Tuesday, November 13, 2012

Rare Disease Legislative Conference


Dear SFA Supporter,

On the last day of February each year, the rare disease community unites to celebrate Rare Disease Awareness Day.  In honor of this day, this community will join forces during the last week of February to draw attention to the special needs of those who are dealing with rare diseases like sarcoma.  While we work tirelessly every day to raise awareness, we see World Rare Disease Day, and the days that lead up to it, as a wonderful opportunity to further educate the public, legislators, and government regulators about the challenges faced by those with sarcoma.

The EveryLife Foundation for Rare Diseases has once again taken the lead in developing two fantastic events during this week and the Sarcoma Foundation of America (SFA) is proud to be a partner with them in this effort.  We would like to invite you to join us in participating in these events to be held in Washington, DC.

Tuesday, February 26, 2013

Rare Disease Legislative Conference: A Rare Disease Legislative Conference will take place on Tuesday, February 26.  This conference aims to educate patient advocates about the legislative process and empower patient advocates to take an active role in the democratic process.

Wednesday, February, 27, 2013

Lobby Day: Patient advocates are invited to take part in meetings on Capitol Hill as a way of educating legislators about the special needs of the rare disease community.  The SFA would love to have members of the sarcoma community on Capitol Hill to share their stories.  Organizers of the event will take care of scheduling the meetings and will provide you with the information that you need to have successful meetings with your Members of Congress and/or their staff.

More information, including a link to the registration site, can be found at http://rareadvocates.org/lobby-day/.  Once you have registered to attend, please let the SFA know of your plans by emailing Lori Hoffman, Manager of Scientific and Government Affairs, atlhoffman@curesarcoma.org.  Additionally, financial assistance for travel costs is available.  Interested parties can apply for travel scholarships at http://rareadvocates.org/travel-scholarship-application/

It takes all of our collective voices to make a difference and raise awareness for rare diseases like sarcoma.  We sincerely hope that you will consider coming to our nation’s capital and join us in this effort.

Tony Ferlenda
Chief Executive Officer

Thursday, November 8, 2012

ACTION ALERT: Tell Congress to Protect the NIH & FDA from Devastating Budget Cuts

The election is over, and the status-quo remains.  Republicans remain in control of the House of Representatives, while Democrats retain control of the Senate and White House.

When Congress returns to work next week for the lame duck session, they must act quickly to protect the National Institutes of Health (NIH) and Food & Drug Administration (FDA) from funding cuts that would paralyze the development of treatments for patients with rare diseases.

Whether you are pleased or disappointed with the outcome of the election, we can all agree that we urgently need Congress to find a bipartisan solution to prevent sequestration.  Please take two minutes today to email your Members of Congress and President Obama to urge them to find a solution that protects rare disease research funding and ensures adequate FDA funding for review and approval of lifesaving treatments.

The potential automatic spending cuts, or sequestration, scheduled to take effect in January 2013, will be devastating for patients as well as the future U.S. leadership of research and development.  Sequestration would slash federal investments in critical health, scientific, medical and biological research aimed at discovering treatments, moving safe and effective new medicines to market, and creating the innovations to grow our economy.

If Congress does not reach an agreement  – which they have been unable to do for a year – automatic, across-the-board cuts of $1 trillion to nearly all government programs will go into effect.  This would mean that the NIH and FDA are each at risk of an 8% budget cut, slowing progress toward developments of new treatments and cures.  We simply cannot allow this to happen.  Have your voice heard!
Please click here to contact your Members of Congress and urge them to protect funding for the Food & Drug Administration & National Institutes of Health! 

For more information on Sequestration visit RareAdvocates.org.

Thursday, June 28, 2012

Calling All Natural Healers...

For all you acupuncturists, massage therapists, and yoga instructors out there, here is an excellent opportunity for you to expand your knowledge on using your art to help cancer patients achieve an improved quality of life. 


8th Annual Oncology Training Conference for Acupuncture, Massage and Yoga Teachers

July 18-20, 2012

The University of Texas MD Anderson Cancer Center
Conference Center – Floor 8, Dan L. Duncan Building (CPB)
1155 Pressler Street, Houston, Texas

Click on the link above for more details.

