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Policy Learning with Observational Data: The Case of Hepatitis C Treatment for HIV/HCV Co-Infected Patients

基于观察数据的政策学习:HIV/HCV 共感染患者的丙肝治疗

Decision-makers frequently must choose a single action from a finite set of alternatives -- for example, physicians selecting a treatment, investors choosing a portfolio risk level, or judges determining sentences. To improve outcomes, policymakers often issue policy rules or guidelines to inform such choices. In this paper, I show how to generally derive policy rules from observational data in a multi-action framework under relatively weak assumptions about the underlying…

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A subgroup-aware scoring approach to the study of effect modification in observational studies

观察研究中效应修饰的子群感知评分方法

Effect modification means the size of a treatment effect varies with an observed covariate. Generally speaking, a larger treatment effect with more stable error terms is less sensitive to bias. Thus, we might be able to conclude that a study is less sensitive to unmeasured bias by using these subgroups experiencing larger treatment effects. Lee et al. (2018) proposed the submax method that leverages the joint distribution of test statistics from subgroups to draw a firmer…

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Nesting a Target Study within a Target Trial: A Framework for Evaluating Intervention Effects on Disparities

在目标试验中嵌入目标研究:评估干预对群体差距的影响

We present a novel framework (TS+TT) to nest a Target Study (TS) within a Target Trial (TT) for evaluating the effects of interventions on disparities. The TS component grounds the measurement of disparity in ethical assumptions, based on the concept of allowability, and anchors it to an explicit population within calendar time. It specifies an enrollment plan of stratified sampling of eligible persons to yield a sample where social groups are distributionally similar on…

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Nearest-Neighbour Matching on Unbounded Supports and Covariate Shift Transfer

无界支撑上的最近邻匹配与协变量漂移迁移

Expectations of multivariate functions with missing labels occur in various fields such as transfer learning and average treatment effects. Although non-parametric estimators based on nearest-neighbour matching are frequently used in this context, the existing literature assumes that the covariates live in some well-shaped compact subset of $\R^d$, with densities that are bounded away from zero. In this paper, we show that the usual rates of convergence can be achieved with…

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Asymptotic inference with flexible covariate adjustment under rerandomization and stratified rerandomization

再随机化与分层再随机化下灵活协变量调整的渐近推断

Rerandomization is an effective treatment allocation procedure to control for baseline covariate imbalance. For estimating the average treatment effect, rerandomization has been previously shown to improve the precision of the unadjusted and the linearly-adjusted estimators over simple randomization without compromising consistency. However, it remains unclear whether such results apply more generally to the class of M-estimators, including the g-computation formula with…

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Mean-field Variational Bayes for Sparse Probit Regression

稀疏Probit回归的均值场变分贝叶斯方法

We consider Bayesian variable selection for binary outcomes under a probit link with a spike-and-slab prior on the regression coefficients. Motivated by the computational challenges encountered by Markov chain Monte Carlo (MCMC) samplers in high-dimensional regimes, we develop a mean-field variational Bayes approximation in which all variational factors admit closed-form updates, and the evidence lower bound is available in closed form. This, in turn, allows the development…

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NMF-FFB: Non-negative matrix factorization with feedforward-feedback structure

NMF-FFB:具有前馈-反馈结构的非负矩阵分解

Non-negative matrix factorization (NMF) approximates a non-negative endogenous data matrix as $Y_1 \approx XB$, with non-negative latent components $X$ and coefficients $B$. Standard covariate-aware NMF is feedforward: $B$ depends only on exogenous variables $Y_2$, with no latent feedback among endogenous variables. We propose NMF-FFB (NMF with feedforward-feedback structure), an exploratory data-fitting framework that embeds the simultaneous equation $B = Θ_1 Y_1 + Θ_2 Y_2$…

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Re-examining and calibrating weighted survival analysis for causal inference

重新审视并校准用于因果推断的加权生存分析

Causal inference with time-to-event outcomes is fundamental in various scientific studies. In a static setup with fitted propensity scores, weighted Kaplan-Meier estimation for survival probabilities and weighted Breslow-Peto estimation for hazard ratios have been widely used, but their statistical properties have been overlooked or studied only to a limited extent. We re-examine the weighted Kaplan-Meier method by formally linking it with the general framework of augmented…

