arXiv subjects

Ying-Qi Zhao

Publications and source records attributed to Ying-Qi Zhao.

14 recordsLinked to original sources

arXiv2026

Primal--Dual Alternating Neural Learning for Timely Classification with Performance Guarantees

Timely risk classification is essential in many clinical monitoring settings, where decisions must balance the benefit of classifying patients early for subsequent intervention against the value of observing additional data. Yet most existing statistical and machine-learning methods are designed for fully observed trajectories and offer limited control over key operating characteristics such as sensitivity, specificity, and monitoring cost. We cast the sequential classification problem within a multi-objective optimization framework targeting these three criteria. We characterize the optimal decision rule through a value recursion that quantifies, at each time point, the trade-off between immediate classification and continued monitoring. To estimate the rule from data, we formulate a constrained optimization problem that maximizes specificity while enforcing prespecified sensitivity and monitoring-cost constraints. We then develop an estimation procedure that employs a recurrent neural network to approximate the evolving value processes and a primal--dual updating scheme to satisfy the performance constraints. Through simulation studies and an application to continuous glucose monitoring for hypoglycemia risk prediction, we demonstrate that the proposed method yields accurate and timely sequential decision rules that adhere to the desired operating characteristics.

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arXiv2026

Inference with non-differentiable surrogate loss in a general high-dimensional classification framework

Penalized empirical risk minimization with a surrogate loss function is often used to learn a high-dimensional linear decision rule in classification problems. Although much of the literature focus on the generalization error, there is a lack of inference procedures for identifying the driving factors of the estimated decision rule, especially when the surrogate loss is non-differentiable. We propose a kernel-smoothed decorrelated score to construct hypothesis tests and interval estimators for a linear decision rule estimated using a piece-wise linear surrogate loss, which has a discontinuous gradient and non-regular Hessian. Specifically, we adopt kernel approximations to smooth the discontinuous gradient near discontinuity points and approximate the non-regular Hessian of the surrogate loss. In applications where additional nuisance parameters are involved, we propose a novel cross-fitted version to accommodate flexible nuisance estimates and kernel approximations. We establish the limiting distribution of the kernel-smoothed decorrelated score and its cross-fitted version in a high-dimensional setup. Simulation and real data analysis are conducted to demonstrate the validity and the superiority of the proposed method.

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arXiv2026

Developing Performance-Guaranteed Biomarker Combination Rules with Integrated External Information under Practical Constraint

In clinical practice, there is significant interest in integrating novel biomarkers with existing clinical data to construct interpretable and robust decision rules. Motivated by the need to improve decision-making for early disease detection, we propose a framework for developing an optimal biomarker-based clinical decision rule that is both clinically meaningful and practically feasible. Specifically, our procedure constructs a linear decision rule designed to achieve optimal performance among class of linear rules by maximizing the true positive rate while adhering to a pre-specified positive predictive value constraint. Additionally, our method can adaptively incorporate individual risk information from external source to enhance performance when such information is beneficial. We establish the asymptotic properties of our proposed estimator and compare to the standard approach used in practice through extensive simulation studies. Results indicate that our approach offers strong finite-sample performance. We also apply the proposed methods to develop biomarker-based screening rules for pancreatic ductal adenocarcinoma (PDAC) among new-onset diabetes (NOD) patients.

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arXiv2026

Nested Instrumental Variables Analysis: Switcher Average Treatment Effect, Identification, Efficient Estimation and Generalizability

Instrumental variables (IVs) are widely used to estimate causal effects from non-randomized data. A canonical example is a randomized trial with noncompliance, in which the randomized treatment assignment serves as an IV for the non-ignorable treatment received. Under a monotonicity assumption, a valid IV nonparametrically identifies the average treatment effect among a latent complier subgroup, whose generalizability is often under debate. In many studies, there exist multiple versions of an IV, for instance, different nudges to take the same treatment in different study sites in a multicenter clinical trial. These different versions of an IV may result in different compliance rates and offer a unique opportunity to study IV estimates' generalizability. In this article, we introduce a novel nested IV assumption and study identification of the average treatment effect among two latent subgroups: always-compliers and switchers, who are defined based on the joint potential treatment received under two versions of a binary IV. We derive the efficient influence function for the SWitcher Average Treatment Effect (SWATE) under a nonparametric model and propose efficient estimators. We then propose formal statistical tests of the generalizability of IV estimates under the nested IV framework. The proposed tests are flexible nonparametric generalizations of classical overidentification tests that allow estimating nuisance parameters using machine learning tools. We apply the proposed method to the Prostate, Lung, Colorectal and Ovarian (PLCO) Cancer Screening Trial and study the causal effect of colorectal cancer screening and its generalizability.

