Strengthening Pediatric Drug Testing and Accountability for Rare Disease Treatments.
This Act aims to increase the availability of safe and effective drugs for children, particularly those with rare diseases and cancer, by strengthening pediatric study requirements. It mandates that "orphan drugs" (for rare diseases) must undergo pediatric testing if they offer a meaningful therapeutic benefit to children. The law also clarifies enforcement procedures, ensuring drug companies are held accountable for failing to conduct required studies unless they demonstrate due diligence.
Key points
Increased Pediatric Testing: Pediatric study requirements are extended to include orphan drugs (for rare diseases) if they offer a meaningful therapeutic benefit to children.
Clearer Accountability: The FDA must prove a lack of "due diligence" before penalizing companies for failing to complete required pediatric studies.
Automatic Waivers: The FDA must create a public list of diseases where pediatric studies are impossible or highly impractical, granting automatic waivers for those conditions.
Funding Boost: Authorizes the NIH to allocate up to 1% of certain research funds for pediatric drug studies from 2026 through 2030.
Introduced
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