Applying Regulatory Science to Develop Safe and Effective Medicines for Neonates: Report of the US Food and Drug Administration First Annual Neonatal Scientific Workshop, October 28-29, 2014

Martin Offringa; Jonathan M Davis; Mark A Turner; Robert Ward; Ralph Bax; Sam Maldonado; Vikram Sinha; Susan K McCune; Anne Zajicek; Daniel K Benjamin Jr; Christina Bucci-Rechtweg; Robert M Nelson

doi:10.1177/2168479015597730

Applying Regulatory Science to Develop Safe and Effective Medicines for Neonates: Report of the US Food and Drug Administration First Annual Neonatal Scientific Workshop, October 28-29, 2014

Ther Innov Regul Sci. 2015 Sep;49(5):623-631. doi: 10.1177/2168479015597730.

Authors

Affiliations

¹ 1 Child Health Evaluative Sciences, Hospital for Sick Children, Toronto, Ontario, Canada.
² 2 Department of Pediatrics and the Tufts Clinical and Translational Science Institute, The Floating Hospital for Children at Tufts Medical Center, Boston, MA, USA.
³ 3 Department of Women's and Children's Health, Institute of Translational Medicine, University of Liverpool, Liverpool, UK.
⁴ 4 Department of Pediatrics and Pharmacology/Toxicology, University of Utah, Salt Lake City, UT, USA.
⁵ 5 Paediatric Medicines, Product Development Scientific Support Department, European Medicines Agency, London, UK.
⁶ 6 Janssen Research & Development, CHILD-Child Health Innovation Leadership Department, Raritan, NJ, USA.
⁷ 7 Division of Pharmacometrics, Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, Food and Drug Administration, Silver Spring, MD, USA.
⁸ 8 Office of Translational Sciences, Center for Drug Evaluation and Research, Food and Drug Administration, Silver Spring, MD, USA.
⁹ 9 Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD, USA.
¹⁰ 10 Duke Clinical Research Institute, Duke University, Durham, NC, USA.
¹¹ 11 Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA.
¹² 12 Office of Pediatric Therapeutics, Food and Drug Administration, Silver Spring, MD, USA.

PMID: 30227031
DOI: 10.1177/2168479015597730

Abstract

The First Annual Neonatal Scientific Workshop focused on the needs of the neonate by addressing the basic question: what information is required to inform decision making both at the regulatory level and at the bedside? Priority therapeutic areas include neonatal lung, brain, and gastrointestinal injury, retinopathy of prematurity, sepsis, and neonatal abstinence syndrome. Scientific progress in these therapeutic areas, regulatory standards, and the acceptable design and conduct of clinical trials must be aligned. This report will review potential approaches to enhancing neonatal drug development.

Keywords: adaptive designs; biomarkers; clinical outcomes; extrapolation; pediatric trials.