Defining Pathways for Development of Disease-Modifying Therapies in Children With Type 1 Diabetes: A Consensus Report

Authors: Diane K. Wherrett, Jane L. Chiang, Alan M. Delamater, Linda A. DiMeglio, Stephen E. Gitelman, Peter A. Gottlieb, Kevan C. Herold, Daniel J. Lovell, Trevor J. Orchard, Christopher M. Ryan, Desmond Schatz, David Wendler, Carla J. Greenbaum, the Type 1 Diabetes TrialNet Study Group - Diabetes Care 2015 cited by 92

Emerging data suggest that type 1 diabetes is a more aggressive disease in children than in adults, with important differences in pathophysiology and clinical course. Therefore, the efficacy of disease-modifying therapies may be different in the two populations. Understanding the developmental and regulatory pathways for type 1 diabetes-modifying therapies in children will enable industry, academia, funders, advocacy groups, and regulators to translate new science to clinical care. This consensus report characterizes the fundamental differences in type 1 diabetes between children and adults and proposes a thoughtful approach to better understand the development and regulatory pathways for type 1 diabetes therapies.