Alternate approaches for pediatric type 1 diabetes drug development and potential regulatory approval: a perspective.

The incidence and prevalence of pediatric type 1 diabetes are increasing globally, including in the U.S. While the increasing number of cases of pediatric diabetes makes expeditious availability of new medical products and therapies for diabetes care essential, there have been many barriers encountered in bringing some drugs and devices to pediatric patients who may benefit. Newer insulins have been studied and approved for use in children. However, hurdles exist in the inclusion of children in studies of therapies aimed at preventing β-cell loss in those with new-onset diabetes and those at risk for type 1 diabetes. This Perspective focuses on potential solutions to the challenges experienced in bringing new drugs for pediatric type 1 diabetes to marketing approval. Given their central importance as the users of medical products, patient perspectives are included along with scientific and regulatory considerations.