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Covariate adjustment in subgroup analyses of randomized clinical trials: A propensity score approach

随机临床试验亚组分析中的协变量调整:倾向得分方法

Background: Subgroup analyses are frequently conducted in randomized clinical trials to assess evidence of heterogeneous treatment effect across patient subpopulations. Although randomization balances covariates within subgroups in expectation, chance imbalance may be amplified in small subgroups and harm the precision. Covariate adjustment in overall analysis of RCT is often conducted, via either ANCOVA or propensity score weighting, but for subgroup analysis has been…

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Model-Assisted Inference for Covariate-Specific Treatment Effects with High-dimensional Data

高维数据下协变量特定处理效应的模型辅助推断

Covariate-specific treatment effects (CSTEs) represent heterogeneous treatment effects across subpopulations defined by certain selected covariates. In this article, we consider marginal structural models where CSTEs are linearly represented using a set of basis functions of the selected covariates. We develop a new approach in high-dimensional settings to obtain not only doubly robust point estimators of CSTEs, but also model-assisted confidence intervals, which are valid…

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Inference for multi-valued heterogeneous treatment effects when the number of treated units is small

处理组单位较少时多值异质性处理效应的推断

We propose a method for conducting asymptotically valid inference for treatment effects in a multi-valued treatment framework where the number of units in the treatment arms can be small and do not grow with the sample size. We accomplish this by casting the model as a semi-/non-parametric conditional quantile model and using known finite sample results about the law of the indicator function that defines the conditional quantile. Our framework allows for structural…

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Two Sample Unconditional Quantile Effect

两样本无条件分位数效应

This paper proposes a new framework to evaluate unconditional quantile effects (UQE) in a data combination model. The UQE measures the effect of a marginal counterfactual change in the unconditional distribution of a covariate on quantiles of the unconditional distribution of a target outcome. Under rank similarity and conditional independence assumptions, we provide a set of identification results for UQEs when the target covariate is continuously distributed and when it is…

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Threshold Martingales and the Evolution of Forecasts

阈值鞅与预测分布的演化

This paper introduces a martingale that characterizes two properties of evolving forecast distributions. Ideal forecasts of a future event behave as martingales, sequen- tially updating the forecast to leverage the available information as the future event approaches. The threshold martingale introduced here measures the proportion of the forecast distribution lying below a threshold. In addition to being calibrated, a threshold martingale has quadratic variation that…

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Asymptotic Properties of Penalized Spline Estimators in Concave Extended Linear Models: Rates of Convergence

凹扩展线性模型中惩罚样条估计量的渐近性质:收敛速度

This paper develops a general theory on rates of convergence of penalized spline estimators for function estimation when the likelihood functional is concave in candidate functions, where the likelihood is interpreted in a broad sense that includes conditional likelihood, quasi-likelihood, and pseudo-likelihood. The theory allows all feasible combinations of the spline degree, the penalty order, and the smoothness of the unknown functions. According to this theory, the…

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Bootstrap Inference for Quantile Treatment Effects in Randomized Experiments with Matched Pairs

配对随机实验中分位数处理效应的自助法推断

This paper examines methods of inference concerning quantile treatment effects (QTEs) in randomized experiments with matched-pairs designs (MPDs). Standard multiplier bootstrap inference fails to capture the negative dependence of observations within each pair and is therefore conservative. Analytical inference involves estimating multiple functional quantities that require several tuning parameters. Instead, this paper proposes two bootstrap methods that can consistently…

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Robust Estimation of Heterogeneous Treatment Effects using Electronic Health Record Data

利用电子健康记录稳健估计异质性处理效应

Estimation of heterogeneous treatment effects is an essential component of precision medicine. Model and algorithm-based methods have been developed within the causal inference framework to achieve valid estimation and inference. Existing methods such as the A-learner, R-learner, modified covariates method (with and without efficiency augmentation), inverse propensity score weighting, and augmented inverse propensity score weighting have been proposed mostly under the square…

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A nonparametric instrumental approach to endogeneity in competing risks models

竞争风险模型中处理内生性的非参数工具变量方法

This paper discusses endogenous treatment models with duration outcomes, competing risks and random right censoring. The endogeneity issue is solved using a discrete instrumental variable. We show that the competing risks model generates a non-parametric quantile instrumental regression problem. The cause-specific cumulative incidence, the cause-specific hazard and the subdistribution hazard can be recovered from the regression function. A distinguishing feature of the model…

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Probabilistic water demand forecasting using quantile regression algorithms

利用分位数回归算法进行城市用水需求概率预测

Machine and statistical learning algorithms can be reliably automated and applied at scale. Therefore, they can constitute a considerable asset for designing practical forecasting systems, such as those related to urban water demand. Quantile regression algorithms are statistical and machine learning algorithms that can provide probabilistic forecasts in a straightforward way, and have not been applied so far for urban water demand forecasting. In this work, we aim to fill…

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Incorporating External Data into the Analysis of Clinical Trials via Bayesian Additive Regression Trees

利用贝叶斯加性回归树将外部数据纳入临床试验分析

Most clinical trials involve the comparison of a new treatment to a control arm (e.g., the standard of care) and the estimation of a treatment effect. External data, including historical clinical trial data and real-world observational data, are commonly available for the control arm. Borrowing information from external data holds the promise of improving the estimation of relevant parameters and increasing the power of detecting a treatment effect if it exists. In this…

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On a log-symmetric quantile tobit model applied to female labor supply data

用于女性劳动供给数据的对数对称分位数 Tobit 模型

The classic censored regression model (tobit model) has been widely used in the economic literature. This model assumes normality for the error distribution and is not recommended for cases where positive skewness is present. Moreover, in regression analysis, it is well-known that a quantile regression approach allows us to study the influences of the explanatory variables on the dependent variable considering different quantiles. Therefore, we propose in this paper a…

