Statement from the Cystic Fibrosis Foundation on Passage of the EXPERRT Act in House of Representatives

Statement from the Cystic Fibrosis Foundation on Passage of the EXPERRT Act in
                           House of Representatives

PR Newswire

BETHESDA, Md., June 20, 2012

Legislation Includes Measures to Increase Patient and Expert Participation in
FDA Review of Rare Disease Medicines

BETHESDA, Md., June 20, 2012 /PRNewswire-USNewswire/ --The Cystic Fibrosis
Foundation issued the following statement in response to passage of
legislation today in the House of Representatives to expand consultation
between the Food and Drug Administration (FDA) and external rare disease
experts and patient advocates during the FDA drug approval process.

The bill is known as the Expanding and Promoting Expertise in Review of Rare
Treatments Act (EXPERRT). It was introduced by Reps. Edward Markey (D-MA), Tom
Marino (R-PA) and Cliff Stearns (R-FL). EXPERRT passed as part of the Food
and Drug Administration Safety and Innovation Act, which reauthorizes the
FDA's user-fee program that funds its drug and device evaluation.

"The Cystic Fibrosis Foundation applauds the approval of the EXPERRT Act,
which will help expedite the approval of safe and effective new rare-disease
drugs and treatments for patients by ensuring that the FDA has the most
complete information during its evaluation," said Robert J. Beall, Ph.D.,
president and CEO of the Cystic Fibrosis Foundation. "This legislation is
critically important to the 30,000 Americans with cystic fibrosis and millions
more with rare diseases who desperately need access to sophisticated therapies
for their complex conditions. We extend our special thanks to Representatives
Markey, Marino and Stearns for the critical work they have done to advance
this legislation."

"The inclusion of the EXPERRT Act in the FDA Safety and Innovation Act ensures
that sharing knowledge amongst the FDA and individuals with expertise in rare
diseases will become common practice," said Markey, co-chair of the
Congressional Cystic Fibrosis Caucus. "Information about the severity of
diseases, the risks patients may be willing to take, or improved clinical
trial designs will help facilitate the review process and bring new treatments
for rare diseases like cystic fibrosis to market quicker and more easily."

"EXPERRT will have the FDA consult with experts in rare diseases and will
ensure that they have access to the knowledge needed when dealing with drug
approvals for diseases where FDA may lack expertise in the subject matter,"
added Stearns, co-chair of the Congressional Cystic Fibrosis Caucus.

Marino, who also co-chairs the Congressional Cystic Fibrosis Caucus, said: "I
applaud the inclusion of the EXPERRT Act of 2012 in the final agreement of the
reauthorization of the Food and Drug Administration's user fee programs that
passed the House today. As the father of a daughter who has cystic fibrosis,
I understand firsthand how critical it is that we continue to work to ensure
that the voice of patients and other experts are an integral part of the drug
review and approval process. This bipartisan legislation will allow those
voices to be heard and will allow the FDA to have the most complete
information from those most affected by devastating rare diseases."

Passage of this legislation follows the recent approval of Kalydeco™, a cystic
fibrosis drug developed by Vertex Pharmaceuticals with major financial,
scientific and clinical support from the Cystic Fibrosis Foundation.
Kalydeco's swift approval, announced by the FDA just three months after it was
submitted for review, was a result of the overwhelming, conclusive clinical
evidence of its safety and efficacy. The review process benefited greatly from
expertise provided by the Foundation and its partners, as well as patients and
other experts. Kalydeco is the first CF drug to address the underlying cause
of the disease in select CF patient groups.

Beall continued: "As new promising cystic fibrosis treatments come through the
pipeline, we hope they will be reviewed with the same speed and agility as
Kalydeco. Our aim is to establish the best practice we saw with Kalydeco's
review as the standard, not only for cystic fibrosis treatments, but for all
rare disease drugs."

About the Cystic Fibrosis Foundation

The Cystic Fibrosis Foundation is the world's leader in the search for a cure
for cystic fibrosis. The Foundation funds more cystic fibrosis research than
any other organization, and nearly every CF drug available today was made
possible because of Foundation support. Based in Bethesda, Md., the Foundation
also supports and accredits a national care center network that has been
recognized by the National Institutes of Health as a model of care for a
chronic disease. The Cystic Fibrosis Foundation is a donor-supported nonprofit
organization. For more information, go to

SOURCE Cystic Fibrosis Foundation

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