通过模拟研究来比较使用危险比点估计或置信区间限制的时间到事件终点的额外效益评估方法
Christopher A Büsch1, Marietta Kirchner1, Rouven Behnisch1
1Institute of Medical Biometry (IMBI), Department Medical Biometry, Heidelberg University, Heidelberg, Germany.
概括
我们比较了评估新疗法的临床益处的三种方法 (ASCO,ESMO,IQWiG). IQWiG和ASCO比ESMO更保守,这可能会过度补偿治疗效应.
科学领域:
- 卫生经济学 卫生经济学
- 临床试验分析
- 生物统计学 生物统计学
背景情况:
- 三种方法对时间到事件的终点评估临床益处:ASCO (危险比率点估计),ESMO (下方95%HR置信区间) 和IQWiG (上方95%HR置信区间).
- 这些框架旨在对新批准的治疗方法进行公正的评估.
研究的目的:
- 描述和比较ASCO,ESMO和IQWiG的好处评估框架.
- 为了确定与ESMO和IQWiG类别相一致的ASCO分数,以便进行公平的比较.
主要方法:
- 一个全面的模拟研究使用斯皮尔曼的相关性和描述性统计评估方法.
- 一个加权的科恩·卡帕方法确定了与ESMO/IQWiG类别相对应的ASCO值.
主要成果:
- 在ASCO和IQWiG之间发现了高度的正相关性.
- 在ASCO和ESMO之间观察到较低的正相关性.
- 针对ESMO (<17, 1720, 2024, >24) 和IQWiG (21和38的截止值) 定义了特定的ASCO分数门.
结论:
- IQWiG和ASCO比ESMO更保守,ESMO倾向于授予最大类别.
- ASCO与IQWiG具有共同的特点,而ESMO是最自由的评估方法.
- 延迟治疗效应和研究功率影响所有方法,ESMO受影响最大.
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