Joining forces: a call for greater collaboration to study new medicines in children and adolescents with type 2 diabetes.

Abstract

The number of individuals with type 2 diabetes (T2D) is increasing worldwide and is projected to rise further over the next decades (1). The prevalence of T2D diabetes in the pediatric population is also rising and in the U.S. one in three new cases of diabetes mellitus diagnosed in patients younger than 18 years of age is T2D (2,3). In contrast, the prevalence of T2D in the European pediatric population remains relatively low (4–7). Diabetes complications in children and adolescents may be evident as early as 2 years after diagnosis (8). Data from the Treatment Options for type 2 Diabetes in Adolescents and Youth (TODAY) study suggest almost half of children and adolescents with T2D in the U.S. have suboptimal glycemic control after 4–5 years of treatment with metformin monotherapy (9) and that hypertension, dyslipidemia, and early microvascular complications (e.g., nephropathy and retinopathy) are highly prevalent in these patients (10–12). There is therefore a need to make additional safe and effective treatment options available to youths with T2D. Therapeutic options to treat pediatric T2D in the U.S. are limited, as only 2 of the 12 classes of glucose-lowering agents with an approved adult T2D indication have a pediatric indication. The U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) enforce legislation that requires pharmaceutical companies to define how they will establish the safety and efficacy of new therapeutic products for use in children and adolescents, age less than 18 years (13–15). Under current U.S. and European (EU) laws, new therapeutic products with the potential to benefit children are required to be studied unless a waiver is granted. Although FDA and EMA pediatric processes differ, pharmaceutical companies use a single pediatric …