Introduction to the Pediatric PPK (PD) Study Design Template and Analyses of the FDA Pediatric Database Clinical Pharmacology Subcommittee Advisory Committee for Pharmaceutical Sciences April 22-23, 2003 Rockville, Maryland

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Introduction to the Pediatric PPK (PD) Study
Design Template and Analyses of the FDA Pediatric
DatabaseClinical Pharmacology
SubcommitteeAdvisory Committee for
Pharmaceutical SciencesApril 22-23,
2003Rockville, Maryland

• Lawrence J. Lesko, Ph.D.
• Director, Office of Clinical Pharmacology and
  Biopharmaceutics
• Center for Drug Evaluation and Research
• Food and Drug Administration

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Pediatric Rule or Best Pharmaceuticals for
Children Act

• Use adult clinical data to draw conclusions about
  the efficacy, safety and dosing of drugs in
  pediatric patients
• avoids large scale pediatric clinical trials
• basis to decide what studies to conduct
• expedites access to drugs for children
• cost-effective
• generally successful in meeting goals

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Some Questions Always Need Assessment

• Is it reasonable to assume a similar PK-PD
  relationship as adults
• need to develop standard methods for specific
  drugs and drug classes
• What is the appropriate dose?
• rely on PK studies
• full exposure profiles or sparse samples
• dosing intended to achieve exposure similar to
  adults
• standardized PPK study design template would be
  useful (Dr. Peter Lee)

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What Can Be Learned From the Studies Conducted
Under the Pediatric Rule?

• Evaluation of the FDA pediatric database
• age groups, PK data, elimination pathways,
  clinical endpoints
• Presentation of research objectives to generate
  knowledge from database
• look at the underlying mechanisms (age-related)
  where exposure differences exist between children
  and adults
• goal is to improve or revise the pediatric
  decision tree to better identify studies need in
  children
• Dr. Gene Williams