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  1. FDA In Brief

FDA in Brief: FDA issues draft guidance regarding clinical trial design for newborns

July 31, 2019

Media Inquiries

  Amanda Turney

“Because of the challenges of studying investigational medical products in newborns, health care professionals often are left to administer drugs in neonatal intensive care units (NICUs) for uses that are not approved in neonates, relying on their medical expertise to extrapolate data on dosing, safety and efficacy for this very young and vulnerable patient population. Yet, these newborns have unique health factors -- not only their age, but the underlying disease that can affect their treatment. The FDA is committed to gaining more information on the safety and efficacy of existing therapies given to our youngest patients, and we also want to encourage the development of new therapies, so today we are issuing our first draft guidance regarding specific considerations on the design of clinical trials involving newborns,” said Susan McCune, M.D., Director, Office of Pediatric Therapeutics, U.S. Food and Drug Administration.“Today’s draft guidance provides clarity for industry regarding clinical trial study design, drug dosing and analysis considerations. We are encouraging industry that when designing these studies, there should be input from a multidisciplinary team involved in neonatal care, including parents. We hope today’s recommendations will encourage drug development for newborns, and ultimately help families make more informed decisions about the care of their babies.”

Today, the U.S. Food and Drug Administration issued the draft guidance, General Clinical Pharmacology Considerations for Neonatal Studies for Drugs and Biological Products. The neonatal period is defined as the day of birth plus 27 days for full-term infants and as the day of birth through the expected date of delivery plus 27 days for preterm infants. The draft guidance provides the FDA’s current thinking on clinical pharmacology considerations for neonatal studies for drugs and biological products. It discusses neonatal subgroup classification, dose selection and study design and analysis considerations for the conduct of neonatal clinical pharmacology studies.


The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

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