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Pediatric Drug Development Initiatives

Pediatric Drug Development Initiatives. Lisa Mathis, M.D. Acting Director Division of Pediatric Drug Development October 20, 2005. Pediatric Initiatives. Pediatric Research Equity Act, December 3, 2003 Best Pharmaceuticals for Children Act, January 4, 2002

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Pediatric Drug Development Initiatives

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  1. Pediatric Drug Development Initiatives Lisa Mathis, M.D. Acting Director Division of Pediatric Drug Development October 20, 2005

  2. Pediatric Initiatives • Pediatric Research Equity Act, December 3, 2003 • Best Pharmaceuticals for Children Act, January 4, 2002 Both laws are intended to support and encourage drug development in the pediatric population

  3. PREA studies are mandatory • BPCA studies are voluntary

  4. Why Both PREA and BPCA? • Distinction between the scope of studies requested under BPCA and required under PREA • PREA specific to indication in submission • BPCA can ask for “off-label” indications • Sildenafil Citrate (Viagra) • Pediatric studies required by the Pediatric Rule were waived • Written Request was issued

  5. Pediatric Research Equity Act (PREA) • Became law December 3, 2003 • PREA is the codification of the 1998 Pediatric Rule • Drugs and Biologics affected • Not applicable to drugs with Orphan Designation

  6. PREA • One of two laws intended to promote the study of drugs and biologics in pediatric patients • Studies prevent pediatric patients from being a “study of one” • Studies in the pediatric population are REQUIRED, but only for the indication that was studied in adults

  7. PREA • Pediatric Assessment required for certain applications unless waived or deferred • Draft Guidance recently issued (9/7/05)

  8. PREA • Pediatric Assessment contains • data adequate to assess the safety and effectiveness of the drug or biological product, and • data to support dosing and administration for each pediatric subpopulation

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

  10. Full Waiver Waiver granted when: • Necessary studies impossible or highly impracticable; • Strong evidence suggests the drug or biologic would be ineffective or unsafe; or • Product does not represent a meaningful therapeutic benefit over existing therapies AND is not likely to be used in a substantial number of pediatric patients

  11. “Substantial Number” • PREA does not define substantial number • FDA generally has considered 50,000 patients to be a substantial number • FDA will take into consideration the nature and severity of the condition when making this determination

  12. Partial Waiver Partial Waiver is a special waiver for a pediatric age group (i.e. less than 6 months of age) and is granted when: • The criteria for a full waiver applies to that age group; or • Reasonable attempts to produce a pediatric formulation necessary for that age group have failed

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

  14. Deferral • A deferral is granted when a pediatric assessment is needed, but permits submission of the pediatric assessment after submission of NDA/BLA • Reasons for deferral • Drug or biologic is ready for approval in adults; • Need additional safety data; or • There is another appropriate reason for deferral

  15. PREA • Not as flexible as BPCA • Indications for required pediatric studies are limited to the indications in a given submission • An assessment would be waived under PREA in submissions for the treatment of a condition that occurs only in adults • Prostate, breast cancer

  16. Best Pharmaceuticals for Children Act (BPCA) • Became law January 4, 2002 • Renewed authority (FDAMA) to grant six months of marketing exclusivity to Sponsors who conduct and submit studies in response to a Written Request. • Includes an additional mechanism for obtaining information for the use of off-patent drugs in pediatric patients

  17. PREA and BPCA:Togetherness

  18. Goal • The goal of both PREA and BPCA: • Obtain information from studies about the use of medications in the pediatric population. • Obtain studies for both common and rare conditions. • Disseminate information about the safe and efficacious use of medications in children.

  19. PREA vs. BPCA PREA • Studies mandatory • Required studies only on drug/indication under review • Studies for orphan indications not required • Applies to drugs and biologics • Sunsets October 1, 2007 BPCA • Studies voluntary • Studies on entire active moiety • WR may be issued for orphan indications • Applies only to drugs • Sunsets October 1, 2007

  20. To Contact Division of Pediatric Drug Development: Peds Line: 301-796-2200 Internet: www.fda.gov/cder/pediatric Email: pdit@cder.fda.gov

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