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The premise of independence among subjects in the same cluster/group often fails in practice, and models that rely on such untenable assumption can produce misleading results. To overcome this severe deficiency, we introduce a new regression model to handle overdispersed and correlated clustered counts. To account for correlation within clusters, we propose a Poisson regression model where the observations within the same cluster are driven by the same latent random effect that follows the Birnbaum-Saunders distribution with a parameter that controls the strength of dependence among the individuals. This novel multivariate count model is called Clustered Poisson Birnbaum-Saunders (CPBS) regression. As illustrated in this paper, the CPBS model is analytically tractable, and its moment structure can be explicitly obtained. Estimation of parameters is performed through the maximum likelihood method, and an Expectation-Maximization (EM) algorithm is also developed. Simulation results to evaluate the finite-sample performance of our proposed estimators are presented. We also discuss diagnostic tools for checking model adequacy. An empirical application concerning the number of inpatient admissions by individuals to hospital emergency rooms, from the Medical Expenditure Panel Survey (MEPS) conducted by the United States Agency for Health Research and Quality, illustrates the usefulness of our proposed methodology.

The sequential treatment decisions made by physicians to treat chronic diseases are formalized in the statistical literature as dynamic treatment regimes. To date, methods for dynamic treatment regimes have been developed under the assumption that observation times, i.e., treatment and outcome monitoring times, are determined by study investigators. That assumption is often not satisfied in electronic health records data in which the outcome, the observation times, and the treatment mechanism are associated with patients' characteristics. The treatment and observation processes can lead to spurious associations between the treatment of interest and the outcome to be optimized under the dynamic treatment regime if not adequately considered in the analysis. We address these associations by incorporating two inverse weights that are functions of a patient's covariates into dynamic weighted ordinary least squares to develop optimal single stage dynamic treatment regimes, known as individualized treatment rules. We show empirically that our methodology yields consistent, multiply robust estimators. In a cohort of new users of antidepressant drugs from the United Kingdom's Clinical Practice Research Datalink, the proposed method is used to develop an optimal treatment rule that chooses between two antidepressants to optimize a utility function related to the change in body mass index.

There has been significant attention given to developing data-driven methods for tailoring patient care based on individual patient characteristics. Dynamic treatment regimes formalize this through a sequence of decision rules that map patient information to a suggested treatment. The data for estimating and evaluating treatment regimes are ideally gathered through the use of Sequential Multiple Assignment Randomized Trials (SMARTs) though longitudinal observational studies are commonly used due to the potentially prohibitive costs of conducting a SMART. These studies are typically sized for simple comparisons of fixed treatment sequences or, in the case of observational studies, a priori sample size calculations are often not performed. We develop sample size procedures for the estimation of dynamic treatment regimes from observational studies. Our approach uses pilot data to ensure a study will have sufficient power for comparing the value of the optimal regime, i.e. the expected outcome if all patients in the population were treated by following the optimal regime, with a known comparison mean. Our approach also ensures the value of the estimated optimal treatment regime is within an a priori set range of the value of the true optimal regime with a high probability. We examine the performance of the proposed procedure with a simulation study and use it to size a study for reducing depressive symptoms using data from electronic health records.

The increasing size of data generated by smartphones and IoT devices motivated the development of Federated Learning (FL), a framework for on-device collaborative training of machine learning models. First efforts in FL focused on learning a single global model with good average performance across clients, but the global model may be arbitrarily bad for a given client, due to the inherent heterogeneity of local data distributions. Federated multi-task learning (MTL) approaches can learn personalized models by formulating an opportune penalized optimization problem. The penalization term can capture complex relations among personalized models, but eschews clear statistical assumptions about local data distributions. In this work, we propose to study federated MTL under the flexible assumption that each local data distribution is a mixture of unknown underlying distributions. This assumption encompasses most of the existing personalized FL approaches and leads to federated EM-like algorithms for both client-server and fully decentralized settings. Moreover, it provides a principled way to serve personalized models to clients not seen at training time. The algorithms' convergence is analyzed through a novel federated surrogate optimization framework, which can be of general interest. Experimental results on FL benchmarks show that our approach provides models with higher accuracy and fairness than state-of-the-art methods.

Observational studies can play a useful role in assessing the comparative effectiveness of competing treatments. In a clinical trial the randomization of participants to treatment and control groups generally results in well-balanced groups with respect to possible confounders, which makes the analysis straightforward. However, when analysing observational data, the potential for unmeasured confounding makes comparing treatment effects much more challenging. Causal inference methods such as the Instrumental Variable and Prior Even Rate Ratio approaches make it possible to circumvent the need to adjust for confounding factors that have not been measured in the data or measured with error. Direct confounder adjustment via multivariable regression and Propensity score matching also have considerable utility. Each method relies on a different set of assumptions and leverages different data. In this paper, we describe the assumptions of each method and assess the impact of violating these assumptions in a simulation study. We propose the prior outcome augmented Instrumental Variable method that leverages data from before and after treatment initiation, and is robust to the violation of key assumptions. Finally, we propose the use of a heterogeneity statistic to decide two or more estimates are statistically similar, taking into account their correlation. We illustrate our causal framework to assess the risk of genital infection in patients prescribed Sodium-glucose Co-transporter-2 inhibitors versus Dipeptidyl Peptidase-4 inhibitors as second-line treatment for type 2 diabets using observational data from the Clinical Practice Research Datalink.

