FDA package misses separate opportunity to include Markey bill to close loophole in flawed medical device approval process
WASHINGTON, D.C. – Today, the House Energy and Commerce Committee approved legislation from Congressman Edward J. Markey (D-Mass.), which promotes access to treatments for rare diseases by expanding opportunities for rare disease experts to provide input to the Food and Drug Administration (FDA). Rep. Markey’s bill, H.R. 4156 The Expanding and Promoting Expertise in the Review of Rare Treatments (EXPERRT) Act of 2012, was included as part of legislation to reauthorize the FDA’s Medical Device and User Fee Act. However, the legislative package passed today does not include any provisions to address the faulty FDA approval process that enables defective medical devices to be implanted in patients, causing serious injury and death. In February, Rep. Markey introduced H.R. 3847, the Safety Of Untested and New Devices (SOUND Devices) Act to close a major loophole in the device approval process and allow FDA to reject a device if it is based on an earlier product that had to be recalled from the market for causing serious harm to patients.
Rep. Markey’s EXPERRT Act provides researchers, academics, patient advocates, and other rare disease experts a larger role in FDA’s review of new rare disease treatments. Today’s medical breakthroughs don’t often come in the form of blockbuster drugs; they generally target rare diseases or even small subsets of rare diseases. The EXPERRT Act will provide support for reviewers at the FDA who are being asked to review a growing pipeline of very specialized treatments.
“The inclusion of the EXPERRT Act in the larger FDA User Fee Act package ensures that sharing knowledge amongst the FDA and individuals with expertise in rare diseases will become common practice,” said Rep. Markey, senior member of the Energy and Commerce Committee. “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 rare disease treatments to market quicker and more easily.”
Rep. Markey’s EXPERRT legislation was inspired by FDA’s recent approval of a treatment for cystic fibrosis manufactured by the Massachusetts-based Vertex Pharmaceuticals, Inc. During the product’s review, the Cystic Fibrosis Foundation lent their knowledge about the disease and the design of clinical trials to facilitate the review process. FDA approved the new treatment, Kalydeco, in just three months – one of the fastest review times in FDA’s history. Rep. Markey is the Chairman of the Congressional Cystic Fibrosis Caucus.
“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,” said Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation. “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.”
The EXPERRT Act is supported by 64 rare disease groups, including the Cystic Fibrosis Foundation and the National Organization for Rare Disorders. A letter of support can be found HERE.
While Rep. Markey praised the inclusion of several provisions he authored, he expressed his disappointment that the legislative package fails to include the SOUND Devices Act and misses an opportunity to improve the safety of medical devices and protect patients from serious harm.  In February, Rep. Markey released the comprehensive new report titled “Defective Devices, Destroyed Lives: Loophole Leaves Patients Unprotected from Flawed Medical Devices” that highlights the federal loophole that requires the FDA to clear medical devices that demonstrate their similarity to an earlier model, even if that previous model was recalled for a major safety defect.
“While this important legislative package contains several provisions that I have authored, I am disappointed that it misses an important opportunity to vastly improve the safety of medical devices and protect patients from serious harm. As long as this loophole remains open, patients will continue to be at serious risk of injury from defective medical devices that shouldn’t be on the market.”