Pediatric Drug Development. 2nd Edition

  • ID: 2516971
  • Book
  • 624 Pages
  • John Wiley and Sons Ltd
1 of 4

Most medicines have never been adequately tested for safety and efficacy in pediatric populations and preterm, infants and children are particularly vulnerable to adverse drug reactions.Pediatric Drug Development: Concepts and Applications, Second Edition, addresses the unique challenges in conducting effective drug research and development in pediatric populations.

This new edition covers the legal and ethical issues of consent and assent, the additional legal and safety protections for children, and the appropriate methods of surveillance and assessment for children of varying ages and maturity, particularly for patient reported outcomes. It includes new developments in biomarkers and surrogate endpoints, developmental pharmacology and other novel aspects of global pediatric drug development. It also encompasses the new regulatory initiatives across EU, US and ROW designed to encourage improved access to safe and effective medicines for children globally.

From an international team of expert contributors Pediatric Drug Development: Concepts and Applications is the practical guide to all aspects of the research and development of safe and effective medicines for children.

Note: Product cover images may vary from those shown
2 of 4

List of Contributors

Preface

Part I: PAST, PRESENT, AND FUTURE OF PEDIATRIC DRUG DEVELOPMENT
 
1 Pediatric Drug Development and Therapeutics: Continued Progress for Better Drugs for ChildrenAndrew E. Mulberg, Lisa Mathis, Julia Dunne and Dianne Murphy

2 History of Children and the Development of Regulations at the FDARobert M. Ward and Steven Hirschfeld

3 Population Dynamics, Demographics, and Disease Burden of Infants and Children Across the WorldAshley Malins and Christopher–Paul Milne

4 Pharmaceutical Economics and Market Access for Pediatric  MedicationsChristopher–Paul Milne and Ashley Malins

5 The Global Pediatric Market and Drivers of Pediatric Drug DevelopmentE. Michael D. Scott

6 Industry Benchmarks in Pediatric Clinical TrialsCarolyn A. Campen and Cindy Levy–Petelinkar

Part II: ETHICAL UNDERPINNINGS
 
7 Ethical and practical considerations in conducting neonatal researchMichelle Roth–Cline and Robert M. Nelson

8 Ethical considerations in conducting pediatric researchMichelle Roth–Cline and Robert M. Nelson

9 The Consent and Assent Process in Pediatric Clinical TrialsM. Renee Simar

Part III: REGULATORY GUIDELINES FOR PEDIATRIC DRUG DEVELOPMENT
 
10 A Sponsor s Perspective of US RegulationsSamuel Maldonado

11 FDA experience of extrapolation of efficacy to the pediatric population from adult and other dataJulia Dunne, William J. Rodriguez and Dianne Murphy

12 Accelerated Approval and other regulatory approaches to the development of drugs for serious or life–threatening diseases in pediatricsTherese Cvetkovich

13 Rare Diseases and Orphan DrugsAnne R. Pariser and Lynne P. Yao

14 European PerspectiveAgnès Saint–Raymond

15 Five years of pediatric legislation in the European UnionAgnès Saint–Raymond and Ralf Herold

16 Japanese PerspectiveHidefumi Nakamura and Shunsuke Ono

17 Pediatric Device Development in the United StatesPriya Venkataraman–Rao and Joy Samuels–Reid

Part IV: PRECLINICAL SAFETY ASSESSMENT
 
18 Introduction and OverviewTimothy P. Coogan and Melissa S. Tassinari

19 Juvenile Animal Toxicity Assessments: Decision Strategies and Study DesignLuc M. De Schaepdrijver,  Graham P. Bailey, Timothy P. Coogan and Jennifer L. Ingram–Ross

20 Absorption, Distribution, Metabolism and Excretion (ADME) and Pharmacokinetic Assessments in Juvenile AnimalsLoeckie L. de Zwart, Johan G. Monbaliu and Pieter P. Annaert

21 A Global Regulatory PerspectiveKaren Davis–Bruno, Jacqueline Carleer, Beatriz Silva Lima and Melissa S. Tassinari

22 Disease specific models to enhance Pediatric drug developmentNiraj R. Mehta and Sruthi King

Part V: PHARMACOLOGICAL PRINCIPLES IN PEDIATRIC DRUG DEVELOPMENT
 
23 Pediatric Clinical Pharmacology in Regulatory and Drug Development Sciences:  Lessons learned and the Path ForwardGilbert J. Burckart and Jeremiah Momper

