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CF Foundation Applauds Passage of the EXPERRT Act in House of Representatives to Increase Patient and Expert Participation in FDA Review of Rare Disease Drugs

June 21, 2012

The Cystic Fibrosis Foundation applauds the passage of the Expanding and Promoting Expertise in the Review of Rare Treatments Act, better known as the “EXPERRT” Act, in the U.S. House of Representatives.

Introduced by Reps. Edward Markey (D-MA), Tom Marino (R-PA) and Cliff Stearns (R-FL), the legislation enables expanded consultation between the Food and Drug Administration (FDA) and external rare disease experts and patient advocates during the FDA drug approval process.

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 EXPERRT Act 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.”

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 benefitted 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.
 
“As new promising cystic fibrosis treatments come through the pipeline, we hope they will be reviewed with the same speed and agility as Kalydeco,” said Beall. “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.”

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