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

BETHESDA, Md., April 25, 2012 /PRNewswire-USNewswire/ -- The Cystic Fibrosis Foundation issued the following statement regarding Senate committee 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 tens of thousands of Americans with cystic fibrosis and millions more with rare diseases, the Cystic Fibrosis Foundation applauds Senator Whitehouse, Chairman Harkin and Ranking Member Enzi for the significant work they are doing to enhance the FDA review process and speed patient access to new treatments.

Ensuring that patients and external experts on rare diseases are part of the FDA review process makes certain that reviewers have the most complete information when evaluating new treatments. The voice of the patient and the expert must be heard as more sophisticated therapies for complex, rare diseases are advanced."

Background on the EXPERRT Act

• On April 25, 2012, the Senate Health, Education, Labor and Pensions (HELP) Committee 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, introduced by Senator Sheldon Whitehouse (D-RI), was considered as part of a legislative package to reauthorize the FDA's system for evaluating new prescription drugs and devices.
• 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 benefitted 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.
• Representatives Edward Markey (D-MA), Tom Marino (R-PA), and Cliff Stearns (R-FL) are spearheading companion legislation in the House of Representatives.

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