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 (

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

Rare Disease Day Cocktail Reception & Documentary Screening

Please join RDLA for our 4th Annual Rare Disease Cocktail Reception & Screening of the HBO Documentary Film Life According to Sam to kick-off Rare Disease Day Week in Washington, DC.

Tuesday, February 25, 2014
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11/15 Congressional Hearing “Reviewing FDA’s Implementation of FDASIA”

Friday, November 15, 2013 – 10:00am  Health Subcommittee  – 2322 Rayburn House Office Building, Washington DC
Many members of Congress brought up concerns about how FDASIA implementation is affecting Rare Disease Drug Development. Dr. Woodcock, FDA’s Director of the Center for Drug Evaluation & Research (CDER) explained how the rare diseases programs at the FDA have been hindered due to Sequestration.

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Sept. 12: Rare Disease Congressional Caucus Briefing Videos

RDCC-Horizontal-CMYKRDLA and Shire in coordination with Rare Disease Caucus Co-Chairs Representatives Leonard Lance (R-NJ) and Joe Crowley (D-NY) hosted a briefing on Thursday, September 12, 2013 

Orphans In Jeopardy:  Threats to the Development of Rare Disease Treatments 

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9/10 Press Briefing Hosted by the Coalition for Accessible Treatments

New Study Release:  How Limiting Cost Sharing for Specialty Drugs Would Impact Insurance Premiums
Tuesday, September 10, 2013 • 2:00 pm
2456 Rayburn House Office Building

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9/10-11: Progress through Partnership: The 2013 NINDS Nonprofit Forum

The National Institute of Neurological Disorders and Stroke (NINDS) at the NIH invites members of nonprofit organizations to the Institute’s 2013 Nonprofit Forum. The Forum provides an opportunity for nonprofit leaders to network with colleagues and to engage in discussions with NINDS staff.

Natcher Conference Center, Bethesda, MD September 10 and 11, 2013

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Sept. 10: FDA Patient Network Annual Meeting

Demystifying FDA: An Exploration of Drug Development

The meeting will serve as a forum for FDA’s patient stakeholders and the general public, including health professionals, academia, and industry to learn about regulatory issues related to drug development, analyze where in the process patient input may be most practical and most valuable, and explore practicable approaches to incorporating meaningful patient input that will represent broad patient perspectives in medical product development and regulatory decision-making.

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Sept. 11: RDLA Luncheon & Conference Call

Advocates September Luncheon & Conference Call
Wednesday, Sept. 11, 2013   12:00 – 1:30 pm Eastern

750 9th Street, NW, Washington, DC 6th floor Office of Akerman Senterfitt – Washington Conference Room
lunch provided

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Sept. 12: 2013 National Health Research Forum

Time: 11:30 a.m. to 3 p.m.:  The Research!America National Health Research Forum engages leaders in government, industry, academia, patient advocacy organizations and the media in a dialogue concerning research to improve health and health care. It is a singular event aimed at confronting issues that bear on our nation’s capacity to maintain its global leadership in the medical research arena, and on the health, safety, and prosperity of the American people.

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