用于HTA决策的替代终点评估方法:ISPOR工作组的一份良好实践报告
Sylwia Bujkiewicz1, Oriana Ciani2, Bart Heeg3
1Biostatistics Research Group, Division of Public Health and Epidemiology, School of Medicine, University of Leicester, Leicester, UK.
概括
验证的代用终点对于临床试验决策至关重要. 本报告提供了在医疗技术评估 (HTA) 中评估代用终点的最佳实践,以减少不确定性.
科学领域:
- 卫生经济学与研究成果研究成果
- 临床试验方法论 临床试验方法论
- 生物统计学 生物统计学
背景情况:
- 替代终点通常用于临床试验,但缺乏用于预测患者相关结果的验证.
- 这种不确定性影响了监管机构,医疗技术评估 (HTA) 机构和付款人的决策.
- 缺乏验证会增加医疗保健决策中的不确定性.
研究的目的:
- 为 HTA 决策提供关于评估替代终点的最佳实践的建议.
- 针对不同证据水平的替代终点验证方法.
- 引导在临床研究和医疗保健政策中适当使用代用终点.
主要方法:
- 替代终点验证的方法指南的审查和综合.
- 应用统计方法,包括元分析 (个人参与者数据和汇总数据) 和多变量元分析模型.
- 纳入贝叶斯的方法有限的数据和通用性,并考虑现实世界的数据.
- 调整代孕分析与健康经济模型,包括场景和敏感性分析.
主要成果:
- 建议多变量元分析模型用于强大的替代终点评估.
- 建议贝叶斯的方法和现实世界的数据来解决数据的局限性和通用性.
- 强调将健康经济模型与代孕分析对齐,以评估可信性.
- 提出场景和灵敏度分析,以探索代孕关系中的不确定性.
结论:
- 经过验证的代用终点对于可靠的HTA和临床决策至关重要.
- 坚持推的评估方法可以减少不确定性,提高证据的质量.
- 本报告为HTA的替代终点评估提供了最佳实践框架.
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