4 papers
From Cumulative Weights to Marginal Density Ratios: Per-Protocol Estimation in Sequential Target Trial Emulation
Zern Ke, Mingshi Cui, Feng Dai +3
Sequential target trial emulation evaluates eligibility at multiple baseline times to emulate a sequence of randomized trials using observational data. Estimating per-protocol effe…
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…
Mitigating the Winner's Curse While Controlling Multiplicity: e-Process Methods for Anytime-Valid Inference in Dose-Ranging Trials
Victor K. de la Pena, Fangyuan Lin, Demissie Alemayehu +1
Phase II dose-ranging trials often report the largest observed dose-control effect while inspecting accumulating data repeatedly. This creates two coupled distortions: selection op…
Advancing Evidence Generation in Biomedical Research Using Natural Hermite and Propensity Score Indices: Applications to External Control Arms
Javier Cabrera, Berhanu Alemayehu, Demissie Alemayehu +1
When it is not feasible to conduct randomized controlled trials (RCTs), the use of external control arms based on real-world data (RWD) may be a viable option. However, challenges…