Friday, April 6, 2012

URGENT - Please take action TODAY - Tell the Senate to include the FAST Rare Disease Language in PDUFA

The Senate’s discussion draft for the Prescription Drug User Fee Act (PDUFA) was released late 
Wednesday.  Unfortunately, the rare disease language from the ULTRA/FAST Acts that allows improved
 access to the Accelerated Approval process for rare diseases was not included.

We need your help to ensure the language will be included in the second draft of PDUFA.  
Senators Harkin (D-IA) and Enzi (R-WY), as the Chairman and Ranking Member of the 
Senate HELP Committee, have the power to include this important language in PDUFA.  
Please take 5 minutes TODAY to call Chairman Harkin’s and Ranking Member Enzi’s offices and 
tell them why this is important to you.

We will only be successful if YOU take ACTION immediately!  Senators Harkin & Enzi need to 
receive hundreds of calls before 5pm EST today from advocates to understand that it is important for 
rare disease patients.  YOU can make the difference!  Make your calls and then please share this action
 alert via email, Twitter and Facebook.

Thank you for you for taking action and for your continued support.
Below are the numbers for Harkin & Enzi’s offices.  Please speak with the staff member who answers the
 phone, using the script below.  If no one answers, leave a voicemail.  If the voicemail box is full, call one
 of the district offices.  Keep trying until you are sure they received your message.  If you want a direct 
response, you should leave your contact information and specifically request a written response.  
Be respectful to the staff, but express your urgent concern.  
Chairman Harkin (D-IA) 
DC Office:  202-224-3254
District Offices: 319-365-4504, 563-322-1338, 515-284-4574, 563-582-2130, 712-252-1550      
(if you live in Iowa, please say so on the call)

Ranking Member Enzi’s (R-WY)
DC Office:   (202) 224-3424      Toll-Free:  (888) 250-1879     
District Offices: (307)261-6572, (307)772-2477, (307)527-9444, (307)682-6268, (307)739-9507      
(if you live in Wyoming, please say so on the call)

Sample Scripts:
Parent/Family/Caregiver/Friend:  I am a parent of a child with a rare disease that has no treatment.  
I am calling you because I’m concerned that the Senate’s version of PDUFA does not include language
 to improve access the Accelerated Approval Pathway for rare diseases.  This language, found in HR 4132
 the FAST Act is essential to spur the development of lifesaving treatments for my child.  Please make sure
 the rare disease language is included in the final version of PDUFA.  My child will die without treatment.
Patient:  I’m calling today because I have a rare disease and I deserve a treatment.  The Senate has failed
 to give rare diseases access to the Accelerated Approval pathway in the draft PDUFA language.  
The language being circulated is missing essential language from HR 4132 the Faster Access to 
Specialized Treatments Act that fixes a catch-22 for rare diseases.  I demand that the Senate listen to the 
voices of the patients!
Physician/researcher/industry:  I am a physician working to treat rare diseases calling because I’m 
concerned that the Senate failed to include language in the PDUFA draft that allows rare diseases to 
have access to the Accelerated Approval pathway.  These diseases are so rare that it will be extremely 
difficult to ever get this treatment to patients without access to the Accelerated Approval Pathway. 
 Please include the language from HR 4132 that was included in the House version of PDUFA.
Please share how your calls went on our Facebook event page:  http://on.fb.me/TreatmentsNow or 
respond to this email letting us know that you took action. 
More information on PDFUA:
The Breakthrough Therapies & EXPERRT Acts were included in the Senate’s draft along with the 
Accelerated Approval language in TREAT (S.2113).  While this is very important for rare disease patients,
 it is not enough.  The language, as it is written in the Senate version does not encourage the FDA to allow
 achievable criteria to qualify surrogate endpoints for rare diseases, leaving us with the same limited access
 to Accelerated Approval pathway that we have now and does not place any pressure on the FDA to 
change the status quo.

Currently, the House draft version of PDUFA does include the ULTRA/FAST language that is needed to
 spur the development of treatment for rare diseases.  While the Senate does not include our language, 
the discussion draft is not final; we still have time to influence the Senate’s language.  We must have 
our language in both the House & Senate versions of PDUFA or we risk having our language debated 
in Conference Committee where we have little influence and anything could happen.  In order to be
 includes the ULTRA language in both the House & the Senate.


Matthew Alsante
Executive Director

Address postal inquiries to:
Sarcoma Foundation of America
9899 Main Street, Suite 204
Damascus, MD 20872
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