Senators advocate blockbuster remedy for rare drug woes

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Lawmakers will soon get a chance to start pondering a bill that would dangle some blockbuster cash as an incentive to developers to take on the risk of developing new therapies for rare children's diseases.

The new law, which was proffered last week, would give pharma companies a shot at quicker approvals of blockbusters if they take on new drug programs that tackle rare pediatric diseases. Adding even a few months to a drugmaker's window of market opportunity could add hundreds of millions of dollars to a blockbuster drug's potential revenue.

As The Hill notes, the new bill essentially extends a three-year-old law that provides priority review vouchers to companies which work on treatments for rare tropical diseases. But the bill closes a loophole that gives biopharma companies access to vouchers on drugs that have already been approved. Supporters in Congress say they want to make sure that they're encouraging development of new therapies.

Kids v Cancer, which celebrated the new bill, says that in the past 20 years the FDA has approved only one drug cancer drug that was expressly developed for the pediatric market. "A priority for Kids v Cancer is to find ways to work with pharmaceutical companies to develop treatments for children with cancer and other rare diseases," said Nancy Goodman, the executive director of Kids v Cancer. "This legislation aligns government and private sector interests and will be a major step forward in addressing the unmet needs of children with devastating illnesses, including life-threatening cancer."

"We are falling woefully and inadequately short in our efforts to cure and treat rare and neglected pediatric diseases and conditions," said Senator Sherrod Brown, who introduced the bill with Sam Brownback and Al Franken.

- read The Hill's story
- here's the Kids v Cancer release

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