The Creating Hope Act, Section 908 of the FDA Safety and Innovation Act of 2012, 21 U.S.C. Sec 360ff.

On July 9, 2012, the President signed into law the FDA Safety and Innovation Act and with it, the first legislation to address the dire need for new drugs developed expressly for children with cancer and other life threatening diseases — the Rare Pediatric Disease Priority Review Voucher Program, otherwise known as the Creating Hope Act.

The Creating Hope Act has wildly exceeded our hopes.  On Valentines Day, February 14, 2014, the FDA awarded the first Creating Hope Act voucher to BioMarin for Vimizim to treat Morquio A syndrome.

Then, on March 10, 2015, the FDA awarded the second voucher to United Therapeutics for Unituxin, the most exciting advance in neuroblastoma, a pediatric cancer,  in 40 years, according to the New England Journal of Medicine.

The third voucher was awarded to Asklepion Pharmaceuticals for the approval of Cholbam to treat kids with rare bile acid synthesis disorders.

In addition, two vouchers have now sold at attractive valuations.  BioMarin sold its voucher on July 31, 2014 to Sanofi/Regeneron for $67.5M.  Then, on November 19, 2014, Knights Therapeutics sold to Gillead, a tropical disease voucher, which has the same valuation as a pediatric voucher, for a whopping $125M!

These valuations are proof of the principle that good risk adjusted returns on investment can be made by investing in drug development for pediatric rare diseases, including cancer. These successes are already having a profound impact on the landscape of pediatric cancer research.

We have heard from numerous companies that are now increasing the priority they put on pediatric drug development because of this market incentive. Moreover, we have heard from many academic centers and philanthropic organizations that intend to use this program to finance their current and future research.

Pursuant to the terms of the Creating Hope Act, the program will expire a year after the third voucher, which we now know will be March 14, 2016.  Kids v Cancer is now working with Congressmen G.K. Butterfield, Michael McCaul and Chris Van Hollen on the Advancing Hope Act, HR 1507, which was introduced on March 25, 2015, to permanently reauthorize the Creating Hope Act.

 

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