Archives for: 2016

Washington, D.C., September 30, 2016 – Today, President Obama signed the Advancing Hope Act (S. 1878), extending the Rare Pediatric Disease Priority Review Voucher (PRV) program until December 31. “On behalf of the 15 million children with rare diseases, we thank the President for signing the Advancing Hope Act so that some of our most vulnerable […]

Washington, D.C., September 29, 2016 – The National Organization for Rare Disorders (NORD), the leading independent nonprofit organization representing the 30 million Americans with rare diseases, released the following statement from President and CEO Peter L. Saltonstall following President Obama’s signature of the Continuing Resolution today. “On behalf of the 30 million Americans with rare diseases, […]

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 […]

More than 300 organizations join forces to ask Congress to provide robust, sustained, and predictable budget increases for the NIH Washington, D.C., September 22, 2016— The National Organization for Rare Disorders (NORD) is one of more than 300 organizations participating in the Rally for Medical Research Hill Day on September 22, uniting millions of Americans […]

The Hill, a top U.S. political website, read by Congressional offices, the White House, political pundits, and more, today published the following op-ed by NORD President and CEO, Peter L. Saltonstall, urging Congress to reauthorize the Rare Pediatric Disease Priority Review Voucher (PRV) program. The PRV program encourages the development of therapies for children with […]

The National Organization for Rare Disorders submitted comments to the Institute for Clinical and Economic Review (ICER) in response to their call for comments on how to improve their value framework development process. You can find NORD’s comments here. People with rare diseases have a uniquely important role to play in value framework development. Almost every input […]

By Peter L. Saltonstall One of the most successful pieces of legislation ever passed by the Congress is the Orphan Drug Act of 1983 (ODA).  It was intended to stimulate the research and development of new therapies for rare diseases, which were generally neglected by the research community and the drug industry. Since 1983, more […]

NORD has published a new guide to help people and caregivers living with rare diseases understand expanded access and how to access unapproved medicines. In the guide, NORD answers frequently asked questions including how to determine if you are eligible for expanded access, how to apply, and what you can expect throughout the application period and […]

On July 15, the Food and Drug Administration released the performance goals letter for the reauthorization of the Prescription Drug User Fee Act, also known as PDUFA VI.  The PDUFA program is the agreement between the FDA and the pharmaceutical industry to pay user fees to facilitate the review of pharmaceutical products. PDUFA must be reauthorized […]

UPDATE:  July 11, 2016 Illinois HB 4576 is close to passing!  This bill would establish a Rare Disease Commission that would give patients – many of whom are too young or sick to speak for themselves – a voice in the state government.  The bill is awaiting Governor Bruce Rauner’s signature before it is passed […]