(Bethesda, Md.) -- 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 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.

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.  

Media Contact

Laurie Fink, national director of media relations: 301-841-2602; lfink@cff.org