Faculty Collaboration Database

Doubly Robust Estimators of the Restricted Mean Time in Favor Estimands in Individual- and Cluster-Randomized Trials. Stat Med 2026 Jun;45(13-14):e70599

Date

05/26/2026

Pubmed ID

42186819

DOI

10.1002/sim.70599

Scopus ID

2-s2.0-105040332313 (requires institutional sign-in at Scopus site)

Abstract

Progressive multi-state survival outcomes are common in trials with recurrent or sequential events and require treatment effect estimands that remain interpretable without proportional intensity or Markov assumptions. The restricted mean time in favor of treatment (RMT-IF) extends the restricted mean survival time to ordered multi-state processes and provides such an interpretable estimand. However, existing RMT-IF methods are nonparametric, assume covariate-independent censoring for independent observations, and do not accommodate cluster-randomized trials (CRTs), limiting both efficiency and applicability. We develop a class of doubly robust estimators for RMT-IF under right censoring using an augmented inverse-probability weighting framework that combines stage-specific outcome regression with arm-specific censoring models, yielding consistency when either nuisance model is correctly specified. We further extend the framework to CRTs by formalizing both cluster-level and individual-level average RMT-IF estimands to address informative cluster size and by constructing corresponding doubly robust estimators that account for within-cluster correlation. For inference, we employ model-agnostic jackknife variance estimators in both individually randomized and cluster-randomized settings. Extensive simulation studies demonstrate finite-sample performance, and the methods are illustrated using two randomized trial examples.

Author List

Fang X, Wang B, Tong G, Hu L, Ma S, Li F

Author

Xi Fang Assistant Professor in the Data Science Institute department at Medical College of Wisconsin

MESH terms used to index this publication - Major topics in bold

Cluster Analysis
Computer Simulation
Data Interpretation, Statistical
Humans
Models, Statistical
Randomized Controlled Trials as Topic
Survival Analysis