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Quality of Life (QOL) outcomes are important in the management of chronic illnesses. In studies of efficacies of treatments or intervention modalities, QOL scales multi-dimensional constructs are routinely used as primary endpoints. The standard data analysis strategy computes composite (average) overall and domain scores, and conducts a mixed-model analysis for evaluating efficacy or monitoring medical conditions as if these scores were in continuous metric scale. However, assumptions of parametric models like continuity and homoscedastivity can be violated in many cases. Furthermore, it is even more challenging when there are missing values on some of the variables. In this paper, we propose a purely nonparametric approach in the sense that meaningful and, yet, nonparametric effect size measures are developed. We propose estimator for the effect size and develop the asymptotic properties. Our methods are shown to be particularly effective in the presence of some form of clustering and/or missing values. Inferential procedures are derived from the asymptotic theory. The Asthma Randomized Trial of Indoor Wood Smoke data will be used to illustrate the applications of the proposed methods. The data was collected from a three-arm randomized trial which evaluated interventions targeting biomass smoke particulate matter from older model residential wood stoves in homes that have children with asthma.

To further develop the statistical inference problem for heterogeneous treatment effects, this paper builds on Breiman's (2001) random forest tree (RFT)and Wager et al.'s (2018) causal tree to parameterize the nonparametric problem using the excellent statistical properties of classical OLS and the division of local linear intervals based on covariate quantile points, while preserving the random forest trees with the advantages of constructible confidence intervals and asymptotic normality properties [Athey and Imbens (2016),Efron (2014),Wager et al.(2014)\citep{wager2014asymptotic}], we propose a decision tree using quantile classification according to fixed rules combined with polynomial estimation of local samples, which we call the quantile local linear causal tree (QLPRT) and forest (QLPRF).

Data from both a randomized trial and an observational study are sometimes simultaneously available for evaluating the effect of an intervention. The randomized data typically allows for reliable estimation of average treatment effects but may be limited in sample size and patient heterogeneity for estimating conditional average treatment effects for a broad range of patients. Estimates from the observational study can potentially compensate for these limitations, but there may be concerns about whether confounding and treatment effect heterogeneity have been adequately addressed. We propose an approach for combining conditional treatment effect estimators from each source such that it aggressively weights toward the randomized estimator when bias in the observational estimator is detected. This allows the combination to be consistent for a conditional causal effect, regardless of whether assumptions required for consistent estimation in the observational study are satisfied. When the bias is negligible, the estimators from each source are combined for optimal efficiency. We show the problem can be formulated as a penalized least squares problem and consider its asymptotic properties. Simulations demonstrate the robustness and efficiency of the method in finite samples, in scenarios with bias or no bias in the observational estimator. We illustrate the method by estimating the effects of hormone replacement therapy on the risk of developing coronary heart disease in data from the Women's Health Initiative.

We aim to make inferences about a smooth, finite-dimensional parameter by fusing data from multiple sources together. Previous works have studied the estimation of a variety of parameters in similar data fusion settings, including in the estimation of the average treatment effect, optimal treatment rule, and average reward, with the majority of them merging one historical data source with covariates, actions, and rewards and one data source of the same covariates. In this work, we consider the general case where one or more data sources align with each part of the distribution of the target population, for example, the conditional distribution of the reward given actions and covariates. We describe potential gains in efficiency that can arise from fusing these data sources together in a single analysis, which we characterize by a reduction in the semiparametric efficiency bound. We also provide a general means to construct estimators that achieve these bounds. In numerical experiments, we show marked improvements in efficiency from using our proposed estimators rather than their natural alternatives. Finally, we illustrate the magnitude of efficiency gains that can be realized in vaccine immunogenicity studies by fusing data from two HIV vaccine trials.

Identification theory for causal effects in causal models associated with hidden variable directed acyclic graphs (DAGs) is well studied. However, the corresponding algorithms are underused due to the complexity of estimating the identifying functionals they output. In this work, we bridge the gap between identification and estimation of population-level causal effects involving a single treatment and a single outcome. We derive influence function based estimators that exhibit double robustness for the identified effects in a large class of hidden variable DAGs where the treatment satisfies a simple graphical criterion; this class includes models yielding the adjustment and front-door functionals as special cases. We also provide necessary and sufficient conditions under which the statistical model of a hidden variable DAG is nonparametrically saturated and implies no equality constraints on the observed data distribution. Further, we derive an important class of hidden variable DAGs that imply observed data distributions observationally equivalent (up to equality constraints) to fully observed DAGs. In these classes of DAGs, we derive estimators that achieve the semiparametric efficiency bounds for the target of interest where the treatment satisfies our graphical criterion. Finally, we provide a sound and complete identification algorithm that directly yields a weight based estimation strategy for any identifiable effect in hidden variable causal models.

