Essentials of Translational Pediatric Drug Development. From Past Needs to Future Opportunities

Table of Contents

SECTION 1: From past to current needs in pediatric drug research
1. Historical perspective
2. The needs from the regulatory authorities’ perspective worldwide initiatives
3. The needs from the pharmaceutical industry’s perspective
4. The needs from the clinicians’ perspective
5. The needs from the parents’/children’ perspective

SECTION 2: Designing pediatric drug research: from bench to bedside and back
6. Different approaches: concepts and principles
7. Application of in vitro models
8. Application of preclinical juvenile animal models
9. Application of pediatric adapted modelling and simulation approaches
10. Application of multi-omics approaches
11. Generation and interpretation of big data
12. Designing pediatric clinical trials using different approaches from different stakeholder perspectives

SECTION 3: Improving the performance of pediatric drug research: big changes start with small steps
13. General principles of performing pediatric clinical drug trials
14. Regulatory considerations in the design and conduct of paediatric clinical trials
15. Performing clinical drug trials in acute and critically ill neonates and children
16. Performing clinical drug trials in chronically ill children
17. Performing clinical drug trials in pediatric rare diseases
18. Performing clinical drug trials in pediatric oncology
19. Pediatric drug formulations
20. Worldwide network initiatives in improving pediatric drug research
21. The need for an ethical framework to conduct pediatric drug research
22. The patients’/parents’ voice within pediatric drug research

SECTION 4: Future perspectives in pediatric drug research: the road to better drugs for children
23. Future of pediatric drug research from different stakeholder perspectives
24. Concluding thoughts

Authors

Elke Gasthuys Postdoctoral researcher, Ghent University, Belgium. 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 Pediatrician-neonatologist and Clinical Pharmacologist, Professor, KU Leuven, Belgium; Senior clinical consultant, Erasmus MC Rotterdam, The Netherlands. 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 Full-time Academic Staff Member, Ghent University, Belgium. 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 Professor of Neonatology and Research Delivery, University of Liverpool, UK. 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.