PediatricDrugDevelopmentADecadeofProgress.pptVIP

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 2003 PREA Review Written Request Process Discuss statistics, goals available resources 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 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 1997 FDAMA Pediatric Provisions Created pediatric exclusivity incentives based on Written Request from FDA Sunset on January 1, 2002 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 FDAMA “Priority List” Issued by FDA Being on the priority list did not require a WR to be issued Sunset on January 1, 2002 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 Best Pharmaceuticals for Children Act Establishes a process for studying both “off-patent” and “on-patent” drugs Mandates that FDA and NIH collaborate in the study of “off-patent” and “on-patent” drugs that industry does not want to study Two Paths to a Written Request FDA generates and issues a WR PPSR sent t