Main description:

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.

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