Congress Reauthorized the Creating Hope Act! Over $1 billion in incentives for pediatric rare disease drug development!
Thank you to our sponsors, Congressmen Butterfield, McCaul and Van Hollen, and Senators Casey and Isakson, for their commitment to children with cancer and other rare diseases. Thank you to the FDA for executing this critical program. The reauthorization is part of the Omnibus Appropriations Act that President Obama signed into law in December, 2015.
We aim to achieve permanent reauthorization, beyond the Omnibus’ new sunset of September 30, 2016 by supporting passage of Congress’ 21st Century Cures, which includes reauthorization of the Creating Hope Act. A thank you from all sick children to Chairman Upton, Congresswoman DeGette, and Congressmen Butterfield, McCaul and Van Hollen.
We are also pleased to report that on March 9, 2016, the Senate H.E.L.P. committee will mark up the reauthorization of the Creating Hope Act, S. 1878, as introduced by Senators Casey and Isakson in the Advancing Hope Act. Thank you Senators Casey and Isakson.
We urge the Senate to permanently reauthorize the Creating Hope Act so that drug companies can rely on these incentives in their long term business development plans for new pediatric rare disease drugs.
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 known 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.