Pediatric Research Equity Act June 9, 2004

1. Pediatric Research Equity ActJune 9, 2004 Shirley Murphy, M.D. Division Director Division of Pediatric Drug Development

2. PediatricResearchEquityAct

3. PREA • Became law December 3, 2003 • Legislation mimics Pediatric Rule • Requires pediatric studies of certain drugs & biological products unless waived or deferred • Retroactive for all applications back to April 1, 1999

4. PREA • Not applicable to drugs with orphan designation • FDA Guidance under development • Establishes Pediatric Advisory Committee

5. PREA Legislation Drugs & Biologics Meetings at appropriate times Retroactive 4/1/99 Establishes AC Rule Regulation Drugs & Biologics Meeting requirements at specific times PREA vs. Rule

6. PREA Studies are required Orphan drugs designated exempt Studies limited to drug/indication under development BPCA Studies are voluntary Includes orphan drugs and orphan drug indications Studies on whole moiety PREA vs. BPCA

7. PREA • Pediatric Assessment required for applications: • New ingredient • New indication • New dosage form • New dosing regimen • New route of administration

8. PREA Pediatric Assessment must contain: • Data adequate to assess the safety and effectiveness of the drug or biological product, and • Data to support dosing and administration for each relevant pediatric subpopulation

9. PREA Similar course of disease or effect: • Effectiveness can be extrapolated from adequate and well-controlled studies in adults, when supplemented with other information (safety, PK-PD) • Extrapolation from one age group to another age group where appropriate

10. PREA Deferral granted when: • Drug or biologic is ready for approval in adults; OR • Additional safety and effectiveness data determined to be necessary; • Tracked as phase IV commitment

11. PREA Full Waiver granted when: • Condition doesn’t occur in children • Necessary studies impossible or highly impracticable; • Strong evidence suggests the drug or biologic would be ineffective or unsafe; or • Drug or biologic does not represent a meaningful therapeutic benefit over existing therapies and is not likely to be used in a substantial number of pediatric patients

12. PREA Partial Waiver granted when: • A subset of pediatric patients cannot be studied • Reasonable attempts to produce a pediatric formulation necessary for that age group have failed

13. PREA Labeling Requirement: • If full or partial waiver is granted because there is evidence that the drug or biologic would be ineffective or unsafe, the information shall be included in the label

14. PREA Sponsor declines issued WR Refer NIH Foundation No Funds Sponsor required to do studies May be considered misbranded Rule Sponsor doesn’t agree to do required studies May be considered misbranded or unapproved Process for legal recourse PREA vs. RuleMarketed Drugs

15. PREA Pediatric Advisory Committee • Established in Office of the Commissioner • Responsibilities include: • Advice and recommendations on matters relating to pediatric therapeutics including: • Pediatric research conducted under off-patent process • Identification of research priorities and the need for additional treatments of specific diseases or conditions • Ethics, design and analysis of clinical trials related to pediatrics

16. PREA Pediatric Advisory Committee • Responsibilities include: • Adverse Event Reporting as related to drugs granted pediatric exclusivity • Labeling Dispute Resolution • Subpart D referrals under 50.54

17. Summary PREABPCA New Pediatric Information in Labels

18. To Contact Us: OPT Line: 301-827-9218 301-827-4001 (fax) OCTAP Line: 301-827-7777 Peds Line: 301-594-PEDS Internet: www.fda.gov/cder/pediatrics Email: opt@fda.gov pdit@cder.fda.gov