How to Conduct Clinical Trials in Children: A Tutorial

Abstract

Despite a growing interest in, and commitment to, implementing pediatric clinical trials, approximately one in every five trials in children fails because of inappropriate study design, suboptimal experiment planning, or inadequate participant enrollment. This tutorial, presented from the perspectives of seasoned pediatric investigators, an experienced research coordinator, and an established pediatric clinical trials network, is designed to provide practical guidance for successfully implementing pediatric clinical trials at an academic center or another comparable institution.

Figure 1

Evolution in pediatric drug legislature; image created with content from Tolbert et al.1 FDA, US Food and Drug Administration.

Figure 2

Mean plasma concentrations (means ± SD) of fexofenadine (FEX) after oral administration of FEX 60 mg with water 600 mL, apple juice (AJ) 150 mL, followed by water 450 mL, AJ 300 mL, followed by water 300 mL, and AJ 600 mL. N = 10; image adapted from Luo et al.,12 https://doi.org/10.1111/cts.12400, is licensed under CC BY‐NC‐ND 4.0. ^©2016 The authors.

Figure 3

Extrapolation in pediatric drug development, decision tree from the US Food and Drug Administration; image borrowed from https://www.fda.gov/ScienceResearch/SpecialTopics/PediatricTherapeuticsResearch/ucm106614.htm,47 public domain.

Figure 4

Checklist of key elements for successful pediatric clinical trial design and execution; pharmacokinetics (PKs), pharmacodynamics (PDs). CFR, Code of Federal Regulations; DME, drug metabolizing enzyme; FDA, US Food and Drug Administration; IRB, institutional review board; PTN, Pediatric Trials Network.