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Title: Expanding treatment options for youth with type 2 diabetes: Current problems and proposed solutions: A white paper from the NICHD Diabetes Working Group

item TAMBORLANE, WILLIAM - Yale School Of Medicine
item HAYMOND, MOREY - Children'S Nutrition Research Center (CNRC)
item DUNGER, DAVID - University Of Cambridge
item SHANKAR, RAVI - Merck Research Laboratories
item GUBITOSI-KLUG, ROSE - Rainbow Babies And Children’s Hospital
item BETHIN, KATHLEEN - Children'S Hospital - Buffalo, New York
item KARRES, JANINA - European Medicines Agency
item TOMASI, PAOLO - European Medicines Agency
item LIBMAN, INGRID - University Of Pittsburgh Medical Center
item HALE, PAULA - Novo Nordisk, Inc
item PORTMAN, RONALD - Novartis Institutes
item KLINGENSMITH, GEORGEANNA - University Of Colorado
item REED, MICHAEL - Akron Children'S Hospital
item BLUMER, JEFFREY - University Of Toledo
item GIACOIA, GEORGE - National Institutes Of Health (NIH)

Submitted to: Diabetes Care
Publication Type: Popular Publication
Publication Acceptance Date: 12/8/2015
Publication Date: 3/1/2016
Citation: Tamborlane, W.V., Haymond, M.W., Dunger, D., Shankar, R., Gubitosi-Klug, R., Bethin, K., Karres, J., Tomasi, P., Libman, I., Hale, P.H., Portman, R., Klingensmith, G., Reed, M., Blumer, J., Giacoia, G. 2016. Expanding treatment options for youth with type 2 diabetes: Current problems and proposed solutions: A white paper from the NICHD Diabetes Working Group. Diabetes Care. 39(3):323-329.

Interpretive Summary:

Technical Abstract: The Best Pharmaceuticals for Children Act of 2002 mandated that the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) carries out critical reviews of the gaps in knowledge and unmet needs regarding safe and effective pharmacologic treatment of infants, children, and adolescents in a broad range of disease areas. In 2012, NICHD selected diabetes mellitus as one of the pediatric disorders for review. Dr. William V. Tamborlane was named chair, and Dr. Linda DiMeglio, vice-chair, of the Diabetes Working Group. Together with Dr. George Giacoia of NICHD, they assembled a distinguished group of medical experts in childhood diabetes, including clinicians/clinical investigators from leading academic centers and from industry and representatives from the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA), and the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), to carry out this review. It is very important to note that the views expressed in this article, as well as in other reports from the Diabetes Working Group, are the personal views of the authors and may not be understood or quoted as being made on behalf of or reflecting the position of the FDA or EMA or any of the organizations or pharmaceutical companies represented in our working group. The large Diabetes Working Group was divided into five committees: Type 1 Diabetes (T1D): Therapeutics, Type 2 Diabetes (T2D): Therapeutics, T1D: Natural History and Biomarkers, T2D: Natural History and Biomarkers, and Diabetes Pharmacology. The consensus of the T2D Therapeutics Committee was that its efforts should address the crisis in care that clinicians face in treating this disorder in adolescents. Despite a plethora of new drug classes and new agents within each class that have been approved for use in adults with T2D, in 2015, metformin and insulin remain the only drugs that are approved by the FDA and EMA for use in patients with T2D <18 years of age. Although there are obstacles to the successful completion of phase 3 studies of new treatment modalities in a number of childhood disorders, the magnitude of the problem is particularly challenging in studying youth with T2D. In this white paper, we describe the population of pediatric patients with T2D in the U.S. and Europe, address the limitations of current therapy for youth with T2D, and summarize how inclusion and exclusion criteria, study design, and investigator-related issues have made it especially challenging to complete the investigation plans for new drugs that have been submitted to the regulatory agencies to fulfill pediatric study requirements. We have also developed a proposal, described here, that may better facilitate the collection of adequate and well-controlled pediatric efficacy and safety clinical trial data to inform clinicians regarding the pediatric use of new drugs to treat T2D.