23 March 2012

Advocacy in Action: The EXXPERT Act

There is a really important bill coming through right now in the House that I have been asked to help share information about...  

It is the "Expanding and Promoting Expertise in the Review of Rare Treatments (EXPERRT) Act" (H.R. 4156) in the House of Representatives. 

This EXPERRT Act legislation aims to enhance the Food and Drug Administration’s (FDA) rare disease drug review process by including systematic consultation with experts and patient advocates.  

According to the Cystic Fibrosis Foundation, the bipartisan EXPERRT Act of 2012 would complement FDA’s existing authority by allowing "rare disease experts, academic researchers, health professional organizations, patient advocates and care providers greater opportunity to offer input and advice to the FDA on rare diseases and drugs and by helping FDA officials navigate a rapidly changing scientific landscape, ensuring that they have the most up-to-date, specialized information when making decisions about rare disease treatments under review."

"The EXPERRT Act makes it easier for review staff to access specialized input and expertise on issues like the risks and benefits of potential therapies, the design of clinical trials and medical needs not met by existing treatments.  This increased collaboration will make the FDA review and approval process more efficient, allowing patients to access needed treatments more quickly."

This is a bill that, while it will help those with Cystic Fibrosis, will help all of those with diseases waiting for medications to be approved by the FDA. 

There is an easy way for those in support of this EXPERRT Act can help get it passed:

1. Follow this link and send a pre-written letter to Congress. 
2. It asks your representatives to support a no-cost bipartisan bill that will save many lives.
3. It does this by making the FDA work more efficiently and more effectively
4. It WILL bring a cure to Cystic Fibrosis that much closer when passed.
5. All you need is 2 minutes to have your voice heard and to make a difference

(Thanks to my friend Peter for putting the following simple steps together for me to share!)

The EXPERRT Act is expected to reach the House floor to be voted on in the next two weeks.  So, I am watching closely to see what happens!  


  1. Wow - Bennett is so lucky to have you as his mom and all you do for him!

    I found your blog through Tube Fed Kids Deserve to eat site. My daughter is scheduled to go to the inpatient program at Baylor's Our Children's House in Dallas in April.

    I appreciate your blog because I have already skimmed over your son's time there, and it looks like he is eating a lot better. This gives me encouragement.

    I know you're busy, but I'd love to hear if overall you're happy with the treatment he received at OCH at Baylor.

    I've read from other parents who are NOT supportive of Baylor's program, but I am still planning to go and hopeful!
    Thanks! Elizabeth

  2. Done and done! I love doing what I can to help!

  3. Wow! They make it so easy. Love and miss you all.


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