Development of Drug Therapies for Newborns and Children: The Scientific and Regulatory Imperatives

Yeruk Lily Mulugeta; Anne Zajicek; Jeff Barrett; Hari Cheryl Sachs; Susan McCune; Vikram Sinha; Lynne Yao

doi:10.1016/j.pcl.2017.08.015

Development of Drug Therapies for Newborns and Children: The Scientific and Regulatory Imperatives

Pediatr Clin North Am. 2017 Dec;64(6):1185-1196. doi: 10.1016/j.pcl.2017.08.015.

Authors

Yeruk Lily Mulugeta¹, Anne Zajicek², Jeff Barrett³, Hari Cheryl Sachs⁴, Susan McCune⁴, Vikram Sinha⁵, Lynne Yao⁴

Affiliations

¹ Division of Pediatric and Maternal Health, CDER US Food and Drug Administration, 10903 New Hampshire Avenue, Silver Spring, MD 20903, USA. Electronic address: yeruk.mulugeta@fda.hhs.gov.
² Obstetric and Pediatric Pharmacology and Therapeutics Branch, Eunice Kennedy Shriver National Institute of Child Health and Human Development, 6710B Rockledge Drive Room 2334, MSC 7002, Bethesda, MD 20817, USA.
³ Sanofi Pharmaceuticals, Discovery Drive, Swiftwater, PA 18370, USA.
⁴ Division of Pediatric and Maternal Health, CDER US Food and Drug Administration, 10903 New Hampshire Avenue, Silver Spring, MD 20903, USA.
⁵ Merck & Co., Inc, PO Box 1000, UG4D-48, North Wales, PA 19454, USA.

Abstract

Pediatric legislation has generated information about the efficacy, safety, and dosing of more than 600 products in children. Extrapolation of adult efficacy data has been an integral part of pediatric drug development. Advances in our understanding of physiology and pharmacology have improved the approach to pediatric dose selection. However, a high percentage of pediatric trials do not meet their primary efficacy endpoint. Delays in initiating completing pediatric studies persist. This article describes these advances and provides innovative approaches to optimize pediatric drug development.

Keywords: Dosing; Extrapolation; Neonatal; Pediatric; Regulations; Research.

Publication types

Review

MeSH terms

Adult
Age Factors
Child
Dose-Response Relationship, Drug*
Drug Industry*
Humans
Infant, Newborn
Pediatrics
Research Design
United States
United States Food and Drug Administration

Grants and funding

FD999999/Intramural FDA HHS/United States