Introduction
Due to the challenges associated with studying investigational medical products in neonates, healthcare professionals must often resort to administering drugs in neonatal intensive care units (NICUs) off-label. While numerous pediatric legislative initiatives have been passed worldwide, neonates are still largely treated with medicines that have not been specifically studied or approved in this young, vulnerable population. In fact, it has been over 25 years since a novel medicine that improves outcomes has been developed for premature neonates.
The U.S. Food and Drug Administration (FDA) defines the neonatal period 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. These neonates have unique health factors – not just their young age and complex pathophysiology, but also their underlying diseases – which can affect treatment.
In this white paper, we explore the landscape of neonatal studies, from regulatory guidance and design considerations to challenges associated with study conduct and safety reporting. With improved understanding of the unique nuances of research in neonates, sponsors can increase their likelihood of developing and executing successful neonatal clinical trials.