Adaptive trial design and interim decision-making using incomplete longitudinal measurements: Methods and application to myasthenia gravis
Sample size re-estimation designs using a promising zone framework are widely used adaptive trial methodologies that guide study continuation or modification during interim analyses. Conventional implementations often base interim calculations solely on participants with available primary endpoints, overlooking predictive information from baseline and earlier visits. This underutilization can lead to inefficient interim decision-making. In this work, we adapt semi-parametric efficient estimators...