Headed to president’s desk before Sept. 30 deadline

U.S. Senator Johnny Isakson, R-Ga., today applauded the U.S. House of Representatives for unanimously passing legislation to extend a critical program that will help bring faster treatments and cures to children with rare diseases.

The legislation will extend the Food and Drug Administration’s Pediatric Rare Disease Priority Review Voucher program through December 31, 2016, to continue incentivizing the research and development of life-saving treatments for rare pediatric diseases. This program was previously set to expire this Friday, September 30, 2016. The legislation, a short-term version of Isakson’s Advancing Hope Act, passed the U.S. Senate unanimously on September 21, 2016, and with today’s House action, it now heads to the president’s desk for his signature.

“I am pleased that the House of Representatives took immediate action to prevent this program from expiring,” said Isakson, a member of the Senate health committee. “I look forward to a longer-term extension of the Pediatric Rare Disease Priority Review Voucher Program, along with passage of the broader legislation that supports medical research and innovation. We must take the right steps to help find the right cures for our youngest patients.”

“If you’re the parent of a child with a devastating rare disease, Senators Isakson and Casey’s program increases the likelihood that your child will be able to take a drug that will help him or her,” Senate health committee Chairman Lamar Alexander, R-Tenn., said. “I’m grateful that the House has acted quickly to pass the Senate’s agreement, and I hope the president will sign this extension without delay to prevent this crucial 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.”

Isakson last week spoke on the Senate floor to urge support for his legislation, the Advancing Hope Act, and to share a story about Alexa Rohrbach, a young Georgian suffering from a rare cancer who came to see Isakson in 2004 to ask for his help in finding a cure. Rohrbach passed away in 2008, but Isakson said that her memory is “a reminder of why getting a result on bills like this one is as critical as anything we could ever do in the United States Senate.”

Isakson introduced the Advancing Hope Act of 2015, with Sens. Bob Casey, D-Pa., Sherrod Brown, D-Ohio, and Mark Kirk, R-Ill. It was passed out of the Senate health committee on March 9, 2016, during the committee’s second of three meetings on the Senate “Cures” agenda to deliver safe and effective treatments and cures to patients more quickly.

The legislation continues a program that was set to expire on September 30, 2016, that spurs research and investment for the development of treatments that do not currently exist for rare pediatric diseases. If a company develops a drug for one of these diseases, and it’s approved, the company gets a voucher they can keep or sell that will speed up the review of another drug.

Related:

Senate Passes Short-term Agreement to Continue Accelerated Treatments, Cures for Children with Rare Diseases

Isakson Discusses Advancing Hope Act of 2015 at Senate HELP Committee Hearing