Enhancing Clinical Trial Development for Pediatric Kidney Diseases

• Type of Material: Article
• Authors: H. William Schnaper, Northwestern University; Joseph T. Flynn, Seattle Children's Hospital; Coleman Gross, Relypsa Inc; Anne b. Cropp, Echelon Pharma Solutions, LLC; Bastian Dehmel, Amgen Europe GmbH; Leah B Patel, Mallinckrodt Pharmaceuticals; Larry Greenbaum, Emory University; Elisabeth Houtsmuller, PCORI; Frederick Kaskel, Montefiore Medical Center; Marva Moxey-Mims, National Institutes of Health; Karen Nowak, Mallinckrodt Pharmaceut; Douglas Silverstein, U.S. Food and Drug Administration; Aliza Thompson, U.S. Food and Drug Administration; Lynne Yao, U.S. Food and Drug Administration; Edress Darsey, Pfizer Corporation; William E. Smoyer, The Research Institute at Nationwide Children’s Hospital
• Language: English
• Date: 2017-11-01
• Publisher: Nature Publishing Group: Open Access Hybrid Model Option A
• Publication Version: Author Accepted Manuscript (After Peer Review)
• Copyright Statement: © 2017 International Pediatric Research Foundation, Inc.
• Final Published Version (URL): http://dx.doi.org/10.1038/pr.2017.180
• Title of Journal or Parent Work: Pediatric Research
• ISSN: 0031-3998
• Volume: 82
• Issue: 5
• Start Page: 727
• End Page: 732
• Grant/Funding Information: Supported in part by National Institutes of Health's National Center for Advancing Translational Sciences, Grants Number UL1TR001422 (H.W.S) and UL1TR001070 (W.E.S.)
• Keywords: ACT; Pediatrics; CHILDREN; Life Sciences & Biomedicine; Science & Technology
• Research Categories: Engineering, Biomedical; Health Sciences, Medicine and Surgery

Abstract

The conduct of clinical trials in small pediatric subspecialties such as pediatric nephrology is hampered by both clinical demands on the pediatric nephrologist and the small number of appropriate patients available for such studies. The American Society of Pediatric Nephrology Therapeutics Development Committee (TDC) was established to (1) identify the various stakeholders with interests and/or expertise related to clinical trials in children with kidney disease and (2) develop more effective partnerships among all parties regarding strategies for successful clinical trial development and execution. This article discusses the rationale, structure, and function of the TDC, the status of progress toward its goals, and the insights gained to date that may be useful for other subspecialties that face similar challenges.