This bill encourages pharmaceutical companies to develop and test drugs for children, particularly for rare diseases. It removes an exemption that previously allowed orphan drugs (medications for rare conditions) to skip pediatric study requirements, meaning companies must now conduct pediatric research on these drugs or request a waiver explaining why studies are impossible or impractical. The FDA must issue guidance within 18 months on how these new requirements will work, hold a public meeting to get input from industry and patient groups, and consider using real-world evidence to help meet these requirements. The bill also doubles federal funding for pediatric drug studies from $25 million to $50 million annually through 2027, strengthens enforcement by allowing the FDA to take action against companies that fail to complete required pediatric studies, and requires the FDA to report annually on penalties and settlements related to pediatric study violations. The Government Accountability Office must submit a report within four years assessing the impact on rare disease drug development and pediatric drug labeling.
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