Kids v Cancer Announces "The Advancing Hope Act" To Reauthorize The Pediatric Priority Review Voucher Program

23 Mar, 2015, 20:56 ET from Kids v Cancer

WASHINGTON, March 23, 2015 /PRNewswire-USNewswire/ -- Kids v Cancer is pleased to announce that today, Representatives GK Butterfield, Mike McCaul, Chris Van Hollen, Mike Kelly and Steve Cohen have introduced the Advancing Hope Act to reauthorize the pediatric priority review voucher program of the Creating Hope Act, currently set to expire on March 14, 2016.

Kids v Cancer, a pediatric cancer non-profit, championed the Rare Disease Priority Review Voucher Program (also known as the Creating Hope Act, 21 USC 360ff), which created priority review vouchers as incentives for drug development for rare pediatric diseases, including pediatric cancers.

The Act was passed as a pilot program and has so far awarded vouchers to three companies: BioMarin for Vimizim to treat Morquo A Syndrome; United Therapeutics for Unituxin to treat a pediatric cancer, neuroblastoma; and Asklepion for Cholbam to treat rare bile acid synthesis disorder.

The most recent voucher, which was a tropical disease voucher and has an equivalent value to a pediatric voucher, sold for $125M.

"This is what I had envisioned and hoped for when I started working on the Creating Hope Act six years ago," said Nancy Goodman, Founder of Kids v Cancer.  Goodman's passion for advancing in the Creating Hope Act grew from her experience with the inadequate 40-year old treatments her son, Jacob, received.  Jacob died of a pediatric cancer at age 10."

"It is critical to create incentives for companies to develop drugs expressly for children with cancer and other life threatening illnesses," Goodman said.  "The Creating Hope Act and the Advancing Hope Act are a wonderful way to keep fighting for Jacob and millions of other children. We have proven the concept, now let's make it permanent."

Goodman noted that the Advancing Hope Act is an important first step toward bringing life saving drugs to children, but a more complete response is required.

With this in mind, Kids v Cancer is working on the Kids' Innovative Drugs (KIDS) Initiative to enable kids with cancer to benefit from the almost 900 drugs in the adult cancer pipeline.  "We think one of the best ways to help kids with cancer is to ensure that they can be treated with the most promising drugs, even if those drugs are sometimes drugs developed for adult cancers but not yet approved for juvenile use, or drugs that were developed for adult cancers but abandoned and may work for children," said Goodman.

Kids v Cancer is a 501(c)(3) organization focused on changing the landscape of pediatric cancer research by identifying structural impediments at key junctures in the research process – new drugs, tissue donation and access to funding – and developing strategies to address them.

SOURCE Kids v Cancer



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