1. ¹Division of Pediatric Clinical Pharmacology and Medical Toxicology, The Children's Mercy Hospitals and Clinics, Kansas City, Missouri, USA
2. ²Department of Pediatrics, University of Missouri-Kansas City, School of Medicine, Kansas City, Missouri, USA
3. ³Division of Pediatric Pharmacology, Rainbow Babies and Children's Hospital, Cleveland, Ohio, USA
4. ⁴Department of Pediatrics and Pharmacology, Case Western Reserve University School of Medicine, Cleveland, Ohio, USA
5. ⁵Section of Clinical Pediatric Clinical Pharmacology and Toxicology, Arkansas Children's Hospital, Little Rock, Arkansas, USA
6. ⁶Department of Pediatrics, University of Arkansas for Medical Sciences, Little Rock, Arkansas, USA

Correspondence: SM Abdel-Rahman, (srahman@cmh.edu)

Abstract

Over the past decade, there has been a heightened awareness of the need to include children in the drug development process. With this awareness has come an expansion of the infrastructure for conducting studies in children and an increase in the sponsorship of pediatric clinical trials. However, the growth in pediatric research has, in many cases, not been accompanied by an increase in the involvement of trained pediatric investigators when it comes to trial design and/or interpretation. Pediatric phase I/II protocols continue to span a spectrum from those that are carefully constructed to those that are poorly designed. This paper highlights the basic elements of phase I/II protocols that merit unique consideration when the clinical trial involves children. Illustrations are provided from our experience, which highlight problems that may arise when trials are not designed with the pediatric patient in mind.