Trial Design

By integrating advanced trial designs, we will address the challenges of small patient populations and heterogeneous disease presentations, ensuring rare disease trials are both scientifically rigorous and accessible. 

This initiative will support trial setup and delivery, working in collaboration with researchers, clinicians, and regulatory bodies to streamline the design, approval, and execution of rare disease trials. A key focus will be developing trial documentation and governance frameworks, ensuring seamless trial implementation alongside patient and public involvement (PPIE) strategies to promote diverse and representative recruitment. As the LifeArc Translational Challenge for Rare Disease progresses, this research area will expand its scope to support a broader range of trials across the initiative. 

By pioneering efficient, patient-focused trial methodologies, we aim to accelerate the availability of new treatments for rare diseases. These trial designs will use innovate methodologies around Bayesian statistical methods, synthetic control experience and casual inference to leverage complex trial designs for the greatest result.