Rare Disease Congressional Caucus Briefing Nov. 2012

RDLANational Health Council, and Shire, in coordination with Rare Disease Caucus Co-Chairs Representatives Leonard Lance (R-NJ) and Joe Crowley (D-NY) hosted a briefing on Wednesday, November 14, 2012

Read more

NIH Webinar 10/17 on Expansion of Undiagnosed Diseases Program

Open To All: NIH Webinar Today To Provide Input From Patient Perspective

The NIH Office of Rare Diseases Research-NCATS and the National Human Genome Research Institute are hosting a webinar at 2:30 p.m. ET today to provide information to the patient community about a new NIH initiative to expand the Undiagnosed Diseases Program.

Read more

EveryLife Foundation Workshop #4: Developing Guidance and Policy Recommendations for Accelerated Approval in Rare Diseases

Please save the date for the next workshop in the Rare Disease Workshop Series:  Thursday, November 15, 2012 at the Sofitel Hotel in Washington DC.

 Now is the ideal time to establish policy and guidance recommendations on Accelerated Approval in rare diseases. The Food and Drug Administration Safety and Innovation Act, S. 3187, which was signed by the President in July, includes the need to develop a guidance on the implementation of updated Accelerated Approval practices.

Read more

2012 RARE Patient Advocacy Summit – Session #1 – Political Advocacy – You Have The Power!

A discussion about the power you have in political advocacy at the federal, state and local levels. One voice – yours! – makes a world of difference …

Read more

Friends-Brookings Conference on Clinical Cancer Research

Friends of Cancer Research & the Brookings Institute:
November 14, 2012 – 8:30am- 3:00pm – The Washington Marriott – Washington, DC
Morning Keynote: Harold Varmus, M.D., Director, National Cancer Institute
Afternoon Keynote: Margaret Hamburg, M.D., Commissioner, U.S. Food and Drug Administration

Read more

Patient-Focused Drug Development – Oct. 25th FDA Public Meeting

As part of the performance commitments of the fifth authorization of the Prescription Drug User Fee Act (PDUFA V), FDA has published a notice in the Federal Register related to Patient-Focused Drug Development, an initiative that provides for a more systematic approach to obtaining the patient perspective on certain disease areas in PDUFA V.

Read more

Opportunity to Learn From AIDS Activists – Documentary Screenings

ACT UP and their colleagues fought for nine years before winning life-saving medications. They organized a mass movement, they took to the streets, they made art, they made noise, they made a difference. Their tools included clear demands, arresting graphics, media savvy, and an ability to learn from their mistakes and refine their strategies. And a sense of humor, when appropriate, in combination with their urgent and ethical message.

Read more

POLITICO Pro’s Deep Dive: The Future of the FDA

The POLITICO Pro Health care team takes an in-depth look at the future and policies of the FDA.  Special guests include: Friends of Cancer Research’s Dr. Jeff Allen; The Pew Charitable Health Group’s Allan Coukell; Biotechnology Industry Organization’s Sara Radcliffe and the FDA’s Dr. Janet Woodcock.

Read more

Rare Disease Lobby Day 2012

In February, RDLA hosted its first lobby day, bringing 70 patient advocates to Washington, D.C. to advocate for the Creating Hope Act and the Unlocking Lifesaving Treatments for Rare Diseases Act (ULTRA), which was later included into the Faster Access to Specialized Therapies (FAST) Act. Both provisions were included in FDASIA.


You are invited to join us in Washington, DC for our Rare Disease Lobby Day on Tuesday, February 28, 2012.  RDLA is partnering with the EveryLife Foundation for Rare Diseases, RARE Project, Sarcoma Foundation of America, Darlington House and ViroPharma to ensure that rare disease advocates are present on Capitol Hill during Rare Disease Week! Join us and make sure Congress hears your voice!

