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)
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
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.
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
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.