Andrew E. (Division Deputy Director, Gastroenterology and Inborn Error Products, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD, USA) Mulberg
Meer over de auteurs
Andrew E. (Division Deputy Director, Gastroenterology and Inborn Error Products, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD, USA) Mulberg,
AE Mulberg,
Dianne (Director, Office of Pediatric Therapeutics, Office of the Commissioner, US Food and Drug Administration, Silver Spring, MD, USA) Murphy,
Julia (Vigilance and Risk Management in Medicines (VRMM), Medicines and Healthcare Products Regulatory Agency, London, UK) Dunne,
Lisa L. (Executive Director for Global Regulatory Affairs and Safety (GRAAS), Amgen Incorporated, Thousand Oaks, CA, USA) Mathis
e.a.
John Wiley & Sons
2e druk, 2013
9781118312155
Pediatric Drug Development Concepts and Applications 2e
Specificaties
Gebonden, 624 blz.
|
Engels
John Wiley & Sons |
2e druk, 2013
ISBN13: 9781118312155
Rubricering
Verwachte levertijd ongeveer 9 werkdagen
Samenvatting
Pediatric Drug Development, Second Edition, encompasses the new regulatory initiatives across EU, US and ROW designed to encourage improved access to safe and effective medicines for children. It includes new developments in biomarkers and surrogate endpoints, developmental pharmacology and other novel aspects of pediatric drug development.
Specificaties
ISBN13:9781118312155
Taal:Engels
Bindwijze:gebonden
Aantal pagina's:624
Uitgever:John Wiley & Sons
Druk:2
Inhoudsopgave
<p>List of Contributors</p>
<p>Preface</p>
<p>Part I: PAST, PRESENT, AND FUTURE OF PEDIATRIC DRUG DEVELOPMENT<br /> <br /> 1 Pediatric Drug Development and Therapeutics: Continued Progress for Better Drugs for Children<br /> Andrew E. Mulberg, Lisa Mathis, Julia Dunne and Dianne Murphy</p>
<p>2 History of Children and the Development of Regulations at the FDA<br /> Robert M. Ward and Steven Hirschfeld</p>
<p>3 Population Dynamics, Demographics, and Disease Burden of Infants and Children Across the World<br /> Ashley Malins and Christopher–Paul Milne</p>
<p>4 Pharmaceutical Economics and Market Access for Pediatric Medications<br /> Christopher–Paul Milne and Ashley Malins</p>
<p>5 The Global Pediatric Market and Drivers of Pediatric Drug Development<br /> E. Michael D. Scott</p>
<p>6 Industry Benchmarks in Pediatric Clinical Trials<br /> Carolyn A. Campen and Cindy Levy–Petelinkar</p>
<p>Part II: ETHICAL UNDERPINNINGS<br /> <br /> 7 Ethical and practical considerations in conducting neonatal research<br /> Michelle Roth–Cline and Robert M. Nelson</p>
<p>8 Ethical considerations in conducting pediatric research <br /> Michelle Roth–Cline and Robert M. Nelson</p>
<p>9 The Consent and Assent Process in Pediatric Clinical Trials<br /> M. Renee Simar</p>
<p>Part III: REGULATORY GUIDELINES FOR PEDIATRIC DRUG DEVELOPMENT<br /> <br /> 10 A Sponsor s Perspective of US Regulations<br /> Samuel Maldonado</p>
<p>11 FDA experience of extrapolation of efficacy to the pediatric population from adult and other data<br /> Julia Dunne, William J. Rodriguez and Dianne Murphy</p>
<p>12 Accelerated Approval and other regulatory approaches to the development of drugs for serious or life–threatening diseases in pediatrics<br /> Therese Cvetkovich</p>
<p>13 Rare Diseases and Orphan Drugs<br /> Anne R. Pariser and Lynne P. Yao</p>
<p>14 European Perspective<br /> Agnès Saint–Raymond</p>
<p>15 Five years of pediatric legislation in the European Union<br /> Agnès Saint–Raymond and Ralf Herold</p>
