[Congressional Record Volume 160, Number 123 (Friday, August 1, 2014)]
[Extensions of Remarks]
[Pages E1321-E1322]
From the Congressional Record Online through the Government Publishing Office [www.gpo.gov]




       FIRST RARE PEDIATRIC DISEASE PRIORITY REVIEW VOUCHER SOLD

                                  _____
                                 

                         HON. MICHAEL T. McCAUL

                                of texas

                    in the house of representatives

                         Friday, August 1, 2014

  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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