Wednesday, June 27, 2012

Bipartisan Action Offers Rare Disease Patients Hope

A NORD press release today announced the successful results of two years of work by NORD and its membership.  I wrote about the House’s passage a few months ago, and today, after the Senate passage, the bill goes to the President for his signature.  This is a major step forward and will hopefully help those of living with rare disorders. 

The Kennedy’s Disease Association thanks all those that worked so hard to get this bill passed.


Washington DC-----Millions of Americans who have serious rare diseases and no treatment may face a brighter future as a result of a bill approved today by the U.S. Senate, according to the National Organization for Rare Disorders (NORD).

"We salute the Senate for decisive and bipartisan action in approving the FDA Safety and Innovation Act (S. 3187)," said Peter L. Saltonstall, president and CEO of NORD.  "And we applaud the House of Representatives for approving in a similarly bipartisan manner the House version of this bill last week."

Saltonstall said the legislation, which now goes to President Obama for his signature, provides broad-ranging and innovative measures to speed the development of safe and effective treatments for people who desperately need them.

"Only about 250 of the nearly 7,000 diseases considered rare in the U.S. have therapies," he said.  "But treatments are desperately needed because most rare diseases are serious, many are life-threatening, and about two-thirds of the patients are children."

He added that "NORD and its members have worked long and hard to raise awareness of the challenges of living with rare diseases.  We are grateful for the hard work and thoughtful deliberation that both the House and Senate have brought to this process."

Among others, some features of the bill that Saltonstall said were especially important to NORD and the rare disease patient community include:
  • enhancing accelerated patient access to new medical treatments
  • encouraging the development of Humanitarian Use Devices, or medical devices for small patient populations
  • providing for accelerated development of "breakthrough therapies" -- or ones that show early promise
  • enhanced consultation with rare disease medical experts and
  • a rare pediatric disease priority review voucher incentive program

The bill approved by the Senate today and the one approved by the House last week are the result of a process that has been underway for nearly two years and for which NORD has provided significant input, the reauthorization of the Prescription Drug User Fee Act (PDUFA). This legislation, which authorizes the Food and Drug Administration (FDA) to collect user fees from companies seeking to have products reviewed, must be reauthorized every five years.

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