[Congressional Record Volume 162, Number 147 (Wednesday, September 28, 2016)]
[Senate]
[Page S6209]
From the Congressional Record Online through the Government Publishing Office [www.gpo.gov]




                         THE ADVANCING HOPE ACT

  Mr. CASEY. Mr. President, today I wish to speak about S. 1878, the 
Advancing Hope Act. This is a bill I introduced with the support of my 
Republican cosponsor, Senator Johnny Isakson. This is a bipartisan bill 
that brings hope to some of our most vulnerable citizens: children 
living with rare diseases.
  Despite significant unmet medical need, private companies seldom 
pursue new therapies for rare diseases because it requires making an 
investment in products that will likely not recoup the high costs 
associated with their research, development, marketing, and 
distribution. Developing products for children is particularly 
challenging because of the difficulties associated with conducting 
clinical trials in this population.
  So, several years ago, former Senator Brownback authored the Creating 
Hope Act with Senator Sherrod Brown. In 2011, I became the Senate 
leader on this bill, which provided an incentive for drug developers to 
pursue therapies for rare pediatric diseases. The goal was to bring 
hope to the millions of American children living with a rare disease.
  Provisions based on the Creating Hope Act were included in the Food 
and Drug Administration Safety and Innovation Act, which became law in 
2012. The new FDA program established three ``priority review 
vouchers'' that would be awarded to companies who develop a new drug 
for a rare pediatric disease. A company that earns a voucher can then 
sell it to another company, which can use the voucher to speed up the 
FDA's review time for one of its own new drugs. Companies that earn and 
then sell their vouchers can use that money to fund additional drug 
development to treat rare pediatric diseases. So far, seven vouchers 
have been awarded, including on the 19th of this month.
  However, the pediatric priority review program is due to expire on 
September 30, just days away. In fact, it would have expired in March 
of this year, but Congress passed an extension through the end of 
fiscal year 2016 as the House and Senate worked on legislation to 
extend and improve the program. In July of 2015, I introduced S. 1878, 
the Advancing Hope Act, with Senator Isakson, to extend the pediatric 
priority review program. We had extensive consideration of the bill in 
the Committee on Health, Education, Labor, and Pensions, which voted 
favorably on the Advancing Hope Act in April 2016. Thanks to an 
agreement we reached here in the Senate on September 21, we were able 
to pass S. 1878 with an extension of the program through the end of 
this year, which included important policy changes to the program. I 
would like to thank my colleagues for working with me on this 
agreement. I hope that we will be able to come to further agreement 
later this year for a longer extension to the program.
  The pediatric priority review program is important for families, and 
a longer extension is warranted. If this program is allowed to lapse, 
Congress will have broken faith with these children with rare diseases.
  Last year, I went to the Children's Hospital of Pittsburgh and met 
with the Rinaldi family. I met Jennie Rinaldi and her daughter Adelyn, 
who was receiving treatment at the hospital. Adelyn was born with 
congenital hypophosphatasia, an extremely rare bone disorder. There are 
only a handful of children in the world with this disease.
  At the time, Adelyn was receiving an experimental therapy for her 
condition. That drug, Strensiq, was later approved by the FDA in 
October 2015, and the drug sponsor received a priority review voucher. 
Strensiq is the first drug to treat hypophosphatasia. There are no 
other options. It is now available commercially, and Adelyn continues 
to receive treatment.
  Just imagine for a moment the uncertainty that families like the 
Rinaldis live with every day. We owe it to these families to give them 
the peace of mind in knowing that this important incentive for drug 
development will continue. We cannot let this program expire. I am 
pleased that the House passed S. 1878 yesterday, and I hope that we can 
continue to work in good faith on a longer-term extension before the 
end of the year.
  We need to provide certainty for drug developers so that they can 
count on this incentive when deciding to invest the time and money into 
drugs for rare pediatric diseases. We need to provide hope for the 
other children like Adelyn. On behalf of these children, we must 
incentivize companies to take on the challenges of developing new 
treatments for rare pediatric diseases.

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