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arXiv2023

Efficient surrogate-assisted inference for patient-reported outcome measures with complex missing mechanism

Patient-reported outcome (PRO) measures are increasingly collected as a means of measuring healthcare quality and value. The capability to predict such measures enables patient-provider shared decision making and the delivery of patient-centered care. However, due to their voluntary nature, PRO measures often suffer from a high missing rate, and the missingness may depend on many patient factors. Under such a complex missing mechanism, statistical inference of the parameters in prediction models for PRO measures is challenging, especially when flexible imputation models such as machine learning or nonparametric methods are used. Specifically, the slow convergence rate of the flexible imputation model may lead to non-negligible bias, and the traditional missing propensity, capable of removing such a bias, is hard to estimate due to the complex missing mechanism. To efficiently infer the parameters of interest, we propose to use an informative surrogate that can lead to a flexible imputation model lying in a low-dimensional subspace. To remove the bias due to the flexible imputation model, we identify a class of weighting functions as alternatives to the traditional propensity score and estimate the low-dimensional one within the identified function class. Based on the estimated low-dimensional weighting function, we construct a one-step debiased estimator without using any information of the true missing propensity. We establish the asymptotic normality of the one-step debiased estimator. Simulation and an application to real-world data demonstrate the superiority of the proposed method.

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arXiv2022

Handling Nonmonotone Missing Data with Available Complete-Case Missing Value Assumption

Nonmonotone missing data is a common problem in scientific studies. The conventional ignorability and missing-at-random (MAR) conditions are unlikely to hold for nonmonotone missing data and data analysis can be very challenging with few complete data. In this paper, we introduce the available complete-case missing value (ACCMV) assumption for handling nonmonotone and missing-not-at-random (MNAR) problems. Our ACCMV assumption is applicable to data set with a small set of complete observations and we show that the ACCMV assumption leads to nonparametric identification of the distribution for the variables of interest. We further propose an inverse probability weighting estimator, a regression adjustment estimator, and a multiply-robust estimator for estimating a parameter of interest. We studied the underlying asymptotic and efficiency theories of the proposed estimators. We show the validity of our method with simulation studies and further illustrate the applicability of our method by applying it to a diabetes data set from electronic health records.

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arXiv2022

Long-term effect estimation when combining clinical trial and observational follow-up datasets

Combining experimental and observational follow-up datasets has received a lot of attention lately. In a time-to-event setting, recent work has used medicare claims to extend the follow-up period for participants in a prostate cancer clinical trial. This allows the estimation of the long-term effect that cannot be estimated by clinical trial data alone. In this paper, we study the estimation of long-term effect when participants in a clinical trial are linked to an observational follow-up dataset with incomplete data. Such data linkages are often incomplete for various reasons. We formulate incomplete linkages as a missing data problem with careful considerations of the relationship between the linkage status and the missing data mechanism. We use the popular Cox proportional hazard model as a working model to define the long-term effect. We propose a conditional linking at random (CLAR) assumption and an inverse probability of linkage weighting (IPLW) partial likelihood estimator. We show that our IPLW partial likelihood estimator is consistent and asymptotically normal. We further extend our approach to incorporate time-dependent covariates. Simulations results confirm the validity of our method, and we further apply our methods to the SWOG study.

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arXiv2021

Estimation and inference on high-dimensional individualized treatment rule in observational data using split-and-pooled de-correlated score

With the increasing adoption of electronic health records, there is an increasing interest in developing individualized treatment rules, which recommend treatments according to patients' characteristics, from large observational data. However, there is a lack of valid inference procedures for such rules developed from this type of data in the presence of high-dimensional covariates. In this work, we develop a penalized doubly robust method to estimate the optimal individualized treatment rule from high-dimensional data. We propose a split-and-pooled de-correlated score to construct hypothesis tests and confidence intervals. Our proposal utilizes the data splitting to conquer the slow convergence rate of nuisance parameter estimations, such as non-parametric methods for outcome regression or propensity models. We establish the limiting distributions of the split-and-pooled de-correlated score test and the corresponding one-step estimator in high-dimensional setting. Simulation and real data analysis are conducted to demonstrate the superiority of the proposed method.

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arXiv2020

Near-optimal Individualized Treatment Recommendations

Individualized treatment recommendation (ITR) is an important analytic framework for precision medicine. The goal is to assign proper treatments to patients based on their individual characteristics. From the machine learning perspective, the solution to an ITR problem can be formulated as a weighted classification problem to maximize the average benefit that patients receive from the recommended treatments. Several methods have been proposed for ITR in both binary and multicategory treatment setups. In practice, one may prefer a more flexible recommendation with multiple treatment options. This motivates us to develop methods to obtain a set of near-optimal individualized treatment recommendations alternative to each other, called alternative individualized treatment recommendations (A-ITR). We propose two methods to estimate the optimal A-ITR within the outcome weighted learning (OWL) framework. We show the consistency of these methods and obtain an upper bound for the risk between the theoretically optimal recommendation and the estimated one. We also conduct simulation studies, and apply our methods to a real data set for Type 2 diabetic patients with injectable antidiabetic treatments. These numerical studies have shown the usefulness of the proposed A-ITR framework. We develop a R package aitr which can be found at https://github.com/menghaomiao/aitr.

