WASHINGTON, D.C. - U.S. Senator Kay R. Hagan (NC) today honored Rare Disease Day and urged her colleagues to join her in supporting the Transforming the Regulatory Environment to Accelerate Access to Treatments (TREAT) Act to get targeted treatments to patients with rare or life-threatening diseases, in a quick and safe manner.

"More than one in ten Americans suffer from rare diseases and of the 7,000 known rare diseases, fewer than 250 have FDA-approved therapies," said Hagan. "For patients suffering from rare diseases for which there are no current treatments, medical advances can't come fast enough. Today, on International Rare Disease Day I urge my colleagues to join me in supporting the TREAT Act. This bill creates a consistent process and a clear, safe and effective pathway at FDA that will encourage the development and accelerate the approval of innovative treatments for patients with rare and life-threatening diseases. Supporting the TREAT Act is a perfect way to honor Rare Disease Day."

The TREAT Act accelerates 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.

The bill enhances FDA's access to external scientific and medical expertise. It allows the FDA Commissioner to better utilize waivers when potential conflicts of interest are outweighed by the need to have input from leading medical and scientific experts. It also recommends that patient advocacy and disease research organizations have more representation on FDA advisory committees. This is critical in areas where research is on the cutting edge.

The TREAT Act advances regulatory science within the FDA. It ensures that drug sponsors are provided explanations when their drugs are turned down so that they might address concerns, improve the treatment, and get approved medications to patients more quickly.

The TREAT Act updates the FDA Mission Statement to reflect FDA's role in advancing medical innovation while promoting and strengthening the agency's safety and effectiveness standards. The bill establishes new positions to promote innovation, integration and oversight as well as a Management Review Board to help FDA keep pace with advancing medical innovation and ensure that safety precautions are adhered to.

For more background on the TREAT Act please click here.