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通用机器学习子群识别的统计性能保证AI 解读

Across a wide array of disciplines, many researchers use machine learning (ML) algorithms to identify a subgroup of individuals who are likely to benefit from a treatment the most (``exceptional responders'') or those who are harmed by it. A common approach to this subgroup identification problem consists of two steps. First, researchers estimate the conditional average treatment effect (CATE) using an ML algorithm. Next, they use the estimated CATE to select those…

优化随机临床试验处理分配的自适应设计AI 解读

The treatment assignment mechanism in a randomized clinical trial can be optimized for statistical efficiency within a specified class of randomization mechanisms. Optimal designs of this type have been characterized in terms of the variances of potential outcomes conditional on baseline covariates. Approximating these optimal designs requires information about the conditional variance functions, which is often unavailable or unreliable at the design stage. As a practical…

利用高维经验似然进行平均处理效应的多重稳健推断AI 解读

In this paper, we develop a multiply robust inference procedure of the average treatment effect (ATE) for data with high-dimensional covariates. We consider the case where it is difficult to correctly specify a single parametric model for the propensity scores (PS). For example, the target population is formed from heterogeneous sources with different treatment assignment mechanisms. We propose a novel high-dimensional empirical likelihood weighting method under soft…

具有延迟治疗效应的随机免疫治疗试验贝叶斯最优二期设计AI 解读

Immunotherapy has transformed cancer treatment, yet its delayed therapeutic effects often lead to non-proportional hazards, rendering many conventional phase II designs underpowered and prone to type I error inflation. To address this issue, we propose a novel Bayesian Optimal Phase II design (DTE-BOP2) that explicitly models the uncertainty in the separation timing of treatment effect. The treatment separation timepoint (denoted by S) is endowed with a truncated-Gamma…

具有信息性处理时点的因果效应估计AI 解读

Epidemiological studies are often concerned with estimating causal effects of a sequence of treatment decisions on survival outcomes. In many settings, treatment decisions do not occur at fixed, pre-specified followup times. Rather, timing varies across subjects in ways that may be informative of subsequent treatment decisions and potential outcomes. Awareness of the issue and its potential solutions is lacking in the literature, which motivate this work. Here, we formalize…

边际上的处理效应：每个人都处于边际AI 解读

This paper develops a framework for identifying treatment effects when a policy simultaneously alters both the incentive to participate and the outcome of interest -- such as hiring decisions and wages in response to employment subsidies; or working decisions and wages in response to job trainings. This framework was inspired by my PhD project on a Belgian reform that subsidised first-time hiring, inducing entry by marginal firms yet meanwhile changing the wages they pay.…

回归不连续设计中的异质性处理效应发现AI 解读

The paper proposes a causal supervised machine learning algorithm to uncover treatment effect heterogeneity in sharp and fuzzy regression discontinuity (RD) designs. We develop a criterion for building an honest ``regression discontinuity tree'', where each leaf contains the RD estimate of a treatment conditional on the values of some pre-treatment covariates. It is a priori unknown which covariates are relevant for capturing treatment effect heterogeneity, and it is the…

模型误设下的稳健通用推断AI 解读

In statistical inference, it is rarely realistic that the hypothesized statistical model is well-specified, and consequently it is important to understand the effects of misspecification on inferential procedures. When the hypothesized statistical model is misspecified, the natural target of inference is a projection of the data generating distribution onto the model. We present a general method for constructing valid confidence sets for such projections, under weak…

多组临床试验中死亡截断结局的双重稳健估计与敏感性分析AI 解读

In clinical trials, the observation of participant outcomes may frequently be hindered by death, leading to ambiguity in defining a scientifically meaningful final outcome for those who die. Principal stratification methods are valuable tools for addressing the average causal effect among always-survivors, i.e., the average treatment effect among a subpopulation defined as those who would survive regardless of treatment assignment. Although robust methods for the…

乘法工具变量模型下非线性反事实泛函的推断AI 解读

Instrumental variable (IV) methods play a central role in causal inference, particularly in settings where treatment assignment is confounded by unobserved variables. IV methods have been extensively developed in recent years and applied across diverse domains, from economics to epidemiology. In this work, we study the recently introduced multiplicative IV (MIV) model and demonstrate its utility for causal inference beyond the average treatment effect. In particular, we show…

高维混杂下的因果机器学习方法与交叉拟合AI 解读

Observational epidemiological studies commonly seek to estimate the causal effect of an exposure on an outcome. Adjustment for potential confounding bias in modern studies is challenging due to the presence of high-dimensional confounding, which occurs when there are many confounders relative to sample size or complex relationships between continuous confounders and exposure and outcome. Doubly robust methods such as Augmented Inverse Probability Weighting (AIPW) and…

何时可以进行因果推断？未测量混杂的统计检验AI 解读

This paper clarifies a fundamental difference between causal inference and traditional statistical inference by formalizing a mathematical distinction between their respective parameters. We connect two major approaches to causal inference, the potential outcomes framework and causal structure graphs, which are typically studied separately. While the unconfoundedness assumption in the potential outcomes framework cannot be assessed from an observational dataset alone, causal…

