Congressman Michael McCaul

Representing the 10th District of Texas

McCaul Statement on Passage of the 21st Century Cures Act

Jul 10, 2015
Press Release
“It’s not very often that Congress passes landmark, visionary legislation that has the chance to truly improve and save lives. The 21st Century Cures Act is one of those rare bills.”

McCaul Statement on Passage of the 21st Century Cures Act
“It’s not very often that Congress passes landmark, visionary legislation that has the chance to truly improve and save lives. The 21st Century Cures Act is one of those rare bills.”

WASHINGTON, DC – Today, following passage of H.R. 6, the 21st Century Cures Act, Representative Michael McCaul (TX-10) issued the following statement about the bill including two provisions which he spearheaded:

“It’s not very often that Congress passes landmark, visionary legislation that has the chance to truly improve and save lives” said McCaul.  “The 21st Century Cures Act is one of those rare bills.  I congratulate Chairman Upton, Representative DeGette and all of the Members who have worked so tirelessly over the past year on this extraordinary undertaking.  The 21st Century Cures Act will reverse the decade-long decline in much-needed federal investments in biomedical research at the National Institute of Health.  It also includes two provisions I am especially proud of – one to incentivize the development of new treatments for children with rare pediatric diseases through the reauthorization of the Creating Hope Act, and another which includes key provisions of the Andrea Sloan CURE Act to expand patient access to potentially lifesaving treatments and provide more regulatory certainty for drug innovators that hold them back from participating in the FDA’s compassionate use process.”

Reauthorization of the Creating Hope Act

Section 2152 of the 21st Century Cures Act reauthorizes the rare pediatric diseasepriority review voucher incentive program, which was first proposed by Representative McCaul as part of the bipartisan Creating Hope Act of 2011.  Under this program, biopharmaceutical companies are incentivized to develop treatments for rare diseases that are often less profitable than treatments for more common medical conditions.  Specifically, companies can receive a priority review voucher if they develop novel treatments for a rare pediatric disease, such as childhood cancer and sickle cell disease, entitling the company to a priority six month review of another new drug application that would otherwise be reviewed under the FDA’s standard ten month review period.  This shortened review time, which can lead to earlier market entry, is valuable to companies who can either choose to exercise the voucher on one of their own products or sell it to another company.  Vouchers has been sold for as much as $245 million.  Since its inception, the program has helped bring three new drugs to market for children with rare pediatric diseases, including the first-ever drug approved by the FDA to treat high-risk neuroblastoma, a particularly deadly childhood cancer. 

Andrea Sloan CURE Act

Sections 2082 and 2083 of the 21st Century Cures Act are modeled after H.R. 909, theAndrea Sloan CURE Act, sponsored by Representative McCaul. Named after Austin resident Andrea Sloan, who passed away in 2014 while fighting for access to a potentially life-saving treatment, this bill would make important reforms to the FDA’scompassionate use process which allows patients to access experimental drugs when all other options have been exhausted.  Sec. 2082 will ensure that biopharmaceutical companies have publicly accessible compassionate use policies, including information for patients about how to make a request for access.  Sec. 2083 requires the FDA to finalize its May 2013 draft compassionate use guidance for industry and clarify how it interprets and uses adverse drug event data in compassionate use cases – a key regulatory gray area which discourages many companies from providing patients with access to experimental drugs when appropriate.

You can watch Representative McCaul’s floor speech in favor of the bill here.

 

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