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Sep. 23, 2016

TOPIC: Advocacy, Press Releases

Senate Passes Short-Term Agreement to Accelerate Treatments for 15 Million Sick Children with Rare Diseases

Posted by Jennifer Huron

Unanimous vote extends the Rare Pediatric Disease Priority Review Voucher Program with important policy changes through December

Washington, D.C., September 23, 2016 – On Thursday, the Senate reached an important compromise to accelerate new treatments to help 15 million sick children with rare diseases.

Led by Senators Robert Casey (D-PA), Johnny Isakson (R-GA), and Lamar Alexander (R-TN), the Senate voted unanimously to extend the Rare Pediatric Disease Priority Review Voucher (PRV) Program until the end of 2016.  The Rare Pediatric Disease PRV, supported by the National Organization for Rare Disorders (NORD), is the Food and Drug Administration’s program that incentivizes the research and development of life-saving treatments for rare pediatric diseases. The program was set to expire at the end of this month.

Before voting to extend the program, Sens. Casey, Isakson, and Alexander had attempted to pass the Advancing Hope Act (S.1878), which would have reauthorized the PRV for six years. While this vote failed, they achieved a critical compromise to extend the program and include all of the policy changes included in S.1878, including an important definition change for “rare pediatric disease.”

“The Advancing Hope Act changes the statutory definition for rare pediatric diseases because it takes into account morbidity and incidence rather than overly relying on prevalence data,” said Peter L. Saltonstall, president and CEO of NORD.  “NORD has pushed for this important change for nearly two years so that all deserving rare pediatric diseases are included.”

The compromise bill has moved over to the House with a vote likely next week.  The Senate Republicans have also included the extension of the PRV program in their Continuing Resolution proposal released yesterday.

“We urge members of the House to join their Senate colleagues and unanimously support this legislation that puts children first and protects our most vulnerable citizens,” added Saltonstall.

NORD applauds Sens. Casey, Isakson, and Alexander, and the hundreds of advocates who joined in advocating for the reauthorization of the Rare Pediatric Disease PRV program.

“With the extension, we will need advocates to join us and fight this battle again in November before the extension expires,” said Saltonstall.

The National Organization for Rare Disorders (NORD) is a 501(c)(3) independent charity and the leading independent advocacy organization representing all patients and families affected by rare diseases.

Sign up to receive policy-related news and alerts from NORD in your email.

5 Responses to “Senate Passes Short-Term Agreement to Accelerate Treatments for 15 Million Sick Children with Rare Diseases”

  1. Theresa wildes says:

    My four year old daughter has a rare incurable primary immunedeciency (cvid) and arthritis. Our family doesn’t qualify for state insurance and my daughters infusions, hospital stays, chest port surgery and other survives , amubalance trips to the hospital, genetic testing , labs, medications and many more are costly. I honest feel if anyone child has a rare disease and life threatening should have free health insurance. We can’t afford or will survive if we do not get help. I and my husband works full time and I have 4 children and two step that we provide for. Please we need your assistance. Thank you. I want to fight this with you all and would love to chat with you.

  2. Janice Smith says:

    It is very important for continuation to find a cure for these very rare terminally diseases in our children’s best interest and for that child’s extended siblings etc.

  3. Janice Smith says:

    Congratulations on getting a compromise in this very important work in finding a cure

  4. Lara Stoller says:

    I am so glad to hear the good news for this bill. I have a rare disease myself called, “MoyaMoya.” It affects 1 in every 2 million people. Mostly children. In the state of Massachusetts where I live there is a bill sitting in the Senate waiting to be passed for a bill for my disease. It was passed in the House. In my state May 6th is National MoyaMoya Day! I am so happy. There is no cure or medication for my disease. It mainly affects children and adults. In my lifetime there will probably be no cure, but they should put a lot of effort for the children. It affects the major arteries in the brain and causes strokes in adults and strokes and seizures in children. Only cure is surgery. Once you have it on one side it spreads to the other. I only had 1 surgery. It also causes migraine headches. No child should have to suffer like this! Please help me and I am glad as to what is happenening for rare diseases.

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