Content:
Chapter 1 Introduction: Pediatric Drug Development and Therapeutics: Continued Progress for Better Drugs for Children (pages 3–5): Andrew E. Mulberg, Lisa L. Mathis, Julia Dunne and Dianne Murphy
Chapter 2 History of Children and the Development of Regulations at the FDA (pages 6–15): Steven Hirschfeld and Robert M. Ward
Chapter 3 Population Dynamics, Demographics and Disease Burden of Infants and Children across the World (pages 15–36): Ashley J. Malins and Christopher‐Paul Milne
Chapter 4 Pharmaceutical Economics and Market Access for Pediatric Medications (pages 37–56): Christopher‐Paul Milne and Ashley J. Malins
Chapter 5 The Global Pediatric Market and Drivers of Pediatric Drug Development (pages 57–67): E. Michael and D. Scott
Chapter 6 Industry Benchmarks in Pediatric Clinical Trials (pages 68–70): Carolyn A. Campen and Cindy Levy‐Petelinkar
Chapter 7 Ethical and Practical Considerations in Conducting Neonatal Research (pages 73–82): Michelle D. Roth‐Cline and Robert M. Nelson
Chapter 8 Ethical Considerations in Conducting Pediatric Research (pages 83–93): Michelle D. Roth‐Cline and Robert M. Nelson
Chapter 9 The Consent and Assent Process in Pediatric Clinical Trials (pages 94–104): M. Renee Simar
Chapter 10 A Sponsor's Perspective of US Regulations (pages 107–109): Samuel Maldonado
Chapter 11 FDA Experience of Extrapolation of Efficacy to the Pediatric Population from Adult and Other Data (pages 110–122): Julia Dunne, William J. Rodriguez and Dianne Murphy
Chapter 12 Accelerated Approval and Other Regulatory Approaches to Drug Development for Serious Diseases in Pediatrics (pages 123–129): Therese Cvetkovich
Chapter 13 Rare Diseases and Orphan Drugs (pages 130–148): Anne R. Pariser and Lynne P. Yao
Chapter 14 European Perspective (pages 149–155): Agnès Saint‐Raymond
Chapter 15 Five Years of Pediatric Legislation in the European Union (pages 156–169): Agnès Saint‐Raymond and Ralf Herold
Chapter 16 The Japanese Perspective (pages 170–176): Hidefumi Nakamura and Shunsuke Ono
Chapter 17 Pediatric Device Development in the United States (pages 177–191): Priya Venkataraman‐Rao and Joy H. Samuels‐Reid
Chapter 18 Preclinical Safety Assessment: Introduction and Overview (pages 195–200): Timothy P. Coogan and Melissa S. Tassinari
Chapter 19 Juvenile Animal Toxicity Assessments: Decision Strategies and Study Design (pages 201–221): Luc M. De Schaepdrijver, Graham P. Bailey, Timothy P. Coogan and Jennifer L. Ingram‐Ross
Chapter 20 Absorption, Distribution, Metabolism and Excretion (ADME) and Pharmacokinetic Assessments in Juvenile Animals (pages 222–245): Loeckie L. de Zwart, Johan G. Monbaliu and Pieter P. Annaert
Chapter 21 A Global Regulatory Perspective (pages 246–256): Karen L. Davis‐Bruno, Jacqueline Carleer, Beatriz Silva Lima and Melissa S. Tassinari
Chapter 22 Disease‐Specific Models to Enhance Pediatric Drug Development (pages 257–267): Niraj R. Mehta and Sruthi Tallapragada King
Chapter 23 Pediatric Clinical Pharmacology in Regulatory and Drug Development Sciences: Lessons Learned and the Path Forward (pages 271–280): Gilbert J. Burckart and Jeremiah D. Momper
Chapter 24 Development and Clinical Trial Design (pages 281–291): Kathleen A. Neville, Ralph E. Kauffman and Susan M. Abdel‐Rahman
Chapter 25 Developmental Hepatic Pharmacology in Pediatrics (pages 292–305): Martin Otto Behm
Chapter 26 Applications of Population Pharmacokinetics for Pediatric Drug Development (pages 306–315): Jeremiah D. Momper, Gilbert J. Burckart and Pravin Jadhav
Chapter 27 Applications of Pharmacogenomics to Pediatric Drug Development (pages 316–331): Gilbert J. Burckart, Dionna Green and Padmaja Mummaneni
Chapter 28 Pharmacometrics Applications to Pediatric Trials (pages 332–341): Devin Pastoor, Mallika Lala and Jogarao V.S. Gobburu
Chapter 29 Brain and Central Nervous System Development: Physiological Considerations for Assessment of Long‐Term Safety (pages 345–354): Kachi Illoh
Chapter 30 Cognitive Development Considerations for Long‐term Safety Exposures in Children (pages 355–382): Mary Pipan, Paul P. Wang and Rebecca Thompson Penna
Chapter 31 Cardiovascular Issues and the QT Interval (pages 383–394): Bert Suys and Luc Dekie
Chapter 32 Pediatric Bone and Adult Bone – Physiological Differences (pages 396–408): Francisco A. Sylvester and Erica L. Wynn
Chapter 33 Issues Related to Organ Development: Renal (pages 409–418): Katia Boven
Chapter 34 Growth and Physical Development (pages 419–437): Alisha J. Rovner and Babette S. Zemel
Chapter 35 Development of Drugs for Pediatric Cancers (pages 438–452): Kristen M. Snyder, Martha Donoghue, Whitney S. Helms and Gregory Reaman
Chapter 36 Recruitment and Retention in Pediatric Clinical Trials: Focus on Pediatric Research Networks in the US and EU (pages 455–471): Rosalind L. Smyth, Geetinder Kaur, Daniel K. Benjamin and Matthew M. Laughon
Chapter 37 Recruitment and Retention of Minority Populations in Clinical Trials (pages 472–476): Benjamin Ortiz and Sergio Guerrero
Chapter 38 Conducting Clinical Trials in Developing and Emerging Countries: Review and Case Study (pages 477–481): Kevin D. Hill and Jennifer S. Li
Chapter 39 Globalization of Pediatric Clinical Trials with a Focus on Emerging Countries (pages 482–513): Alexandar Cvetkovich Muntañola
Chapter 40 Clinical Laboratory Testing in Clinical Trials for Pediatric Subjects (pages 517–529): Andrew E. Mulberg and Ethan D. Hausman
Chapter 41 Surrogate Endpoints: Application in Pediatric Clinical Trials (pages 530–538): Geert Molenberghs
Chapter 42 Clinical Outcome Assessments for Clinical Trials in Children (pages 539–548): Elektra J. Papadopoulos, Donald L. Patrick, Melissa S. Tassinari, Andrew E. Mulberg, Carla Epps, Anne R. Pariser and Laurie B. Burke
Chapter 43 Challenges Evaluating the Safety of Pediatric Therapeutic Agents in Clinical Trials and Post‐Marketing Surveillance (pages 549–556): Ann W. McMahon, Chris Feudtner and Dianne Murphy
Chapter 44 Biomarkers and Endpoints in Rare Diseases (pages 557–561): Lynne P. Yao and Anne R. Pariser
Chapter 45 Formulation, Chemistry and Manufacturing Controls (pages 565–575): Gerard P. McNally and Aniruddha M. Railkar
Chapter 46 Drug Delivery Challenges for the Pediatric Patient: Novel Forms for Consideration (pages 576–582): Anne Zajicek
Chapter 47 The Jelly Bean Test: A Novel Technique to Help Children Swallow Medications (pages 583–587): Robbyn E. Sockolow and Aliza B. Solomon