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Extremal points of Lorenz curves and applications to inequality analysis

洛伦兹曲线的极点及其在不平等分析中的应用

We find the set of extremal points of Lorenz curves with fixed Gini index and compute the maximal $L^1$-distance between Lorenz curves with given values of their Gini coefficients. As an application we introduce a bidimensional index that simultaneously measures relative inequality and dissimilarity between two populations. This proposal employs the Gini indices of the variables and an $L^1$-distance between their Lorenz curves. The index takes values in a right-angled…

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Panel semiparametric quantile regression neural network for electricity consumption forecasting

用于电力消费预测的面板半参数分位数回归神经网络

China has made great achievements in electric power industry during the long-term deepening of reform and opening up. However, the complex regional economic, social and natural conditions, electricity resources are not evenly distributed, which accounts for the electricity deficiency in some regions of China. It is desirable to develop a robust electricity forecasting model. Motivated by which, we propose a Panel Semiparametric Quantile Regression Neural Network (PSQRNN) by…

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Semiparametric counterfactual density estimation

半参数反事实密度估计

Causal effects are often characterized with averages, which can give an incomplete picture of the underlying counterfactual distributions. Here we consider estimating the entire counterfactual density and generic functionals thereof. We focus on two kinds of target parameters. The first is a density approximation, defined by a projection onto a finite-dimensional model using a generalized distance metric, which includes f-divergences as well as $L_p$ norms. The second is the…

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lpdensity: Local Polynomial Density Estimation and Inference

lpdensity:局部多项式密度估计与推断

Density estimation and inference methods are widely used in empirical work. When the underlying distribution has compact support, conventional kernel-based density estimators are no longer consistent near or at the boundary because of their well-known boundary bias. Alternative smoothing methods are available to handle boundary points in density estimation, but they all require additional tuning parameter choices or other typically ad hoc modifications depending on the…

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Global jump filters and realized volatility

全局跳跃过滤与已实现波动率

For a semimartingale with jumps, we propose a new estimation method for integrated volatility, i.e., the quadratic variation of the continuous martingale part, based on the global jump filter proposed by Inatsugu and Yoshida [8]. To decide whether each increment of the process has jumps, the global jump filter adopts the upper $α$-quantile of the absolute increments as the threshold. This jump filter is called global since it uses all the observations to classify one…

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Shape-Enforcing Operators for Point and Interval Estimators

点估计与区间估计的形状约束算子

A common problem in econometrics, statistics, and machine learning is to estimate and make inference on functions that satisfy shape restrictions. For example, distribution functions are nondecreasing and range between zero and one, height growth charts are nondecreasing in age, and production functions are nondecreasing and quasi-concave in input quantities. We propose a method to enforce these restrictions ex post on point and interval estimates of the target function by…

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Dilation bootstrap

扩张自助法

We propose a methodology for constructing confidence regions with partially identified models of general form. The region is obtained by inverting a test of internal consistency of the econometric structure. We develop a dilation bootstrap methodology to deal with sampling uncertainty without reference to the hypothesized economic structure. It requires bootstrapping the quantile process for univariate data and a novel generalization of the latter to higher dimensions. Once…

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Adaptive Random Bandwidth for Inference in CAViaR Models

用于 CAViaR 模型推断的自适应随机带宽

This paper investigates the size performance of Wald tests for CAViaR models (Engle and Manganelli, 2004). We find that the usual estimation strategy on test statistics yields inaccuracies. Indeed, we show that existing density estimation methods cannot adapt to the time-variation in the conditional probability densities of CAViaR models. Consequently, we develop a method called adaptive random bandwidth which can approximate time-varying conditional probability densities…

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Nonparametric Analysis of Delayed Treatment Effects using Single-Crossing Constraints

利用单交叉约束非参数分析延迟治疗效应

Clinical trials involving novel immuno-oncology (IO) therapies frequently exhibit survival profiles which violate the proportional hazards assumption due to a delay in treatment effect, and in such settings, the survival curves in the two treatment arms may have a crossing before the two curves eventually separate. To flexibly model such scenarios, we describe a nonparametric approach for estimating the treatment arm-specific survival functions which constrains these two…

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Local Regression Distribution Estimators

局部回归分布估计量

This paper investigates the large sample properties of local regression distribution estimators, which include a class of boundary adaptive density estimators as a prime example. First, we establish a pointwise Gaussian large sample distributional approximation in a unified way, allowing for both boundary and interior evaluation points simultaneously. Using this result, we study the asymptotic efficiency of the estimators, and show that a carefully crafted minimum distance…

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Regression Discontinuity Design with Many Thresholds

具有多个阈值的断点回归设计

Numerous empirical studies employ regression discontinuity designs with multiple cutoffs and heterogeneous treatments. A common practice is to normalize all the cutoffs to zero and estimate one effect. This procedure identifies the average treatment effect (ATE) on the observed distribution of individuals local to existing cutoffs. However, researchers often want to make inferences on more meaningful ATEs, computed over general counterfactual distributions of individuals,…

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Uniform Post Selection Inference for LAD Regression and Other Z-estimation problems

LAD 回归及其他 Z 估计问题的一致有效选择后推断

We develop uniformly valid confidence regions for regression coefficients in a high-dimensional sparse median regression model with homoscedastic errors. Our methods are based on a moment equation that is immunized against non-regular estimation of the nuisance part of the median regression function by using Neyman's orthogonalization. We establish that the resulting instrumental median regression estimator of a target regression coefficient is asymptotically normally…

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