Bill to Accelerate Development of Treatments for Patients with Unmet Medical Needs

WASHINGTON, Feb. 15, 2012 /PRNewswire-USNewswire/ -- The National Organization for Rare Disorders (NORD) supports proposed legislation introduced today by U.S. Senator Kay Hagan (NC) intended to accelerate the development of medical treatments for people with unmet medical needs.

Known as the TREAT Act, the bill focuses on regulatory innovation in areas such as the accelerated approval and fast-track processes to speed the development of therapies for patients with serious or life-threatening diseases.

"We've worked very hard with Senator Hagan and her staff to ensure that this bill would accomplish the dual challenges of accelerating the process while also keeping in place important safeguards related to safety and efficacy," said NORD President and CEO Peter L. Saltonstall.  "We are grateful to Senator Hagan and her staff for listening to the concerns and needs of the patient community.  We look forward to supporting their efforts as this bill makes its way through Congress and … we hope … ultimately is implemented."

NORD represents the 1 in 10 Americans who have rare diseases, most of whom currently have no Food and Drug Administration (FDA)-approved treatment.  Since most rare diseases are serious, and the patients are children in about two-thirds of the cases, there is a particular sense of urgency in accelerating the process of bringing safe, effective new therapies to market, Saltonstall said.

Of 7,000 known rare diseases, fewer than 250 currently have FDA-approved therapies.  The TREAT Act is intended to accelerate the review and approval process for medicines that:

• Treat an unmet medical need
• Significantly advance the standard of care or
• Are highly targeted therapies for serious or life-threatening diseases or conditions

SOURCE National Organization for Rare Disorders (NORD)

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