38 members of Congress sign letter to FDA calling for consistent approval processes across agency, engagement with rare disease experts


Washington (March 4, 2014) – Senator Edward J. Markey (D-Mass.) today sent a letter signed by 38 members of Congress calling on the Food and Drug Administration (FDA) to further support innovative therapies for rare diseases and unmet medical needs, by improving consistency and predictability in the approval review process. Passed in 2012 by Congress, the Food and Drug Administration Safety and Innovation Act (FDASIA) laid the groundwork for approval of 27 new medicines in 2013 alone, nearly half of which used one of FDA’s expedited approval pathways available for drugs that fill an unmet medical need. The Senators and Representatives call on the FDA to build on this success by continuing to ensure potential new medicines are guided and reviewed consistently across the agency, in order to bring more novel treatments for rare diseases into the drug development pipeline and ultimately to market. The lawmakers also call on the FDA to use its existing authority provided under FDASIA to seek input from external experts, including patients, researchers, and physicians, on rare diseases and other conditions to help the agency better understand the rapidly changing scientific landscape and ensure that it has the best, most complete information when making decisions about treatments under review. This authority, which provides rare disease experts a larger role in FDA’s review of new rare disease treatments, was authored by Senator Markey and included in FDASIA.


“Many families continue to struggle with too few options to manage serious and life-threatening diseases,” write the Senators and Representatives in the letter to FDA Commissioner Hamburg. “The development of new and more effective medical treatments often comes too slow for the individuals and the families of those who are currently afflicted.  Innovation of new and safe drugs is especially urgent for rare diseases, for which either no approved therapeutics or no cures currently exist.”  


“A larger, faster and more predictable pipeline for the approval of new and promising drugs can be a lifeline for families struggling with rare and life-threatening diseases,” said Senator Markey. “I join my colleagues in urging the FDA to prioritize new and promising treatments to help government, industry and, ultimately, families treat and manage these serious diseases.”    


A link to the letter to the FDA can be found HERE


The letter is signed by Senators Markey, Kelly Ayotte, Jeanne Shaheen, Elizabeth Warren, Charles E. Schumer, Maria Cantwell, Roger Wicker, Sherrod Brown, Ron Johnson, Jeff Sessions; and Representatives Jim McGovern, Tom Marino, Carol Shea-Porter, Jiim McDermott, Spencer Bachus, Bill Keating, Louie Gohmert, Yvette Clark, Michael McCaul, Adam Smith, Suzan DelBene, Suzanne Bonamici, Jared Polis, Chris Smith, Jim Hines, Tammy Duckworth, Katherine Clark, Mike Capuano, Mike Simpson, Stephen F. Lynch, Sam Johnson, Bob Goodlatte, Niki Tsongas, Pat Tiberi, Mike Doyle, Joe Kennedy, John Tierney, and Richard Neal.