arXiv Statistics @arxiv_stats@qoto.org

Parameter inference is essential when interpreting observational data using mathematical models. Standard inference methods for differential equation models typically rely on obtaining repeated numerical solutions of the differential equation(s). Recent results have explored how numerical truncation error can have major, detrimental, and sometimes hidden impacts on likelihood-based inference by introducing false local maxima into the log-likelihood function. We present a straightforward approach for inference that eliminates the need for solving the underlying differential equations, thereby completely avoiding the impact of truncation error. Open-access Jupyter notebooks, available on GitHub, allow others to implement this method for a broad class of widely-used models to interpret biological data.

Hierarchical Archimedean copulas (HACs) are multivariate uniform distributions constructed by nesting Archimedean copulas into one another, and provide a flexible approach to modeling non-exchangeable data. However, this flexibility in the model structure may lead to over-fitting when the model estimation procedure is not performed properly. In this paper, we examine the problem of structure estimation and more generally on the selection of a parsimonious model from the hypothesis testing perspective. Formal tests for structural hypotheses concerning HACs have been lacking so far, most likely due to the restrictions on their associated parameter space which hinders the use of standard inference methodology. Building on previously developed asymptotic methods for these non-standard parameter spaces, we provide an asymptotic stochastic representation for the maximum likelihood estimators of (potentially) overparametrized HACs, which we then use to formulate a likelihood ratio test for certain common structural hypotheses. Additionally, we also derive analytical expressions for the first- and second-order partial derivatives of two-level HACs based on Clayton and Gumbel generators, as well as general numerical approximation schemes for the Fisher information matrix.

Micro-randomized trials (MRTs) are increasingly utilized for optimizing mobile health interventions, with the causal excursion effect (CEE) as a central quantity for evaluating interventions under policies that deviate from the experimental policy. However, MRT often contains missing data due to reasons such as missed self-reports or participants not wearing sensors, which can bias CEE estimation. In this paper, we propose a two-stage, doubly robust estimator for CEE in MRTs when longitudinal outcomes are missing at random, accommodating continuous, binary, and count outcomes. Our two-stage approach allows for both parametric and nonparametric modeling options for two nuisance parameters: the missingness model and the outcome regression. We demonstrate that our estimator is doubly robust, achieving consistency and asymptotic normality if either the missingness or the outcome regression model is correctly specified. Simulation studies further validate the estimator's desirable finite-sample performance. We apply the method to HeartSteps, an MRT for developing mobile health interventions that promote physical activity.

Observational studies provide invaluable opportunities to draw causal inference, but they may suffer from biases due to pretreatment difference between treated and control units. Matching is a popular approach to reduce observed covariate imbalance. To tackle unmeasured confounding, a sensitivity analysis is often conducted to investigate how robust a causal conclusion is to the strength of unmeasured confounding. For matched observational studies, Rosenbaum proposed a sensitivity analysis framework that uses the randomization of treatment assignments as the "reasoned basis" and imposes no model assumptions on the potential outcomes as well as their dependence on the observed and unobserved confounding factors. However, this otherwise appealing framework requires exact matching to guarantee its validity, which is hard to achieve in practice. In this paper we provide an alternative inferential framework that shares the same procedure as Rosenbaum's approach but relies on a different justification. Our framework allows flexible matching algorithms and utilizes alternative source of randomness, in particular random permutations of potential outcomes instead of treatment assignments, to guarantee statistical validity.

Modeling observations as random distributions embedded within Wasserstein spaces is becoming increasingly popular across scientific fields, as it captures the variability and geometric structure of the data more effectively. However, the distinct geometry and unique properties of Wasserstein space pose challenges to the application of conventional statistical tools, which are primarily designed for Euclidean spaces. Consequently, adapting and developing new methodologies for analysis within Wasserstein spaces has become essential. The space of distributions on $\mathbb{R}^d$ with $d>1$ is not linear, and ''mimic'' the geometry of a Riemannian manifold. In this paper, we extend the concept of statistical depth to distribution-valued data, introducing the notion of {\it Wasserstein spatial depth}. This new measure provides a way to rank and order distributions, enabling the development of order-based clustering techniques and inferential tools. We show that Wasserstein spatial depth (WSD) preserves critical properties of conventional statistical depths, notably, ranging within $[0,1]$, transformation invariance, vanishing at infinity, reaching a maximum at the geometric median, and continuity. Additionally, the population WSD has a straightforward plug-in estimator based on sampled empirical distributions. We establish the estimator's consistency and asymptotic normality. Extensive simulation and real-data application showcase the practical efficacy of WSD.