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Leveraging heterogeneity for identifiability: Bayesian order-based learning of multiple DAGs

利用异质性实现可识别性:多个有向无环图的贝叶斯排序学习

We propose a joint order-based scoring framework for causal structure learning of directed acyclic graph (DAG) models under heterogeneous data settings. We show that leveraging heterogeneity improves the accuracy of causal ordering estimation. In the most favorable case, the causal ordering is identifiable up to two permutations. Building on this framework, we propose an order-based Bayesian method for Gaussian DAG models and establish its theoretical properties in the…

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Bipartite causal inference with interference, time series data, and a random network

具有干扰、时间序列数据与随机网络的二部因果推断

In bipartite causal inference with interference, interventional units might receive treatment or control, and they might affect the outcome of outcome units through their connections on a bipartite network. We study bipartite causal inference with interference based on observational data across time and a changing bipartite network. Under an exposure mapping framework, we define the immediate and carryover causal effects for each outcome unit, representing contrasts of…

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Improving the Efficiency of Subgroup Analysis in Randomized Controlled Trials with TMLE

利用 TMLE 提高随机对照试验子组分析的效率

Subgroup analyses within randomized controlled trials are often underpowered due to limited sample sizes. We address this challenge by leveraging trial participants outside the subgroup of interest to augment estimation within the subgroup. Specifically, we study two Targeted Maximum Likelihood Estimators (TMLEs) that borrow information from non-subgroup participants within the same trial: a TMLE with pooled regression (TMLE-PR) and an Adaptive Targeted Maximum Likelihood…

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Closed-form estimation and inference for panels with attrition and refreshment samples

存在样本流失与补充样本的面板数据闭式估计与推断

It has long been established that, if a panel dataset suffers from attrition, auxiliary (refreshment) sampling restores full identification under additional assumptions that still allow for nontrivial attrition mechanisms. Such identification results rely on implausible assumptions about the attrition process or lead to theoretically and computationally challenging estimation procedures. We propose an alternative identifying assumption that, despite its nonparametric nature,…

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A comparison of methods for designing hybrid type 2 cluster-randomized trials with continuous effectiveness and implementation endpoints

具有连续效果与实施终点的二型混合整群随机试验设计方法比较

Hybrid type 2 studies are gaining popularity for their ability to assess both implementation and health outcomes as co-primary endpoints. Often conducted as cluster-randomized trials (CRTs), five design methods can validly power these studies: p-value adjustment methods, combined outcomes approach, single weighted 1-DF test, disjunctive 2-DF test, and conjunctive test. We compared these methods theoretically and numerically. Theoretical comparisons of power equations allowed…

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Nonparametric inference for sublevel-set probabilities of conditional average treatment effect functions

条件平均处理效应函数下水平集概率的非参数推断

The average treatment effect can obscure important heterogeneity when individuals respond differently to a treatment. While the conditional average treatment effect (CATE) function captures such heterogeneity, it is difficult to communicate when it depends on many covariates. Sublevels sets of a multivariate CATE function are equally complicated objects, but the probability of a sublevel set of a CATE function is a single number with a simple interpretation as the proportion…

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Sensitivity Analysis for the Test-Negative Design

检测阴性设计的敏感性分析

The test-negative design has become popular for evaluating the effectiveness of post-licensure vaccines using observational data. In addition to its logistical convenience on data collection, the design is also believed to control for the differential health-care-seeking behavior between vaccinated and unvaccinated individuals, an important while often unmeasured confounder between the vaccination and infection. Hence, the design has been employed routinely to monitor…

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Generalizing conditional average treatment effects from nested randomized trials to all trial-eligible individuals

将嵌套随机试验的条件平均处理效应推广至全部符合资格者

Randomized controlled trials often enroll participants whose characteristics differ from those of a target population, which can limit the generalizability of the estimated treatment effects when effect modifiers differ across populations. While existing generalizability methods primarily focus on estimating the average treatment effect (ATE) in the target population, such summaries may obscure important heterogeneity that is relevant for clinical and policy decision-making.…

