Pediatric Drug Development: Concepts and Applications Book

Past, Present and Future of Pediatric Drug Development.

1. Introduction: Need for a New Model for Children (Mulberg, Silber, and van den anker).

2. History of Pediatric Drug Development and Therapeutics-(Stephen Spielberg).

3. Perspectives on Pediatric Clinical Trials: The Good, the Bad, and the Ugly (Greg Koski).

4. Population Dynamics and Demographics of Infants and Children Across the World (Milne).

5. Pharmaceutical Economics and Applications to Pediatric Business Case Development (Milne).

6. Pediatric Drug Strategy and Development: Considerations for the Pediatric Marketplace (Clothier).

7. Industry Benchmarks in Pediatric Clinical Trials (Petelinkar-Levy, and Campen).

8. Novel organizational strategies for advancing pediatric products (Lombardi).

Ethical Underpinnings:.

9. Additional Protections for Children Enrolled in Clinical Investigations (Nelson).

10. Additional Protections for Pregnant Women, Fetus and Neonates (Baer).

11. Ethical Principles of Pediatric Research and Drug Development - A Guide Through National and International Frameworks and Applications to a Worldwide Perspective (Rose).

Past, Present and Future Regulatory Guidances for Pediatric Drug Development: Stimulation of Pediatric Drug Research by Regulatory Authorities.

12. US perspective (Maldonado).

13. Europe perspective (Rose).

14. Japan perspective (Nakamura).

15. Regulatory Considerations for Study of Generic Drugs under Best Pharmaceuticals for Children Act:NICHD and FDA Collaboration (Goldstein and Colleagues).

Preclinical toxicology: Overview and Introduction (Coogan).

16. Preclinical Juvenile Toxicity Assessments and Study Designs (Luc M. De Schaepdrijver).

17. Absorption, Distribution, Metabolism and Excretion (ADME) and Pharmacokinetic Assessments in Juvenile Animals: (Loeckie L. de Zwart).

Pharmacological principles in Pediatric Drug development.

18. Pediatric Clinical Pharmacology: Why, Where, How, When? (van den Anker).

19. Developmental Pharmacology Issues: Neonates, Infants and Children (van den Anker).

20. Developmental Hepatic Pharmacology in Pediatrics (Behm).

21. Applications of Pharmacogenomics to Study design in Pediatrics (Cohen, and Ness).

22. General Principles of Population Pharmacokinetics in Pediatrics (samtani, and Kimko).

23. Development of Oncology Drugs for Children (Rackoff, Adamson).

Clinical Trial Operations: Understanding Differences between Pediatric and Adult Study Subjects: Developmental Issues Related to Organ Development and Endpoint Choice.

24. CNS and Brain: Physiological Maturation: (Schneider).

25. Cognitive developmental considerations for long term exposures (Pipan).

26. Cardiovascular and QTc Issues (Suys, and Dekie).

27. Kidney (Boven).

28. Growth and Physical Maturation (Rovner, and Zemel).

Clinical Trial Operations and Good Clinical Practices (GCP):.

29. The Consent Process in Pediatric Clinical Trials (Simar).

30. Recruitment and retention in Pediatric Clinical trials (Simar).

31. Pediatric Good Clinical Practices (GCP) (Procaccino).

32. Considerations and Barriers to Pediatric Patient Recruitment Strategy for Clinical Trials (Niesz).

33. Conducting Clinical Trials in Developing Countries: A Case Study (Friedland, and Miller).

34. Global Clinical trials and Pediatric recruitment (Muntanola).

35. Collaborations with Academia and Clincal Research Centers (CRCs) (Goldstein).

Clinical Efficacy and Safety Endpoints.

36. Laboratory Monitoring of Efficacy and Safety Parameters (Mulberg, and Maldonado).

37. Breath Testing in Pediatrics (Veeremans).

38. Surrogate Markers (Orman).

39. Patient Related Outcomes (PROs) (Kleinman, and Rothman).

40. Safety Monitoring of Pediatric Clinical Trials (Hetherington).

41. Post-Marketing Safety Surveillance (Kaplan).

Formulation/Chemistry/Manufacturing Controls.

42. Liquids, Suspensions, Tablets: (McNally, Railkar).

43. Drug Delivery Challenges for the Pediatric Patient: Novel forms for Consideration (Roche).

44. Drug Delivery Challenges for the Pediatric Patient: Oral Forms for Consideration (Hoy).

45. The Jelly Bean Test: A Novel Technique To Help Children Swallow Medications (Sockolow).

Case Studies: Successes for Children.

46. A Case Study of Psychiatric Research in Children—The Risperdal Example (Haas).

47. Topamax (Ness).

48. Ascent Pediatrics (Clement).

49. DCI Pancrecarb (Sipos, Humphries).

Appendices (culled from various chapters for listing).

1. Relevant Code of Federal Regulations and FDA Guidances for Conduct in Pediatric Clinical Trials.

a. Application of General IRB Regulations to Pediatric Research .

b. FDA Algorithm for Extrapolation.

c. Algorithm for Protocol Assessment under 21 CFR 50, Subpart D .

2. Paradigm for Recruitment and Retention in Pediatric Clinical Trials.

3. Normal Blood Values.

a. Neonatal, Pediatric, and Adult Populations.

b. Hematologic Values in Children and Adults.

c. Serum and Plasma Chemistry Values in Children and Adults.

4. Values of mean (range) eGFR for infants and children of various ages using classic clearance techniques..

5. Examples of substances secreted or reabsorbed by active mechanisms in the proximal tubule.

6. Local and national neonatal and pediatric Clinical Research Networks (IECRN).

7. Ontogeny of drug metabolizing enzymes in liver of several animal species.

8. Summary of Developmental Changes in Drug Disposition in Human Infant and Child.

9. Ontogeny of Oran transporters in Animals.

10. Developmental events in Animals.

11. Dosing routes in neonatal / juvenile animal species.

12. Reflex ontogeny and behavioural testing battery in Nonclinical Testing of Animals.

13. Commonly used QT correction formulae.

14. Tanner Staging.

a. boys.

b. girls.

15. Median Ages in Years at Entry into Tanner Stage in Girls.

16. Median Ages in Years at Entry into Tanner Stage in Boys.

17. Recommended Reference Data for the Assessment of Growth and Nutritional Status.

Index.