将试验衍生治疗效应应用于现实世界的人口:在建模复杂危险时,将成本效益估计概括为模型
Ian Koblbauer1, Daniel Prieto-Alhambra2, Edward Burn3
1Centre for Statistics in Medicine, Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences, University of Oxford, Oxford, England, UK.
概括
将试验的成本效益推广到现实世界的人口中需要复杂危险的灵活模型. 准确地捕捉外部基线中的生存率对于可靠的经济评估至关重要.
科学领域:
- 卫生经济学 卫生经济学
- 生物统计学 生物统计学
- 临床试验分析
背景情况:
- 基于试验的成本效益估计对现实世界人口的概括性对于医疗保健决策至关重要.
- 时间到事件数据中的复杂危险对使用聚合数据进行经济建模提出了挑战.
研究的目的:
- 为应用试验衍生的相对治疗效应应用于外部现实世界的基线,特别是复杂危险的方法提供概述.
- 用头癌治疗的成本效益分析来证明这些方法.
主要方法:
- 提出了在存在复杂危险的情况下将试验衍生的相对效应应用于现实世界的基线的方法.
- 作为一个激励的例子,利用了一项发表的关于头癌治疗的研究的成本效益分析.
主要成果:
- 复杂的基线危险和不成比例的影响需要灵活的建模来准确估计生存时间.
- 关于试验和现实世界人口之间的共同生存分布的假设显著影响了生存率和成本效益估计.
- 建模时间依赖与比例相对效应对估计的影响较小.
结论:
- 当将试验效应推广到真实世界的人口时,准确估计参考治疗生存率至关重要,这会影响成本效益.
- 在评估具有决策复杂风险的多个数据源时,模型复杂性和充分性之间的平衡是必不可少的.
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