基于 HAL 的因果剂量反应曲线插件估计与逐点渐近正态性AI 解读

Estimating and obtaining reliable inference for the marginally adjusted causal dose-response curve for continuous treatments without relying on parametric assumptions is a well-known statistical challenge. Parametric models risk introducing significant bias through model misspecification, compromising the accurate representation of the underlying data and dose-response relationship. On the other hand, nonparametric models face difficulties as the dose-response curve is not…

检查引导随机化：灵活透明的受限随机化实验设计框架AI 解读

Randomized experiments are considered the gold standard for estimating causal effects. However, out of the set of possible randomized assignments, some may be likely to produce poor effect estimates and misleading conclusions. Restricted randomization is an experimental design strategy that filters out undesirable treatment assignments, but its application has primarily been limited to ensuring covariate balance in two-arm studies where the target estimand is the average…

结局误分类与非随机验证子集下整群随机试验的平均处理效应估计AI 解读

Randomized trials are viewed as the benchmark for assessing causal effects of treatments on outcomes of interest. Nonetheless, challenges such as measurement error can undermine the standard causal assumptions for randomized trials. In ASPIRE, a cluster-randomized trial, pediatric primary care clinics were assigned to one of two treatments aimed at promoting clinician delivery of a secure firearm program to parents during well-child visits. A key outcome of interest is thus…

基于匹配的组平均处理效应非参数估计AI 解读

Heterogeneous treatment effects, which vary according to individual covariates, are crucial in fields such as personalized medicine and tailored treatment strategies. In many applications, rather than considering the heterogeneity induced by all covariates, practitioners focus on a few key covariates to develop tailored treatment decisions. Based on this, we aim to estimate the group average treatment effects (GATEs), which represent heterogeneous treatment effects across…

未知干扰结构下纵向数据的因果推断AI 解读

In longitudinal studies where units are embedded in space or a social network, interference may arise, meaning that a unit's outcome can depend on treatment histories of others. The presence of interference poses significant challenges for causal inference, particularly when the interference structure -- how a unit's outcome responds to others' influences -- is complex, heterogeneous, and unknown to researchers. This paper develops a general framework for identifying and…

内生暴露与轻尾误差下的因果效应识别和推断AI 解读

Endogeneity poses significant challenges in causal inference across various research domains. This paper proposes a novel approach to identify and estimate causal effects in the presence of endogeneity. We consider a structural equation with endogenous exposures and an additive error term. Assuming the light-tailedness of the error term, we show that the causal effect can be identified by contrasting extreme conditional quantiles of the outcome given the exposures. Unlike…

基于汇总数据消除赢家诅咒的稳健孟德尔随机化AI 解读

In the past decade, the increased availability of genome-wide association studies summary data has popularized Mendelian Randomization (MR) for conducting causal inference. MR analyses, incorporating genetic variants as instrumental variables, are known for their robustness against reverse causation bias and unmeasured confounders. Nevertheless, classical MR analyses utilizing summary data may still produce biased causal effect estimates due to the winner's curse and…

用于平均处理效应估计的因子辅助双重深度学习AI 解读

We investigate the problem of estimating the average treatment effect (ATE) under a very general setup where the covariates can be high-dimensional, highly correlated, and can have sparse nonlinear effects on the propensity and outcome models. We present the use of a Double Deep Learning strategy for estimation, which involves combining recently developed factor-augmented deep learning-based estimators, FAST-NN, for both the response functions and propensity scores to…

统计学的挑战：因果关系与因果推断的十二项挑战AI 解读

Causality and causal inference have emerged as core research areas at the interface of modern statistics and domains including biomedical sciences, social sciences, computer science, and beyond. The field's inherently interdisciplinary nature -- particularly the central role of incorporating domain knowledge -- creates a rich and varied set of statistical challenges. Much progress has been made, especially in the last three decades, but there remain many open questions. Our…

不完全区组设计的设计本位因果推断AI 解读

Researchers often turn to block randomization to increase the precision of their inference or due to practical considerations, such as in multisite trials. However, if the number of treatments under consideration is large it might not be feasible or practical to assign all treatments within each block. We develop novel inference results under the finite-population design-based framework for natural alternatives to the complete block design that do not require reducing the…

用随机矩阵理论重新考察多工具变量问题AI 解读

Instrumental variables estimation with many instruments is biased. Traditional bias-adjustments are closely connected to the Silverstein equation. Based on the theory of random matrices, we show that Ridge estimation of the first-stage parameters reduces the implicit price of bias-adjustments. This leads to a trade-off, allowing for less costly estimation of the causal effect, which comes along with improved asymptotic properties. Our theoretical results nest existing ones…

随机实验中实现无偏机器学习协变量调整的条件交叉拟合AI 解读

Randomized experiments are the gold standard for estimating the average treatment effect (ATE). While covariate adjustment can reduce the asymptotic variances of the unbiased Horvitz-Thompson estimators for the ATE, it suffers from finite-sample biases due to data reuse in both prediction and estimation. Traditional sample-splitting and cross-fitting methods can address the problem of data reuse and obtain unbiased estimators. However, they require that the data are…