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

Recent changes at the FDA, along with ongoing discussions around regulatory harmonization and convergence, present new opportunities to evolve frameworks governing pediatric drug development. The term "therapeutic orphans" emerged in 1968, highlighting the exclusion of children from drug approvals, which ultimately prompted the establishment of pediatric pharmaceutical regulations in both the U.S. and EU. These regulations were designed to protect minors by ensuring that medications used in pediatric populations are safe and effective. However, the implementation of these laws has led to some concerns about the necessity and appropriateness of certain pediatric studies, particularly as clinical advancements have at times occurred through off-label use.

In light of recent discussions on drug development practices and the evolving landscape of pediatric healthcare, this panel seeks to provide a balanced examination of the historical context and implications of pediatric drug development. We will explore the origins of current regulatory frameworks, assess the impact of existing requirements on research innovation, and engage in a constructive dialogue about potential policy reforms. By considering diverse perspectives, we aim to identify strategies that can enhance the advancement of pediatric medicine while ensuring the safety and well-being of young patients.

Panelists include:
Dr. Raquel R. Mura
Founder, RGM Life Sciences Consulting

Dr. Evaristus Nwulia
Professor & Chair, Medical Institute Review Board
Howard University

Dr. Klaus Rose
Chief Executive Officer, Klausrose Consulting Pediatric Drug Development

Moderator:
Dr. Earl Ettienne
Assistant Dean & Co-Chair, Medical Instit. Review Board
Howard University