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Estimation and Inference in Boundary Discontinuity Designs: Location-Based Methods

边界断点设计的估计与推断:基于位置的方法

Boundary discontinuity designs are used to learn about causal treatment effects along a continuous assignment boundary that splits units into control and treatment groups according to a bivariate location score. We analyze location-based local polynomial treatment effect estimators that directly employ the bivariate score of each unit. We develop pointwise and uniform estimation and inference methods for the \textit{Boundary Average Treatment Effect Curve} (BATEC), as well…

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Finite-sample bias-variance tradeoff with variables related to trial participation inserted into causal forest models for ensuring generalizability

因果森林纳入试验参与变量时的有限样本偏差—方差权衡

Estimating conditional average treatment effects (CATE) from randomized controlled trials (RCTs) and generalizing them to broader populations is essential for personalizing treatment rules but is complicated by selection bias due to trial participation and potentially high dimensional covariates. We evaluated finite sample bias variance tradeoff for Causal Forest based CATE estimation strategies to address the selection bias. Identification theory suggests unbiased CATE…

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Individualized Treatment Allocation in Sequential Network Games

序贯网络博弈中的个体化处理分配

Designing individualized allocation of treatments so as to maximize the equilibrium welfare of interacting agents has many policy-relevant applications. Focusing on sequential decision games of interacting agents, this paper develops a method to obtain optimal treatment assignment rules that maximize a social welfare criterion by evaluating stationary distributions of outcomes. Stationary distributions in sequential decision games are given by Gibbs distributions, which are…

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Partial Identification of the Valuation Distribution in Sequential English Auctions

序贯英式拍卖中估值分布的部分识别

This paper extends the incomplete model of Haile and Tamer (2003) from static English auctions to sequential English auctions. Because bidders may wait for future opportunities, the static condition that bidders do not let rivals win at beatable prices need not hold. We replace it with a dynamic opportunity-cost restriction, yielding nonparametric valuation bounds without solving a dynamic equilibrium. Sharp bounds are also characterized. We propose a novel moment-condition…

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Causal Multi-Task Demand Learning

因果多任务需求学习

We study a canonical multi-task demand-learning problem motivated by retail pricing, where a firm seeks to estimate heterogeneous linear price-response functions across multiple decision contexts. Each context is described by rich covariates but exhibits limited price variation, motivating transfer learning across tasks. A central challenge in leveraging cross-task transfer is endogeneity: prices may be arbitrarily correlated with unobserved task-level demand determinants…

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Double/debiased machine learning of quantile treatment effects on long-term outcomes in clinical trials

临床试验长期结局分位数处理效应的双重/去偏机器学习

Long-term outcomes are often unavailable in randomized clinical trials, although short-term surrogate outcomes are commonly observed. External observational data may contain the long-term outcome, but causal comparisons based on such data alone are vulnerable to confounding. Existing surrogate-based data integration methods for long-term outcomes have focused primarily on average treatment effects. We study estimation of quantile treatment effects for long-term outcomes in…

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Robust and Data-Adaptive Integration of Nonconcurrent Data in Platform Trials via Gaussian Processes

通过高斯过程稳健、自适应地整合平台试验非同期数据

A platform trial is an innovative clinical trial design that enables simultaneous and continuous evaluation of multiple treatments within a single master protocol. Existing robust methods restrict analyses to concurrently randomized participants due to concerns that including nonconcurrent data may introduce bias from temporal trends. However, this exclusion represents a missed opportunity to improve efficiency. We propose a Gaussian process framework for incorporating…

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Reluctant Transfer Learning in Penalized Regressions for Individualized Treatment Rules under Effect Heterogeneity

效应异质性下个体化治疗规则的惩罚回归审慎迁移学习

Estimating individualized treatment rules (ITRs) is fundamental to precision medicine, where the goal is to tailor treatment decisions to individual patient characteristics. While numerous methods have been developed for ITR estimation, there is limited research on model updating that accounts for shifted treatment-covariate relationships in the ITR setting. In practice, models trained on source data must be updated for new (target) datasets that exhibit shifts in treatment…

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