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Pediatric Drug Development: A Decade of Progress: 1994-2004

Pediatric Drug Development: A Decade of Progress: 1994-2004. Susan K. Cummins, MD, MPH Medical Team Leader Division of Pediatric Drug Development Center for Drug Evaluation and Research. Today’s Talk. Review legal milestones of the last decade 1994 Pediatric Rule 1997 FDAMA 2002 BPCA

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Pediatric Drug Development: A Decade of Progress: 1994-2004

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  1. Pediatric Drug Development: A Decade of Progress: 1994-2004 Susan K. Cummins, MD, MPH Medical Team Leader Division of Pediatric Drug Development Center for Drug Evaluation and Research

  2. Today’s Talk • Review legal milestones of the last decade • 1994 Pediatric Rule • 1997 FDAMA • 2002 BPCA • 2003 PREA • Review Written Request Process • Discuss statistics, goals & available resources

  3. Acronyms • FDAMA – Food & Drug Admin Modernization Act • BPCA – Best Pharmaceuticals for Children Act • PREA – Pediatric Research Equity Act • WR – Written Request • PPSR – Proposed Pediatric Study Request

  4. 1994 Pediatric Regulations • Required sponsor review of available pediatric data to determine whether existing data was adequate to support pediatric labeling • No clinical studies required • Introduced concept of extrapolation of efficacy data from adults to children

  5. 1997 FDAMA Pediatric Provisions • Created pediatric exclusivity incentives based on Written Request from FDA • Sunset on January 1, 2002

  6. What is “Pediatric Exclusivity”? • An additional 6-month period during which a sponsor retains exclusive marketing control of all forms of a drug product line • Requires either an existing patent or existing exclusivity • Confers to the entire moiety • Substantial financial incentive • Delays introduction of generic products

  7. FDAMA “Priority List” • Issued by FDA • Being on the priority list did not require a WR to be issued • Sunset on January 1, 2002

  8. 2002 Best Pharmaceuticals for Children Act • Became law January 4, 2002 • Re-authorizes exclusivity incentive program under FDAMA • Includes an additional mechanism for obtaining information on safe and efficacious use of drugs in pediatric populations for off-patent drugs

  9. Two Paths to a Written Request • FDA generates and issues a WR • PPSR sent to FDA by industry • FDA accepts proposal and issues WR • FDA modifies proposal and issues WR • FDA does not accept proposal and issues inadequate letter

  10. Written Request • A (legal) document sent by the FDA to sponsors requesting studies in the pediatric population • Specifies: • indication • population • type of studies • safety parameters • longer term follow-up • timeframe for response

  11. FDA issues Inadequate Letter Process for the Study of On-Patent Drugs under BPCA Industry submits Proposed Pediatric Study Request FDA determines if there is a public health benefit no yes FDA issues Written Request

  12. Process for the Study of On-Patent Drugs under BPCA (cont.) FDA issues Written Request Industry has 180 days to respond no Industry declines to conduct studies yes Industry agrees to conduct studies Possible Referral to Foundation for NIH

  13. Process for the Study of On-Patent Drugs under BPCA (cont.) Referral to Foundation for NIH Foundation lacks funds to conduct studies Foundation conducts studies WR referred to NIH for inclusion on List

  14. FDA On-Patent Drug Exclusivity Process Background Research/Extensive Literature Review Sponsor Proposed Pediatric Development of Written Request Study Request (PPSR) by Reviewing Division Reviewed by PdIT FDA WR Issued Sponsor Completes Studies or FDA Exclusivity Determined by Exclusivity Board (60-90 days) Action on Application (6-10 months) – includes label negotiations with sponsor

  15. Progress: Industry Response as of January 2004 • Proposals from Industry 336 • FDA-issued Written Requests 283 • Exclusivity Determinations 101 • Exclusivity Granted 91 • New Labels 63

  16. 2003 Pediatric Research Equity Act • Became law December 3, 2003 • Legislation mimics Pediatric Rule • Retroactive for all applications back to April 1, 1999

  17. Pediatric Research Equity Act • Requires pediatric studies of certain drugs & biological products • New indication • New dosage form • New route • New dosing regimen • New active ingredient • FDA Guidance under development • Establishes Pediatric Advisory Committee

  18. Goal On-Patent Exclusivity Process and Off-Patent BPCA Process New Pediatric Information in Label

  19. Link to Peds page FDA Web Page www.fda.gov

  20. Pediatric Web Page

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