Application of the 1998 Pediatric Rule to Oncology

1
Application of the 1998 Pediatric Rule to Oncology

• Steven Hirschfeld, MD PhD
• CDR U.S. Public Health Service
• Medical Officer
• Division of Oncology Drug Products
• Center for Drug Evaluation and Research
• Food and Drug Administration

2
Brief History

• Pediatric therapeutic development has never been
  as thorough or robust as adult therapeutic
  development
• Many therapies are administered to children
  without adequate study
• Many therapies are not made available for
  pediatric study until after adult marketing
  studies are completed

3
Pathways to Development

• Conventional
• Pre-clinical ---gt Adult - - - gt Pediatric
• Possible
• Pre-clinical ---gt Adult Pediatric
• Pre-clinical ---gt Pediatric - - -gt Adult

4
FDA Pediatric Initiatives

• 1994 Rule established principle of extrapolation
  of efficacy data from adult population to
  pediatric population if certain conditions were
  met
• 1997 Exclusivity Extension in FDAMA

5
1998 Pediatric Rule

• Mandates pediatric studies if the indication for
  an application under review can be found in
  children
• Applies to drugs and biologicals
• If the indication does not apply to children,
  then a waiver can be granted
• Does not specifically address extrapolation of
  efficacy- raises issue of Are studies
  warranted?
• Does not delay development for adult indication

6
Question for committee

• How should the 1998 Rule be applied for solid
  tumors and central nervous system malignancies?

7
Goals

• Recommendations for adult indications that should
  trigger the Pediatric Rule
• Recommendations for adult indications that should
  be waived from compliance
• Recommendations for general principles that may
  used to apply the Pediatric Rule

8
Example of a principle

• If a lesion is necessary for establishing or
  maintaining the malignant phenotype and if a
  therapy is directed against that lesion, then
  studies in tumors where the lesion occurs and has
  the same critical role are warranted