Failed Pediatric Drug Development Trials

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


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


  • Pharmaceutical Preparations