Essentials of Translational Pediatric Drug Development
From Past Needs to Future Opportunities
- 1st Edition - July 22, 2024
- Imprint: Academic Press
- 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
Section 1: From past to current needs in pediatric drug research
1. Preface: overview of pediatric drug development and pharmacovigilance
MARK A. TURNER, ELKE GASTHUYS, LIEN DOSSCHE AND KAREL ALLEGAERT
2. Historical perspective
PAULINE DE BRUYNE, FACUNDO GARCIA-BOURNISSEN AND SHINYA ITO
3. Parents' and children's needs in the drug development process
MARIA CAVALLER-BELLAUBI, ARIANE WEINMAN, ERIC VERMEULEN, DIMITRIS ATHANASSIOU, MARIETTE DRIESSENS, ANITA KIENESBERGER AND MARIA BORRELL-PAGES
4. The needs from the regulatory authorities’ perspective – current status and worldwide initiatives
AGNES V. KLEIN AND JUNKO SATO
5. Optimal pediatric therapeutic development – the partnership between patients/families/industry and academia: the pharmaceutical industry’s perspective
SOLANGE CORRIOL-ROHOU, KATHARINE CHENG AND SUSAN MCCUNE
6. The needs, challenges and opportunities from the academic researchers’ perspective
EVA DEGRAEUWE, HIDEFUMI NAKAMURA, JOHAN WALLE AND GREGORY KEARNS
7. The needs from a health care provider’s perspective
SAMARDZIC JANKO, SCHRIER LENNEKE, BIEKE TACK, LIEN DOSSCHE, FACUNDO GARCIA BOURNISSEN AND
JOHN VAN DEN ANKER
Section 2: Designing pediatric drug research: from bench to bedside and back
8. Application of in vitro models
SAGNIK CHATTERJEE, BHAGWAT PRASAD AND PIETER ANNAERT
9. Application of preclinical juvenile animal models
ELINE HERMANS, JAAN TOELEN, VENTRELLA DOMENICO, DE SCHAEPDRIJVER LUC, KATIE TURNER, SISKA CROUBELS AND DEVREESE MATHIAS
10. Application of pediatric adapted modelling and simulation approaches
PIETER-JAN DE SUTTER, JONAS LANGERAERT, ROBIN MICHELET AND AN VERMEULEN
11. The promise of omics approaches for pediatric drug development
SIGRID VERHELST, TESS GOESSENS, ROGER PERO-GASCON, MARTHE DE BOEVRE, FRANCIS IMPENS, DIETER DEFORCE AND MAARTEN DHAENENS
12. Generation and interpretation of big data in pediatric drug development
JEFF BARRETT, AMANDA BORENS AND SOLANGE CORRIOL-ROHOU
13. Investigational Medicinal Product considerations in pediatric clinical drug trials
MANDY WAN, SAMANTHA CARMICHAEL AND CIARA DUFFY
14. Co-designing pediatric clinical trials with multi-stakeholders: a step by step approach
JENNIFER PRESTON, SALLY ANNE DEWS, SOPHIE EVETT,ADIT BASSI, DAISY BARRICK AND SAMMY AINSWORTH
Section 3: Improving the performance of pediatric drug research: big changes start with small steps
15. Principles of performing pediatric clinical drug trials
EVA DEGRAEUWE, JOHAN WALLE, KIM JENNINGS AND PIRKKO LEPOLA
16. Regulatory considerations in the design and conduct of pediatric clinical trials
MELANIE E. BHATNAGAR, GILBERT BURCKART, DONNA L. SNYDER, RALPH BAX, LYNNE YAO AND DIONNA GREEN
17. Ethical considerations in the design and conduct of pediatric clinical trials
PIRKKO LEPOLA, ROBERT NELSON AND KENJI MATSUI
