Butterfield Applauds First Voucher Approval Under His ‘Creating Hope Act'

Feb 24, 2014
Press Release

WASHINGTON, DC – U.S. Representative G. K. Butterfield (NC-01), a member of the House Energy and Commerce Subcommittee on Health, today applauded the U.S. Food and Drug Administration’s (FDA) approval of the first voucher made available under legislation he successfully included in the Prescription Drug User Fee Act (PDUFA).  The voucher was issued for Vimizim, a pharmaceutical drug that will be used to treat a rare childhood disorder called “Mucopolysaccharidosis” that leads to problems with bone development, growth, and movement.  Representatives Butterfield and Michael McCaul (R-TX) co-sponsored the Creating Hope Act, which was passed and signed into law by the President in 2012 as part of the PDUFA reauthorization. The provision provides market incentives to pharmaceutical companies to develop new drugs for children with rare pediatric diseases, such as childhood cancers and sickle cell.

“Passing the Creating Hope Act marked the first time the Congress acted to directly address the lack of drug development for rare pediatric disorders,” said Butterfield.  “I am pleased that the FDA is already issuing vouchers to manufactures for the development of drugs that will help children cope and survive these rare illnesses.”

The Creating Hope Act expanded the cost-neutral Food and Drug Administration priority review voucher (PRV) program, allowing pharmaceutical companies to expedite FDA review of more profitable drugs in return for developing treatments for rare pediatric diseases.  Since 1980, the FDA has approved only one new drug for treatment of childhood cancer, 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.