As the volume and complexity of data continue to expand across various scientific disciplines, the need for robust methods to account for the multiplicity of comparisons has grown widespread. A popular measure of type 1 error rate in multiple testing literature is the false discovery rate (FDR). The FDR provides a powerful and practical approach to large-scale multiple testing and has been successfully used in a wide range of applications. The concept of FDR has gained wide acceptance in the statistical community and various methods has been proposed to control the FDR. In this work, we review the latest developments in FDR control methodologies. We also develop a conceptual framework to better describe this vast literature; understand its intuition and key ideas; and provide guidance for the researcher interested in both the application and development of the methodology.

Testing for mediation effect poses a challenge since the null hypothesis (i.e., the absence of mediation effects) is composite, making most existing mediation tests quite conservative and often underpowered. In this work, we propose a subsampling-based procedure to construct a test statistic whose asymptotic null distribution is pivotal and remains the same regardless of the three null cases encountered in mediation analysis. The method, when combined with the popular Sobel test, leads to an accurate size control under the null. We further introduce a Cauchy combination test to construct p-values from different subsample splits, which reduces variability in the testing results and increases detection power. Through numerical studies, our approach has demonstrated a more accurate size and higher detection power than the competing classical and contemporary methods.

This paper addresses the challenge of improving finite sample performance in Ranking and Selection by developing a Bahadur-Rao type expansion for the Probability of Correct Selection (PCS). While traditional large deviations approximations captures PCS behavior in the asymptotic regime, they can lack precision in finite sample settings. Our approach enhances PCS approximation under limited simulation budgets, providing more accurate characterization of optimal sampling ratios and optimality conditions dependent of budgets. Algorithmically, we propose a novel finite budget allocation (FCBA) policy, which sequentially estimates the optimality conditions and accordingly balances the sampling ratios. We illustrate numerically on toy examples that our FCBA policy achieves superior PCS performance compared to tested traditional methods. As an extension, we note that the non-monotonic PCS behavior described in the literature for low-confidence scenarios can be attributed to the negligence of simultaneous incorrect binary comparisons in PCS approximations. We provide a refined expansion and a tailored allocation strategy to handle low-confidence scenarios, addressing the non-monotonicity issue.

The general applicability and ease of use of the pseudo-marginal Metropolis--Hastings (PMMH) algorithm, and particle Metropolis--Hastings in particular, makes it a popular method for inference on discretely observed Markovian stochastic processes. The performance of these algorithms and, in the case of particle Metropolis--Hastings, the trade off between improved mixing through increased accuracy of the estimator and the computational cost were investigated independently in two papers, both published in 2015. Each suggested choosing the number of particles so that the variance of the logarithm of the estimator of the posterior at a fixed sensible parameter value is approximately 1. This advice has been widely and successfully adopted. We provide new, remarkably simple upper and lower bounds on the asymptotic variance of PMMH algorithms. The bounds explain how blindly following the 2015 advice can hide serious issues with the algorithm and they strongly suggest an alternative criterion. In most situations our guidelines and those from 2015 closely coincide; however, when the two differ it is safer to follow the new guidance. An extension of one of our bounds shows how the use of correlated proposals can fundamentally shift the properties of pseudo-marginal algorithms, so that asymptotic variances that were infinite under the PMMH kernel become finite.

