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AI 根据所提供的论文材料归类:摘要直接研究条件平均治疗效应及治疗受益者筛选,属于因果推断。

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正在核对关联版本…

代码、数据与补充材料

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关注这篇论文的作者
Jiajun Liu
Ke Zhu
Xiaofei Wang

利用辅助外部数据识别治疗受益者的共形筛选框架

研究问题
如何利用随机试验与外部数据筛选条件治疗效应超过临床阈值的候选者,并处理同时筛选多人时的不确定性与多重检验问题?
方法与思路
作者将条件平均治疗效应筛选转为多重检验,用外部数据训练模型、随机试验数据校准共形 p 值,再以 BH 程序调整。
材料陈述的贡献
摘要称该框架可搭配不同学习器,模拟中维持错误发现率控制;检验功效取决于基础学习器和外部数据可比性,并提供肺癌案例。
阅读关注点
应核对共形校准和 BH 调整的有效性条件、试验与外部数据的分工,以及受益阈值设定和肺癌案例中的效应解释。
阅读边界
这是 arXiv 预印本,同行评审及接收未核实;摘要未说明错误发现率保证的具体条件,条件平均获益也不能直接等同于个人真实获益。

原文摘要

Identifying patients who are likely to benefit from a treatment is central to precision medicine and can guide follow-up trials, enrichment designs, and individualized decisions. Although randomized controlled trials (RCTs) provide evidence on efficacy, they are usually powered to estimate average treatment effects rather than patient-level benefit. Meanwhile, artificial intelligence and machine learning methods offer flexible tools for estimating heterogeneous treatment effects, especially when augmented by real-world data (RWD). However, in practice, these estimated effects are often translated into decisions through simple ranking or thresholding rules, which can ignore uncertainty and multiplicity when many patients are evaluated simultaneously. Motivated by this, we propose a model-agnostic conformal inference framework for uncertainty-aware beneficiary selection. The framework reformulates CATE-based treatment-benefit selection as a multiple-testing problem. For each candidate, we test whether the conditional treatment benefit exceeds a clinically meaningful threshold and construct a conformal p-value using RCT-based calibration. These p-values are then adjusted by the Benjamini-Hochberg procedure to control the false discovery rate (FDR) among selected beneficiaries. To improve efficiency when RCT sample sizes are limited, external data, such as RWD, can be used to train flexible treatment effect models, while conformal calibration remains anchored in the RCT data. It can be paired with conventional machine learning algorithms and emerging tabular foundation models. Simulations show that the framework maintains FDR control, with power depending on the base learner and external-data comparability. A case study in early-stage non-small-cell lung cancer illustrates how the method identifies candidate profiles with evidence of benefit from limited resection to reduce overtreatment.

笔记与分组
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