Transition Plan for a Leading Nation

Pediatric Medical Product Innovation

The administration should work to expand children’s access to Food and Drug Administration (FDA)-approved drugs and devices. Despite progress made by the Best Pharmaceuticals for Children Act (BPCA) and Pediatric Research Equity Act (PREA), roughly 50 percent of all drugs in children still lack FDA-approved pediatric information. Similarly, medical device innovation continues to lag about a decade behind that for adults.

Ensure that drugs for rare diseases are studied appropriately in children. FDA should act quickly to remove the PREA exemption for orphan drugs, as orphan drug approvals are increasing, and therefore, so are the number of new drugs that are exempt from pediatric study requirements. An FDA study recently showed that 36 percent of approved orphan indications relevant to children were incompletely labeled for children, with 23 percent containing no pediatric information whatsoever.

Improve compliance with pediatric study requirements. FDA needs additional enforcement tools to ensure that critical pediatric studies required by PREA are completed in a timely manner.

Expand treatment options for neonates. While some progress has been made with regards to neonatal studies, more must be done as studies for neonates continue to lag behind those for other children.

Prioritize the development of medical devices that are appropriately labeled for children. FDA should create an internal pediatric device review committee to provide consultation and advice to device review offices and device manufacturers with the goal of increasing the number medical and surgical devices approved for use in children.

Ensure clinical trial participants reflect a more diverse population of children. FDA should create and implement a strategy to ensure that drug and device clinical trial participants are sufficiently diverse especially when the product being studied is intended to be used to treat a condition that disproportionately affects Black or Hispanic children.

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American Academy of Pediatrics