Lobby Training Breakfast
7:30a.m. – 9:30a.m.  Darlington House 1610 20th ST NW Washington DC
Learn about the Rare Disease Congressional Caucus and legislation to help spur the development of treatments for rare disease patients, including the ULTRA Act and Creating Hope Act 

Hill Meetings with your Representatives

10:00a.m. – 5:00p.m. on Capitol Hill
Transportation will be provided from the Darlington House to Capitol Hill
Each advocate will be scheduled for at least three meetings

Happy Hour Lobby Day Celebration
5:30p.m. – 7:00p.m.  Darlington House 1610 20th ST NW Washington DC
Join us to share your meeting experiences and feedback from Congress
The happy hour includes appetizers & a no host bar

Please click YES below to register for this event.  You must provide your home address in order for us to help schedule a meeting with your Members of Congress:

Financial Assistance Available:  To help offset travel costs, $500 travel scholarships are available to advocates who wish to attend and have financial need.  To apply, please click here to download the application.  A limited number of scholarships are available.  Scholarships will be distributed in order to ensure a diverse representation of the rare disease community can participate.  Please return completed applications via email to patientadvocate@kakkis.org or fax at 415-884-0562.

HOTEL:  We have reserved a block of hotel rooms for Monday & Tuesday nights at The Embassy Row Hotel at the discounted rates of $169 single or $189 double per night.  The Hotel is located in Dupont Circle a 5 minute walk from the Darlington House.  To receive the discounted rate, please book directly with the Hotel by calling 202-265-1600 and mentioning the group “Rare Disease Day Lobby Day.”  Please book your hotel room immediately as we will not be able to guarantee this rate once our block of rooms is filled.  Discounted rate expires on Feb. 20th.

If you can’t join us in DC, please stay tuned for Action Alerts!  We will email you ways to participate in Rare Disease Day from your home or office by making calls and sending emails to your Members of Congress and sending emails to support legislation that will help spur the development of lifesaving treatments!

Don’t miss these other great Rare Disease Day events in DC!
You can participate in most of them from your home or office:


Monday, February 27, 2012

RDLA Luncheon Meeting & Conference Call

12:30 – 2:00p.m. at 750 9th Street NW, Suite 750 Washington, DC


2nd Annual RDLA Cocktail Reception and Movie Screening 

5:30p.m – 9:00p.m at the West End Cinema, 2301 M Street NW, Washington DC

Featuring:  The Documentary “Fight to Live” with panel discussion led by Academy Award Winning Director Barbara Kopple


Wednesday, February 29, 2012

Rare Disease Day at NIH (RDD@NIH) 

Clinical Center’s Masur Auditorium, 9000 Rockville Pike, Building 10, Bethesda, MD
The National Institutes of Health (NIH) will celebrate the fifth annual Rare Disease Day with a day-long celebration. Attendance is free, open to the public and will be webcasted.


Take a LEAP for Complex Illness

8pm at the Dunes 1402 Meridian Place NW, Washington, DC

Featuring performances by Jonny Grave & the Tombstones, Benny, & Nelly Kate.
To benefit MarbleRoad a 501c3 non-profit: connecting people who have complex illnesses with resources to improve their lives.


Thursday, March 1, 2012

FDA Rare Disease Patient Advocacy Day

White Oak Campus, 10903 New Hampshire Ave, Silver Spring, MD
The Food and Drug Administration (FDA) will celebrate the fifth annual Rare Disease Day by hosting a “FDA Rare Disease Patient Advocacy Day” to engage and educate the rare disease community on regulatory processes related to rare diseases. Attendance is free. Registration is required. The event will be webcasted


Rare Disease Day 2012 “Fight to Live” Documentary Screening

2nd Annual RDLA Cocktail Reception and Movie Screening
Monday, February 27, 2012, West End Cinema, Washington DC

Featuring:  The Documentary “Fight to Live” with panel discussion led by Academy Award Winning Director Barbara Kopple

“In a country that defends freedom as the core of American values, director Barbara Kopple explores the absolute powers of the FDA over access to investigational drugs that may be the only chance at survival for terminally ill patients.  Patient and public safety is jeopardized when breakthroughs are lost or delayed by an insurmountable process – with a cost tallied in human lives.  Through an intimate journey with patients, physicians, researchers, advocates and FDA overseers, Kopple asks the question: What rights should you have in a life or death battle to survive versus what decisions should the government make for you? It’s a question we all must answer before we each face our own Fight to Live.”

Read more