Essentials of Translational Pediatric Drug Development

Elke Gasthuys is a Postdoctoral researcher at Ghent University, focusing on advanced modelling and simulations tools (PopPK/PD, PBPK, Systems biology) within drug research. Her main focus lies within the fields of inflammatory bowel disease; cystic fibrosis and enuresis, with special emphasis on the pediatric population. Her PhD thesis focused on developing a juvenile conventional piglet model at the Faculty of Veterinary Sciences, Ghent University, Belgium. Her research project was embedded within a broader research project (SAFEPEDRUG), aiming to improve pediatric drug research by integrating juvenile animal models and modelling and simulation tools to adapt pediatric clinical trial designs. Karel Allegaert is a pediatrician-neonatologist and clinical pharmacologist, appointed as Professor at KU Leuven, Belgium and as senior clinical consultant at Erasmus MC Rotterdam, the Netherlands. His main clinical research focuses on perinatal and developmental pharmacology. This includes active involvement in clinical studies, as well as educational and consultancies to different stakeholders, like authorities and industry. He is an author, reviewer and editor for a broad range of journals and textbooks in the fields of clinical pharmacology and pediatrics. These activities have resulted in relevant awards like the Galenus clinical researcher award (Belgium, 2009) for neonatal clinical pharmacology research and an appointment as ordinary member of the Royal Academy of Medicine, Belgium (2015). Lien Dossche is a full-time academic staff member at Ghent University, focusing on clinical activities as a Pediatrician and clinical research. After obtaining a medical degree at Ghent University in 2012, she started her specialization in Pediatrics at the Ghent University Hospital. Following her training as a Pediatrician, she completed a fellowship in Pediatric Nephrology at the Great Ormond Street Hospital in London. Since March 2015, she has combined her clinical activities with translational clinical research as a PhD candidate, focusing on the use of desmopressin in children with enuresis. She is a member of the IPNA (International Pediatric Nephrology Association), the ESPN (European Society of Paediatric Nephrology) and the ESPN Working Group on CAKUT/UTI/Bladder Dysfunction. Mark Turner is a Professor of Neonatology and Research Delivery at the University of Liverpool, UK. He has worked clinically for 30 years and has experience in many aspects of pediatric drug development including excipients, formulations, drug safety, and development and implementation of Pediatric Investigation Plans. He has led Phase 0 (microdosing) through Phase 3 studies. Currently, he co-leads European and international initiatives that promote a strategic approach to pediatric drug development through the development of research standards and infrastructure for clinical trials networks. He works in public private partnership with industry, regulators, academia, and advocates for babies, children, young people and their families. He was elected as an Honorary Fellow of the Faculty of Pharmaceutical Medicine.