<p>16 Japanese Perspective<br /> Hidefumi Nakamura and Shunsuke Ono</p>
<p>17 Pediatric Device Development in the United States<br /> Priya Venkataraman–Rao and Joy Samuels–Reid</p>
<p>Part IV: PRECLINICAL SAFETY ASSESSMENT<br /> <br /> 18 Introduction and Overview<br /> Timothy P. Coogan and Melissa S. Tassinari</p>
<p>19 Juvenile Animal Toxicity Assessments: Decision Strategies and Study Design<br /> Luc M. De Schaepdrijver, Graham P. Bailey, Timothy P. Coogan and Jennifer L. Ingram–Ross</p>
<p>20 Absorption, Distribution, Metabolism and Excretion (ADME) and Pharmacokinetic Assessments in Juvenile Animals<br /> Loeckie L. de Zwart, Johan G. Monbaliu and Pieter P. Annaert</p>
<p>21 A Global Regulatory Perspective<br /> Karen Davis–Bruno, Jacqueline Carleer, Beatriz Silva Lima and Melissa S. Tassinari</p>
<p>22 Disease specific models to enhance Pediatric drug development<br /> Niraj R. Mehta and Sruthi King</p>
<p>Part V: PHARMACOLOGICAL PRINCIPLES IN PEDIATRIC DRUG DEVELOPMENT<br /> <br /> 23 Pediatric Clinical Pharmacology in Regulatory and Drug Development Sciences: Lessons learned and the Path Forward<br /> Gilbert J. Burckart and Jeremiah Momper</p>
<p>24 Development and Clinical Trial Design<br /> Kathleen A. Neville, Ralph E. Kauffman, and Susan M. Abdel–Rahman</p>
<p>25 Developmental Hepatic Pharmacology in Pediatrics<br /> Martin Otto Behm</p>
<p>26 Applications of Population Pharmacokinetics for Pediatric Drug Development<br /> Jeremiah Momper, Gilbert J. Burckart and Pravin Jadhav</p>
<p>27 Applications of Pharmacogenomics to Pediatric Drug Development <br /> Gilbert J. Burckart, Dionna Green and Padmaja Mummaneni</p>
<p>28 Pharmacometrics Applications to Pediatric Trials<br /> Devin Pastoor, Mallika Lala, Jogarao V.S. Gobburu</p>
<p>Part VI: CLINICAL TRIAL OPERATIONS<br /> <br /> 29 Brain and Central Nervous System Development: Physiological Considerations for Assessment of Long–Term Safety<br /> Kachikwu Illoh</p>
<p>30 Cognitive Development Considerations for Long–Term Safety Exposures in Children<br /> Mary Pipan, Paul Wang, and Rebecca Thompson–Penna</p>
<p>31 Cardiovascular and QTc Issues<br /> Bert Suys and Luc Dekie</p>
<p>32 Pediatric bone and adult bone physiological differences<br /> Francisco A. Sylvester and Erica L. Wynn</p>
<p>33 Renal Function Issues<br /> Katia Boven</p>
<p>34 Growth and Physical Development<br /> Alisha J. Rovner and Babette S. Zemel</p>
<p>35 Development of Drugs for Pediatric Cancers<br /> Kristen M. Snyder, Martha Donoghue, Whitney S. Helms, and Gregory Reaman</p>
<p>Part VII: CLINICAL TRIAL OPERATIONS AND GOOD CLINICAL TRIALS<br /> <br /> 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</p>
<p>37 Recruitment and Retention of Minority populations in Clinical Trials<br /> Benjamin Ortiz and Sergio Guerrero</p>
<p>38 Conducting Clinical Trials in Developing and Emerging Countries: Review and Case Study<br /> Kevin D. Hill and Jennifer S. Li</p>
<p>39 The Importance of Geographic Differences in Pediatric Clinical Trials<br /> Alexandar Cvetkovich Muntañola</p>
<p>Part VIII: CLINICAL EFFICACY AND SAFETY ENDPOINTS<br /> <br /> 40 Clinical Laboratory Testing in Clinical Trials for Pediatric Subjects<br /> Andrew E. Mulberg and Ethan D. Hausman<br /> <br /> 41 Surrogate Endpoints: Application in Pediatric Clinical Trials<br /> Geert Molenberghs</p>
<p>42 Clinical Outcome Assessments for Clinical Trials in Children<br /> Elektra J. Papadopoulos, Donald L. Patrick, Melissa S. Tassinari, Andrew E. Mulberg, Carla Epps, Anne R. Pariser and Laurie B. Burke</p>
