Pediatric Drug Development: Concepts and Applications (v. 1)

✍ Scribed by Andrew E. Mulberg, Steven A. Silber, John N. van den Anker

• Publisher: Wiley-Blackwell
• Year: 2009
• Tongue: English
• Leaves: 705
• Edition: 1
• Category: Library

✦ Synopsis

Pediatric Drug Development: Concepts and Applications is designed as a reference and textbook and is meant to address the science of differences between the pediatric and adult subject in the development of pharmaceutical products. Considered are the ethics and medical needs of proper understanding the pediatric and adult differences, the business case for proper development of drugs for children, as well as the technical feasibility studies and processes that are necessary for a proper pediatric drug development program. The applications of these approaches will benefit all stakeholders and ultimately not only educate but also provide better and safer drugs for pediatric patients.

✦ Table of Contents

PEDIATRIC DRUG DEVELOPMENT......Page 5
CONTENTS......Page 9
PREFACE......Page 15
CONTRIBUTORS......Page 17
PART I PAST, PRESENT, AND FUTURE OF PEDIATRIC DRUG DEVELOPMENT......Page 23
1 A New Model for Children......Page 25
2 History of Pediatric Drug Development and Therapeutics......Page 29
3 Perspectives on Pediatric Clinical Trials: The Good, the Bad, and the Ugly......Page 33
4 Population Dynamics, Demographics, and Disease Burden of Infants and Children Across the World......Page 43
5 Pharmaceutical Economics and Applications to Pediatrics: Business Case Development......Page 61
6 Pediatric Market Dynamics......Page 81
7 Industry Benchmarks in Pediatric Clinical Trials......Page 87
8 Novel Organizational Strategies for Advancing Pediatric Products: Business Case Development......Page 91
PART II ETHICAL UNDERPINNINGS......Page 101
9 Additional Protections for Children Enrolled in Clinical Investigations......Page 103
10 Ethical Issues in Neonatal Drug Development......Page 125
11 Ethical Principles of Pediatric Research and Drug Development: A Guide Through National and International Frameworks and Applications to a Worldwide Perspective......Page 137
PART III REGULATORY GUIDELINES FOR PEDIATRIC DRUG DEVELOPMENT: STIMULATION OF PEDIATRIC DRUG RESEARCH BY REGULATORY AUTHORITIES......Page 153
12 United States Perspective......Page 155
13 European Perspective......Page 159
14 Japanese Perspective......Page 175
15 Regulatory Considerations for Study of Generic Drugs Under Best Pharmaceuticals for Children Act: NICHD and FDA Collaboration......Page 187
PART IV PRECLINICAL SAFETY ASSESSMENT......Page 195
16 Introduction and Overview......Page 197
17 Preclinical Juvenile Toxicity Assessments and Study Designs......Page 203
18 Absorption, Distribution, Metabolism, and Excretion (ADME) and Pharmacokinetic Assessments in Juvenile Animals......Page 223
PART V PHARMACOLOGICAL PRINCIPLES IN PEDIATRIC DRUG DEVELOPMENT......Page 245
19 Pediatric Clinical Pharmacology: Why, Where, How, When?......Page 247
20 Developmental Pharmacology Issues: Neonates, Infants, and Children......Page 253
21 Developmental Hepatic Pharmacology in Pediatrics......Page 265
22 Applications of Pharmacogenomics to Study Design in Pediatrics......Page 281
23 General Principles of Population Pharmacokinetics in Pediatrics......Page 299
24 Development of Oncology Drugs for Children......Page 307
PART VI CLINICAL TRIAL OPERATIONS: UNDERSTANDING DIFFERENCES BETWEEN PEDIATRIC AND ADULT STUDY SUBJECTS—DEVELOPMENT ISSUES RELATED TO ORGAN DEVELOPMENT AND ENDPOINT CHOICES......Page 321
25 Brain and Central Nervous System Development: Physiological Considerations for Assessment of Long-Term Safety......Page 323
26 Cognitive Development Considerations for Long-Term Safety Exposures in Children......Page 335
27 Cardiovascular and QTc Issues......Page 359
28 Renal Function Issues......Page 373
29 Growth and Physical Maturation......Page 385
PART VII CLINICAL TRIAL OPERATIONS AND GOOD CLINICAL TRIALS......Page 407
30 The Consent Process in Pediatric Clinical Trials......Page 409
31 Recruitment and Retention in Pediatric Clinical Trials......Page 427
32 Certification of Pediatric Clinical Investigators......Page 439
33 Considerations and Barriers for Pediatric Patient Recruitment Strategy for Clinical Trials......Page 441
34 Conducting Clinical Trials in Developing Countries: A Case Study......Page 453
35 The Importance of Geographic Differences in Pediatric Clinical Trials......Page 459
36 Partnering with Industry: Academic Medical Centers and Clinical Research Centers......Page 473
PART VIII CLINICAL EFFICACY AND SAFETY ENDPOINTS......Page 487
37 Laboratory Monitoring of Efficacy and Safety Parameters in Clinical Trials for Pediatric Subjects......Page 489
38 Breath Testing in Pediatrics......Page 503
39 Surrogate Endpoints: Application in Pediatric Clinical Trials......Page 523
40 Patient-Reported Outcomes in Pediatric Drug Development......Page 535
41 Safety Monitoring in Pediatric Clinical Trials......Page 547
42 Pharmacovigilance: Assessing Safety Postapproval......Page 565
PART IX FORMULATION, CHEMISTRY, AND MANUFACTURING CONTROLS......Page 573
43 Formulation of Pediatric Dosage Forms......Page 575
44 Drug Delivery Challenges for the Pediatric Patient: Novel Forms for Consideration......Page 589
45 Oral Drug Delivery Challenges for the Pediatric Patient......Page 595
46 The Jelly Bean Test: A Novel Technique to Help Children Swallow Medications......Page 603
PART X CASE STUDIES: SUCCESSES FOR CHILDREN......Page 609
47 A Case Study of Psychiatric Research in Children: The Risperdal(®) Example......Page 611
48 Topiramate Case Study......Page 621
49 Ascent Pediatrics: A Model for Successful Pediatric Drug Development......Page 627
50 Case Study for the Development of an Enteric-Coated and High-Buffered Pancreatic Enzyme Product......Page 633
APPENDIX Reference Tables and Figures......Page 647
INDEX......Page 679