Pharmacotherapy for Minors: Pediatric Drug Development from Good Intentions to Global Abuse

The concept of “therapeutic orphans” arose in 1968 when children were excluded from drug approvals, leading to the development of pediatric pharmaceutical laws in the U.S. and EU. While intended to protect minors, these regulations have led to excessive and often unnecessary pediatric studies, as most clinical breakthroughs historically occurred off-label. This panel aims to critically assess the origins of pediatric drug development, interrogate excessive regulatory requirements, and suggest new policy approaches to ensure advancement in the science.