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

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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

Agenda:

  • 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

RDLA’s June Webinar/Conference Call

 

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

Rare Disease Congressional Caucus Briefing May 7th


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

“Access to Care & Therapies in the New Healthcare System: A Rare Disease Perspective”

Wednesday May 7, 2014

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New Op-Ed With Details on 21st Century Cures Initiative

by Rep. Fred Upton,U.S. Congressman representing Michigan’s 6th District &  Rep. Diana DeGette, Chief Deputy Whip

Originally published by The Huffington Post on May 6th:  Talk to any American adolescent and it quickly becomes clear that keeping pace with 21st century technology is a constant challenge. But staying on top of today’s technology goes far beyond trending hashtags and Instagram likes; it presents a unique opportunity to bring researchers, innovators, caregivers, and patients together in a new way that advances our collective understanding of disease and how we combat it.

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Rare Disease Advocate featured in CNN article about Compassionate Use

The program that allows patients to use experimental drugs not yet approved by the FDA is called “compassionate use”. Sandy Barker wonders, as do many others, if there might be a way to make it a little more compassionate.    

The CNN article gives a detailed overview of the compassionate use approval use process, including how it can be improved. It also illustrates how drug companies are increasingly concerned about how patients and parents of patients are using social media as a tactic in gaining approval for compassionate use.

RDLA’s April Advocate Luncheon/Conference Call

Advocate’s Luncheon/Conference Call

Wednesday, April 23rd, 2014 12 noon to 1:30 pm

Location: Offices of Polsinelli 1401 Eye Street NW 8th floor Washington, DC,  20005

Girl, 7, with Rare Disease Stands Beside Congressman as He pushes for More Funding

Stephanie stood beside Congressman Mike Fitzpatrick when he called for continued research funding for Tuberous Sclerosis Complex (TSC).

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2014 Rare Disease Legislative Conference Presentations

RDLA’s 2014 Legislative Conference took place on Feb. 25 at the National Press Club in Washington D.C. Over 160 rare disease advocates were in attendance, with 25 speakers educating and training the advocates how to make their voices heard. Click on the title to view the video from the conference.

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