Single-arm trials are an important study design for evaluating drug efficacy and safety without enrolling patients into a control arm. Although they do not provide the gold-standard evidence of randomized controlled trials, they are increasingly used in clinical development as they offer an efficient, ethical, and practical alternative. A wide variety of approaches can be used to construct control comparators and estimate treatment effects, from fixed comparators informed by clinical knowledge to data-based and model-based patient-level comparators, also known as synthetic controls. Powerful and flexible machine learning models can allow outcome-model-based synthetic controls to overcome key limitations of direct data-based approaches, yield more robust estimates of treatment effects, and provide a principled way to incorporate corrections or encode additional assumptions when external data are not directly comparable. In this work, we argue that outcome-model-based synthetic control arms are an important tool for single-arm trials. We focus on digital twins, personalized predictions of disease progression generated from machine learning models trained on historical datasets, which naturally leverage these flexible approaches. We review doubly robust estimators, present power and sample size formulas, and discuss trade-offs in selecting historical data for training and analysis. We also outline practical considerations for deploying digital twins within the framework of recent FDA draft guidance on the use of artificial intelligence in drug development. Finally, we reanalyze data from trials in amyotrophic lateral sclerosis and Huntington's disease to demonstrate the proposed methods.

Cross-domain recommendation forms a crucial component in recommendation systems. It leverages auxiliary information through source domain tasks or features to enhance target domain recommendations. However, incorporating inconsistent source domain tasks may result in insufficient cross-domain modeling or negative transfer. While incorporating source domain features without considering the underlying causal relationships may limit their contribution to final predictions. Thus, a natural idea is to directly train a cross-domain representation on a causality-labeled dataset from the source to target domain. Yet this direction has been rarely explored, as identifying unbiased real causal labels is highly challenging in real-world scenarios. In this work, we attempt to take a first step in this direction by proposing a causality-enhanced framework, named CE-CDR. Specifically, we first reformulate the cross-domain recommendation as a causal graph for principled guidance. We then construct a causality-aware dataset heuristically. Subsequently, we derive a theoretically unbiased Partial Label Causal Loss to generalize beyond the biased causality-aware dataset to unseen cross-domain patterns, yielding an enriched cross-domain representation, which is then fed into the target model to enhance target-domain recommendations. Theoretical and empirical analyses, as well as extensive experiments, demonstrate the rationality and effectiveness of CE-CDR and its general applicability as a model-agnostic plugin. Moreover, it has been deployed in production since April 2025, showing its practical value in real-world applications.

Unbiased Learning to Rank (ULTR) aims to leverage biased implicit user feedback (e.g., click) to optimize an unbiased ranking model. The effectiveness of the existing ULTR methods has primarily been validated on synthetic datasets. However, their performance on real-world click data remains unclear. Recently, Baidu released a large publicly available dataset of their web search logs. Subsequently, the NTCIR-17 ULTRE-2 task released a subset dataset extracted from it. We conduct experiments on commonly used or effective ULTR methods on this subset to determine whether they maintain their effectiveness. In this paper, we propose a Contextual Dual Learning Algorithm with Listwise Distillation (CDLA-LD) to simultaneously address both position bias and contextual bias. We utilize a listwise-input ranking model to obtain reconstructed feature vectors incorporating local contextual information and employ the Dual Learning Algorithm (DLA) method to jointly train this ranking model and a propensity model to address position bias. As this ranking model learns the interaction information within the documents list of the training set, to enhance the ranking model's generalization ability, we additionally train a pointwise-input ranking model to learn the listwise-input ranking model's capability for relevance judgment in a listwise manner. Extensive experiments and analysis confirm the effectiveness of our approach.

Recommender systems often suffer from selection bias as users tend to rate their preferred items. The datasets collected under such conditions exhibit entries missing not at random and thus are not randomized-controlled trials representing the target population. To address this challenge, a doubly robust estimator and its enhanced variants have been proposed as they ensure unbiasedness when accurate imputed errors or predicted propensities are provided. However, we argue that existing estimators rely on miscalibrated imputed errors and propensity scores as they depend on rudimentary models for estimation. We provide theoretical insights into how miscalibrated imputation and propensity models may limit the effectiveness of doubly robust estimators and validate our theorems using real-world datasets. On this basis, we propose a Doubly Calibrated Estimator that involves the calibration of both the imputation and propensity models. To achieve this, we introduce calibration experts that consider different logit distributions across users. Moreover, we devise a tri-level joint learning framework, allowing the simultaneous optimization of calibration experts alongside prediction and imputation models. Through extensive experiments on real-world datasets, we demonstrate the superiority of the Doubly Calibrated Estimator in the context of debiased recommendation tasks.

