Rare Disease Congressional Caucus Briefing: Urgent Healthcare Policy Needs of the Rare Disease Community

Rare Disease Legislative Advocates & the National MPS Society in coordination with Rare Disease Congressional Caucus Co-Chairs:
Representatives Leonard Lance (R-NJ) and Joe Crowley (D-NY), held a briefing,

Urgent Healthcare Policy Needs of the Rare Disease Community

Thursday, February 26th, 2015

Russell Caucus Room

Lunch Provided by event sponsors AbbVie, Alexion, Raptor, & Shire
Moderator:  Anthony J. Castaldo, President, US Hereditary Angiodema Association (HAEA)
  • Limited Access to Specialists in Private Insurance Networks, Stephanie Bozarth, National MPS Society
  • Coverage Gaps for Medically Necessary Foods, Nicole Dreyer-Gavin, PKU Parent Advocate
  • Medicare Coverage Challenges for Cystinosis Patients, Kristina Broadbelt, Patient Advocate
  • Step-wise Therapies: Negative Implications for the Health of Rare Disease Patients, Anthony J. Castaldo, President, US Heriditary Angiodema Association


Briefing Hosted by:


October RDLA Meeting

Please join us for an
Advocates Luncheon/Conference Call

Thursday, October 16th, 2014
12 noon to 1 p.m. EST

RDLA DC Office
1101 14th Street NW, Suite 700
Washington, DC,  20005

Lunch will be provided

Click the titles to view PowerPoint Sides:

Please click here to listen to the audio recording of the call

Mark your Calendars!

BIO Patient & Health Advocacy Summit: Oct 14-15 2014, in DC


BIO is throwing their Patient & Health Advocacy Summit, titled “The Power of Partnerships” this October 14-15 in Washington, DC.

The event voluntary brings health organizations and the biotechnology industry together for two days of policy-driven panel discussions, best practice seminars and invaluable networking opportunities. The summit will feature speeches from Janet Woodcock, who serves as Director of the FDA center for Drug Evaluation, Representatives Diana Degette (D-CO) and Joe Pitts (R-PA), and many more.

To register and find out more, clickbit.ly/biosummit

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) ”
Read more

Nominate someone today for a RareVoice Award!


Rare Disease Legislative Advocates is pleased to open nominations for the 3rd Annual rarevoicelogoweb

A celebration to honor advocates who give rare disease patients a voice on Capitol Hill

Thursday, November 13, 2014 Arena Stage in Washington, DC

www.RareVoiceAwards.org RareVoice Award nominations are open to the public.  We encourage the community to nominate individuals and organizations who advocate for legislation and policy that benefits the rare disease community.  If you know of someone who has been a “Voice” for the rare disease community and should be honored for their work, please click here to submit your nomination.  We have nominations open for three award categories, Member of Congress or Congressional Staff, Patient Advocate or Patient Organization, and Government Agency Staff.  Deadline to submit nominations is  Friday August 29, 2014. Sponsorship Opportunities are available, contact Julia@RareAdvocates.org.   Thank you to our sponsors:

Gala Sponsor Mashup

Rare Disease Congressional Caucus Briefing


Rare Disease Legislative Advocates in coordination with Rare Disease Congressional Caucus Co-Chairs

Representatives Leonard Lance (R-NJ) and Joe Crowley (D-NY), will host a briefing on

Implementation of the Rare Disease Provisions in the
Food & Drug Administration Safety and Innovation Act of 2012 (FDASIA) PL 112-144

Wednesday, September 17, 2014

12:00 noon – 1:30 pm

Rayburn House Office Building, Room: B-318

Lunch Provided by event sponsors Amicus & 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, TBD, Novartis Therapeutics
  • 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 Patient Advocate
  • FDA’s Strategic Plan for Accelerating the Development of Therapies for Pediatric Rare Diseases – TBD, FDA
  • Emerging Biotech Perspective of the combined impact of FDASIA, TBD, Amicus Therapeutics

Limited Seating, RSVP to attend: To register click here.

Briefing Hosted by:

Sept. Caucus briefing logo mashup

Sept. 17th & 18th Rally for Medical Research Lobby Day

Medical Research is an issue that the rare disease community cares deeply about. If you would like to participate in this year’s rally: register here

Read more

Bio Patient & Health Advocacy Summit

The event will be held October 14-15,2014 at the Mayflower Renaissance Hotel in Washington, DC. To register, please visit this link. For those traveling from out of town, BIO has a limited number of rooms reserved in the BIO room block (available here).

Bio Patient Health Advocacy Summit

In-District Lobby Days

One of the most important parts of being a rare disease advocate is building relationships with your Members of Congress, your state and local legislators, and other advocates in your community. Click below to register for RDLA’s In-District Lobby Days

For help and questions contact Andy Russell @ 415 884-0223, arussell@everylifefoundation.org

 July 11th Deadline to Register to Participate Read more

RDLA’s June Webinar/Conference Call


Tuesday, June 17th, 2014
11:30 a.m. to 12:30 p.m. EST