Mandatory Pediatric Testing for Orphan Drugs: Increased Funding and Penalties for Non-Compliance.
This Act aims to encourage innovation in the development of pediatric drugs and improve labeling for rare disease drugs (orphan drugs). It removes the previous exemption for orphan drugs from mandatory pediatric study requirements and doubles the funding for the pediatric research program. The law also subjects pharmaceutical companies to penalties for failing to comply with study requirements and mandates the FDA to issue guidance on the application of these new rules.
Key points
Mandatory Pediatric Studies: The exemption from pediatric study requirements for orphan drugs is removed, requiring companies to conduct studies for these indications.
Penalties for Non-Compliance: The Act removes an exception that protected orphan drugs from penalties, meaning companies are now subject to enforcement actions for failing to complete required studies.
Increased Research Funding: Funding for the pediatric drug study program is increased from $25 million to $50 million annually for fiscal years 2023 through 2027.
New FDA Guidance: The FDA must issue guidance on how the new requirements apply, including criteria for granting waivers and the use of real-world evidence.
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