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.

• Section 1: From past to current needs in pediatric drug research1. Preface: overview of pediatric drug development and pharmacovigilance2. Historical perspective3. Parents' and children's needs in the drug development process4. The needs from the regulatory authorities’ perspective – current status and worldwide initiatives5. Optimal pediatric therapeutic development – the partnership between patients/families/industry and academia: the pharmaceutical industry’s perspective6. The needs, challenges and opportunities from the academic researchers’ perspective7. The needs from a health care provider’s perspectiveSection 2: Designing pediatric drug research: from bench to bedside and back8. Application of in vitro models9. Application of preclinical juvenile animal models10. Application of pediatric adapted modelling and simulation approaches11. The promise of omics approaches for pediatric drug development12. Generation and interpretation of big data in pediatric drug development13. Investigational Medicinal Product considerations in pediatric clinical drug trials14. Co-designing pediatric clinical trials with multi-stakeholders: a step by step approachSection 3: Improving the performance of pediatric drug research: big changes start with small steps15. Principles of performing pediatric clinical drug trials16. Regulatory considerations in the design and conduct of pediatric clinical trials17. Ethical considerations in the design and conduct of pediatric clinical trials18. Performing clinical drug trials in acute and critically ill neonates and children19. Performing clinical drug trials in children with a rare disease20. Policy of pediatric oncology drug development21. Practice of pediatric oncology drug development22. Pediatric drug formulations23. Worldwide network initiatives in improving pediatric drug research24. The patients’/parents’ voice within pediatric drug research25. Pediatric drug development issues during public health emergenciesSection 4: Future perspectives in pediatric drug research: the road to better drugs for children26. Future of pediatric drug research from different stakeholder perspectives

"This book discusses ethical considerations in pediatric clinical trials, pediatric trials for children with rare diseases, and cancer. It discusses these issues not only for the US but also for the European Union and worldwide network efforts for improving pediatric drug research.... The editors and authors of the book are credible experts in their fields based on their credentials. This book covers all matters of pediatric drug development. The book describes and discusses the three different approaches to pediatric drug development and the special issues and needs that arise due to the needs of pediatric patients.... It includes references and further readings at the end of each chapter.... The book provides a good, detailed background and description of the pediatric drug development process in the U.S. and in Europe and the special issues with pediatric drug development." --©Doody’s Review Service, 2024, Judith Klevan, B.A Biology B.S. Pharmacy (Einstein Medical Center)