2 papers
stat.ME2026
Towards Best Practices for Covariate Adjustment in Regulatory Trials: From Fixed to Data-Adaptive Approaches
Laura B. Balzer, Lei Nie, Issa J. Dahabreh +15
While randomization justifies the use of unadjusted effect estimators in randomized trials, there is growing interest in covariate adjustment to improve precision. Adjusting for ba…
stat.ME2026
A CV-TMLE global test approach to improve power in rare disease clinical studies with multiple-component endpoints
Tianyue Zhou, Susan Gruber, Hana Lee +3
Rare disease trials face unique statistical challenges due to limited patient populations and heterogeneous clinical manifestations among patients. Multiple endpoints are often nec…