The Creating Hope Act to incentivize pediatric rare disease drug development was passed in 2012 as a pilot program. After a resoundingly successful few years, the Creating Hope Act is now set to expire on March 18, 2016.
We are thrilled to report that in June, 2015, The U.S. House of Representatives passed 21st Century Cures, H.R. 6, and with it, the reauthorization of the Creating Hope Act, otherwise known as the Rare Pediatric Disease Priority Review Voucher Program, 21 U.S.C. Sec 360ff.
A thank you from all sick children to Chairman Upton, Congresswoman DeGette, and Congressmen Butterfield and McCaul for their leadership in ensuring the reauthorization of the Creating Hope Act.
We are also pleased to report that Senator Casey and Senator Isakson have introduced the Advancing Hope Act, S. 1878, to reauthorize the Creating Hope Act. We urge the Senate to pass this bill as a provision of the Innovations for Healthier Americans Bill or otherwise.
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, four 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. Most recently, United Therapeutics has sold their pediatric priority review voucher to AbbVie for $350M!
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.