Essentials of translational pediatric drug development : from past needs to future opportunities /

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.

Bibliographic Details
Corporate Author: ScienceDirect (Online service)
Other Authors: Gasthuys, Elke (Editor), Allegaert, Karel (Editor), Dossche, Lien (Editor), Turner, Mark (Neonatologist) (Editor)
Format: eBook
Language:English
Published: London, United Kingdom ; San Diego, CA, United States ; Cambridge, MA, United States : Elsevier, Academic Press, an Imprint of Elsevier, [2024]
Subjects:
Online Access:Connect to the full text of this electronic book

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245 0 0 |a Essentials of translational pediatric drug development :  |b from past needs to future opportunities /  |c edited by Elke Gasthuys, Karel Allegaert, Lien Dossche, Mark Turner. 
264 1 |a London, United Kingdom ;  |a San Diego, CA, United States ;  |a Cambridge, MA, United States :  |b Elsevier, Academic Press, an Imprint of Elsevier,  |c [2024] 
264 4 |c ©2024 
300 |a 1 online resource :  |b illustrations (some color) 
336 |a text  |b txt  |2 rdacontent 
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347 |a text file  |2 rdaft 
504 |a Includes bibliographical references and index. 
588 0 |a Online resource; title from PDF title page (ScienceDirect, viewed August 7, 2024). 
505 0 |a Front Cover -- ESSENTIALS OF TRANSLATIONAL PEDIATRIC DRUG DEVELOPMENT -- ESSENTIALS OF TRANSLATIONAL PEDIATRIC DRUG DEVELOPMENT: FROM PAST NEEDS TO FUTURE OPPORTUNITIES -- Copyright -- Contents -- Contributors -- I -- From past to current needs in pediatric drug research -- 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 -- Pharmacovigilance -- Pharmacovigilance tailored to children -- Doing studies -- The importance of collaboration -- Not in scope -- References -- 2 -- Historical perspective -- Labeling of medicines -- Therapeutic orphans -- Pediatric medicines initiatives -- Worldwide initiatives to stimulate pediatric clinical trials -- The US legislation -- The European Pediatric Regulation -- "Better Medicines for Children" resolution of the World Health Assembly -- Pediatric medicines initiatives in other countries across the globe -- ICH E11: Harmonizing the development of pediatric medicines -- Pediatric medicine networks -- Pediatric clinical pharmacology -- References -- 3 -- Parents' and children's needs in the drug development process -- Introduction: Unmet needs for pediatric patients -- Pediatric drug development -- What are the benefits of pediatric and clinical trial regulations? -- Pediatric regulation in the United States (US) and in the European Union (EU) -- Novel EU clinical trial regulation -- Research -- Agenda setting and translational research -- Pediatric patient-centered clinical trials -- Clinical trials at home -- Clinical trial awareness and communication -- Consent for clinical research -- Data sharing -- Registries and data platforms. 
505 8 |a The specific parents/children's needs for accessing early diagnosis/screening programs, innovative treatments, clinical tri ... -- Early diagnosis -- Innovative treatments -- Access to cross-border clinical trials -- Compassionate use (early access) programs -- The specific parents/children's needs for empowerment and training to be active actors in research and for their health -- Empowerment and training -- The role of patient organizations -- Involvement of children/young patients -- Pediatric networks -- Authors' final reflections -- References -- Further reading -- 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 -- Acts/laws -- Data -- Data requirements -- Data scope -- Preclinical or nonclinical -- Clinical -- Instructions for the use of a therapeutic -- Burden of proof -- How regulators work? -- Regulators' profile -- Communication -- Between regulatory agencies -- With other stakeholders -- Follow-up file submission -- Decision-making -- Post-marketing -- Has the regulatory approach changed over the years and what will the future bring? -- Concluding thoughts -- References -- Further reading -- Additional interesting references -- 5 -- Optimal pediatric drug development- the partnership between patients/parents, academia, regulatory authorities ... -- Pharmaceutical industry's participation in pediatric drug development -- Pharmaceutical industry involvement in all phases of pediatric drug development -- Drug discovery -- Preclinical research -- Development of pediatric formulations -- Clinical research -- Postmarketing monitoring -- Partnership between the pharmaceutical industry and other stakeholders within pediatric drug research -- Patients/parents. 
