Issue Date: October 17, 2024
Issued by: Center for Biologics Evaluation and Research
Topic(s): Clinical – Medical, Pediatric Product Development
Status: Final
Considerations for Long-Term Clinical Neurodevelopmental Safety Studies in Neonatal Product Development: Guidance for Industry
Office of the Commissioner, Office of the Chief Medical Officer, Office of Pediatric Therapeutics The purpose of this guidance is to provide a framework for considering whether and what type of long-term neurologic, sensory and developmental evaluations could be useful to support a determination of safety of a drug, biological product, or device (referred to as ‘medical product’ in this guidance) for use in neonates, and if so, which domains of neurodevelopment may be most applicable. This guidance will not specifically address efficacy or effectiveness assessments for products primarily intended to improve neurologic outcomes, e.g., neuroprotective agents. This guidance is focused on long-term evaluations of neurodevelopmental safety. Although assessments of nephrotoxicity, pulmonary toxicity, and toxicity to other tissues and organs may also be warranted in neonatal medical product development, the approach to those assessments is outside the scope of this guidance. Additional Guidance Resources Neonatal Neurodevelopmental Safety Studies – Guidance Snapshot Neonatal Neurodevelopmental Safety Studies – Podcast Neonatal Neurodevelopmental Safety Studies – Podcast Transcript
Download the Guidance Document
