Essentials of Translational Pediatric Drug Development
From Past Needs to Future Opportunities
- 1st Edition - July 22, 2024
- Editors: Elke Gasthuys, Karel Allegaert, Lien Dossche, Mark Turner
- Language: English
- Paperback ISBN:9 7 8 - 0 - 3 2 3 - 8 8 4 5 9 - 4
- eBook ISBN:9 7 8 - 0 - 3 2 3 - 9 0 4 2 9 - 2
Essentials of Translational Pediatric Drug Development: From Past Needs to Future Opportunities provides integrated and up-to-date insights relevant for both translational resear… Read more
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Request a sales quoteEssentials 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.
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.
- Covers both theoretical and practical aspects of translational pediatric drug development
- Approaches the topic from different stakeholder perspectives (academics, industry, regulators, clinicians and patient/parent advocacy groups)
- Offers best practices and future perspectives for the improvement of translational pediatric drug development
Translational researchers and clinicians active in the field of pediatric drug development, Pediatricians, pharmacists, general practitioners, health care professionals involved in childcare and pediatric development, clinical trial personnel, patient/parent advocacy groups, institutional review boards, regulatory authorities, and pharmaceutical industry
- Cover image
- Title page
- Table of Contents
- Copyright
- Contributors
- Section I. From past to current needs in pediatric drug research
- Chapter 1. Preface: Overview of pediatric drug development and pharmacovigilance
- Introduction
- Elements of drug development
- Approaches to drug development
- Clinical pediatric drug development
- Foundational research
- Goals of drug development
- Doing studies
- The importance of collaboration
- Not in scope
- Chapter 2. Historical perspective
- Labeling of medicines
- Therapeutic orphans
- Pediatric medicines initiatives
- Pediatric medicine networks
- Pediatric clinical pharmacology
- Chapter 3. Parents' and children's needs in the drug development process
- Introduction: Unmet needs for pediatric patients
- Pediatric drug development
- The specific parents/children's needs for accessing early diagnosis/screening programs, innovative treatments, clinical trials, and compassionate use programs
- The specific parents/children's needs for empowerment and training to be active actors in research and for their health
- Authors' final reflections
- Chapter 4. The needs from the regulatory authorities' perspective—current status and worldwide initiatives
- Introduction
- Definitions
- The regulatory requirements for the drug submission process
- Acts, laws, and guidance
- Data
- Data scope
- Burden of proof
- How regulators work?
- Has the regulatory approach changed over the years and what will the future bring?
- Concluding thoughts
- Chapter 5. Optimal pediatric drug development— the partnership between patients/parents, academia, regulatory authorities and industry: The pharmaceutical industry's perspective
- Pharmaceutical industry's participation in pediatric drug development
- Pharmaceutical industry involvement in all phases of pediatric drug development
- Preclinical research
- Development of pediatric formulations
- Clinical research
- Postmarketing monitoring
- Partnership between the pharmaceutical industry and other stakeholders within pediatric drug research
- Patients/parents
- Academia/clinicians in clinical practice and research
- Regulatory authorities
- Co-operation among pharmaceutical companies
- Organization of pharmaceutical industry pediatric drug research
- The future of pharmaceutical industry pediatric drug research
- Chapter 6. The needs, challenges, and opportunities from the academic researchers' perspective
- Academia participate in translational pediatric drug research
- Roles of academia within pediatric drug research
- Interplay between academia and other stakeholders within pediatric drug research
- Organization of academic pediatric drug research
- Conclusions: The future for academia participating in pediatric drug research
- Chapter 7. The health care provider's needs in the pediatric drug development process
- Introduction and background of prescribing medicines for children
- Current daily clinical practice: Challenges and (practical) approaches
- Practical approaches to current prescribing challenges
- Future perspectives: Needs and priorities
- Section II. Designing pediatric drug research: From bench to bedside and back
- Chapter 8. Application of in vitro models for pediatric translational research
- Introduction
- Methods
- In vitro models for protein activity measurements
- ADME protein abundance measurements on pediatric tissues
- Discussion
- Chapter 9. Application of preclinical juvenile animal models
- Introduction
- Regulatory perspective
- Animal species and age categories to be considered
- Study design
- Reproducibility and translatability of animal research
- Conclusions
- Chapter 10. Application of pediatric-adapted modeling and simulation approaches
- Quantitative pharmacology in pediatric drug development
- Allometric approaches
- Physiologically based pharmacokinetic modeling
- Population pharmacokinetics
- Moving pediatric adapted modeling and simulation approaches toward maturity
- Conclusion
- Chapter 11. The promise of omics approaches for pediatric drug development
- Introducing OMICS
- Chapter 12. Generation and interpretation of big data in pediatric drug development
- Introduction
- RWD and RWE definitions
- Sources of real-world data—overview
- Quality and privacy challenges for pediatric RWD
- Legislations and organized efforts that have encouraged the development of RWD
- Regulatory guidance documents and laws
- Existing RWD guidelines
- ICH guidelines
- HIPAA/GDPR and patient confidentiality and access concerns/sensitive variables
- Institutional review boards/research ethics committees (IRB/REC)
- Applications and examples
- Workforce and resource needs
- Conclusions
- Chapter 13. Investigational medicinal products considerations in pediatric clinical drug trials
- Introduction
- Investigational products
- Challenges in investigational product supply chain
- What is a drug?
