13 papers
Towards Best Practices for Covariate Adjustment in Regulatory Trials: From Fixed to Data-Adaptive Approaches
Laura B. Balzer, Lei Nie, Issa J. Dahabreh +15
The paper discusses how to improve precision in randomized clinical trials by using covariate adjustment, comparing traditional fixed parametric methods with flexible data‑adaptive…
Targeted maximum likelihood estimation for longitudinal two-stage designs with outcome subsampling
Kirsten E. Landsiedel, Maya L. Petersen, Mark J. van der Laan
We consider efficient estimation of causal parameters in longitudinal two-stage designs with outcome subsampling, motivated by resampling designs in HIV-related mortality studies.…
Higher-Order Efficient Estimators: A Review and Simulation-Based Benchmark Study
Zeyi Wang, Mark J. van der Laan
Higher-order efficient estimators extend standard first-order semiparametric estimators by replacing second-order residuals with third- or higher-order terms, potentially enabling…
Improving the Efficiency of Subgroup Analysis in Randomized Controlled Trials with TMLE
Sky Qiu, Nerissa Nance, Rachael Phillips +3
Subgroup analyses within randomized controlled trials are often underpowered due to limited sample sizes. We address this challenge by leveraging trial participants outside the sub…
Estimation of treatment effects in presence of differential use of post-randomization concomitant medication with time-to-event outcomes
Helene C. W. Rytgaard, Edwin Fong, Jens M. Tarp +3
In placebo-controlled randomized trials, the post-randomization use of concomitant medications may be higher in the placebo arm than in the treatment arm. This may dilute the full…
Externally Controlled Trials: A Review of Design and Borrowing Through a Causal Lens
Ke Zhu, Rima Izem, Peng Yang +8
Externally controlled trials (ECTs) are increasingly used when randomized controls are infeasible, unethical, or insufficient, including applications in rare diseases, oncology, pe…