<p>43 Challenges in Evaluating the Safety of Pediatric Therapeutic Agents in Trials and Post–Marketing Studies<br /> Ann W. McMahon, Chris Feudtner and Dianne Murphy</p>
<p>44 Endpoints in Rare Diseases<br /> Anne R. Pariser and Lynne P. Yao</p>
<p>Part IX: FORMULATION, CHEMISTRY, AND MANUFACTURING CONTROLS<br /> <br /> 45 Formulation, Chemistry, and Manufacturing Controls<br /> Gerard P. McNally and Aniruddha M. Railkar</p>
<p>46 Drug Delivery Challenges for the Pediatric Patient: Novel Forms for Consideration<br /> Ann Zajicek<br /> <br /> 47 The Jelly Bean Test: A Novel Technique To Help Children Swallow Medications<br /> Robbyn E. Sockolow and Aliza B. Solomon</p>
<p>Index</p>
<p>Preface</p>
<p>Part I: PAST, PRESENT, AND FUTURE OF PEDIATRIC DRUG DEVELOPMENT<br /> <br /> 1 Pediatric Drug Development and Therapeutics: Continued Progress for Better Drugs for Children<br /> Andrew E. Mulberg, Lisa Mathis, Julia Dunne and Dianne Murphy</p>
<p>2 History of Children and the Development of Regulations at the FDA<br /> Robert M. Ward and Steven Hirschfeld</p>
<p>3 Population Dynamics, Demographics, and Disease Burden of Infants and Children Across the World<br /> Ashley Malins and Christopher–Paul Milne</p>
<p>4 Pharmaceutical Economics and Market Access for Pediatric Medications<br /> Christopher–Paul Milne and Ashley Malins</p>
<p>5 The Global Pediatric Market and Drivers of Pediatric Drug Development<br /> E. Michael D. Scott</p>
<p>6 Industry Benchmarks in Pediatric Clinical Trials<br /> Carolyn A. Campen and Cindy Levy–Petelinkar</p>
<p>Part II: ETHICAL UNDERPINNINGS<br /> <br /> 7 Ethical and practical considerations in conducting neonatal research<br /> Michelle Roth–Cline and Robert M. Nelson</p>
<p>8 Ethical considerations in conducting pediatric research <br /> Michelle Roth–Cline and Robert M. Nelson</p>
<p>9 The Consent and Assent Process in Pediatric Clinical Trials<br /> M. Renee Simar</p>
<p>Part III: REGULATORY GUIDELINES FOR PEDIATRIC DRUG DEVELOPMENT<br /> <br /> 10 A Sponsor s Perspective of US Regulations<br /> Samuel Maldonado</p>
<p>11 FDA experience of extrapolation of efficacy to the pediatric population from adult and other data<br /> Julia Dunne, William J. Rodriguez and Dianne Murphy</p>
<p>12 Accelerated Approval and other regulatory approaches to the development of drugs for serious or life–threatening diseases in pediatrics<br /> Therese Cvetkovich</p>
<p>13 Rare Diseases and Orphan Drugs<br /> Anne R. Pariser and Lynne P. Yao</p>
<p>14 European Perspective<br /> Agnès Saint–Raymond</p>
<p>15 Five years of pediatric legislation in the European Union<br /> Agnès Saint–Raymond and Ralf Herold</p>
<p>16 Japanese Perspective<br /> Hidefumi Nakamura and Shunsuke Ono</p>
<p>17 Pediatric Device Development in the United States<br /> Priya Venkataraman–Rao and Joy Samuels–Reid</p>
<p>Part IV: PRECLINICAL SAFETY ASSESSMENT<br /> <br /> 18 Introduction and Overview<br /> Timothy P. Coogan and Melissa S. Tassinari</p>
<p>19 Juvenile Animal Toxicity Assessments: Decision Strategies and Study Design<br /> Luc M. De Schaepdrijver, Graham P. Bailey, Timothy P. Coogan and Jennifer L. Ingram–Ross</p>
<p>20 Absorption, Distribution, Metabolism and Excretion (ADME) and Pharmacokinetic Assessments in Juvenile Animals<br /> Loeckie L. de Zwart, Johan G. Monbaliu and Pieter P. Annaert</p>
<p>21 A Global Regulatory Perspective<br /> Karen Davis–Bruno, Jacqueline Carleer, Beatriz Silva Lima and Melissa S. Tassinari</p>
