title: Defining pathways for development of disease-modifying therapies in children with type 1 diabetes: a consensus report.
abstract: 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.
authors.affiliation: Department of Pediatrics, University of Toronto, Toronto, Canada.
authors.forename: Diane K
authors.initials: DK
authors.lastname: Wherrett
authors.affiliation: American Diabetes Association, Alexandria, VA.
authors.forename: Jane L
authors.initials: JL
authors.lastname: Chiang
authors.affiliation: Department of Pediatrics, University of Miami, Miami, FL.
authors.forename: Alan M
authors.initials: AM
authors.lastname: Delamater
authors.affiliation: Department of Pediatrics, Indiana University, Indianapolis, IN.
authors.forename: Linda A
authors.initials: LA
authors.lastname: DiMeglio
authors.affiliation: Department of Pediatrics, University of California, San Francisco, San Francisco, CA.
authors.forename: Stephen E
authors.initials: SE
authors.lastname: Gitelman
authors.affiliation: Departments of Medicine and Pediatrics, University of Colorado, Denver, CO.
authors.forename: Peter A
authors.initials: PA
authors.lastname: Gottlieb
authors.affiliation: Department of Immunobiology, Yale University, New Haven, CT.
authors.forename: Kevan C
authors.initials: KC
authors.lastname: Herold
authors.affiliation: Division of Rheumatology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH.
authors.forename: Daniel J
authors.initials: DJ
authors.lastname: Lovell
authors.affiliation: Graduate School of Public Health, University of Pittsburgh, Pittsburgh, PA.
authors.forename: Trevor J
authors.initials: TJ
authors.lastname: Orchard
authors.affiliation: Department of Psychiatry, University of Pittsburgh, Pittsburgh, PA.
authors.forename: Christopher M
authors.initials: CM
authors.lastname: Ryan
authors.affiliation: Department of Pediatrics, University of Florida, Gainesville, FL.
authors.forename: Desmond A
authors.initials: DA
authors.lastname: Schatz
authors.affiliation: Department of Bioethics, National Institutes of Health, Bethesda, MD.
authors.forename: David S
authors.initials: DS
authors.lastname: Wendler
authors.affiliation: Diabetes Research Program, Benaroya Research Institute, Seattle, WA cjgreen@benaroyaresearch.org.
authors.forename: Carla J
authors.initials: CJ
authors.lastname: Greenbaum
mesh_terms: D000328:Adult; D000367:Age Factors; D002648:Child; D002986:Clinical Trials as Topic / Q000331:legislation & jurisprudence / Q000379:methods; D003072:Cognition Disorders / Q000209:etiology; D017281:Cost of Illness; D003922:Diabetes Mellitus, Type 1 / Q000097:blood / Q000150:complications / Q000188:drug therapy*; D018450:Disease Progression; D004992:Ethics, Medical; D006442:Glycated Hemoglobin / Q000378:metabolism; D006801:Humans; D007004:Hypoglycemic Agents / Q000627:therapeutic use*; D007328:Insulin / Q000378:metabolism; D000078790:Insulin Secretion; D009965:Orphan Drug Production; D011788:Quality of Life; D016896:Treatment Outcome
chemical_list: D006442:Glycated Hemoglobin; D007004:Hypoglycemic Agents; D007328:Insulin