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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Hearing Notice – 21st Century Cures: PCAST’s Report on Drug Innovation

The Subcommittee on Health will hold a hearing on Tuesday, May 20, 2014, at 10:00 a.m. in 2322 Rayburn House Office Building.  The hearing is entitled “21st Century Cures: The President’s Council of Advisors on Science and Technology (PCAST) Report on Drug Innovation.”  Witnesses will be announced and are by invitation only.  The hearing webcast will be available at http://energycommerce.house.gov/.

A bipartisan staff briefing will be held on Monday, May 19, 2014, at 4:00 p.m. in 2218 Rayburn House Office Building.  The Republican staff briefing will follow immediately after the bipartisan briefing in 2218 Rayburn.  The Democratic staff briefing will follow immediately after the bipartisan briefing in 2322 Rayburn.

If you have any questions concerning this hearing, please contact John Stone, Carly McWilliams, Paul Edattel, Brenda Destro, or Katie Novaria at (202) 225-2927.

RDLA’s May Webinar/Conference Call

Wednesday, May 21, 2014 

Click here to listen and view a recording of the call

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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

Monthly Meetings

Join us for our next Legislative Webinar on Tuesday, May 17th, 2016

Click here to register!


Once a month RDLA convenes in person and/or over the phone to discuss legislation and developments that affect the rare disease community. The meeting/conference calls are essentially a clearing house for legislation and participating in the calls does not imply support for any of the policy proposals or legislation that are discussed or promoted at meetings.

2016 RDLA Call Schedule:

2015 RDLA Calls: click for agendas and PowerPoint presentations

2014 RDLA Calls: click for agendas and PowerPoint presentations

SCAD Alliance at Rare Disease week 2014

“As anticipated, Rare Disease Day 2014 events did not disappoint. We are taking the information we gained and are busy reaching out to new contacts to advance the mission of SCAD Alliance.” – SCAD Alliance

Read more about SCAD Alliance at Rare Disease Week in Washington D.C. 2014

RDLA Conference Call/Webinar March 2014

Our monthly conference call/webinar took place on March 20th.  You can find the agenda & slides from the call below:

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Senate Briefing on Rare Diseases

“Rare Diseases in a Changing Healthcare Landscape,” Thursday, March 20, 2014; 2 – 3:30 p.m. ET in The Capitol Visitors Center, Room SVC 212-10.

All rare disease stakeholders are invited to a Capitol Hill briefing to update Congressional staff and others on current issues related to rare diseases and orphan products.

The briefing is hosted by NORD, if you have any questions please contact Diane Dorman, VP of Public Policy for NORD (ddorman@rarediseases.org).

NJ State House Event

Join Rare New Jersey, for a hour of learning and discussion in honor of Rare Disease Day. Click here to learn more and how to RSVP: 2014 RDD invitation

Feb. 27th Rare Disease Congressional Caucus Briefing


Rare Disease Legislative Advocates, National Organization for Rare Disorders & Jonah’s Just Begun Foundation to Cure Sanfilippo, in coordination with
Rare Disease Congressional Caucus Co-Chairs
Representatives Leonard Lance (R-NJ) and Joe Crowley (D-NY),
hosted a briefing featuring

Special Guest Jonny Lee Miller, who stars as Sherlock Holmes in the hit TV show Elementary,
will help us unlock the mystery of the
“Science Behind Rare Disease Policy”

Thursday February 27, 2014
12:00 noon – 1:30 pm
Capitol Visitors Center, Room: HVC – 201

Lunch Provided by event sponsors Genzyme & Shire

Moderator:  Kay Holcombe, Senior Policy Analyst, Genzyme


  • P.J. Brooks, Ph.D., Health Science Administrator – Office of Rare Diseases Research & Division of Metabolism and Health Effects, NIAAA,  NIH
  • Marshall Summar, MD, NORD Board Member, Division Chief, Genetics and Metabolism at Children’s National Medical Center and Faculty, Children’s Research Institute, Center for Genetic Medicine Research
  • Emil Kakkis, MD, PhD, President EveryLife Foundation for Rare Diseases
  • Hugh Hempel, Founder, Addi & Cassi Fund
  • Jill Wood, CEO, Jonah’s Just Begun to Cure Sanfilippo
  • Jonny Lee Miller, Actor, Patient Advocate



Briefing Sponsors:

logos for breifing