USA – FDA final guidance targets neonatal pharmacology studies

The US Food and Drug Administration (FDA) issued final guidance this week to assist sponsors in developing clinical pharmacology studies for neonatal populations. The guidance is meant to address gaps in neonatal labeling and encourage the development of therapies that are “unique to neonates.”

The guidance is tailored to sponsors developing these studies for investigational new drug applications (INDs), new drug applications (NDAs), biologics license applications (BLAs) and supplements to those applications.

The guidance revises a draft version issued in August 2019. Changes include adding immunogenicity as a factor to consider when designing these studies, providing additional information on total volume to be administered for parenteral formulations, and clarification on using microsampling methodology.

“Given that most drugs used in neonatal intensive care units (NICUs) are used off-label, it is important that drug information be obtained in neonates to address gaps in neonatal labeling,” states the guidance.”…