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FDA News: Issue 15-1, July 2022

Author: [AUTHOR] Published on 7/29/2022 12:00:00 AM


On July 27, 2022, the US Food and Drug Administration (FDA) announced the availability of a final guidance for industry entitled General Clinical Pharmacology Considerations for Neonatal Studies for Drugs and Biological Products. This guidance is intended to assist sponsors of investigational new drug applications (INDs) and applicants of new drug applications (NDAs), biologics license applications (BLAs), and supplements to such applications who are planning to conduct clinical studies in neonatal populations.

Effectiveness, safety, or dose-finding studies in neonates involve assessing clinical pharmacology information, such as information regarding a product’s pharmacokinetics (PK) and pharmacodynamics (PD) to inform dose selection and individualization. During in utero development, there are significant physiological changes in the fetus involving the maturation of organs and tissues, including enzyme systems, receptors, transporters, and neurotransmitters. The normal developmental trajectory of these systems is altered by preterm delivery. Postnatal development can also be adversely affected by concurrent illnesses, resulting in altered maturation of organs and tissues and affecting the systems responsible for product absorption (A), distribution (D), metabolism (M), and excretion (E), known collectively as ADME.

To facilitate the development of drugs for use in neonates, this guidance describes these unique developmental considerations as well as other intrinsic and extrinsic factors that can independently alter the pharmacokinetic (exposure) and pharmacodynamic (response) characteristics of a drug. Additionally, 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.

Detailed planning of neonatal studies should include input from a multidisciplinary team involved in neonatal care, including parents and NICU nurses, in the early stages of study design to provide their perspectives of study feasibility and the potential impact of study participation on neonates and their families. This guidance provides recommendations regarding approaches and design features for neonatal clinical pharmacology studies, including ethical considerations and coupling prior knowledge and data obtained from adult, preclinical, and other pediatric studies with innovative quantitative approaches, to help predict neonatal doses and optimize clinical trial design. 

The “General Clinical Pharmacology Considerations for Neonatal Studies for Drugs and Biological Products” guidance is available at: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/general-clinical-pharmacology-considerations-neonatal-studies-drugs-and-biological-products-guidance. Please refer to the final guidance for more details. To support transparent communication and dissemination of FDA guidance documents, FDA also provides Guidance Snapshots and podcasts as communication tools to provide highlights from guidance documents using visuals and plain language. Please find the Guidance Snapshot and podcast for the General Clinical Pharmacology Considerations for Neonatal Studies for Drugs and Biological Products guidance here: https://www.fda.gov/drugs/guidances-drugs/guidance-snapshot-pilot.


The Office of Clinical Pharmacology (OCP) is pleased to offer the e-mail subscription service Clinical Pharmacology Corner. This is a free service from FDA to provide occasional updates from OCP regarding newly approved therapies, new regulatory and scholarly publications, upcoming events and other items of interest. Subscribe today at https://public.govdelivery.com/accounts/USFDA/subscriber/topics and select Clinical Pharmacology Corner under Drugs.

We always welcome your thoughts regarding the format, content, and utility of this communication. Comments may be sent via email to ocp@fda.hhs.gov.

This communication was prepared by Office of Clinical Pharmacology, Office of Translational Sciences, CDER, FDA.

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