Back to: FDA Clinical Investigator Training Course (CITC) 2024
Presenter
Lynne Yao, MD
Director
Division of Pediatrics and Maternal Health (DPMH)
Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine (ORPURM)
Office of New Drugs (OND)
Center for Drug Evaluation and Research (CDER)
US Food and Drug Administration (FDA)
Lynne Yao, MD, is the Director, Division of Pediatric and Maternal Health in the Office of New Drugs, Center for Drug Evaluation and Research. Dr. Yao received a B.S. degree in Biology from Yale University, and an M.D. degree from the George Washington University School of Medicine. She is board certified in both Pediatrics and Pediatric Nephrology. Prior to joining FDA, Dr. Yao was the Director of Dialysis and Associate Pediatric Residency Program Director at the Inova Fairfax Hospital for Children in Fairfax, VA. She has been with the FDA since 2008. The Division of Pediatric and Maternal Health oversees quality initiatives which promote and necessitate the study of drug and biological products in the pediatric population; and improve collection of data to support the safe use of drugs and biological products in pregnant and lactating individuals. She collaborates with numerous stakeholders both inside and outside of FDA to advance development of safe and effective therapies for children, and pregnant and lactating women.
Abstract
The presentation addresses crucial considerations for including specific populations in clinical trials, focusing on children, pregnant and lactating individuals, and enhancing diversity. Historically, children were largely excluded from trials, leading to significant off-label drug use; however, laws like the Best Pharmaceuticals for Children Act (BPCA) and Pediatric Research Equity Act (PREA) now either incentivize or require pediatric studies, resulting in over 1,100 pediatric-specific label changes. While approval standards remain consistent, special considerations for children include ethical issues, feasibility challenges, formulation development, and the application of pediatric extrapolation, which leverages adult data when similarities exist. For pregnant and lactating individuals, despite historical exclusion, there is a growing recognition that they can be safely included, with considerations for safety margins from non-clinical studies and prospect of direct benefit. The presentation also highlights efforts to enhance diversity in clinical trials, emphasizing that studies should reflect the population most likely to use the drug, utilizing strategies like decentralized trials to improve accessibility. Ultimately, the speaker advocates that these populations are protected through research, not from research, ensuring access to appropriately evaluated medicines.