Butterfield Seeks to Make Expediting Treatments for Rare Pediatric Diseases Permanent through Introduction of Bipartisan Advancing Hope Act

Mar 23, 2015
Press Release

WASHINGTON, DC – Congressman G. K. Butterfield (NC-01) today introduced the Advancing Hope Act of 2015, a bipartisan bill that will make permanent the Rare Disease Priority Review Voucher Program, which was created through the inclusion of the Creating Hope Act in the Prescription Drug User Fee Act (PDUFA) reauthorization. 

The Creating Hope Act, which was signed into law by President Obama in 2012 as part of the PDUFA reauthorization, expanded the cost-neutral Food and Drug Administration (FDA) priority review voucher (PRV) program, allowing pharmaceutical companies to expedite FDA review of more profitable drugs in return for developing treatments for rare diseases. 

Prior to the Creating Hope Act, the FDA had approved only one new drug for treatment of childhood cancer in the past 35 years, compared to dozens for adults.  Despite this significant unmet medical need, in the past pharmaceutical companies have been reluctant to develop drugs for rare pediatric diseases because it required making an investment in products that are unlikely to cover the high costs associated with its research, development, marketing, and distribution.  In addition, developing products for children was particularly challenging because of the difficulties associated with conducting clinical trials on this patient population. 

The Creating Hope Act helped to ease this process by incentivizing and fast-tracking much-needed pediatric drugs to market.  The bill, however, only provided three vouchers to be used to expedite treatments for rare diseases through the FDA approval process.  The Advancing Hope Act seeks to remove the three-voucher cap and would clear the way for more therapies to be approved by FDA in an expedited fashion so long as they are used to treat rare diseases.  It will also modify the definition of “rare pediatric disease” to include “any form of sickle cell disease” and “any pediatric cancers.”

“The Creating Hope Act was a historic bill that’s been wildly successful in directly addressing the lack of drug development for rare disorders,” Congressman Butterfield said.  “In a matter of months we were able to safely fast-track important rare disease treatments to market, something many thought would take years.  Let’s expand on this success by quickly passing the Advancing Hope Act so that rare disease treatments can reach the people that need them the most.”

Representatives Mike McCaul (R-TX), Chris Van Hollen (D-MD), Mike Kelly (R-PA), and Steve Cohen (D-TN) are all original cosponsors of this bill.