Direct Learning of Treatment-Benefit Rankings for Restricted Mean Survival Time in Randomized Trials with Censoring

原文摘要

In precision medicine, treatment decisions often depend more on identifying which patients are most likely to benefit from treatment than on accurately estimating benefit for every patient. However, existing methods estimate patient-specific treatment effects and derive patient rankings as a secondary step. We propose a direct ranking approach for conditional RMST treatment benefit in randomized trials with right-censored outcomes. We first construct a censoring-adjusted orthogonal RMST pseudo-outcome whose conditional expectation equals the true conditional RMST difference. Then, we compare patients pairwise and optimize a smooth ranking loss that directly targets treatment-benefit ordering. The pairwise criterion incorporates orthogonal corrections for estimation of the event and censoring distributions. We show that the population minimizer of the proposed loss induces the same ordering as the true conditional RMST treatment benefit and that the resulting estimating equation is Neyman-orthogonal to nuisance survival and censoring models. In simulation studies with nonlinear treatment-effect heterogeneity and moderate to heavy censoring, the proposed approach improved rank correlation and treatment-benefit enrichment relative to both plug-in RMST estimation and regression of the same RMST pseudo-outcome. Application to a randomized breast cancer trial demonstrated improved identification of patients with greater treatment benefit. In conclusion, the proposed framework combines orthogonal survival learning with pairwise ranking and may be particularly useful in precision medicine settings where treatment prioritization and patient selection are the primary goals.

摘要来源：arXiv