What to Do When Medical Evidence Can Only Be Generated Through Routine Data: An Example From Pediatric Oncology

Children undergoing allogeneic stem cell transplantation often receive off-label rituximab treatment for Epstein-Barr virus reactivation, using adult dosing without pediatric evidence. This project aims to develop a clinical decision support tool (CDSS) that provides evidence-based dosing scenarios by analyzing real-world patient data. The CDSS integrates clinical and laboratory data across institutions and includes dynamic models that guide therapy decisions while incorporating feedback to improve over time. It addresses a critical evidence gap for vulnerable populations and represents a scalable approach for individualized care. The project also highlights the need for regulatory and infrastructural adaptations to support data-driven, post-approval evidence generation.