Description

Book Synopsis

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 globa

Table of Contents

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

Pediatric Drug Development

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    A Hardback by Andrew E. Mulberg, Dianne Murphy, Julia Dunne

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      View other formats and editions of Pediatric Drug Development by Andrew E. Mulberg

      Publisher: John Wiley and Sons Ltd
      Publication Date: 09/08/2013
      ISBN13: 9781118312155, 978-1118312155
      ISBN10: 1118312155
      Also in:
      Medical specialties, branches of medicine Paediatric medicine

      Description

      Book Synopsis

      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 globa

      Table of Contents

      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

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