VERY FEW DRUGS APPROVED FOR PATIENTS WITH LIFE THREATENING, RARE AND ULTRA-RARE DISEASES

WASHINGTON, DEC. 20, 2011 – “A rare disease in the United States is a condition that affects fewer than 200,000 people, and according to the U.S. Food and Drug Administration (FDA) about 30 million Americans are afflicted with a rare disease,” said Rep. Cliff Stearns (R-FL).  “Because the diseases are rare, there is a disparity in access to drugs and treatments for these rare diseases.  In addition, under normal market conditions there is no incentive for the pharmaceutical industry to develop and market drugs for patients suffering from rare and ultra-rare diseases.  That is why these drugs are called ‘orphan’ drugs.”   

In 1992, the FDA created an Accelerated Approval process to make new drug available to treat dire conditions that lack alternative therapies. However, the modern FDA’s approval rate for drugs or medical devices has slowed immensely.  Explained Stearns, “I held a hearing earlier this year on medical devices and in discussions we found that the FDA has not used all the tools available to them to help bring new drugs to market to treat rare and ultra-rare diseases.”

“I have introduced H.R. 3737, the Unlocking Lifesaving Treatments for Rare-Diseases Act (ULTRA), to promote the discovery and development of safe and effective drugs and treatments to prevent, diagnose, or treat rare and ultra-rare diseases,” added Stearns.  “This legislation will improve access to the existing accelerated approval pathway for patients with life threatening ultra-rare genetic diseases with the added attribute of promoting private investment in new biotechnology companies and job growth in the United States.”

Over 90 groups advocating for rare and ultra-rare disease patients have endorsed Stearns’ legislation.