JAMA Pediatrics (2014)

Innovative drugs to treat pediatric diseases

The development of safe and effective pediatric drugs continues to fall short. The paucity of new therapies is particularly stark for rare diseases, which disproportionately affect children and collectively affect an estimated 25 million people in the United States and 30 million in Europe. This paper shines a light on a new federal incentive program for pediatric drug development that has, to date, attracted little attention but may have substantial implications for clinicians and patients.

(cite: Hwang TJ, Bourgeois FT. New regulatory paradigms for innovative drugs to treat pediatric diseases. JAMA Pediatrics 2014; 168(10): 879-880.)