Rare Diseases Congressional Caucus Briefing Sept.17th

Rare Disease Legislative Advocates & Kids v Cancer in coordination with Rare Disease Congressional Caucus Co-Chairs
Representatives Leonard Lance (R-NJ) and Joe Crowley (D-NY), will host a briefing

“Implementation of the Rare Disease Provisions in the
Food & Drug Administration Safety and Innovation Act of 2012 (FDASIA) ”

Wednesday September 17th, 2014
12:00 pm – 1:30 pm

Rayburn House Office Building, Room B-318 
Lunch will be provided by event sponsors Amicus, Alexion & Shire


  • Sec. 901. Enhancement of accelerated patient access to new medical treatments: Accelerated Approval for rare diseases, Emil Kakkis, MD PhD, EveryLife Foundation for Rare Diseases
  • Sec. 902. Breakthrough therapies, Shanthi Ganeshan, Novartis
  • Sec. 903. Consultation with external experts on rare diseases, Stephanie Krenrich, Cystic Fibrosis Foundation
  • Sec. 908. Rare pediatric disease priority review voucher incentive program, Nancy Goodman, Kids v Cancer
  • Sec. 1137. Patient participation in medical product discussions, Allison Greenstein, Narcolepsy Network
  • FDA’s Strategic Plan for Accelerating the Development of Therapies for Pediatric Rare Diseases – Dr. Gayatri Rao, Office of Ophan Product Development FDA
  • Emerging Biotech Perspective of the combined impact of FDASIA – Jayne Gershkowitz, Amicus Therapeutics
Limited Seating, RSVP to attend:
Briefing Hosted by: