Rare Disease Congressional Caucus Briefing Co-Hosted by Shire and RDLA

September 25, 2013

Shire co-hosted a Rare Disease Congressional Caucus briefing on Thursday, September 12 in conjunction with Rare Disease Legislative Advocates. Approximately 50 people attended the event.

John Crowley, parent advocate and CEO of Amicus Therapeutics, served as moderator. An overview of the speakers and their remarks are below.


  •          Representative Gus Bilirakis (R-FL), House Energy and Commerce Health Subcommittee

o    We need FDA’s accelerated approval process to apply to rare diseases

o    The accelerated approval process was created for HIV, bioterrorism and some cancer drugs – but the rare disease community urgently needs helps

o    Congress intended that the FDA use this accelerated approval process for rare disease

o    One year later, FDA has not approved any rare disease drugs, and we need to hold FDA accountable

o    We are in the process of sending a letter to the FDA, and we have 36 bipartisan co-signers as well as 58 patient advocacy groups

o    We need a clear workable framework for rare disease drugs


  •         Miriam O’Day, Senior Director, Alpha-1 Foundation

o    Congress can help stimulate the business community to invest in rare diseases

o    We need to preserve and protect the ODTC; it’s a critical incentive for these companies to develop new treatments

o    We must continue to create an appetite for drug industry development

o    The Alpha-1 community has taken drug development into its own hands


  •          Tracy VanHoutan, Parent Advocate, Noah’s Hope, Batten Disease Support and Research Association

o    Father of two children diagnosed with Batten disease, Tracy recently learned that a new clinical trial for Batten will be taking place in Europe

o    Batten isn’t the only disease area where clinical trials are being conducted in Europe rather than the U.S.; Tracy was warned this was going to happen by other rare disease patient support groups

o    These companies are investing billions of dollars annually, but the jobs created are being sent overseas

o    The way the FDA looks at rare disease is broken, but it’s not fatally flawed

o    Some big changes need to happen at the FDA and happen quickly for the sake of patients

o    We need to give the FDA the resources they need to make these changes happen

o    Let’s have more conversation on these diseases


  •         Dr. Patrick Scannon, Exectuive Vice President and Chief Scientific Officer, XOMA

o    Our industry needs to be able to rely on FDA timelines that are regular and timely

o    We have voluntarily increased the amount of money we pay to the FDA to help with the approval process

o    Sequestration has slowed the hiring of much needed FDA expert reviewers

o    Many companies are trying to access breakthrough pathways – but this won’t be possible if FDA reviewers don’t exist

o    Rep. Lance has introduced this bill that will take this private money and use it to assist the FDA approval process


Many thanks to the RDLA (Rare Disease Legislative Advocates) and Julia Jenkins for this post.

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