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AAP Responds to Senate Committee Passage of the FDA Reauthorization Act of 2017

5/11/2017 Bill makes progress on pediatric devices, misses key opportunities to improve pediatric drugs

Washington, DC – Today, the U.S. Senate Health, Education, Labor and Pensions (HELP) Committee passed FDA Reauthorization Act of 2017, which reauthorizes U.S. Food and Drug Administration (FDA) user fees and contains provisions related to pediatric drugs and devices. The Academy appreciates the long-standing history of bipartisan support for consideration of the Pediatric Research Equity Act (PREA) and the Best Pharmaceuticals for Children Act (BPCA) as part of user fee reauthorization legislation, but urges lawmakers to continue working to further strengthen these essential laws for children.

"Children are not just little adults; medical devices and medications work differently in children and must be studied specifically for their use," said AAP President Fernando Stein, MD, FAAP. "While the overall legislation misses critical opportunities to ensure that all drugs used in children are studied in children, the bill does make improvements that will advance appropriate and safe medical devices available for children. The Academy thanks Sens. Al Franken (D-Minn.) and Mike Enzi (R-Wyo.) for their work to ensure the provisions of S. 1093, the Pediatric Device Development Act, were included in the reauthorization bill. Their efforts ensure that important pediatric programs at FDA will continue and that FDA will have stronger pediatric expertise to provide to its various reviewing divisions."

However, the FDA Reauthorization Act of 2017 fails to address a concerning loophole that allows certain drugs to forgo pediatric study requirements under PREA because they have been designated as orphan drugs, even if the disease the drug treats is not considered an orphan disease in adults. Drugs for common diseases that also occur in children should be subject to PREA and not receive a blanket exemption simply because the number of children who are affected by that disease is less than 200,000. Last year, 41% of all drugs approved by FDA were designated as orphan drugs, meaning FDA could not apply PREA to any of those drugs. Since the majority of rare diseases occur in childhood, the orphan exemption from PREA has a profound effect on children's health.

The legislation also does not empower FDA to apply PREA to molecularly targeted drugs, failing to adapt the law to continued advances in how drugs are being developed. These drugs are now mainstay of new drug development for cancer, including in children. FDA should be allowed to use PREA to require pediatric studies for a drug when it affects specific molecular targets that are shared between the adult and pediatric disease.

"Much like drug development has evolved over time, so too should our drug laws evolve to better protect children," said Dr. Stein. "Despite the tremendous progress of BPCA and PREA, today approximately 50% of drugs used in children still lack FDA-approved pediatric labeling. Congress should be doing everything possible to ensure that all drugs that are used in children are labeled for their use."

As the bill moves to the Senate floor, the AAP will urge legislators to prioritize children and advance policies to strengthen BPCA and PREA. The AAP also urges Congress to move forward on legislation to revise FDA's over-the-counter (OTC) drug monograph system. The current process for FDA to update OTC monographs is cumbersome and complex, making it hard for FDA to keep up with scientific developments, address safety concerns, and accommodate innovation. Any OTC drugs with ingredients that have been demonstrated to be unsafe or ineffective, like pediatric cough and cold medicines, can legally remain on the market, posing a risk to child health and safety. The AAP worked with the Pew Charitable Trusts to develop principles for FDA to consider when revising its OTC monograph, and the Academy welcomes future opportunities for Congress to consider these recommendations in reforming the OTC monograph process.

"As we evolve our understanding of the best ways to treat devastating diseases, we must ensure that children are not forgotten," said Dr. Stein. "That means we need to support pediatric research to determine the safety and efficacy of drugs and devices in children so that they can benefit from cutting-edge technology and life-saving medical innovations. As the FDA user fee bill advances, the Academy looks forward to working with members of Congress and FDA to make needed improvements to pediatric drug and device laws."


The American Academy of Pediatrics is an organization of 66,000 primary care pediatricians, pediatric medical subspecialists and pediatric surgical specialists dedicated to the health, safety and well-being of infants, children, adolescents and young adults. For more information, visit and follow us on Twitter @AmerAcadPeds.