目的:
- 审查和综合证据验证的替代终点在乳腺癌.
- 评估这些替代终点在癌症药物补偿HTA决策中的作用.
主要方法:
- 文献审查以综合代用终点验证的证据.
- 替代终点效用与整体存活率和生活质量有关的分析.
主要成果:
- 在HER2阳性早期乳腺癌中,无病生存率 (DFS) 是唯一经过验证的代孕终点.
- 病理完整反应 (pCR),无事件生存 (EFS),客观反应率 (ORR) 和无进展生存 (PFS) 缺乏整体生存 (OS) 的验证.
- 生活质量 (QOL) 替代终点尚未确定,一些改善PFS的药物可能会对QOL产生负面影响.
结论:
- 许多用于乳腺癌研究的代用终点不能准确预测整体存活率或生活质量.
- 缺乏经过验证的代用终点使得HTA对公共药物资金的决策变得复杂.
- 这些发现对于HTA机构做出癌症药物补偿决定至关重要.
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