BIO Commends FDA Plan to Establish a Rare Disease Innovation Hub to advance rare disease collaboration, treatments

July 17, 2024 – BIO President and CEO John F. Crowley today released the following statement after the U.S. Food & Drug Administration (FDA) announced a plan to establish a Rare Disease Innovation Hub that will utilize new and collaborative approaches to expedite the development and approval of safe and effective drugs and biologics that treat rare diseases. With one out of every 10 people in the United States affected by a rare disease, BIO is supportive of initiatives that accelerate the FDA’s and industry’s ongoing efforts to improve the lives of millions of people with a rare disease: 

“By elevating the focus and urgency on rare diseases at the FDA, agency senior leaders are sending a strong message to patients, caregivers, and researchers everywhere of the importance of developing newer and better medicines for every person living with a rare disorder. As a parent of two children with a rare disease, I know how difficult it can be to find information – much less treatments – for a rare condition. This announcement from the FDA, and the personal commitment and leadership of Dr. Cavazzoni and Dr. Marks, is a watershed moment for the rare disease community. BIO welcomes this important news and looks forward to engaging with the FDA and providing input into how the innovation hub can be the success that it needs to be for so many who need hope.”

More information about the FDA’s new Rare Disease Innovation Hub can be found on the FDA’s website HERE. The FDA will hold a public meeting this fall to share further information about the new hub and solicit information from all interested parties.