Your Voice Counts!

A Message of Hope and Proof of Concept

On March 8, 2012, my friend Marissa and I joined dozens of other advocates from across the country in Washington D.C. to talk with our nation’s Senators and House Representatives about issues important to the cystic fibrosis (CF) community.  What a thrill it was to go to Capitol Hill on the heels of the FDA’s approval of Kalydeco®; the first medicine to treat the underlying cause of CF for 4% of the CF population (those who have a certain mutatation in their CF gene called the G551D mutation).   Additionally, Kalydeco® has created a road map that will extend to treatment for 100% of CF patients.  Dr. Margaret Hamburg, FDA Commissioner, and Dr. Robert Beall, President and CEO of the Cystic Fibrosis Foundation, hail this as a huge step forward for both personalized medicine and for CF treatment.

Me, Congressman Jim Cooper, and Marissa
March 8, 2012
Image by Cystic Fibrosis Foundation

CFF Agenda

Like last year, we met with our legislators to explain how the National Institutes of Health (NIH) and Food and Drug Administration (FDA) play critical roles in the search for the cure for CF, and why it is so important that funding to these two institutions continues.  Since our meetings, a record 150 co-signers signed onto Congressman Ed Markey’s letter supporting funding for the NIH in Fiscal Year 2013.

Additionally, the CFF’s public policy agenda for 2012 seeks to expand rare drug expertise during the FDA review process.  On March 7, the day before our advocacy meetings, Congressional CF Caucus Co-Chairs Representatives Edward Markey (D-MA), Cliff Stearns (R-FL) and Tom Marino (R-PA) introduced the “Expanding and Promoting Expertise in the Review of Rare Treatments (EXPERRT) Act,” in the House of Representatives. This legislation aims to enhance the FDA’s rare disease drug review process by including systematic consultation with experts and patient advocates.  Consulting with rare disease experts will ensure that the FDA has complete information, allowing drugs to move through the pipeline quickly.  Kalydeco® is an excellent example of this process.  It was granted approval in approximately three months, making it one of the fastest FDA approvals ever.  Simply put, the EXPERRT Act will make best practice standard.

You can help!

Marissa and me at Rep. Blackburn’s office

One of the most enlightening things I learned while in D.C. was that every phone call, every letter, every email gets counted and noticed by our Representatives.  The information gathered in those communications is the baseline those offices use to know what is going on.  Please join Marissa and me in helping CFers and countless others with rare diseases.  Tell your lawmakers to support the Expanding and Promoting Expertise in Review of Rare Treatments (EXPERRT) Act of 2012! Your members of Congress will support the bill if they hear that it is important to you, your friends and your family. Email your legislators today and urge them to support the EXPERRT Act!

Please click here for the quickest, easiest way to make your voice count.

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2 Comments

  1. Hey Blake, I can’t find that note you sent me about the other CF aunt from South Carolina that you met — if you’ve still got her contact let me know.

    It’s been a little crazy here lately, my newest niece spent 3 weeks in the NICU but they sent her home, her gut is working properly and she’s already taking her enzymes like a champ.

    I get so wrapped up in the hardships surrounding CF that I forget about other rare diseases. There are twin 12-yr old boys in my church suffering from Duchenne’s MD, and I have added that disease to my prayer list. I’m trying to donate to both CF and MD now.

    We need cures for so many life-shortening, devastating diseases!

    Reply
  1. Progress! « simplyblake

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