Ensuring Treatment for Children with Orphan Diseases

Overview

Pediatric drug laws, including the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA) have helped to shrink the time between when medicines are approved for adults and for children. However, challenges still remain in pediatric research and development (R&D), including the Orphan Drug Exemption.

Created in collaboration with Treatment Action Group (TAG), this brief illustrates how the Orphan Drug exemption affects children with tuberculosis and outlines solutions to ensure all children benefit from pediatric research and development.