505 8 |a 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 -- References -- 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 -- Drug discovery and development -- Preclinical research -- Clinical research -- Postmarketing pharmacovigilance -- Repurposing -- Interplay between academia and other stakeholders within pediatric drug research -- Pharmaceutical industry -- Governmental authorities -- Healthcare professionals -- Involvement of patients and parents in the clinical research process -- Organization of academic pediatric drug research -- Role of the academic institutions: Education, training, and facilities -- Education/training -- Facilities -- Academic expertise core facilities -- Academic clinical trial units -- Academic technology transfer offices -- Funding -- Open science -- Impact -- Initiatives examples -- Conclusions: The future for academia participating in pediatric drug research -- References -- 7 -- The health care provider's needs in the pediatric drug development process -- Introduction and background of prescribing medicines for children -- Introduction -- Historical aspects and background -- Current daily clinical practice: Challenges and (practical) approaches -- The current clinical situation: Global extent of off-label and unlicensed medicines use in children -- Off-label use -- Unlicensed medicine use -- Insufficient access to on-label medicines -- Practical approaches to current prescribing challenges -- Off-label medicines -- When to prescribe off-label medicines?. 
505 8 |a Challenges when prescribing off-label medicines -- How to prescribe off-label medicines? -- Use of pediatric medicine formularies -- Benefit-risk assessment off-label medicine use in children -- Informed consent -- Unlicensed medicines -- Challenges when prescribing unlicensed medicines -- Possible approaches when prescribing unlicensed medicines -- Pediatric medicine development as a possible solution -- health care providers in collaboration with other stakeholders -- Future perspectives: Needs and priorities -- References -- II -- Designing pediatric drug research: From bench to bedside and back -- 8 -- Application of in vitro models for pediatric translational research -- Introduction -- Methods -- In vitro models for protein activity measurements -- Types of in vitro models in translational pediatric research -- Experimental conditions for activity assays with in vitro pediatric models -- Data sets generated with in vitro pediatric or juvenile ADME models -- In vitro studies with tissues derived from pediatric donors -- In vitro studies with tissues derived from neonatal or juvenile animals -- Translation of in vitro ADME activity data by IVIVE approaches -- ADME protein abundance measurements on pediatric tissues -- Positioning of protein content analysis in translational pediatric research -- Technological state-of-the-art of quantitative proteomics -- Application of quantitative proteomics to reveal ontogeny of DMEs and transport proteins -- Application of proteomics data in relation to in vitro ADME tools -- Discussion -- References -- 9 -- Application of preclinical juvenile animal models -- Introduction -- Regulatory perspective -- Pediatric Investigation Plan and Pediatric Study Plan -- Designing the nonclinical strategy -- Need for a juvenile animal toxicity study -- Timing of juvenile animal toxicity studies. 
505 8 |a Animal species and age categories to be considered -- Mice -- Rat -- Rabbit -- Dog -- Sheep -- Pig -- Nonhuman primates -- Age of the animals -- Study design -- Study end-points -- Route of administration -- Dose selection -- Formulation and excipients -- ADME -- Reproducibility and translatability of animal research -- The reproducibility crisis: the standardization fallacy -- The translatability crisis: animal models -- Conclusions -- References -- 10 -- Application of pediatric-adapted modeling and simulation approaches -- Quantitative pharmacology in pediatric drug development -- Basic PK/PD concepts of small and large molecules -- Exposure-response relationships -- Overview of the available toolbox -- Allometric approaches -- Selecting an allometric exponent -- When is allometric scaling appropriate? -- Allometric scaling from preclinical animal species -- Allometric scaling of monoclonal antibodies -- Physiologically based pharmacokinetic modeling -- Core principles of PBPK modeling -- Building a pediatric-PBPK model -- Step 1: Gather drug-dependent data to define a drug model -- Step 2: Learn and confirm in an adult population -- Step 3: Verify and apply the PBPK model in children -- Pediatric PBPK model applications -- Dose selection and study design -- Drug-drug interactions -- Special pediatric populations and disease effects -- Protein drugs -- Pharmacodynamics and toxicology -- Population pharmacokinetics -- PopPK modeling and simulation -- Integration with allometric scaling and ontogeny -- Linking PK with PD -- Moving pediatric adapted modeling and simulation approaches toward maturity -- Rational selection and application of available modeling and simulation tools -- The pediatric diseased populations -- Pediatric and developmental pharmacodynamics -- Conclusion -- References. 
505 8 |a 11 -- The promise of omics approaches for pediatric drug development. 
520 |a 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. 
650 0 |a Pediatric pharmacology. 
650 0 |a Drug development. 
650 0 |a Drugs  |x Design. 
650 2 |a Drug Design 
650 6 |a Pharmacologie pédiatrique. 
650 6 |a Médicaments  |x Développement. 
650 6 |a Médicaments  |x Conception. 
655 7 |a Electronic books.  |2 local 
700 1 |a Gasthuys, Elke,  |e editor. 
700 1 |a Allegaert, Karel,  |e editor. 
700 1 |a Dossche, Lien,  |e editor. 
700 1 |a Turner, Mark  |c (Neonatologist),  |e editor.  |1 https://id.oclc.org/worldcat/entity/E39PCjHdhpwYWG4hq68TCQb7h3 
710 2 |a ScienceDirect (Online service) 
776 0 8 |i Print version:  |t Essentials of translational pediatric drug development.  |d London : Academic Press, [2024]  |z 9780323884594 
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