- Drug formulation development
- Age-appropriate formulations
- Quality management in pharmaceutical manufacturing
- Good manufacturing practice
- Manufacturing process development and validation
- Blinding in clinical trials
- Investigational product label
- Supply chain management
- Transport, storage, and distribution of investigational products
- Role of the qualified person
- Site logistics
- Risk proportionate approaches to the management of investigational products
- Prospective—From drug substance to investigational product supply
- Conclusions
- Chapter 14. Co-designing pediatric clinical trials with multistakeholders: A step-by-step approach
- Introduction
- The role of CYP in the design and/or conduct of pediatric clinical trials
- Practicalities of involving CYP
- Involving children and young people in a life science sponsored study: A case study
- Public/private partnership—pilot project
- Strategic and operational aspects of PPI within Pfizer
- “How-to” involve CYP in a clinical trial protocol design of an eczema study
- Our approach and lessons learned
- Defining partnership and collaboration goals
- Identifying patient partners and initiating a partnership
- Contracts and confidentiality agreements
- Maintaining confidentiality
- Disclosure of conflicts of interest
- Costings
- Logistics
- Reflections and conclusion
- Section III. Improving the performance of pediatric drug research: Big changes start with small steps
- Chapter 15. Principles of performing pediatric clinical drug trials
- Main principles of conducting clinical trials in children
- Involving child and/or parent within a trial design
- Adapting trial study team for pediatric needs
- Limiting sampling and optimizing outcomes
- Communication optimization between site and sponsor (and research network)
- Designing the pediatric clinical trial
- Multicenter trials
- Site initiation
- Trial start and conduct
- Trial practicalities and limitations
- Monitoring trial quality and patient safety
- Clinical trial closure, reporting, and publications
- Take home messages and future perspectives
- Appendix
- Chapter 16. Regulatory considerations in the design and conduct of pediatric clinical trials
- Introduction to the regulatory perspective on pediatric clinical trials
- What makes pediatric product development unique?
- Approaches for optimizing pediatric product development
- Selecting a pediatric dose
- Considerations for the design and conduct of pediatric clinical therapeutics trials
- Putting it all together: Planning a pediatric product development program
- Communicating with regulatory authorities
- International collaboration and harmonization
- Future directions
- Disclaimer
- Chapter 17. Ethical consideration in the design and conduct of pediatric clinical trials
- Introduction
- Short history of the pediatric clinical research ethics
- Ethical principles of pediatric clinical trials
- Ethical elements of pediatric clinical trials
- Children and their legal representatives
- Design of pediatric clinical trial—ethical perspective
- Concepts of risks, burden, and benefit
- Designing informed consents, assents and patient information
- Recruitment to clinical trials
- Basic elements of the informed consent process
- Respecting child's rights and understanding disagreements
- Ethical review
- Lay summary of clinical trial results
- Chapter 18. Performing clinical drug trials in acute and critically ill neonates and children
- Developmental and pathophysiological changes in critically ill children
- The contemporary approach to drug development in NICU/PICU
- Practical aspects of the design and conduct of research studies
- Ethics
- Policy issues
- Opportunities
- Future perspectives and take home messages
- Chapter 19. Performing clinical drug trials in children with a rare disease
- What conditions are necessary to achieve clinical trial readiness?
