使用真实数据对瘤学试验的模拟:一个系统的文献综述
Jennifer R Rider1,2, Asher Wasserman3, Lukas Slipski2
1Aetion, Inc., New York, New York, USA.
American journal of epidemiology
|September 10, 2024
概括
本系统性审查评估了使用真实世界数据 (RWD) 模拟的瘤随机对照试验 (RCT). 虽然许多仿真结果与RCT相似,但影响性能的因素需要进一步研究.
科学领域:
- 药理流行病学 药理流行病学
- 医疗保健服务研究 医疗服务研究
- 生物统计学 生物统计学
背景情况:
- 现实世界数据 (RWD) 越来越多地用于模拟随机对照试验 (RCT) 研究.
- 评估这些仿真的性能对于理解它们在瘤学研究中的有效性和实用性至关重要.
研究的目的:
- 系统地审查使用RWD发表的瘤学RCT仿真.
- 确定影响这些仿真性能的因素.
主要方法:
- 在PubMed的系统文献搜索中,搜索到2022年的瘤学仿真研究.
- 使用ROBINS-I工具对偏差风险和对目标试验结果进行模拟性能评估的研究评估.
- 分析诸如偏差风险和RCT进入标准的代理等因素.
主要成果:
- 确定了13项使用RWD的瘤仿真研究.
- 大多数研究 (8/13) 存在严重的偏差风险,主要是由于混.
- 在11项有足够细节的研究中,有9项在试验的95%置信区间内显示了仿真危险比率估计.
- 在偏差风险或入口标准代理和仿真性能之间没有明显的趋势.
结论:
- 使用RWD的瘤RCT仿真可以实现与目标试验相匹配的性能.
- 需要进一步开发工具,以全面描述影响仿真性能的因素,包括临床背景.
- 出版偏见和研究人员的自由度可能会影响研究结果,突出需要协议预注册.
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