Statement from the Cystic Fibrosis Foundation on the House Energy and Commerce Committee's Health Subcommittee Approval of the EXPERRT Act

Legislation Would Increase Patient and Expert Participation in FDA Review of Rare Disease Treatments

BETHESDA, Md., May 10, 2012 /PRNewswire-USNewswire/ -- The Cystic Fibrosis Foundation issued the following statement regarding the House Energy and Commerce Committee's Health Subcommittee approval of legislation to include consultation with rare disease experts and patient advocates as part of the U.S. Food and Drug Administration's (FDA) systematic drug review process. [Details provided below.]

Statement from Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation:

"On behalf of the millions of Americans with cystic fibrosis and other rare diseases, we are heartened that the voice of patients and experts will be heard as more sophisticated therapies for complex rare diseases are advanced. The Cystic Fibrosis Foundation applauds members of the House Energy and Commerce Committee's Health Subcommittee and bill sponsors Representatives Edward Markey, Tom Marino and Cliff Stearns for their support of the EXPERRT Act. This legislation will ensure that FDA reviewers have the most complete information when evaluating new treatments and help bring these therapies more quickly to the patients who need them."

Comments from Bill Sponsors

• According to the lead sponsor of the EXPERRT Act, Congressman Edward J. Markey (D-MA): "The inclusion of the EXPERRT Act in the FDA Reform Act ensures that sharing knowledge amongst the FDA and individuals with expertise in rare diseases will become common practice. 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."
• Fellow sponsor Congressman Tom Marino (R-PA) said:  "As the father of a daughter who has a rare disease, I applaud the House Energy and Commerce Committee's Health Subcommittee's inclusion of the Expanding and Promoting Expertise in Review of Rare Treatments (EXPERRT) Act of 2012 in the reauthorization the Prescription Drug User Fee Act (PDUFA).  It is critical that we continue to work to ensure that the voice of patients and other experts are an integral part of the Food and Drug Administration's (FDA) drug review 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 these devastating diseases."
• Sponsor Congressman Cliff Stearns (R-FL) said:  "EXPERRT represents a successful collaboration between the public and private sector in accelerating the process of getting new drugs and treatments to patients with cystic fibrosis.  As the Co-Chair of the Cystic Fibrosis Caucus, I appreciate the efforts of the Caucus's Co-Chair, Rep. Ed Markey, in building support for this measure and I look forward to getting this bill approved quickly by the full Energy and Commerce Committee and to the House floor."

Background on the EXPERRT Act

• On May 10, the House Energy and Commerce Committee's Health Subcommittee approved the EXPERRT Act (Expanding and Promoting Expertise in Review of Rare Treatments Act of 2012). The legislation would enhance the FDA's drug review process by including systematic consultation with rare disease experts and patient advocates.
• The EXPERRT Act was considered as part of the ''Food and Drug Administration Reform Act of 2012,''a legislative package to reauthorize the Prescription Drug User Fee Act (PDUFA), the FDA's system for funding the evaluation of new prescription drugs.
• Introduction of this legislation follows the recent approval of Kalydeco™, a CF drug developed by Vertex Pharmaceuticals with major financial, scientific and clinical support from the CF Foundation. The FDA approved Kalydeco three months after it was submitted for review. This swift review process benefited greatly from expertise provided by the Foundation and its partners, as well as patients and other experts.
• The CF Foundation's aim is to establish the best practice seen with Kalydeco's FDA review as the standard, not only for CF treatments, but for all rare disease drugs.
• Senator Sheldon Whitehouse (D-RI) spearheaded companion legislation in the Senate, which was approved by the Health, Education, Labor, and Pensions (HELP) Committee on April 25.

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 CF 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 CF Foundation is a donor-supported nonprofit organization. For more information, go to www.cff.org.

SOURCE Cystic Fibrosis Foundation

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