Operational and Ethical Perspectives in Pediatric Rare Disease Clinical Research: Making Every Sample Count

A guide to improving rare disease clinical research through ethical design, operational excellence, and patient-centered innovation This white paper explores the unique challenges of rare disease clinical research when conducted in pediatric populations where there is limited patient availability, delays in diagnosis, and geographically dispersed patient pools. In these instances, every interaction with the patient must be scientifically and ethically justified. Trial designs need to […] The pos