BAA - FY25C1FP4 - Genentech

Rare diseases with longitudinal outcomes are an unmet need with only a small percentage of approved treatment. However, challenges in innovative statistical designs for hybrid RCTs (incorporation of external controls in randomized clinical trials) in the longitudinal settings remain to be tackled. As a solution to this, we propose the following aims: Aim 1: Develop causal approaches to handle different missing patterns in the longitudinal settings in the design of hybrid RCTs. Aim 2: Bias correction in designing hybrid RCTs leveraging external controls in longitudinal settings.