Assessing the replicability of RCTs in RWE emulations
评估真实世界证据模拟研究对随机试验的可重复性AI 解读 ↓
← 返回论文列表与筛选
阅读说明
本页整理公开书目信息，不生成学术质量评分。AI 解读根据题名及所注明的摘要或原文片段生成，未完成全文核验，可能有误；具体假设、证明与结论请核对原文。摘要保留来源文本，未经模型改写。
DOI 首次登记不等于接收或发表日期。部分更正、评论等条目没有独立摘要；缺少可靠材料时保留待补状态。
本页为预印本版本；arXiv 收录、作者填写的期刊信息以及作者既有发表经历，都不单独证明本篇已接收。作者发表记录按 OpenAlex 身份与完整姓名、本站覆盖期刊 ISSN 及正式卷期核对，只展示已核实例，档案仍可能误合并。
引用量采用 OpenAlex 单条关联记录，不合计多个记录；关联记录可能包含正式发表版本，不等于此版本单独引用。缺失不视为零，引用指标不代表论文质量。
关注作者按书目中的完整姓名匹配，同名和姓名变体需人工辨别。相关论文先按共享细分方向和具体方法排列，再参考通用方法与发表日期。
分类依据与纠错
AI 根据所提供的论文材料归类：摘要以随机试验及其真实世界模拟中的处理效应为对象，直接关联因果效应证据的统计评估。
反馈分类问题
正在核对关联版本…
代码、数据与补充材料
正在核对对应版本的资源…
评估真实世界证据模拟研究对随机试验的可重复性
- 研究问题
- 真实世界证据模拟研究能否重复随机试验的处理效应，以及如何在两个研究间评估重复成功并兼顾错误率与功效？
- 方法与思路
- 作者比较怀疑性 p 值与两个试验均显著且效应同向的规则，扩展至非劣效试验，并通过反演构造置信区间。
- 材料陈述的贡献
- 摘要称怀疑性 p 值可校准至相同第一类错误率而获得更高功效；应用结论大体相似，但显示更多处理效应证据。
- 阅读关注点
- 应核对重复成功的统计定义、错误率对应的零假设、样本量和效应量如何进入计算，以及非劣效界值与区间解释。
- 阅读边界
- 所给版本为 arXiv 预印本，同行评审或期刊接收未核实；摘要未说明真实世界模拟的混杂控制及功效比较条件。
原文摘要
Background: The standard regulatory approach to assess replication success is the two-trials rule, requiring both the original and the replication study to be significant with effect estimates in the same direction. The sceptical p-value was recently presented as an alternative method for the statistical assessment of the replicability of study results. Methods: We review the statistical properties of the sceptical p-value and compare those to the two-trials rule. We extend the methodology to non-inferiority trials and describe how to invert the sceptical p-value to obtain confidence intervals. We illustrate the performance of the different methods using real-world evidence emulations of randomized, controlled trials (RCTs) conducted within the RCT DUPLICATE initiative. Results: The sceptical p-value depends not only on the two p-values, but also on sample size and effect size of the two studies. It can be calibrated to have the same Type-I error rate as the two-trials rule, but has larger power to detect an existing effect. In the application to the results from the RCT DUPLICATE initiative, the sceptical p- value leads to qualitatively similar results than the two-trials rule, but tends to show more evidence for treatment effects compared to the two-trials rule. Conclusion: The sceptical p-value represents a valid statistical measure to assess the replicability of study results and is especially useful in the context of real-world evidence emulations.