19. Juvenile Animal Toxicity Assessments: Decision Strategies and Study Design

  1. Andrew E. Mulberg MD, FAAP, CPI4,
  2. Dianne Murphy MD, FAAP5,
  3. Julia Dunne MD6 and
  4. Lisa L. Mathis MD7
  1. Luc M. De Schaepdrijver1,
  2. Graham P. Bailey1,
  3. Timothy P. Coogan2 and
  4. Jennifer L. Ingram-Ross3

Published Online: 31 JUL 2013

DOI: 10.1002/9781118312087.ch19

Pediatric Drug Development: Concepts and Applications

Pediatric Drug Development: Concepts and Applications

How to Cite

De Schaepdrijver, L. M., Bailey, G. P., Coogan, T. P. and Ingram-Ross, J. L. (2013) Juvenile Animal Toxicity Assessments: Decision Strategies and Study Design, in Pediatric Drug Development: Concepts and Applications (eds A. E. Mulberg, D. Murphy, J. Dunne and L. L. Mathis), John Wiley & Sons Ltd., Chichester, UK. doi: 10.1002/9781118312087.ch19

Editor Information

  1. 4

    Division Deputy Director, Gastroenterology and Inborn Error Products, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD

  2. 5

    Director, Office of Pediatric Therapeutics, Office of the Commissioner, US Food and Drug Administration, Silver Spring, MD

  3. 6

    Group Manager, Special Populations Unit, Division of Vigilance and Risk Management of Medicines (VRRM), Medicines and Healthcare Products Regulatory Agency, London, UK

  4. 7

    Executive Director for Global Regulatory Affairs and Safety (GRAAS), Amgen Incorporated Thousand Oaks, CA

Author Information

  1. 1

    Drug Safety Sciences, Janssen Research and Development, Beerse, BE

  2. 2

    Biologics Toxicology, Janssen Research and Development LLC, Spring House, PA, USA

  3. 3

    Janssen Research & Development LLC, Raritan, NJ, USA

Publication History

  1. Published Online: 31 JUL 2013
  2. Published Print: 12 AUG 2013

ISBN Information

Print ISBN: 9781118312155

Online ISBN: 9781118312087

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Keywords:

  • biopharmaceuticals;
  • juvenile animal studies;
  • key study designs;
  • specific organ systems;
  • toxicity assessment

Summary

This chapter describes the different study types, key study designs and considerations for toxicity assessment of specific organ systems. It presents a general guide for comparative age categories between different animal species and humans with regard to central nervous system and reproductive organ development. Both the terms “pediatric” and “biopharmaceutical“ share a common element in that each term represents what can be extremely diverse “populations”. A few of the key considerations for biopharmaceuticals as they impact study design are: species selection/crossreactivity; immunogenicity of the biopharmaceutical; pharmacokinetics/toxicokinetics, distribution and elimination. The chapter also talks about the considerations needed in the testing designs for a number of specific target organs or systems. As the knowledge from juvenile animal studies grows, a better understanding of the clinical relevance of findings will be achieved.