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Tianyue Zhou

Publications and source records attributed to Tianyue Zhou.

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Considerations for the Integration of Randomized Controlled Trials and Real-World Data

As clinical decision-making increasingly moves toward individualized and context-specific treatment recommendations, reliance on any single evidence source, randomized or observational, may be insufficient. Principled integration of randomized controlled trials and real-world data, grounded in explicit causal frameworks, offers a path toward evidence that is both internally credible and externally relevant. In this article, we describe distinct objectives for the integration of randomized controlled trials and real-world data and discuss how these objectives shape key design and analytic considerations, illustrating the resulting choices through example estimands. We highlight practical issues that commonly arise in applied settings, including data relevance and curation, cross-source comparability, estimand specification, and sensitivity analysis. We aim for this article to help readers evaluate and implement principled approaches to integrating randomized controlled trials and real-world data in ways that can support more reliable treatment recommendations while maintaining regulatory-grade evidentiary standards.

stat.ME

Task Specialization Fine-Tuning for Contextual Reinforcement Learning

Contextual Reinforcement Learning (CRL) seeks to generalize classical RL by maximizing task coverage across a context space of related tasks. While prior works often train from scratch and rely on either multi-task learning for a single policy or strategically training multiple policies, we advocate for a unified alternative: pretraining a single policy with good initial performance, followed by fine-tuning multiple policies for task specialization. This new paradigm, however, introduces unique challenges, such as heterogeneous marginal returns and sample inefficiency. This raises a critical research question: given a pretrained policy and a constrained budget, how much fine-tuning should each task region receive to enable sample-efficient CRL? To this end, we propose Task Specialization Fine-Tuning (TSFT), an online framework that predicts fine-tuning performance with a simple parametric model and exactly solves the resulting discrete budget allocation problem via integer linear programming. Extensive experiments across diverse decision domains, including combinatorial optimization, continuous control, and LLM fine-tuning, demonstrate that TSFT significantly outperforms baselines in task coverage and approaches oracle performance. Our work charts a new direction for model-based CRL, aligning with the modern pretrain-finetune era.

cs.LG

Towards Best Practices for Covariate Adjustment in Regulatory Trials: From Fixed to Data-Adaptive Approaches

While randomization justifies the use of unadjusted effect estimators in randomized trials, there is growing interest in covariate adjustment to improve precision. Adjusting for baseline variables that are prognostic of the outcome can reduce estimator variance, resulting in narrower confidence intervals and increased statistical power. Recent guidance by the U.S. Food and Drug Administration supports fixed adjustment for prognostic covariates using parametric regression models. However, this guidance does not address more flexible approaches using data-adaptive or machine learning methods. We offer our perspectives on covariate adjustment to improve analytic precision. We focus on estimating the average effect for the target population in trials with minimal outcome missingness. We provide a non-technical overview of effect estimators that are unadjusted and effect estimators using fixed versus data-adaptive adjustment. We offer practical suggestions for conducting adjusted analyses that are data-adaptive, fully pre-specified, transparently and reproducibly implemented, robust to model misspecification, and guaranteed to improve precision relative to unadjusted analyses --- all while preserving statistical validity and the causal effect of interest. We hope that sharing our perspectives will foster broader discussion and eventual acceptance of principled, pre-specified, data-adaptive covariate adjustment in randomized trials.

stat.ME

A CV-TMLE global test approach to improve power in rare disease clinical studies with multiple-component endpoints

Rare disease trials face unique statistical challenges due to limited patient populations and heterogeneous clinical manifestations among patients. Multiple endpoints are often necessary to comprehensively capture treatment benefits. A global test is an approach for evaluating whether a treatment has any beneficial effect across multiple endpoints. We propose a new global test based on a weighted composite endpoint. The proposed global test employs shrinkage-based cross-validated targeted maximum likelihood estimation (CV-TMLE) to learn data-adaptive weights that maximize power while maintaining Type I error control. Shrinkage can be tailored to incorporate existing domain knowledge, such as anticipated relative effect sizes. In simulation studies designed to reflect real rare disease trial settings, the proposed procedure demonstrated improved power over standard multiplicity adjustments and classical global tests (such as the O'Brien test), while maintaining nominal Type I error, when effects are heterogeneous across endpoints. The proposed method simultaneously learns an optimal weighted composite outcome and provides an unbiased and efficient targeted maximum likelihood estimator (TMLE) for the average treatment effect (ATE) on that weighted outcome, with valid inference taking into account that the ATE is data dependent.

