Food and Drug Administration Amendments Act of 2007 Reauthorization of Pediatric Initiatives - PowerPoint PPT Presentation

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Food and Drug Administration Amendments Act of 2007 Reauthorization of Pediatric Initiatives PowerPoint Presentation
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Food and Drug Administration Amendments Act of 2007 Reauthorization of Pediatric Initiatives

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  1. Food and Drug Administration Amendments Act of 2007Reauthorization of Pediatric Initiatives Lisa L. Mathis, M.D. Pediatric and Maternal Health Staff Office of New Drugs April 2008

  2. Acronyms • BPCA – Best Pharmaceuticals for Children Act • FDAAA – Food and Drug Administration Amendments Act • PAC – Pediatric Advisory Committee • PREA – Pediatric Research Equity Act • WR – Written Request FDAAA: ODAC Pediatric Subcommittee April 08

  3. Objectives • Brief Overview of Pediatric History at FDA • Major changes to Title V: Best Pharmaceuticals for Children Act of 2007 (BPCA) • Major changes to Title IV: Pediatric Research Equity Act of 2007 (PREA) FDAAA: ODAC Pediatric Subcommittee April 08

  4. FDA and Pediatrics • 1979 Labeling Requirement • 1994 Pediatric Labeling Rule • 1997 FDA Modernization Act (FDAMA) • 1998 Pediatric Rule • 2002 Best Pharmaceuticals for Children Act (BPCA) • 2002 Pediatric Rule Enjoined • 2003 Pediatric Research Equity Act (PREA) • 2007 FDA Amendments Act of 2007 • Pediatric Medical Device Safety and Improvement Act • Pediatric Research Equity Act (PREA) • Best Pharmaceuticals for Children Act (BPCA) FDAAA: ODAC Pediatric Subcommittee April 08

  5. Childhood Cancer Mortality 1975-2003 FDAAA: ODAC Pediatric Subcommittee April 08

  6. BPCA 2007 FDAAA: ODAC Pediatric Subcommittee April 08

  7. Improvements to BPCA BPCA 2007 • Pediatric Subcommittee of the Oncologic Drugs Advisory Committee • Extended Committee to 2012 • May make recommendations directly to the Internal Review Committee on the implementation of BPCA • Expands role of NIH to “pediatric therapeutics” not just drugs BPCA 2003 • Clarified role of the Pediatric Subcommittee of the Oncologic Drugs Advisory Committee • Previouspriority list was for off patent drugs only FDAAA: ODAC Pediatric Subcommittee April 08

  8. Internal Review CommitteePediatric Review Committee (PeRC) • Established to provide oversight to required and requested pediatric activities within CBER/CDER • Expertise mandated • Pediatric Oncology is represented FDAAA: ODAC Pediatric Subcommittee April 08

  9. National Institutes of Health • Expanded role includes establishing a “List of Priority Issues in Pediatric Therapeutics” • Previously NIH was charged with a list of off patent drugs not eligible for market exclusivity • Expert meeting in June-July 2008 • Law requires consideration of available information on drugs and biologics FDAAA: ODAC Pediatric Subcommittee April 08

  10. Improvements to BPCA (cont) BPCA 2007 • Drugs • Studies voluntary • Studies on entire active moiety • May include on & off label indications • WR may be issued for orphan indications • Preclinical studies may be requested • Priority review for all applications BPCA 2003 • Drugs • Studies voluntary • Studies on entire active moiety • Had to choose between on or off label indication • WR may be issued for orphan indications • Only includes studies in pediatrics • Priority Review supplements only FDAAA: ODAC Pediatric Subcommittee April 08

  11. Preclinical Studies • Law states that pediatric studies and preclinical studies may be included in a Written Request • BPCA 2002 only allowed for the request of studies in the pediatric population • Could not previously require a preclinical study as a term of the WR • Law does not allow for preclinical studies only FDAAA: ODAC Pediatric Subcommittee April 08

  12. Improvements to BPCA (cont) BPCA 2007 • All adverse events must be submitted with application • 6 months to review studies and determine if terms of WR met • Must have 9 months of exclusivity at time of determination • Written requests become public • Studies must be added to label • Full reviews posted • Adverse events review for peds BPCA 2003 • No requirement to submit adverse events • 3 months to review studies and determine if terms of WR met • Could submit on day of patent expiration • Written request confidential • Labeling not required • Summaries of reviews posted • Adverse events reviewed for peds Sunset for PREA and BPCA Oct 1, 2012 FDAAA: ODAC Pediatric Subcommittee April 08

  13. PREA 2007 FDAAA: ODAC Pediatric Subcommittee April 08

  14. Improvements to PREAbasics remain the same PREA 2007 • Drugs and biologics • Studies mandatory • Studies for orphan indications exempt • Required studies only on drug/ indication under review PREA 2003 • Drugs and biologics • Studies mandatory • Studies for orphan indications exempt • Required studies only on drug/ indication under review FDAAA: ODAC Pediatric Subcommittee April 08

  15. Required Studies Only on Drug/ Indication Under Review:Application of PREA • Pediatric studies required and a pediatric assessment must be submitted for NDA/BLA or supplements with • New active ingredient • New indication • New dosage form • New dosing regimen or • New route of administration • Applies only to the indication included in the submission • Limits scope of required studies FDAAA: ODAC Pediatric Subcommittee April 08

  16. Application of PREA • Indications that are likely to be studied under PREA • Leukemia/Lymphoma • Supportive care • Pegfilgrastim FDAAA: ODAC Pediatric Subcommittee April 08

  17. Improvements to PREA (con’t) PREA 2007 • Age appropriate formulation required • Study results must be included in labeling • Full reviews posted • Pediatric adverse events reviewed and presented to PAC PREA 2003 • Age appropriate formulation required Sunset for PREA and BPCA Oct 1, 2012 FDAAA: ODAC Pediatric Subcommittee April 08

  18. Conclusions • Reauthorization provides continued incentive and requirement for pediatric studies and pediatric oncology still plays a major role in the new legislation • Despite progress in pediatric oncology drug development specifically, improvements in survival rates have slowed • Scientists in every sector must collaborate using tools available to them to provide new opportunities for treatment • We can use legislation to improve this collaboration FDAAA: ODAC Pediatric Subcommittee April 08