You Are the Expert

Aside

Congressman Tim Murphy of Pennsylvania has a message for the cystic fibrosis community: members of Congress want to know the issues and problems that people with CF face. “No one knows these issues better than you.”

He’s absolutely right. That’s why it’s so important that you join Make Every Breath Count.

 

Sign up for Make Every Breath Count

Make Every Breath Count is the CF Foundation’s national advocacy campaign, during which advocates like you inspire action in our fight against CF by meeting with their members of Congress in their hometowns.

You can help ensure that we are able to continue vital CF research, that new therapies are approved quickly and safely, and that people with CF have adequate coverage to go to their care center and access their treatments.

Congressman Murphy says it best: elected officials are “depending upon you to be the ones to tell us” about these important issues. You are the expert.

It’s easy to get involved and we will help you every step of the way!

Thank you for all that you do. Together, we will continue to make a difference in the lives of all people with CF.

Sincerely,

Cystic Fibrosis Foundation
publicpolicy@cff.org

Rare Diseases Deserve Access to Accelerated Approval – Call Congress TODAY!

The Food & Drug Administration (FDA) recently issued a draft guidance on Expedited Approval Programs which they say fulfills the FDASIA mandate on Accelerated Approval for Rare Diseases, however the guidance does not address specific issues related to rare disease drug development as required by FDASIA.  We need to do better!

Representative Bilirakis (R-FL) is circulating a letter in Congress calling on the FDA to fully implement FDASIA and give rare disease access to Accelerated Approval.  URGENT ACTION is needed from the patient community to ask Members of Congress to sign onto the letter.

Please CLICK HERE to call your Member of Congress It’s easy & only takes 5 minutes.  Just enter your address & your Congress Member’s phone number will be displayed with a sample script you can use to ask your Member to sign onto the letter.

Access to Accelerated Approval brings down the time and cost to development treatments leading to a surge in investment and development activity for even the most rare disorders, giving more patients with rare diseases earlier access to safe and effective treatments. 

To see the Congressional Letter to FDA & to learn more about the issue, click here.

Thank you for taking action!

Emil Kakkis, MD PhD
President & Founder
EveryLife Foundation for Rare Disease