stat.ME

Structure Detection for Contextual Reinforcement Learning

Contextual Reinforcement Learning (CRL) tackles the problem of solving a set of related Contextual Markov Decision Processes (CMDPs) that vary across different context variables. Traditional approaches--independent training and multi-task learning--struggle with either excessive computational costs or negative transfer. A recently proposed multi-policy approach, Model-Based Transfer Learning (MBTL), has demonstrated effectiveness by strategically selecting a few tasks to train and zero-shot transfer. However, CMDPs encompass a wide range of problems, exhibiting structural properties that vary from problem to problem. As such, different task selection strategies are suitable for different CMDPs. In this work, we introduce Structure Detection MBTL (SD-MBTL), a generic framework that dynamically identifies the underlying generalization structure of CMDP and selects an appropriate MBTL algorithm. For instance, we observe Mountain structure in which generalization performance degrades from the training performance of the target task as the context difference increases. We thus propose M/GP-MBTL, which detects the structure and adaptively switches between a Gaussian Process-based approach and a clustering-based approach. Extensive experiments on synthetic data and CRL benchmarks--covering continuous control, traffic control, and agricultural management--show that M/GP-MBTL surpasses the strongest prior method by 12.49% on the aggregated metric. These results highlight the promise of online structure detection for guiding source task selection in complex CRL environments.

cs.LG

The Nah Bandit: Modeling User Non-compliance in Recommendation Systems

Recommendation systems now pervade the digital world, ranging from advertising to entertainment. However, it remains challenging to implement effective recommendation systems in the physical world, such as in mobility or health. This work focuses on a key challenge: in the physical world, it is often easy for the user to opt out of taking any recommendation if they are not to her liking, and to fall back to her baseline behavior. It is thus crucial in cyber-physical recommendation systems to operate with an interaction model that is aware of such user behavior, lest the user abandon the recommendations altogether. This paper thus introduces the Nah Bandit, a tongue-in-cheek reference to describe a Bandit problem where users can say `nah' to the recommendation and opt for their preferred option instead. As such, this problem lies in between a typical bandit setup and supervised learning. We model the user non-compliance by parameterizing an anchoring effect of recommendations on users. We then propose the Expert with Clustering (EWC) algorithm, a hierarchical approach that incorporates feedback from both recommended and non-recommended options to accelerate user preference learning. In a recommendation scenario with $N$ users, $T$ rounds per user, and $K$ clusters, EWC achieves a regret bound of $O(N\sqrt{T\log K} + NT)$, achieving superior theoretical performance in the short term compared to LinUCB algorithm. Experimental results also highlight that EWC outperforms both supervised learning and traditional contextual bandit approaches. This advancement reveals that effective use of non-compliance feedback can accelerate preference learning and improve recommendation accuracy. This work lays the foundation for future research in Nah Bandit, providing a robust framework for more effective recommendation systems.

cs.LG

Expert with Clustering: Hierarchical Online Preference Learning Framework

Emerging mobility systems are increasingly capable of recommending options to mobility users, to guide them towards personalized yet sustainable system outcomes. Even more so than the typical recommendation system, it is crucial to minimize regret, because 1) the mobility options directly affect the lives of the users, and 2) the system sustainability relies on sufficient user participation. In this study, we consider accelerating user preference learning by exploiting a low-dimensional latent space that captures the mobility preferences of users. We introduce a hierarchical contextual bandit framework named Expert with Clustering (EWC), which integrates clustering techniques and prediction with expert advice. EWC efficiently utilizes hierarchical user information and incorporates a novel Loss-guided Distance metric. This metric is instrumental in generating more representative cluster centroids. In a recommendation scenario with $N$ users, $T$ rounds per user, and $K$ options, our algorithm achieves a regret bound of $O(N\sqrt{T\log K} + NT)$. This bound consists of two parts: the first term is the regret from the Hedge algorithm, and the second term depends on the average loss from clustering. To the best of the authors knowledge, this is the first work to analyze the regret of an integrated expert algorithm with k-Means clustering. This regret bound underscores the theoretical and experimental efficacy of EWC, particularly in scenarios that demand rapid learning and adaptation. Experimental results highlight that EWC can substantially reduce regret by 27.57% compared to the LinUCB baseline. Our work offers a data-efficient approach to capturing both individual and collective behaviors, making it highly applicable to contexts with hierarchical structures. We expect the algorithm to be applicable to other settings with layered nuances of user preferences and information.

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