Strengthening Pediatric Drug Testing and Orphan Drug Research Requirements
This law aims to increase the safety and availability of medication information for children, particularly for rare diseases. It mandates that pharmaceutical companies conduct pediatric studies for more drugs, including orphan drugs, unless the FDA determines the assessment offers no meaningful therapeutic benefit. It also strengthens the FDA's enforcement powers while clarifying the due process for companies.
Key points
More drugs for rare diseases (orphan drugs) must now be tested for safety and efficacy in children, providing better information for pediatric care.
The FDA must prove a company lacked "due diligence" before imposing penalties for failing to complete required pediatric studies.
Increased funding authorization (up to 1% of research institute budgets) is provided for pediatric research through the NIH for fiscal years 2025-2027.
Expired
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