Cross-fitted estimators that transport information from the two labeled sources through source-specific density ratios are proposed that establish asymptotically linear inference for TPR and FPR, consistency and pointwise inference for the ROC curve, and asymptotically normal inference for AUC.
Abstract
In transfer-learning settings, a model derived from abundant surrogate labels may be deployed in a target population where gold-standard outcomes are unobserved. Evaluating its target performance is essential for determining whether decisions based on the model remain reliable, yet it is difficult when gold labels are scarce, and covariate distributions differ across data sources. We study a three-sample setting with a small gold-labeled source, a larger surrogate-labeled source, and an unlabeled target. Under conditional transportability, we evaluate the surrogate-derived model against the latent gold-standard outcome in the target population. We propose cross-fitted estimators that transport information from the two labeled sources through source-specific density ratios. We also combine outcome-regression augmentation with a kernel correction for estimating the model near a threshold, accounting for uncertainty from all three samples. We establish asymptotically linear inference for TPR and FPR, consistency and pointwise inference for the ROC curve, and asymptotically normal inference for AUC. Simulations assess bias, coverage, and sensitivity to bandwidth and relative sample sizes. A retrospective temporal validation on Chatbot Arena and a semi-synthetic ACS-Income study provide validation in real-world AI applications.
Distributional mismatch between the data used to construct a statistical procedure and the population to which it is ultimately applied is pervasive in modern data analysis. We study covariate shift, a fundamental instance of this problem, and develop an adaptive importance-weighted model averaging method for prediction when labeled observations are available from a source distribution, whereas only unlabeled covariates are observed from the target distribution. Procedures fitted directly to the source sample generally optimize prediction risk under the source distribution and may therefore be suboptimal for target prediction. Importance weighting by the density ratio between the target and source covariate marginals provides a natural correction, but a small number of large density-ratio values can substantially inflate the variance of the resulting estimator in finite samples. We address this bias-variance trade-off by treating the degree of importance-weighting correction as a source of model uncertainty. Specifically, we construct a family of adaptive importance-weighted least-squares estimators by raising the estimated density ratio to a range of exponents, with the endpoints corresponding to ordinary least squares and standard importance-weighted least squares, and form a data-driven average over these candidates. Under model misspecification, the proposed model averaging estimator is shown to be asymptotically optimal relative to the infeasible best convex combination of the candidate estimators. Under correct specification, a diverging penalty is shown to make the selected weights concentrate near the ordinary least-squares endpoint. Simulations and a real-data application show that the proposed method achieves competitive target-prediction performance across the settings considered.
Randomized trials provide internally valid treatment-effect evidence, but trial participants may not represent the target population. In contrast, observational studies are often closer to the target population, but their treatment assignment may be affected by possible hidden confounding. We develop a robust posterior-drift framework for estimating the average treatment effect in an observational target population when exact conditional-effect transportability may fail. The framework represents observational conditional potential-outcome regressions as their randomized-trial counterparts plus source-specific drifts. The randomized trial serves as an internally valid anchor, while the observational study supplies the target covariate distribution and partial information about the target causal contrast. To account for possible hidden confounding, we consider a Rosenbaum-type uncertainty set induced by a sensitivity parameter on the generalized propensity score and estimate the drift through a minimax worst-case risk criterion. We derive efficiency results in auxiliary regimes, establish uniform concentration and near-optimality guarantees for the minimax estimator, and handle general parametric and smooth nonparametric drift classes. Simulations and an ACTG 175--WIHS application show that the proposed analysis yields more cautious and interpretable target-population effect estimates than exact-transportability analyses.
Statistical prediction models for binary outcomes are becoming increasingly popular. One significant challenge is calibrating these models to suit the characteristics of a target population that is structurally different from the original population. Calibration is especially challenging when there is no training data available from the target population. To address this problem, we propose a novel calibration method, SimCal, which uses synthetic data generated from the model development data in conjunction with marginal statistics from the calibration cohort. We show that expert judgment modeling (EJM) may be used for calibration if cross-sectional data from the target population are available comprising expert judgments about the potential outcome and the covariates. We describe three alternative calibration approaches when calibration data are lacking: similarity-binning averaging (SBA), adaptive calibration of predictions (ACP), and Elkan calibration. In a simulation study, we compare SBA, ACP, Elkan calibration, and SimCal. R code for applying these methods is provided from the re-analysis of data on coronary artery disease. We illustrate all 5 calibration approaches with a real data set for predicting functional outcome after stroke and all approaches but EJM in the re-analysis of the Cleveland Clinic data. None of the approaches performed convincingly well in all situations. SimCal performed well when model parameters were correctly specified. EJM failed on the stroke data. Further research is urgently required for calibration in the absence of calibration data.
E. Di Carluccio, G. Koliopanos, F. M. Ojeda et al.· medRxiv· 0 citations
Average dose-response functions are widely used to summarize causal effects of continuous treatments, but most existing methods assume that the observed sample represents the target population. We study a covariate-shift setting in which covariates, treatment, and outcome are observed in a labelled source sample, while only covariates are observed in the target sample. We develop a two-sample local polynomial regression framework based on pseudo-outcomes that use source outcomes to address confounding and target covariates to define the population of interest. We further propose a source-to-target extension of distance covariance optimal weighting (DCOW), designed to remove treatment-covariate dependence in the source sample while aligning the weighted source covariate distribution with the target population. A central theoretical contribution is a weight-level analysis of this optimization-based procedure: we show that the population criterion identifies the oracle source-to-target weights and that approximate empirical minimizers, including exact minimizers as a special case, converge uniformly to these weights under regularity conditions. We also establish consistency and asymptotic normality of the resulting estimator. Simulations show that the proposed method improves target dose-response estimation relative to DCOW, generalized-propensity-score weighting, entropy balancing, and unweighted alternatives. We illustrate the method in a county-level analysis of PM2.5 exposure and subsequent heart-disease mortality using a source-target validation design.
A decision-aware weak-to-strong (W2S) framework that leverages both labeled and unlabeled data to improve contextual stochastic optimization and establishes a non-asymptotic upper bound on the excess decision risk of W2S and a complementary lower bound for a strong-only benchmark.
Modern generative models increasingly produce distribution-valued outputs, such as predicted cellular responses to genetic perturbations in single-cell genomics. While these models provide valuable auxiliary information, they are inherently imperfect, creating a need for statistical methods that leverage their predictions without relying on their correctness. We propose generation-powered inference (GPI), a general framework for improving inference on distribution-valued parameters using auxiliary generative models. Focusing on Wasserstein barycenters and related distributional functionals, we introduce a function-valued bridge representation that transforms inference in the nonlinear Wasserstein space into estimation of a mean function in a Hilbert space, enabling an augmented estimation framework analogous to prediction-powered inference. We develop a family of GPI estimators with optimal information borrowing, establish consistency, asymptotic normality, and simultaneous confidence bands, and derive valid inference for linear functionals and Wasserstein distances. Simulation studies demonstrate efficiency gains over labeled-data-only methods and robust performance under generative model misspecification. We illustrate the proposed framework using a Perturb-seq study of K562 cells, where synthetic perturbation responses generated by the State foundation model are used to improve inference for pathway-level consensus gene expression distributions associated with perturbations of the 40S ribosome module.
Yijiao Zhang, Hongzhe Li· 0 citations
We use cookies to run the site and, with your consent, for analytics and to show ads.
See our Cookie Policy.
