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  • 1. Pediatric Oncology and the Best Pharmaceuticals for Children Act Lisa L. Mathis, M.D. OND Associate Director Pediatric and Maternal Health Staff Office of New Drugs 27 June 2007
  • 2. Objectives
    • Review Pediatric Legislation with a focus on the Best Pharmaceuticals for Children Act
    • Describe BPCA On Patent Process
    • Discuss Results
  • 3. Pediatric Legislation
    • Voluntary
      • Best Pharmaceuticals for Children Act (BPCA)
        • Signed into law January 4, 2002
        • Renewed pediatric exclusivity incentive originally in FDAMA
    • Mandatory
      • Pediatric Research Equity Act (PREA)
        • Signed December 3, 2003
        • Restored some important aspects from the Pediatric Rule, enjoined in 2002
  • 4. BPCA
    • Renewed Pediatric Exclusivity provision in FDA Modernization Act (FDAMA)
      • Provides 6 months of market exclusivity in return for voluntarily conducting FDA-requested pediatric studies contained in a Written Request (WR) within a specific period. (Written request includes studies needed to label drug in kids)
  • 5. BPCA
    • If a drug is on-patent, or has existing exclusivity (e.g., new molecular entity, orphan), the FDA may issue a Written Request (WR) for clinical studies
      • If the requested studies are conducted and full reports submitted in a manner consistent with WR, they may get 6 months of additional exclusivity
    • Within 90 days of submission, Exclusivity Board determines if Sponsor met terms of WR
  • 6. BPCA
    • Provides mechanism to work with National Institutes of Health to award contracts to study drugs not currently protected by patent or exclusivity (“off-patent process)
    • Provided mechanism for WRs declined by sponsors (for drugs currently protected by patent or exclusivity) to be referred to NIH if the sponsor declines to conduct the studies included in the WR
  • 7. What is in a Written Request?
    • Indication
    • Study type
    • Statistical information appropriate to the type of study
    • Drug information (formulation, dosing)
    • Drug specific safety concerns (what to monitor)
    • Labeling that may result
    • Formats of study reports (full study reports)
    • Timeframe for submitting reports
  • 8. What is Different in an Oncology Written Request?
    • Types of studies to be performed:
      • Phase I dose finding, including pharmacokinetics with doses determined for all appropriate age groups. Number of patients sufficient to achieve the objectives; typically 18-25 patients.
      • Phase II or pilot: enrollment of at least 14 patients with the tumor type.
  • 9. Process for the Study of On-Patent Drugs FDA issues (WR) Written Request Industry agrees to conduct studies Industry declines to conduct studies Referral to Foundation for NIH Industry has 180 days to respond Industry submits a Proposed Pediatric Study Request (PPSR) FDA determines public health benefit to support pediatric studies yes yes no
  • 10. Success of BPCA
    • PPSRs submitted 492
    • Written Requests Issued 340
    • Studies requested 793
    • Exclusivity Determinations 150
    • Exclusivity Granted 136
    • New Labels 131
  • 11. Pediatric Research Equity Act PREA
    • Became law December 3, 2003
    • Legislation mimics Pediatric Rule
    • Requires pediatric studies if condition in adults occurs in children
    • Retroactive for all applications back to April 1, 1999
  • 12. Pediatric Research Equity Act PREA
    • Most pediatric cancers different from adult cancers, thus most oncology drugs receive waivers
    • Supportive therapies (pain, prevention of nausea and vomiting) usually studied or deferred
  • 13. Success of PREA
    • New Labels 55
      • Only 15 of the 40 have new clinical information
    • Post Marketing Commitments 191
  • 14. Post Marketing Commitments
    • Avastin, Bevacizumab - Safety and activity data and to characterize the pharmacokinetics in pediatric patients with relapsed or refractory solid tumors
    • Erbitux, Cetuximab - Dose finding study in children and adolescents who have EGFR-expressing, treatment refractory, pediatric solid tumors.
    • Neulasta, Pegfilgrastim - Chemotherapy in Pediatric Sarcoma Patients
  • 15. Post Marketing Commitments Supportive Care
    • Mycamine, Micafungin Sodium – Study prophylaxis of Candida infections in patients ages 0 to 16 years old undergoing hematopoietic stem cell transplantation
    • Cancidas, Caspofungin – Study treatment of presumed fungal infections in febrile, neutropenic patients
    • Vfend, Voriconazole – Study treatment of candidemia and invasive candidiasis infections
  • 16. Post Marketing Commitments Supportive Care
    • Kepivance, Palifermin - Study decreases the incidence and duration of severe oral mucositis and related sequelae experienced by patients age 3-16 with hematologic malignancies who are receiving myelotoxic therapy.
  • 17. Post Marketing Commitments Supportive Care
    • Aloxi, Palonosetron HCl – Study prevention of acute nausea and vomiting associated with initial and repeat courses of moderately and highly emetogenic cancer chemotherapy (CINV)
    • Emend, Aprepitant – Study prevention of acute and delayed nausea and vomiting associated with initial and repeat courses of highly emetogenic cancer chemotherapy
    • Kytril, Granisetron HCl - Stduy treatment of post operative nausea and vomiting in pediatric patients 2 to 16 years of age
  • 18. Post Marketing Commitments Supportive Care
    • Aranesp, Darbepoetin alfa - Study treatment of anemia in pediatric patientswith non-myeloid malignancies receiving multi-cycle chemotherapy
  • 19. BPCA vs. PREA
    • PREA
    • Studies are required
    • Orphan drugs exempt
    • Studies limited to indication for which sponsor seeks approval in adults
    • applies to § 505 (FD&C Act) (NDA) and § 351 (PHS Act) (BLA) products
    • 10-1-07 Sunset
    • BPCA
    • Studies are voluntary
    • Includes orphan drugs
    • Studies on whole moiety
    • FDA Issues WR
    • Mechanism for funding studies of off-patent moieties
    • Required safety reviews
    • Transparency: Summaries
    • applies to § 505 products only
    • 10-1-07 Sunset
  • 20. Conclusion
    • BPCA and PREA have had a powerful impact on providing important safety, efficacy, and dosing information for drugs used in children.
    • Oncology products have been studied and information has been learned
    • Scientific advancements must be reflected in the studies for oncology drugs
    • New legislation being crafted with support from Oncology groups and may expand types of studies we are able to ask for in pediatrics