18. Performing clinical drug trials in acute and critically ill neonates and children
PIETER A. DE COCK, ANNE SMITS, MEHDI OUALHA, GIDEON STITT, TUULI METSVAHT, NORI SMEETS,
ANGELA AMIGONI, ATHENA F. ZUPPA AND SASKIA N. DE WILDT
19. Performing clinical drug trials in children with a rare disease
VICTORIA HEDLEY, REBECCA LEARY, ANANDO SEN, EMMA HESLOP, VOLKER STRAUB AND ANNA IRVIN
20. Policy of pediatric oncology drug development
LENNEKE SCHRIER, C. MICHEL ZWAAN, CARMELO RIZZARI, BRENDA WEIGEL, NICOLE SCOBIE, HUBERT CARON,
GREGORY REAMAN AND ANDREW DJ. PEARSON
21. Practice of pediatric oncology drug development
LENNEKE SCHRIER, ANDY PEARSON, CARMELO RIZZARI,BRENDA WEIGEL, HUBERT CARON, ALWIN HUITEMA,
NICOLE SCOBIE, GREGORY REAMAN AND C. MICHEL ZWAAN
22. Pediatric drug formulations
HANNAH BATCHELOR, ISSRAA AL OBAIDI, TSUTOMU HARADA AND JUMPEI SAITO
23. Worldwide network initiatives in improving pediatric drug research
SABAH ATTAR, EVA DEGRAEUWE, JOHAN VANDE WALLE, MARK TURNER, HIDEFUMI NAKAMURA AND
THIERRY LACAZE
24. The patients’/parents’ voice within pediatric drug research
BEGONYA NAFRIA ESCALERA, SARAH BERNAYS, MAGDA CONWAY, KENNETH GETZ, LUNGILE JAFTA AND SIMON R. STONES
25. Pediatric drug development issues during public health emergencies
MARIANA KRUGER, MOJI CHRISTIANAH ADEYEYE AND BARBARA E. BIERER
Section 4: Future perspectives in pediatric drug research: the road to better drugs for children
26. Future of pediatric drug research from different stakeholder perspectives
KAREL ALLEGAERT, ELKE GASTHUYS, LIEN DOSSCHE AND MARK TURNER
1. Preface: overview of pediatric drug development and pharmacovigilance
MARK A. TURNER, ELKE GASTHUYS, LIEN DOSSCHE AND KAREL ALLEGAERT
2. Historical perspective
PAULINE DE BRUYNE, FACUNDO GARCIA-BOURNISSEN AND SHINYA ITO
3. Parents' and children's needs in the drug development process
MARIA CAVALLER-BELLAUBI, ARIANE WEINMAN, ERIC VERMEULEN, DIMITRIS ATHANASSIOU, MARIETTE DRIESSENS, ANITA KIENESBERGER AND MARIA BORRELL-PAGES
4. The needs from the regulatory authorities’ perspective – current status and worldwide initiatives
AGNES V. KLEIN AND JUNKO SATO
5. Optimal pediatric therapeutic development – the partnership between patients/families/industry and academia: the pharmaceutical industry’s perspective
SOLANGE CORRIOL-ROHOU, KATHARINE CHENG AND SUSAN MCCUNE
6. The needs, challenges and opportunities from the academic researchers’ perspective
EVA DEGRAEUWE, HIDEFUMI NAKAMURA, JOHAN WALLE AND GREGORY KEARNS
7. The needs from a health care provider’s perspective
SAMARDZIC JANKO, SCHRIER LENNEKE, BIEKE TACK, LIEN DOSSCHE, FACUNDO GARCIA BOURNISSEN AND
JOHN VAN DEN ANKER
Section 2: Designing pediatric drug research: from bench to bedside and back
8. Application of in vitro models
SAGNIK CHATTERJEE, BHAGWAT PRASAD AND PIETER ANNAERT
9. Application of preclinical juvenile animal models
ELINE HERMANS, JAAN TOELEN, VENTRELLA DOMENICO, DE SCHAEPDRIJVER LUC, KATIE TURNER, SISKA CROUBELS AND DEVREESE MATHIAS
