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We consider functional linear regression models where functional outcomes are associated with scalar predictors by coefficient functions with shape constraints, such as monotonicity and convexity, that apply to sub-domains of interest. To validate the partial shape constraints, we propose testing a composite hypothesis of linear functional constraints on regression coefficients. Our approach employs kernel- and spline-based methods within a unified inferential framework, evaluating the statistical significance of the hypothesis by measuring an $L^2$-distance between constrained and unconstrained model fits. In the theoretical study of large-sample analysis under mild conditions, we show that both methods achieve the standard rate of convergence observed in the nonparametric estimation literature. Through numerical experiments of finite-sample analysis, we demonstrate that the type I error rate keeps the significance level as specified across various scenarios and that the power increases with sample size, confirming the consistency of the test procedure under both estimation methods. Our theoretical and numerical results provide researchers the flexibility to choose a method based on computational preference. The practicality of partial shape-constrained inference is illustrated by two data applications: one involving clinical trials of NeuroBloc in type A-resistant cervical dystonia and the other with the National Institute of Mental Health Schizophrenia Study., Comment: 30 pages, 7 figures

Fr\'echet regression has emerged as a promising approach for regression analysis involving non-Euclidean response variables. However, its practical applicability has been hindered by its reliance on ideal scenarios with abundant and noiseless covariate data. In this paper, we present a novel estimation method that tackles these limitations by leveraging the low-rank structure inherent in the covariate matrix. Our proposed framework combines the concepts of global Fr\'echet regression and principal component regression, aiming to improve the efficiency and accuracy of the regression estimator. By incorporating the low-rank structure, our method enables more effective modeling and estimation, particularly in high-dimensional and errors-in-variables regression settings. We provide a theoretical analysis of the proposed estimator's large-sample properties, including a comprehensive rate analysis of bias, variance, and additional variations due to measurement errors. Furthermore, our numerical experiments provide empirical evidence that supports the theoretical findings, demonstrating the superior performance of our approach. Overall, this work introduces a promising framework for regression analysis of non-Euclidean variables, effectively addressing the challenges associated with limited and noisy covariate data, with potential applications in diverse fields., Comment: 36 pages, 3 figures, 1 table. To appear at NeurIPS 2023

Hypothesis testing procedures are developed to assess linear operator constraints in function-on-scalar regression when incomplete functional responses are observed. The approach enables statistical inferences about the shape and other aspects of the functional regression coefficients within a unified framework encompassing three incomplete sampling scenarios: (i) partially observed response functions as curve segments over random sub-intervals of the domain; (ii) discretely observed functional responses with additive measurement errors; and (iii) the composition of former two scenarios, where partially observed response segments are observed discretely with measurement error. The latter scenario has been little explored to date, although such structured data is increasingly common in applications. For statistical inference, deviations from the constraint space are measured via integrated $L^2$-distance between the model estimates from the constrained and unconstrained model spaces. Large sample properties of the proposed test procedure are established, including the consistency, asymptotic distribution and local power of the test statistic. Finite sample power and level of the proposed test are investigated in a simulation study covering a variety of scenarios. The proposed methodologies are illustrated by applications to U.S. obesity prevalence data, analyzing the functional shape of its trends over time, and motion analysis in a study of automotive ergonomics.

Electronic health record (EHR) data are increasingly used for biomedical research, but these data have recognized data quality challenges. Data validation is necessary to use EHR data with confidence, but limited resources typically make complete data validation impossible. Using EHR data, we illustrate prospective, multi-wave, two-phase validation sampling to estimate the association between maternal weight gain during pregnancy and the risks of her child developing obesity or asthma. The optimal validation sampling design depends on the unknown efficient influence functions of regression coefficients of interest. In the first wave of our multi-wave validation design, we estimate the influence function using the unvalidated (phase 1) data to determine our validation sample; then in subsequent waves, we re-estimate the influence function using validated (phase 2) data and update our sampling. For efficiency, estimation combines obesity and asthma sampling frames while calibrating sampling weights using generalized raking. We validated 996 of 10,335 mother-child EHR dyads in 6 sampling waves. Estimated associations between childhood obesity/asthma and maternal weight gain, as well as other covariates, are compared to naive estimates that only use unvalidated data. In some cases, estimates markedly differ, underscoring the importance of efficient validation sampling to obtain accurate estimates incorporating validated data.

Increasingly, medical research is dependent on data collected for non-research purposes, such as electronic health records data (EHR). EHR data and other large databases can be prone to measurement error in key exposures, and unadjusted analyses of error-prone data can bias study results. Validating a subset of records is a cost-effective way of gaining information on the error structure, which in turn can be used to adjust analyses for this error and improve inference. We extend the mean score method for the two-phase analysis of discrete-time survival models, which uses the unvalidated covariates as auxiliary variables that act as surrogates for the unobserved true exposures. This method relies on a two-phase sampling design and an estimation approach that preserves the consistency of complete case regression parameter estimates in the validated subset, with increased precision leveraged from the auxiliary data. Furthermore, we develop optimal sampling strategies which minimize the variance of the mean score estimator for a target exposure under a fixed cost constraint. We consider the setting where an internal pilot is necessary for the optimal design so that the phase two sample is split into a pilot and an adaptive optimal sample. Through simulations and data example, we evaluate efficiency gains of the mean score estimator using the derived optimal validation design compared to balanced and simple random sampling for the phase two sample. We also empirically explore efficiency gains that the proposed discrete optimal design can provide for the Cox proportional hazards model in the setting of a continuous-time survival outcome., Comment: 22 pages, 2 figures, 3 tables, supplementary material

Multiple imputation provides us with efficient estimators in model-based methods for handling missing data under the true model. It is also well-understood that design-based estimators are robust methods that do not require accurately modeling the missing data; however, they can be inefficient. In any applied setting, it is difficult to know whether a missing data model may be good enough to win the bias-efficiency trade-off. Raking of weights is one approach that relies on constructing an auxiliary variable from data observed on the full cohort, which is then used to adjust the weights for the usual Horvitz-Thompson estimator. Computing the optimally efficient raking estimator requires evaluating the expectation of the efficient score given the full cohort data, which is generally infeasible. We demonstrate multiple imputation (MI) as a practical method to compute a raking estimator that will be optimal. We compare this estimator to common parametric and semi-parametric estimators, including standard multiple imputation. We show that while estimators, such as the semi-parametric maximum likelihood and MI estimator, obtain optimal performance under the true model, the proposed raking estimator utilizing MI maintains a better robustness-efficiency trade-off even under mild model misspecification. We also show that the standard raking estimator, without MI, is often competitive with the optimal raking estimator. We demonstrate these properties through several numerical examples and provide a theoretical discussion of conditions for asymptotically superior relative efficiency of the proposed raking estimator., Comment: 24 pages, 3 figures

Physical growth traits can be naturally represented by continuous functions. In a large dataset of infancy growth patterns, we develop a practical approach to infer statistical associations between growth-trajectories and IQ performance in early childhood. The main objective of this study is to show how to assess physical growth curves and detect if particular infancy growth patterns are associated with differences in IQ (Full-scale WASI scores) in later ages using a semi-parametric functional response model. Additionally, we investigate the association between different growth measurements in terms of their cross-correlation with each other, their correlation with later IQ, as well as their time-varying dynamics. This analysis framework can easily incorporate or learn population information in a non-parametric way, rendering the existence of prior population charts partially redundant.