<p>22 Disease specific models to enhance Pediatric drug development<br /> Niraj R. Mehta and Sruthi King</p>
<p>Part V: PHARMACOLOGICAL PRINCIPLES IN PEDIATRIC DRUG DEVELOPMENT<br /> <br /> 23 Pediatric Clinical Pharmacology in Regulatory and Drug Development Sciences: Lessons learned and the Path Forward<br /> Gilbert J. Burckart and Jeremiah Momper</p>
<p>24 Development and Clinical Trial Design<br /> Kathleen A. Neville, Ralph E. Kauffman, and Susan M. Abdel–Rahman</p>
<p>25 Developmental Hepatic Pharmacology in Pediatrics<br /> Martin Otto Behm</p>
<p>26 Applications of Population Pharmacokinetics for Pediatric Drug Development<br /> Jeremiah Momper, Gilbert J. Burckart and Pravin Jadhav</p>
<p>27 Applications of Pharmacogenomics to Pediatric Drug Development <br /> Gilbert J. Burckart, Dionna Green and Padmaja Mummaneni</p>
<p>28 Pharmacometrics Applications to Pediatric Trials<br /> Devin Pastoor, Mallika Lala, Jogarao V.S. Gobburu</p>
<p>Part VI: CLINICAL TRIAL OPERATIONS<br /> <br /> 29 Brain and Central Nervous System Development: Physiological Considerations for Assessment of Long–Term Safety<br /> Kachikwu Illoh</p>
<p>30 Cognitive Development Considerations for Long–Term Safety Exposures in Children<br /> Mary Pipan, Paul Wang, and Rebecca Thompson–Penna</p>
<p>31 Cardiovascular and QTc Issues<br /> Bert Suys and Luc Dekie</p>
<p>32 Pediatric bone and adult bone physiological differences<br /> Francisco A. Sylvester and Erica L. Wynn</p>
<p>33 Renal Function Issues<br /> Katia Boven</p>
<p>34 Growth and Physical Development<br /> Alisha J. Rovner and Babette S. Zemel</p>
<p>35 Development of Drugs for Pediatric Cancers<br /> Kristen M. Snyder, Martha Donoghue, Whitney S. Helms, and Gregory Reaman</p>
<p>Part VII: CLINICAL TRIAL OPERATIONS AND GOOD CLINICAL TRIALS<br /> <br /> 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</p>
<p>37 Recruitment and Retention of Minority populations in Clinical Trials<br /> Benjamin Ortiz and Sergio Guerrero</p>
<p>38 Conducting Clinical Trials in Developing and Emerging Countries: Review and Case Study<br /> Kevin D. Hill and Jennifer S. Li</p>
<p>39 The Importance of Geographic Differences in Pediatric Clinical Trials<br /> Alexandar Cvetkovich Muntañola</p>
<p>Part VIII: CLINICAL EFFICACY AND SAFETY ENDPOINTS<br /> <br /> 40 Clinical Laboratory Testing in Clinical Trials for Pediatric Subjects<br /> Andrew E. Mulberg and Ethan D. Hausman<br /> <br /> 41 Surrogate Endpoints: Application in Pediatric Clinical Trials<br /> Geert Molenberghs</p>
<p>42 Clinical Outcome Assessments for Clinical Trials in Children<br /> Elektra J. Papadopoulos, Donald L. Patrick, Melissa S. Tassinari, Andrew E. Mulberg, Carla Epps, Anne R. Pariser and Laurie B. Burke</p>
<p>43 Challenges in Evaluating the Safety of Pediatric Therapeutic Agents in Trials and Post–Marketing Studies<br /> Ann W. McMahon, Chris Feudtner and Dianne Murphy</p>
<p>44 Endpoints in Rare Diseases<br /> Anne R. Pariser and Lynne P. Yao</p>
<p>Part IX: FORMULATION, CHEMISTRY, AND MANUFACTURING CONTROLS<br /> <br /> 45 Formulation, Chemistry, and Manufacturing Controls<br /> Gerard P. McNally and Aniruddha M. Railkar</p>
<p>46 Drug Delivery Challenges for the Pediatric Patient: Novel Forms for Consideration<br /> Ann Zajicek<br /> <br /> 47 The Jelly Bean Test: A Novel Technique To Help Children Swallow Medications<br /> Robbyn E. Sockolow and Aliza B. Solomon</p>
<p>Index</p>