About
This bill expands the Food and Drug Administration’s (FDA’s) authority with respect to research on rare pediatric diseases, including by permitting the FDA to take enforcement action against drug sponsors that fail to satisfy pediatric study requirements and by reauthorizing programs that support pediatric research.
Specifically, the bill
- modifies requirements relating to molecularly targeted pediatric cancer investigations to permit research on new drugs in combination with active ingredients that have already been approved, provided certain conditions are met;
- permits the FDA to take enforcement action against drug sponsors that fail to comply with pediatric study requirements, if such sponsors demonstrated a lack of due diligence in satisfying the requirement;
- renews the FDA’s authority to award priority review vouchers to sponsors of new products intended to treat rare pediatric diseases through September 30, 2029; and
- reauthorizes through FY2027 certain funding for the National Institutes of Health to support priority pediatric research.
The bill also provides statutory authority for the FDA’s interpretation of the orphan drug exclusivity period. The bill specifies, consistent with FDA regulations, that the seven-year market exclusivity period for drugs for rare diseases or conditions (i.e., orphan drugs) prohibits the approval of the same drug for the same approved use or indication with respect to the disease or condition. (In Catalyst Pharmaceuticals, Inc. v. Becerra, a court rejected the FDA’s interpretation and held that orphan drug exclusivity extends to all uses or indications for the disease or condition.)
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