We show that the nonstandard limiting distribution of HAR test statistics under fixed-b asymptotics is not pivotal (even after studentization) when the data are nonstationarity. It takes the form of a complicated function of Gaussian processes and depends on the integrated local long-run variance and on on the second moments of the relevant series (e.g., of the regressors and errors for the case of the linear regression model). Hence, existing fixed-b inference methods based on stationarity are not theoretically valid in general. The nuisance parameters entering the fixed-b limiting distribution can be consistently estimated under small-b asymptotics but only with nonparametric rate of convergence. Hence, We show that the error in rejection probability (ERP) is an order of magnitude larger than that under stationarity and is also larger than that of HAR tests based on HAC estimators under conventional asymptotics. These theoretical results reconcile with recent finite-sample evidence in Casini (2021) and Casini, Deng and Perron (2021) who showing that fixed-b HAR tests can perform poorly when the data are nonstationary. They can be conservative under the null hypothesis and have non-monotonic power under the alternative hypothesis irrespective of how large the sample size is.

Beta regression model is useful in the analysis of bounded continuous outcomes such as proportions. It is well known that for any regression model, the presence of multicollinearity leads to poor performance of the maximum likelihood estimators. The ridge type estimators have been proposed to alleviate the adverse effects of the multicollinearity. Furthermore, when some of the predictors have insignificant or weak effects on the outcomes, it is desired to recover as much information as possible from these predictors instead of discarding them all together. In this paper we proposed ridge type shrinkage estimators for the low and high dimensional beta regression model, which address the above two issues simultaneously. We compute the biases and variances of the proposed estimators in closed forms and use Monte Carlo simulations to evaluate their performances. The results show that, both in low and high dimensional data, the performance of the proposed estimators are superior to ridge estimators that discard weak or insignificant predictors. We conclude this paper by applying the proposed methods for two real data from econometric and medicine.

The simultaneous estimation of many parameters based on data collected from corresponding studies is a key research problem that has received renewed attention in the high-dimensional setting. Many practical situations involve heterogeneous data where heterogeneity is captured by a nuisance parameter. Effectively pooling information across samples while correctly accounting for heterogeneity presents a significant challenge in large-scale estimation problems. We address this issue by introducing the "Nonparametric Empirical Bayes Structural Tweedie" (NEST) estimator, which efficiently estimates the unknown effect sizes and properly adjusts for heterogeneity via a generalized version of Tweedie's formula. For the normal means problem, NEST simultaneously handles the two main selection biases introduced by heterogeneity: one, the selection bias in the mean, which cannot be effectively corrected without also correcting for, two, selection bias in the variance. Our theoretical results show that NEST has strong asymptotic properties without requiring explicit assumptions about the prior. Extensions to other two-parameter members of the exponential family are discussed. Simulation studies show that NEST outperforms competing methods, with much efficiency gains in many settings. The proposed method is demonstrated on estimating the batting averages of baseball players and Sharpe ratios of mutual fund returns.

This paper studies distributed binary test of statistical independence under communication (information bits) constraints. While testing independence is very relevant in various applications, distributed independence test is particularly useful for event detection in sensor networks where data correlation often occurs among observations of devices in the presence of a signal of interest. By focusing on the case of two devices because of their tractability, we begin by investigating conditions on Type I error probability restrictions under which the minimum Type II error admits an exponential behavior with the sample size. Then, we study the finite sample-size regime of this problem. We derive new upper and lower bounds for the gap between the minimum Type II error and its exponential approximation under different setups, including restrictions imposed on the vanishing Type I error probability. Our theoretical results shed light on the sample-size regimes at which approximations of the Type II error probability via error exponents became informative enough in the sense of predicting well the actual error probability. We finally discuss an application of our results where the gap is evaluated numerically, and we show that exponential approximations are not only tractable but also a valuable proxy for the Type II probability of error in the finite-length regime.

In this work, we consider the distributed optimization of non-smooth convex functions using a network of computing units. We investigate this problem under two regularity assumptions: (1) the Lipschitz continuity of the global objective function, and (2) the Lipschitz continuity of local individual functions. Under the local regularity assumption, we provide the first optimal first-order decentralized algorithm called multi-step primal-dual (MSPD) and its corresponding optimal convergence rate. A notable aspect of this result is that, for non-smooth functions, while the dominant term of the error is in $O(1/\sqrt{t})$, the structure of the communication network only impacts a second-order term in $O(1/t)$, where $t$ is time. In other words, the error due to limits in communication resources decreases at a fast rate even in the case of non-strongly-convex objective functions. Under the global regularity assumption, we provide a simple yet efficient algorithm called distributed randomized smoothing (DRS) based on a local smoothing of the objective function, and show that DRS is within a $d^{1/4}$ multiplicative factor of the optimal convergence rate, where $d$ is the underlying dimension.