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The development of safe and effective pediatric drugs continues to fall short.1 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.2 Since the 1980s, US policymakers have enacted a range of policies to stimulate drug development for rare diseases, defined as those affecting fewer than 200 000 individuals in the United States. The most notable policy is the 1983 Orphan Drug Act, which grants 7 years of marketing exclusivity as well as subsidies and tax credits to pharmaceutical companies bringing a drug for a rare disease to market. While the Orphan Drug Act has been credited with stimulating product development for rare diseases, only one-quarter of orphan drugs were approved explicitly for pediatric indications between 2000 and 2009.3
Hwang TJ, Bourgeois FT. New Regulatory Paradigms for Innovative Drugs to Treat Pediatric Diseases. JAMA Pediatr. 2014;168(10):879-880. doi:10.1001/jamapediatrics.2014.904