First Rare Pediatric Disease Priority Review Voucher Sold

Floor Speech

Mr. McCAUL. Mr. Speaker, across this nation tens of thousands of children live with rare pediatric diseases, such as childhood cancers, that threatens their bright futures. As the chairman of the Childhood Cancer Caucus, I have personally met with dozens of children and their families who are desperate for treatments.

Since the 1980s, only one new treatment has been developed for children with cancer. Sadly, for many other rare pediatric diseases, no Food and Drug Administration (FDA) approved treatment exists at all. That is why I introduced the Creating Hope Act in 2011, to create an incentive for drug companies to develop new treatments for children with rare pediatric diseases, such as pediatric cancers.

Without any cost to the taxpayer, the Creating Hope Act established a priority review voucher (PRV) for rare pediatric diseases as Sec. 908 of the Food and Drug Administration Safety and Innovation Act. As an incentive for developing a new rare pediatric disease treatment, a company can be awarded a PRV by the FDA. The voucher entitles a 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. Companies can also choose to sell or transfer the voucher to another drug sponsor.

On Valentine's Day this year the first rare pediatric disease PRV was awarded to BioMarin for Vimizim to treat Morquio A Syndrome. There are fewer than 800 kids in the United States with Morquio A Syndrome. Vimizim will help children living with this terrible disease to live a longer and healthier life. That's what every kid should have.

And just this week, BioMarin sold its voucher to Regeneron Pharmaceuticals and Sanofi for $67.5 million. This will allow BioMarin to reinvest their new earnings in even more products to treat rare and ultra-rare diseases. And it will allow Regeneron Pharmaceuticals and Sanofi to speed up FDA review of Alirocumab, an experimental drug designed to lower low-density lipoprotein cholesterol in patients who have previously tried other treatments. This is a win-win for the companies, but best of all this will benefit patients.

Mr. Speaker, I believe this news demonstrates the value of this important program. As Dr. Ned Braunstein, Regeneron's Vice President for Regulatory Affairs, said after his company announced that it was buying BioMarin's voucher, the ``decision to acquire and leverage the voucher is clear evidence that this program is a valuable incentive for biopharmaceutical companies.''

Many people deserve credit for helping to make the Creating Hope Act a reality. Congressman FRED UPTON, the Chairman of the Energy and Commerce Committee, Congressman G.K. BUTTERFIELD, Dr. MICHAEL BURGESS, the Vice Chair of the Energy and Commerce Subcommittee on Health, and former Congresswoman Sue Myrick, were all tremendous champions of this law. So too were many countless advocates, but most of all Nancy Goodman. A mother and an advocate, Nancy is the founder and executive director of Kids V Cancer, which is fighting to change the landscape of pediatric research.

More children deserve life-saving treatments which is why I hope to work with my colleagues to make the rare pediatric disease Priority Review Voucher program permanent. As a father I can think of nothing more important than investing in our children's futures.

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