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

ISBN-13: 9780323884594 / Angielski

Elke Gasthuys; Karel Allegaert; Lien Dossche

Opis

Essentials of Translational Pediatric Drug Development: From Past Needs to Future Opportunities provides integrated and up-to-date insights relevant for both translational researchers and clinicians active in the field of pediatric drug development. The book covers all key aspects from different stakeholder perspectives, providing a literature overview and careful reflection on state-of-the-art approaches. It will be an ideal guide for researchers in the field who are designing and performing high quality, innovative pediatric-adapted drug development by helping them define needs/challenges and possible solutions that advance and harmonize pediatric drug development. Despite the broad consensus that children merit the same quality of drug treatment as any other age group, children remain frequently neglected during drug research and development. Even with the adoption of multiple legislations addressing this problem, the lack of efficacy and safety data of marketed as well as newly developed drugs still remain in the pediatric population.

Spis treści

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