Biostatistics Commons^™

Robust Estimation Of The Average Treatment Effect In Alzheimer's Disease Clinical Trials, Michael Rosenblum, Aidan Mcdermont, Elizabeth Colantuoni

Johns Hopkins University, Dept. of Biostatistics Working Papers

The primary analysis of Alzheimer's disease clinical trials often involves a mixed-model repeated measure (MMRM) approach. We consider another estimator of the average treatment effect, called targeted minimum loss based estimation (TMLE). This estimator is more robust to violations of assumptions about missing data than MMRM.

We compare TMLE versus MMRM by analyzing data from a completed Alzheimer's disease trial data set and by simulation studies. The simulations involved different missing data distributions, where loss to followup at a given visit could depend on baseline variables, treatment assignment, and the outcome measured at previous visits. The TMLE generally has improved …