title: Clinical trials in youth-onset type 2 diabetes: needs, barriers, and options.
abstract: Youth-onset type 2 diabetes (T2D) is increasingly recognized as a disorder with substantial risk for long-term metabolic, cardiovascular, and renal morbidity and mortality, as well as individual and societal burden. Recent studies suggest that the disorder differs from adult-onset T2D in a variety of ways and that there is an urgent need for an expanded set of treatment options. However, demographic, economic, and social challenges limit the number of eligible candidates for clinical trials in youth-onset T2D, and a growing number trials mandated by regulatory agencies have created a circumstance in which too many trials are chasing too few eligible participants. A solution to this situation will require novel approaches to clinical trial design incorporating collaboration among clinical investigators, pharmaceutical sponsors, and regulatory agencies. If successful, this approach could also serve as a model for clinical trials in other rare and understudied pediatric disorders.
authors.affiliation: Department of Pediatrics, Children's Hospital Colorado, University of Colorado School of Medicine, 13123 E 16th Avenue, Aurora, CO, USA, Philip.zeitler@childrenscolorado.org.
authors.forename: Philip
authors.initials: P
authors.lastname: Zeitler
authors.forename: Hubert S
authors.initials: HS
authors.lastname: Chou
authors.forename: Kenneth C
authors.initials: KC
authors.lastname: Copeland
authors.forename: Mitchell
authors.initials: M
authors.lastname: Geffner
mesh_terms: D000293:Adolescent; D017668:Age of Onset; D002986:Clinical Trials as Topic / Q000331:legislation & jurisprudence; D003924:Diabetes Mellitus, Type 2 / Q000191:economics / Q000628:therapy*; D006301:Health Services Needs and Demand; D006801:Humans; D012926:Social Control, Formal; D012959:Socioeconomic Factors; D016896:Treatment Outcome