- Challenges in designing and delivering PRD clinical trials and current solutions to address these
- Conclusions
- Chapter 20. Policy of pediatric oncology drug development
- Introduction
- A paradigm shift in drug development for childhood cancers
- Need to consider childhood cancers separately from adult cancers
- The regulatory context and precompetitive multi-stakeholder collaboration
- Equal access to innovation and precision medicine: new models of partnership and funding
- Current challenges/unmet needs
- Future perspectives and take home messages
- Chapter 21. Practice of pediatric oncology drug development
- Need for mechanism-of-action approach in pediatric oncology drug development
- Preclinical research
- Revised framework for early-phase clinical trials
- Currently unmet challenges/needs
- Future perspectives and take home messages
- Chapter 22. Pediatric drug formulations
- The need for formulated drug products
- The need for pediatric formulated drug products
- Historic landscape of pediatric formulations
- Main challenges in developing pediatric formulations
- Relative bioavailability and the introduction of a pediatric product
- Palatability of medicines and acceptability
- Oral liquid dosage forms
- Oral solid dosage forms
- Nasal formulations
- Inhaled formulations
- Rectal formulations
- Cutaneous/transdermal formulations
- Eye and ear formulations
- Parenteral formulations
- Additional considerations for enteral feeding tubes
- Innovations in pediatric formulation development
- Conclusions
- Chapter 23. Worldwide network initiatives in improving pediatric drug research
- Introduction
- The rationale and roles of clinical research networks
- Types of clinical research networks
- Services and use-cases of clinical trial networks
- Challenges
- Future needs and recomendations
- Conclusions
- Supplementary material
- Chapter 24. The patients'/parents' voice within pediatric drug research
- Introduction: conceptual approach regarding the active involvement of CYP involvement in drug development
- Tracing the historical development of the involvement of CYP in pediatric drug development
- Involvement of patients and parents in regulatory activities
- Tools and resources for involving patients and parents in the drug development process
- The role of patients and caregivers in pediatric clinical trials networks
- Diversity and representativeness: they are not “hard to reach,” we need to extend our arms
- Conclusion: standardizing practice to ensure the meaningful and ethical involvement of patients through an accountable process
- Acknowledgments
- Chapter 25. Pediatric drug development issues during public health emergencies
- Introduction and overview
- Considerations during a PHEIC
- Effect of PHEICs on chronic health conditions
- Effect of PHEICs on research
- Children during PHEICs
- Influence of PHEICs on individual liberty and effects on communities
- Ethical foundations of research during PHEICs
- Potential ethical framework for conducting pediatric drug trials during PHEICs
- Clinical trials during PHEICs with a focus on pediatric drug development
- Lessons learned from the COVID-19 pandemic and the effect on pediatric drug development
- Actions before and during a PHEIC
- Potential practical framework for conducting pediatric trials during PHEICs
- Conclusion
- Section IV. Future perspectives in pediatric drug research: The road to better drugs for children
- Chapter 26. The future of pediatric drug research: Science, context, and collaboration
- The science of drug development
- The context of drug development
- Collaboration in drug development
- Conclusion
- Index
- No. of pages: 728
- Language: English
- Edition: 1
- Published: July 22, 2024
- Imprint: Academic Press
- Paperback ISBN: 9780323884594
- eBook ISBN: 9780323904292
EG
Elke Gasthuys
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.
Affiliations and expertise
Postdoctoral researcher, Ghent University, BelgiumKA
Karel Allegaert
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).
Affiliations and expertise
Pediatrician-neonatologist and Clinical Pharmacologist, Professor, KU Leuven, Belgium; Senior clinical consultant, Erasmus MC Rotterdam, The NetherlandsLD
Lien Dossche
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.
Affiliations and expertise
Full-time Academic Staff Member, Ghent University, BelgiumMT
Mark Turner
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.
Affiliations and expertise
Professor of Neonatology and Research Delivery, University of Liverpool, UKRead Essentials of Translational Pediatric Drug Development on ScienceDirect