Sufficient overlap of propensity scores is one of the most critical assumptions in observational studies. In this article, we will cover the severity in statistical inference under such assumption failure with weighting, one of the most dominating causal inference methodologies. Then we propose a simple, yet novel remedy: "mixing" the treated and control groups in the observed dataset. We state that our strategy has three key advantages: (1) Improvement in estimators' accuracy especially in weak overlap, (2) Identical targeting population of treatment effect, (3) High flexibility. We introduce a property of mixed sample that offers a safer inference by implementing onto both traditional and modern weighting methods. We illustrate this with several extensive simulation studies and guide the readers with a real-data analysis for practice.

Hybrid studies allow investigators to simultaneously study an intervention effectiveness outcome and an implementation research outcome. In particular, type 2 hybrid studies support research that places equal importance on both outcomes rather than focusing on one and secondarily on the other (i.e., type 1 and type 3 studies). Hybrid 2 studies introduce the statistical issue of multiple testing, complicated by the fact that they are typically also cluster randomized trials. Standard statistical methods do not apply in this scenario. Here, we describe the design methodologies available for validly powering hybrid type 2 studies and producing reliable sample size calculations in a cluster-randomized design with a focus on binary outcomes. Through a literature search, 18 publications were identified that included methods relevant to the design of hybrid 2 studies. Five methods were identified, two of which did not account for clustering but are extended in this article to do so, namely the combined outcomes approach and the single 1-degree of freedom combined test. Procedures for powering hybrid 2 studies using these five methods are described and illustrated using input parameters inspired by a study from the Community Intervention to Reduce CardiovascuLar Disease in Chicago (CIRCL-Chicago) Implementation Research Center. In this illustrative example, the intervention effectiveness outcome was controlled blood pressure, and the implementation outcome was reach. The conjunctive test resulted in higher power than the popular p-value adjustment methods, and the newly extended combined outcomes and single 1-DF test were found to be the most powerful among all of the tests.

In comparative studies of progressive diseases, such as randomized controlled trials (RCTs), the mean Change From Baseline (CFB) of a continuous outcome at a pre-specified follow-up time across subjects in the target population is a standard estimand used to summarize the overall disease progression. Despite its simplicity in interpretation, the mean CFB may not efficiently capture important features of the trajectory of the mean outcome relevant to the evaluation of the treatment effect of an intervention. Additionally, the estimation of the mean CFB does not use all longitudinal data points. To address these limitations, we propose a class of estimands called Principal Progression Rate (PPR). The PPR is a weighted average of local or instantaneous slope of the trajectory of the population mean during the follow-up. The flexibility of the weight function allows the PPR to cover a broad class of intuitive estimands, including the mean CFB, the slope of ordinary least-square fit to the trajectory, and the area under the curve. We showed that properly chosen PPRs can enhance statistical power over the mean CFB by amplifying the signal of treatment effect and/or improving estimation precision. We evaluated different versions of PPRs and the performance of their estimators through numerical studies. A real dataset was analyzed to demonstrate the advantage of using alternative PPR over the mean CFB.

We introduce a methodology for performing parameter inference in high-dimensional, non-linear diffusion processes. We illustrate its applicability for obtaining insights into the evolution of and relationships between species, including ancestral state reconstruction. Estimation is performed by utilising score matching to approximate diffusion bridges, which are subsequently used in an importance sampler to estimate log-likelihoods. The entire setup is differentiable, allowing gradient ascent on approximated log-likelihoods. This allows both parameter inference and diffusion mean estimation. This novel, numerically stable, score matching-based parameter inference framework is presented and demonstrated on biological two- and three-dimensional morphometry data.

Often in prediction tasks, the predictive model itself can influence the distribution of the target variable, a phenomenon termed performative prediction. Generally, this influence stems from strategic actions taken by stakeholders with a vested interest in predictive models. A key challenge that hinders the widespread adaptation of performative prediction in machine learning is that practitioners are generally unaware of the social impacts of their predictions. To address this gap, we propose a methodology for learning the distribution map that encapsulates the long-term impacts of predictive models on the population. Specifically, we model agents' responses as a cost-adjusted utility maximization problem and propose estimates for said cost. Our approach leverages optimal transport to align pre-model exposure (ex ante) and post-model exposure (ex post) distributions. We provide a rate of convergence for this proposed estimate and assess its quality through empirical demonstrations on a credit-scoring dataset.