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arXiv2019

Sample Size Calculations for SMARTs

Sequential Multiple Assignment Randomized Trials (SMARTs) are considered the gold standard for estimation and evaluation of treatment regimes. SMARTs are typically sized to ensure sufficient power for a simple comparison, e.g., the comparison of two fixed treatment sequences. Estimation of an optimal treatment regime is conducted as part of a secondary and hypothesis-generating analysis with formal evaluation of the estimated optimal regime deferred to a follow-up trial. However, running a follow-up trial to evaluate an estimated optimal treatment regime is costly and time-consuming; furthermore, the estimated optimal regime that is to be evaluated in such a follow-up trial may be far from optimal if the original trial was underpowered for estimation of an optimal regime. We derive sample size procedures for a SMART that ensure: (i) sufficient power for comparing the optimal treatment regime with standard of care; and (ii) the estimated optimal regime is within a given tolerance of the true optimal regime with high-probability. We establish asymptotic validity of the proposed procedures and demonstrate their finite sample performance in a series of simulation experiments.

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arXiv2019

Simultaneous disease mapping and hot spot detection with application to childhood obesity surveillance from electronic health records

Electronic health records (EHRs) have become a platform for data-driven surveillance on a granular level in recent years. In this paper, we make use of EHRs for early prevention of childhood obesity. The proposed method simultaneously provides smooth disease mapping and outlier information for obesity prevalence, which are useful for raising public awareness and facilitating targeted intervention. More precisely, we consider a penalized multilevel generalized linear model. We decompose regional contribution into smooth and sparse signals, which are automatically identified by a combination of fusion and sparse penalties imposed on the likelihood function. In addition, we weigh the proposed likelihood to account for the missingness and potential non-representativeness arising from the EHR data. We develop a novel alternating minimization algorithm, which is computationally efficient, easy to implement, and guarantees convergence. Simulation studies demonstrate superior performance of the proposed method. Finally, we apply our method to the University of Wisconsin Population Health Information Exchange database.

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arXiv2019

Constructing Stabilized Dynamic Treatment Regimes for Censored Data

Stabilized dynamic treatment regimes are sequential decision rules for individual patients that not only adaptive throughout the disease progression but also remain consistent over time in format. The estimation of stabilized dynamic treatment regimes becomes more complicated when the clinical outcome of interest is a survival time subject to censoring. To address this challenge, we propose two novel methods, censored shared-Q-learning and censored shared-O-learning. Both methods incorporate clinical preferences into a qualitative rule, where the parameters indexing the decision rules are shared across different stages and estimated simultaneously. We use extensive simulation studies to demonstrate the superior performance of the proposed methods. The methods are further applied to the Framingham Study to derive treatment rules for cardiovascular disease.

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arXiv2019

Efficient augmentation and relaxation learning for individualized treatment rules using observational data

Individualized treatment rules aim to identify if, when, which, and to whom treatment should be applied. A globally aging population, rising healthcare costs, and increased access to patient-level data have created an urgent need for high-quality estimators of individualized treatment rules that can be applied to observational data. A recent and promising line of research for estimating individualized treatment rules recasts the problem of estimating an optimal treatment rule as a weighted classification problem. We consider a class of estimators for optimal treatment rules that are analogous to convex large-margin classifiers. The proposed class applies to observational data and is doubly-robust in the sense that correct specification of either a propensity or outcome model leads to consistent estimation of the optimal individualized treatment rule. Using techniques from semiparametric efficiency theory, we derive rates of convergence for the proposed estimators and use these rates to characterize the bias-variance trade-off for estimating individualized treatment rules with classification-based methods. Simulation experiments informed by these results demonstrate that it is possible to construct new estimators within the proposed framework that significantly outperform existing ones. We illustrate the proposed methods using data from a labor training program and a study of inflammatory bowel syndrome.

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arXiv2015

Active Clinical Trials for Personalized Medicine

Individualized treatment rules (ITRs) tailor treatments according to individual patient characteristics. They can significantly improve patient care and are thus becoming increasingly popular. The data collected during randomized clinical trials are often used to estimate the optimal ITRs. However, these trials are generally expensive to run, and, moreover, they are not designed to efficiently estimate ITRs. In this paper, we propose a cost-effective estimation method from an active learning perspective. In particular, our method recruits only the "most informative" patients (in terms of learning the optimal ITRs) from an ongoing clinical trial. Simulation studies and real-data examples show that our active clinical trial method significantly improves on competing methods. We derive risk bounds and show that they support these observed empirical advantages.

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