A Conformal Selection Framework for Individual Treatment Beneficiaries with Auxiliary External Data
利用辅助外部数据识别治疗受益者的共形筛选框架AI 解读 ↓
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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.
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We propose methods for estimation and uniform inference for a broad class of causal functions, such as conditional average treatment effects and continuous treatment effects, under multi-way clustering. The causal function is identified as the conditional expectation of a Neyman-orthogonal signal that depends on high-dimensional nuisance parameters. We introduce a two-step procedure: the first step uses machine learning to estimate the nuisance parameters, and the second…
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This paper proposes methods of estimation and uniform inference for a general class of causal functions, such as the conditional average treatment effects and the continuous treatment effects, under multiway clustering. The causal function is identified as a conditional expectation of an adjusted (Neyman-orthogonal) signal that depends on high-dimensional nuisance parameters. We propose a two-step procedure where the first step uses machine learning to estimate the…
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结果条件平均结构导数的识别与自动去偏机器学习
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高维去偏机器学习的有限样本保证
Debiased machine learning (DML) offers an attractive way to estimate treatment effects in observational settings, where identification of causal parameters requires a conditional independence or unconfoundedness assumption, since it allows to control flexibly for a potentially very large number of covariates. This paper gives novel finite-sample guarantees for joint inference on high-dimensional DML, bounding how far the finite-sample distribution of the estimator is from…