SPYCE: A Doubly Robust Estimator for Trials Targeting Early Huntington Disease under Outcome-Dependent Censoring
SPYCE:结果依赖删失下早期亨廷顿病终点的双重稳健估计
Clinical trials for neurodegenerative diseases must identify sensitive endpoints -- outcomes that change rapidly enough to detect treatment effects. In Huntington disease, this requires measuring how outcomes change as participants approach Stage 1. Yet many participants exit studies before reaching this stage, making their time to Stage 1 right-censored. Estimating how outcomes change requires models for both time to Stage 1 and time to study exit. When participants with…