Ensemble methods based on subsampling, such as random forests, are popular in applications due to their high predictive accuracy. Existing literature views a random forest prediction as an infinite-order incomplete U-statistic to quantify its uncertainty. However, these methods focus on a small subsampling size of each tree, which is theoretically valid but practically limited. This paper develops an unbiased variance estimator based on incomplete U-statistics, which allows the tree size to be comparable with the overall sample size, making statistical inference possible in a broader range of real applications. Simulation results demonstrate that our estimators enjoy lower bias and more accurate confidence interval coverage without additional computational costs. We also propose a local smoothing procedure to reduce the variation of our estimator, which shows improved numerical performance when the number of trees is relatively small. Further, we investigate the ratio consistency of our proposed variance estimator under specific scenarios. In particular, we develop a new "double U-statistic" formulation to analyze the Hoeffding decomposition of the estimator's variance.

Dyadic data is often encountered when quantities of interest are associated with the edges of a network. As such it plays an important role in statistics, econometrics and many other data science disciplines. We consider the problem of uniformly estimating a dyadic Lebesgue density function, focusing on nonparametric kernel-based estimators taking the form of dyadic empirical processes. Our main contributions include the minimax-optimal uniform convergence rate of the dyadic kernel density estimator, along with strong approximation results for the associated standardized and Studentized $t$-processes. A consistent variance estimator enables the construction of valid and feasible uniform confidence bands for the unknown density function. A crucial feature of dyadic distributions is that they may be "degenerate" at certain points in the support of the data, a property making our analysis somewhat delicate. Nonetheless our methods for uniform inference remain robust to the potential presence of such points. For implementation purposes, we discuss procedures based on positive semi-definite covariance estimators, mean squared error optimal bandwidth selectors and robust bias-correction techniques. We illustrate the empirical finite-sample performance of our methods both in simulations and with real-world data. Our technical results concerning strong approximations and maximal inequalities are of potential independent interest.

A multilevel adaptive refinement strategy for solving linear elliptic partial differential equations with random data is recalled in this work. The strategy extends the a posteriori error estimation framework introduced by Guignard and Nobile in 2018 (SIAM J. Numer. Anal, 56, 3121--3143) to cover problems with a nonaffine parametric coefficient dependence. A suboptimal, but nonetheless reliable and convenient implementation of the strategy involves approximation of the decoupled PDE problems with a common finite element approximation space. Computational results obtained using such a single-level strategy are presented in part I of this work (Bespalov, Silvester and Xu, arXiv:2109.07320). Results obtained using a potentially more efficient multilevel approximation strategy, where meshes are individually tailored, are discussed herein. The codes used to generate the numerical results are available online.

Causally identifying the effect of digital advertising is challenging, because experimentation is expensive, and observational data lacks random variation. This paper identifies a pervasive source of naturally occurring, quasi-experimental variation in user-level ad-exposure in digital advertising campaigns. It shows how this variation can be utilized by ad-publishers to identify the causal effect of advertising campaigns. The variation pertains to auction throttling, a probabilistic method of budget pacing that is widely used to spread an ad-campaign`s budget over its deployed duration, so that the campaign`s budget is not exceeded or overly concentrated in any one period. The throttling mechanism is implemented by computing a participation probability based on the campaign`s budget spending rate and then including the campaign in a random subset of available ad-auctions each period according to this probability. We show that access to logged-participation probabilities enables identifying the local average treatment effect (LATE) in the ad-campaign. We present a new estimator that leverages this identification strategy and outline a bootstrap procedure for quantifying its variability. We apply our method to real-world ad-campaign data from an e-commerce advertising platform, which uses such throttling for budget pacing. We show our estimate is statistically different from estimates derived using other standard observational methods such as OLS and two-stage least squares estimators. Our estimated conversion lift is 110%, a more plausible number than 600%, the conversion lifts estimated using naive observational methods.

We introduce Monte-Carlo Attention (MCA), a randomized approximation method for reducing the computational cost of self-attention mechanisms in Transformer architectures. MCA exploits the fact that the importance of each token in an input sequence varies with respect to their attention scores; thus, some degree of error can be tolerable when encoding tokens with low attention. Using approximate matrix multiplication, MCA applies different error bounds to encode input tokens such that those with low attention scores are computed with relaxed precision, whereas errors of salient elements are minimized. MCA can operate in parallel with other attention optimization schemes and does not require model modification. We study the theoretical error bounds and demonstrate that MCA reduces attention complexity (in FLOPS) for various Transformer models by up to 11$\times$ in GLUE benchmarks without compromising model accuracy.