In recommender systems, users always choose the favorite items to rate, which leads to data missing not at random and poses a great challenge for unbiased evaluation and learning of prediction models. Currently, the doubly robust (DR) methods have been widely studied and demonstrate superior performance. However, in this paper, we show that DR methods are unstable and have unbounded bias, variance, and generalization bounds to extremely small propensities. Moreover, the fact that DR relies more on extrapolation will lead to suboptimal performance. To address the above limitations while retaining double robustness, we propose a stabilized doubly robust (StableDR) learning approach with a weaker reliance on extrapolation. Theoretical analysis shows that StableDR has bounded bias, variance, and generalization error bound simultaneously under inaccurate imputed errors and arbitrarily small propensities. In addition, we propose a novel learning approach for StableDR that updates the imputation, propensity, and prediction models cyclically, achieving more stable and accurate predictions. Extensive experiments show that our approaches significantly outperform the existing methods.

Causality has been defined with the identification of the cause or causes of a phenomenon by establishing covariation of cause and effect, a time-order relationship with the cause preceding the effect, and the elimination of plausible alternative causes; see Shaughnessy et al. (2000). To claim a specific causal effect between two variables is quite a strong claim. First, there needs to be well-defined treatment and outcome with an established covariance. Second, the treatment must proceed the observed outcome. Third, there must be no other present confounders, i.e., other "treatments" that could have their own causal effect; see Judea (2010). While these conditions are not perfect parameters for inferring a causal relationship between a treatment and outcome, they help researchers remove strong bias from their studies; see Hammerton and Munafò (2021). A causal effect found in a causal inference study is almost never the true causal effect, rather a less-biased estimate that is significantly closer to the true causal effect of the treatment on the outcome. To calculate a true causal effect would require "counterfactual" outcomes that cannot be measured; see Judea (2010). To describe a counterfactual outcome, let us define some treatment Z and an outcome Y.

We are interested in estimating the effect of a treatment applied to individuals at multiple sites, where data is stored locally for each site. Due to privacy constraints, individual-level data cannot be shared across sites; the sites may also have heterogeneous populations and treatment assignment mechanisms. Motivated by these considerations, we develop federated methods to draw inference on the average treatment effects of combined data across sites. Our methods first compute summary statistics locally using propensity scores and then aggregate these statistics across sites to obtain point and variance estimators of average treatment effects. We show that these estimators are consistent and asymptotically normal. To achieve these asymptotic properties, we find that the aggregation schemes need to account for the heterogeneity in treatment assignments and in outcomes across sites. We demonstrate the validity of our federated methods through a comparative study of two large medical claims databases.

Creating a churn propensity model is now pretty standard for data scientists. Today, churn is the most common data science problem in the world, because every company wants recurring revenue. But how do you go from a churn model to churn prevention? It is much harder than it sounds. Suppose you have a machine learning model that can predict churn.

In recommender systems, a common problem is the presence of various biases in the collected data, which deteriorates the generalization ability of the recommendation models and leads to inaccurate predictions. Doubly robust (DR) learning has been studied in many tasks in RS, with the advantage that unbiased learning can be achieved when either a single imputation or a single propensity model is accurate. In this paper, we propose a multiple robust (MR) estimator that can take the advantage of multiple candidate imputation and propensity models to achieve unbiasedness. Specifically, the MR estimator is unbiased when any of the imputation or propensity models, or a linear combination of these models is accurate. Theoretical analysis shows that the proposed MR is an enhanced version of DR when only having a single imputation and propensity model, and has a smaller bias. Inspired by the generalization error bound of MR, we further propose a novel multiple robust learning approach with stabilization. We conduct extensive experiments on real-world and semi-synthetic datasets, which demonstrates the superiority of the proposed approach over state-of-the-art methods.

The propensity model introduced by Jain et al. 2016 has become a standard approach for dealing with missing and long-tail labels in extreme multi-label classification (XMLC). In this paper, we critically revise this approach showing that despite its theoretical soundness, its application in contemporary XMLC works is debatable. We exhaustively discuss the flaws of the propensity-based approach, and present several recipes, some of them related to solutions used in search engines and recommender systems, that we believe constitute promising alternatives to be followed in XMLC.