Pediatric Drug Development
Wiley-Blackwell (Verlag)
978-1-118-31215-5 (ISBN)
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.
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
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 Children
Andrew E. Mulberg, Lisa Mathis, Julia Dunne and Dianne Murphy
2 History of Children and the Development of Regulations at the FDA
Robert M. Ward and Steven Hirschfeld
3 Population Dynamics, Demographics, and Disease Burden of Infants and Children Across the World
Ashley Malins and Christopher-Paul Milne
4 Pharmaceutical Economics and Market Access for Pediatric Medications
Christopher-Paul Milne and Ashley Malins
5 The Global Pediatric Market and Drivers of Pediatric Drug Development
E. Michael D. Scott
6 Industry Benchmarks in Pediatric Clinical Trials
Carolyn A. Campen and Cindy Levy-Petelinkar
Part II: ETHICAL UNDERPINNINGS
7 Ethical and practical considerations in conducting neonatal research
Michelle Roth-Cline and Robert M. Nelson
8 Ethical considerations in conducting pediatric research
Michelle Roth-Cline and Robert M. Nelson
9 The Consent and Assent Process in Pediatric Clinical Trials
M. Renee Simar
Part III: REGULATORY GUIDELINES FOR PEDIATRIC DRUG DEVELOPMENT
10 A Sponsor’s Perspective of US Regulations
Samuel Maldonado
11 FDA experience of extrapolation of efficacy to the pediatric population from adult and other data
Julia 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 pediatrics
Therese Cvetkovich
13 Rare Diseases and Orphan Drugs
Anne R. Pariser and Lynne P. Yao
14 European Perspective
Agnès Saint-Raymond
15 Five years of pediatric legislation in the European Union
Agnès Saint-Raymond and Ralf Herold
16 Japanese Perspective
Hidefumi Nakamura and Shunsuke Ono
17 Pediatric Device Development in the United States
Priya Venkataraman-Rao and Joy Samuels-Reid
Part IV: PRECLINICAL SAFETY ASSESSMENT
18 Introduction and Overview
Timothy P. Coogan and Melissa S. Tassinari
19 Juvenile Animal Toxicity Assessments: Decision Strategies and Study Design
Luc 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 Animals
Loeckie L. de Zwart, Johan G. Monbaliu and Pieter P. Annaert
21 A Global Regulatory Perspective
Karen Davis-Bruno, Jacqueline Carleer, Beatriz Silva Lima and Melissa S. Tassinari
22 Disease specific models to enhance Pediatric drug development
Niraj 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 Forward
Gilbert J. Burckart and Jeremiah Momper
24 Development and Clinical Trial Design
Kathleen A. Neville, Ralph E. Kauffman, and Susan M. Abdel-Rahman
25 Developmental Hepatic Pharmacology in Pediatrics
Martin Otto Behm
26 Applications of Population Pharmacokinetics for Pediatric Drug Development
Jeremiah Momper, Gilbert J. Burckart and Pravin Jadhav
27 Applications of Pharmacogenomics to Pediatric Drug Development
Gilbert J. Burckart, Dionna Green and Padmaja Mummaneni
28 Pharmacometrics Applications to Pediatric Trials
Devin 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 Safety
Kachikwu Illoh
30 Cognitive Development Considerations for Long-Term Safety Exposures in Children
Mary Pipan, Paul Wang, and Rebecca Thompson-Penna
31 Cardiovascular and QTc Issues
Bert Suys and Luc Dekie
32 Pediatric bone and adult bone – physiological differences
Francisco A. Sylvester and Erica L. Wynn
33 Renal Function Issues
Katia Boven
34 Growth and Physical Development
Alisha J. Rovner and Babette S. Zemel
35 Development of Drugs for Pediatric Cancers
Kristen 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 Trials
Benjamin Ortiz and Sergio Guerrero
38 Conducting Clinical Trials in Developing and Emerging Countries: Review and Case Study
Kevin D. Hill and Jennifer S. Li
39 The Importance of Geographic Differences in Pediatric Clinical Trials
Alexandar Cvetkovich Muntañola
Part VIII: CLINICAL EFFICACY AND SAFETY ENDPOINTS
40 Clinical Laboratory Testing in Clinical Trials for Pediatric Subjects
Andrew E. Mulberg and Ethan D. Hausman
41 Surrogate Endpoints: Application in Pediatric Clinical Trials
Geert Molenberghs
42 Clinical Outcome Assessments for Clinical Trials in Children
Elektra 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 Studies
Ann W. McMahon, Chris Feudtner and Dianne Murphy
44 Endpoints in Rare Diseases
Anne R. Pariser and Lynne P. Yao
Part IX: FORMULATION, CHEMISTRY, AND MANUFACTURING CONTROLS
45 Formulation, Chemistry, and Manufacturing Controls
Gerard P. McNally and Aniruddha M. Railkar
46 Drug Delivery Challenges for the Pediatric Patient: Novel Forms for Consideration
Ann Zajicek
47 The Jelly Bean Test: A Novel Technique To Help Children Swallow Medications
Robbyn E. Sockolow and Aliza B. Solomon
Index
| Erscheint lt. Verlag | 19.8.2013 |
|---|---|
| Verlagsort | Hoboken |
| Sprache | englisch |
| Maße | 196 x 254 mm |
| Gewicht | 1184 g |
| Themenwelt | Medizin / Pharmazie ► Medizinische Fachgebiete ► Neurologie |
| Medizin / Pharmazie ► Medizinische Fachgebiete ► Pädiatrie | |
| Medizin / Pharmazie ► Medizinische Fachgebiete ► Pharmakologie / Pharmakotherapie | |
| Medizin / Pharmazie ► Pflege | |
| Technik | |
| ISBN-10 | 1-118-31215-5 / 1118312155 |
| ISBN-13 | 978-1-118-31215-5 / 9781118312155 |
| Zustand | Neuware |
| Haben Sie eine Frage zum Produkt? |
aus dem Bereich
