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Casey, Isakson, Alexander: Senate Extends Program to Encourage New Drugs and Treatments for Children with Rare Diseases

Call for House to quickly extend Casey-Isakson law that St. Jude Children’s Hospital in Memphis says is “key” to helping sick children get access to new treatments

WASHINGTON, D.C., Sept. 22 – Senators today reached agreement on short-term legislation to prevent an end to a successful program driving faster treatments and cures to children with rare diseases. Sens. Bob Casey (D-Pa.), Johnny Isakson (R-Ga.), and Lamar Alexander (R-Tenn.) called on the House to quickly pass the legislation.

Today’s agreement extends a law enacted in 2012 sponsored by Sens. Casey and Isakson to continue through the end of 2016 a program otherwise set to expire this month that provides drug companies an incentive to develop a drug to treat or cure rare pediatric diseases. If a company develops a drug like that, and it’s approved, the company gets a voucher they can keep or sell that will speed up the review of another drug.

“Our nation has an enduring obligation to be there for the children and families who seek treatment for rare diseases,” Senator Casey said. “This legislation will provide certainty for drug developers so that they can count on this incentive when deciding to invest the time and money into developing new drugs for rare pediatric diseases, many of which do not have a single treatment available. I’m pleased that we were able to reach consensus on extending the program through the end of the year and look forward to making additional progress in the coming weeks.”  

“I am extremely thankful that the Senate acted now to keep this critical program from expiring,” said Senator Isakson. “It is our responsibility to ensure that patients who are suffering from the rarest diseases with no treatments have hope for a cure yet to come.”

“If you’re the parent of a child with a devastating rare disease like brain cancer, this program increases the likelihood that your child will be able to take a drug that will help him or her,” Senate health committee Chairman Alexander said. “I’m hopeful that the House will also act to prevent the program from ending this year as the Senate and House work to pass the 21st Century Cures bill and provide a longer term solution so more doctors can tell parents there’s a treatment available to help their little boy or girl.”

St. Jude Children’s Research Hospital in Memphis – which provides care to approximately 7,800 children with cancer and other are life-threatening diseases every year free of charge – wrote in a letter to Senate health committee leaders Alexander and Patty Murray (D-Wash.) that priority review vouchers, like the ones enabled by the Advancing Hope Act, “provide a powerful incentive to stimulate drug development in rare pediatric diseases. These conditions often lack the market opportunity to attract significant investment … [W]e have found access to new drugs a major impediment to the progress needed to improve survival in children with catastrophic diseases including cancer.  …[W]e have witnessed strong evidence that the programs are working. Support for the voucher program is key to facilitating access to new agents important to improving outcomes like pediatric cancer.”

The Advancing Hope Act of 2015, sponsored by Sens. Casey (D-Pa.), Isakson (R-Ga.), Brown (D-Ohio), and Kirk (R-Ill.), was passed out of the Senate health committee during its second of three meetings on the Senate “Cures” agenda to deliver safe and effective treatments and cures to patients more quickly. That legislation continues the program for five years.


For access to this release and Chairman Alexander’s other statements, click here.