基于变化点的分析程序,以提高临床试验中决策成功率,延迟治疗效果
1Department of Bioinformatics and Biostatistics, School of Life Sciences and Biotechnology, SJTU-Yale Joint Center for Biostatistics and Data Science, Shanghai Jiao Tong University, Shanghai, China.
Frontiers in pharmacology
|September 28, 2023
概括
在免疫疗法试验中延迟治疗效应可能导致错误的决定. 本研究引入了变化点分析,以提高中间分析决策成功率,确保可靠的去/不去结果.
科学领域:
- 临床试验 临床试验
- 生物统计学 生物统计学
- 癌症免疫疗法癌症免疫疗法
背景情况:
- 延迟治疗效应在瘤免疫治疗临床试验中很常见,影响了统计能力和解释.
- 这种现象使无或组序列设计中的中间分析复杂化,可能导致错误的去/不去决策.
- 有限的研究解决了延迟影响对中期分析决策成功和缓解策略的影响.
研究的目的:
- 建议和评估一种新的分析程序,以提高临床试验中期分析期间的决策成功率,以延迟治疗效果.
- 解决延迟治疗对适应性试验设计中的统计能力和决策效应所带来的挑战.
主要方法:
- 开发了一个三步分析程序: 1.检测和测试变化点, 2.估计治疗疗效, 3.做出去/不去的决定.
- 利用模拟研究来评估在延迟治疗效应的情况下程序的性能.
- 根据各种假设,比较拟议的方法与未经调整的方法,包括比例危险.
主要成果:
- 建议的变化点分析程序显著提高了在有延迟治疗效应的情况下,在中间分析时的决策成功率.
- 该方法有效控制了I型错误率.
- 当符合比例危险假设时,性能与未调整的方法相比,这表明稳定性.
结论:
- 拟议的基于变化点的分析程序提供了一种可行和有效的方法,用于在临床试验中进行中期分析时,在延迟治疗效果的情况下进行可靠的决策.
- 这种方法可以减轻错误的去/不去决定的风险,从而提高试验的效率和可靠性.
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