24 Development and Clinical Trial DesignKathleen A. Neville, Ralph E. Kauffman, and Susan M. Abdel–Rahman

25 Developmental Hepatic Pharmacology in PediatricsMartin Otto Behm

26 Applications of Population Pharmacokinetics for Pediatric Drug DevelopmentJeremiah Momper, Gilbert J. Burckart and Pravin Jadhav

27 Applications of Pharmacogenomics to Pediatric Drug DevelopmentGilbert J. Burckart, Dionna Green and Padmaja Mummaneni

28 Pharmacometrics Applications to Pediatric TrialsDevin Pastoor, Mallika Lala, Jogarao V.S. Gobburu

Part VI: CLINICAL TRIAL OPERATIONS
 
29 Brain and Central Nervous System Development: Physiological Considerations for Assessment of Long–Term SafetyKachikwu Illoh

30 Cognitive Development Considerations for Long–Term Safety Exposures in ChildrenMary Pipan, Paul Wang,  and Rebecca Thompson–Penna

31 Cardiovascular and QTc IssuesBert Suys and  Luc Dekie

32 Pediatric bone and adult bone physiological differencesFrancisco A. Sylvester and Erica L. Wynn

33 Renal Function IssuesKatia Boven

34 Growth and Physical DevelopmentAlisha J. Rovner and  Babette S. Zemel

35 Development of Drugs for Pediatric CancersKristen M. Snyder, Martha Donoghue, Whitney S. Helms, and Gregory Reaman

Part VII: CLINICAL TRIAL OPERATIONS AND GOOD CLINICAL TRIALS
 
36 Recruitment and Retention in Pediatric Clinical Trials: Focus on Networks  in the US and EU Rosalind L. Smyth, Geetinder Kaur, Daniel Benjamin Jr, and Matthew Laughon

37 Recruitment and Retention of Minority populations in Clinical TrialsBenjamin Ortiz and Sergio Guerrero

38 Conducting Clinical Trials in Developing and Emerging Countries:  Review and Case StudyKevin D. Hill and Jennifer S. Li

39 The Importance of Geographic Differences in Pediatric Clinical TrialsAlexandar Cvetkovich Muntañola

Part VIII: CLINICAL EFFICACY AND SAFETY ENDPOINTS
 
40 Clinical Laboratory Testing in Clinical Trials for Pediatric SubjectsAndrew E. Mulberg and Ethan D. Hausman41 Surrogate Endpoints: Application in Pediatric Clinical TrialsGeert Molenberghs

42 Clinical Outcome Assessments for Clinical Trials in ChildrenElektra J. Papadopoulos,  Donald L. Patrick,  Melissa S. Tassinari,  Andrew E. Mulberg,  Carla Epps,  Anne R. Pariser and  Laurie B. Burke

43 Challenges in Evaluating the Safety of Pediatric Therapeutic Agents in Trials and Post–Marketing StudiesAnn W. McMahon, Chris Feudtner and Dianne Murphy

44 Endpoints in Rare DiseasesAnne R. Pariser and Lynne P. Yao

Part IX: FORMULATION, CHEMISTRY, AND MANUFACTURING CONTROLS
 
45 Formulation, Chemistry, and Manufacturing ControlsGerard P. McNally and Aniruddha M. Railkar

46 Drug Delivery Challenges for the Pediatric Patient: Novel Forms for  ConsiderationAnn Zajicek47 The Jelly Bean Test: A Novel Technique To Help Children Swallow MedicationsRobbyn E. Sockolow and Aliza B. Solomon

Index

Note: Product cover images may vary from those shown
3 of 4

Loading
LOADING...

4 of 4

Andrew E. Mulberg, Division Deputy Director, Gastroenterology and Inborn Error Products, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD, US

Dianne Murphy, Director, Office of Pediatric Therapeutics, Office of the Commissioner, US Food and Drug Administration, Silver Spring, MD, US

Julia Dunne, Vigilance and Risk Management in Medicines (VRMM), Medicines and Healthcare Products Regulatory Agency, London, UK

Lisa L. Mathis, Executive Director for Global Regulatory Affairs and Safety (GRAAS), Amgen Incorporated, Thousand Oaks, California, US

Note: Product cover images may vary from those shown
5 of 4
Note: Product cover images may vary from those shown
Adroll
adroll