Senate bill would encourage drugs targeting rare kids' diseases

A group of bipartisan senators this week introduced legislation to entice drug makers to focus more intently on cures for uncommon children's diseases.

Sponsored by Sens. Sherrod Brown (D-Ohio), Sam Brownback (R-Kan.) and Al Franken (D-Minn.), the Creating Hope Act aims to solve a nagging problem inherent to the market-driven world of pharmaceutical manufacturing: how to encourage the creation of expensive-to-develop drugs that few patients need?

The lawmakers hope to do so by offering expedited FDA approval of potentially popular drugs to pharmaceutical companies that develop treatments for rare pediatric diseases. The idea is that the financial incentive associated with bringing "blockbuster" drugs to the market sooner will encourage companies to work harder researching the lesser needed treatments.

The proposal expands on a 2007 law that offers "priority review vouchers" to companies that develop new drugs for neglected tropical diseases. Drugs yielding vouchers, under the bill, must meet the definition of "rare" established by the Orphan Drug Act, and must not have previous approval from the FDA.

The bill would also prohibit companies from getting vouchers under the 2007 law for drugs already on the market abroad. Closing that loophole would force the companies to create new treatments, rather than simply bringing old ones to market in the U.S.

The National Institutes of Health estimates that there are more than 6,000 diseases qualifying as rare under the Orphan Drug Act, a vast majority of which get little or no drug company research money because the return on investment would be so small.

"We are falling woefully and inadequately short in our efforts to cure and treat rare and neglected pediatric diseases and conditions," Brown said in a statement announcing the bill. 

Kids' healthcare advocates agree. 

"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," said Nancy Goodman, executive director of Kids v Cancer.