"In 1992, the FDA created an Accelerated Approval process to make new drugs available earlier to treat serious diseases and fill an unmet medical need based on a surrogate endpoint," said Rep. Cliff Stearns (R-FL), a senior member of the House Energy and Commerce Committee. "However, the modern FDA's approval rate for drugs and medical devices has slowed immensely. Rep. Ed Towns (R-NY) joined me today in offering H.R. 4132, the Faster Access to Specialized Treatments (FAST) Act. This measure codifies much of the Accelerated Approval regulations into statute and modernizes the program to reflect the amount of medical and scientific innovation that has occurred in the past 20 years." This streamlined Accelerated Approval pathway also will help the rare disease community.
"A rare disease in the United States is a condition that affects fewer than 200,000 people, and according to the FDA about 30 million Americans are afflicted with a rare disease," said Stearns. "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."
Late last year, Stearns and Towns offered 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. Said Stearns, "After getting input on ULTRA, we developed H.R. 4132 and we are working with the Energy and Commerce Committee leadership on adding it to the Prescription Drug User Fee Act (PDUFA) V, which will be considered by the Committee."