Failed Pediatric Drug Development Trials

J D Momper; Y Mulugeta; G J Burckart

doi:10.1002/cpt.142

Failed Pediatric Drug Development Trials

Clin Pharmacol Ther. 2015 Sep;98(3):245-51. doi: 10.1002/cpt.142. Epub 2015 Jun 26.

Authors

J D Momper¹, Y Mulugeta², G J Burckart²

Affiliations

¹ Skaggs School of Pharmacy and Pharmaceutical Sciences, University of California, San Diego, La Jolla, California, USA.
² Pediatric Clinical Pharmacology Staff, Office of Clinical Pharmacology, Office of Translational Sciences, U.S. Food and Drug Administration, Silver Spring, Maryland, USA.

PMID: 25963725
DOI: 10.1002/cpt.142

Abstract

Pediatric product development initiatives have stimulated the development of therapies for children, resulting in improved product labeling, increased identification of adverse events, and development of new pediatric formulations. However, 42% of recently completed pediatric trials have failed to establish either safety or efficacy, leading to an inability to label the product for use in children.(1) Characterizing these failed trials, including common contributing factors, is imperative to designing better pediatric trials in the future.

MeSH terms

Age Factors
Chemistry, Pharmaceutical
Child
Child, Preschool
Clinical Trials as Topic / methods*
Dose-Response Relationship, Drug
Drug Discovery / methods*
Drug Dosage Calculations
Drug-Related Side Effects and Adverse Reactions / etiology
Drug-Related Side Effects and Adverse Reactions / prevention & control
Humans
Infant
Infant, Newborn
Pharmaceutical Preparations / administration & dosage*
Pharmaceutical Preparations / chemistry
Pharmaceutical Preparations / metabolism
Pharmacokinetics
Placebo Effect
Research Design*
Risk Factors
Treatment Failure

Substances

Pharmaceutical Preparations