10. Application of pediatric adapted modelling and simulation approaches
PIETER-JAN DE SUTTER, JONAS LANGERAERT, ROBIN MICHELET AND AN VERMEULEN
11. The promise of omics approaches for pediatric drug development
SIGRID VERHELST, TESS GOESSENS, ROGER PERO-GASCON, MARTHE DE BOEVRE, FRANCIS IMPENS, DIETER DEFORCE AND MAARTEN DHAENENS
12. Generation and interpretation of big data in pediatric drug development
JEFF BARRETT, AMANDA BORENS AND SOLANGE CORRIOL-ROHOU
13. Investigational Medicinal Product considerations in pediatric clinical drug trials
MANDY WAN, SAMANTHA CARMICHAEL AND CIARA DUFFY
14. Co-designing pediatric clinical trials with multi-stakeholders: a step by step approach
JENNIFER PRESTON, SALLY ANNE DEWS, SOPHIE EVETT,ADIT BASSI, DAISY BARRICK AND SAMMY AINSWORTH
Section 3: Improving the performance of pediatric drug research: big changes start with small steps
15. Principles of performing pediatric clinical drug trials
EVA DEGRAEUWE, JOHAN WALLE, KIM JENNINGS AND PIRKKO LEPOLA
16. Regulatory considerations in the design and conduct of pediatric clinical trials
MELANIE E. BHATNAGAR, GILBERT BURCKART, DONNA L. SNYDER, RALPH BAX, LYNNE YAO AND DIONNA GREEN
17. Ethical considerations in the design and conduct of pediatric clinical trials
PIRKKO LEPOLA, ROBERT NELSON AND KENJI MATSUI
18. Performing clinical drug trials in acute and critically ill neonates and children
PIETER A. DE COCK, ANNE SMITS, MEHDI OUALHA, GIDEON STITT, TUULI METSVAHT, NORI SMEETS,
ANGELA AMIGONI, ATHENA F. ZUPPA AND SASKIA N. DE WILDT
19. Performing clinical drug trials in children with a rare disease
VICTORIA HEDLEY, REBECCA LEARY, ANANDO SEN, EMMA HESLOP, VOLKER STRAUB AND ANNA IRVIN
20. Policy of pediatric oncology drug development
LENNEKE SCHRIER, C. MICHEL ZWAAN, CARMELO RIZZARI, BRENDA WEIGEL, NICOLE SCOBIE, HUBERT CARON,
GREGORY REAMAN AND ANDREW DJ. PEARSON
21. Practice of pediatric oncology drug development
LENNEKE SCHRIER, ANDY PEARSON, CARMELO RIZZARI,BRENDA WEIGEL, HUBERT CARON, ALWIN HUITEMA,
NICOLE SCOBIE, GREGORY REAMAN AND C. MICHEL ZWAAN
22. Pediatric drug formulations
HANNAH BATCHELOR, ISSRAA AL OBAIDI, TSUTOMU HARADA AND JUMPEI SAITO
23. Worldwide network initiatives in improving pediatric drug research
SABAH ATTAR, EVA DEGRAEUWE, JOHAN VANDE WALLE, MARK TURNER, HIDEFUMI NAKAMURA AND
THIERRY LACAZE
24. The patients’/parents’ voice within pediatric drug research
BEGONYA NAFRIA ESCALERA, SARAH BERNAYS, MAGDA CONWAY, KENNETH GETZ, LUNGILE JAFTA AND SIMON R. STONES
25. Pediatric drug development issues during public health emergencies
MARIANA KRUGER, MOJI CHRISTIANAH ADEYEYE AND BARBARA E. BIERER
Section 4: Future perspectives in pediatric drug research: the road to better drugs for children
26. Future of pediatric drug research from different stakeholder perspectives
KAREL ALLEGAERT, ELKE GASTHUYS, LIEN DOSSCHE AND MARK TURNER
- Edition: 1
- Published: July 22, 2024
- No. of pages (Paperback): 728
- Imprint: Academic Press
- Language: English
- 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