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Updated: Jun 7, 2026

A Minimally Invasive Method for Intratracheal Instillation of Drugs in Neonatal Rodents to Treat Lung Disease
Published on: August 4, 2021
Use of pharmaceuticals 'off-label' in the neonate
1Department of Anesthesiology, Wake Forest University School of Medicine, Winston-Salem, NC, USA. jtobin@wfubmc.edu
Abstract:
Neonates are frequently not studied in the development of a novel pharmacological agent. With lack of data to support safe and effective use of a new agent in this population, sponsors will not receive approval for labelling the agent for use in this age group from the United States Food and Drug Administration (USFDA). This causes a significant conundrum for the clinician. Neonates are often precluded the benefits of new pharmaceuticals until investigators begin to report their clinical experience with novel agents. This article provides the clinician with an introductory understanding of the approval process of pharmaceuticals in the United States by USFDA. Models of clinical trial design are noted. Examples of anaesthetic and non-anaesthetic agents and their development and use are discussed as either 'labelled' or 'off-label' indications.
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