New RD Hub

On July 17, 2024, the FDA announced that they are establishing a Rare Disease Innovation Hub (the Hub).  The Hub will work with all rare diseases, but will “focus on products intended for smaller populations or for diseases where the natural history is variable and not fully understood.”  This focus is particularly important since development of products for the treatment of these diseases is particularly difficult.  The FDA says that the Hub will have three primary functions: 1) Serve as a single point of connection and engagement with the rare disease community; 2) Enhance intercenter collaboration to address common scientific, clinical and policy issues related to rare disease product development; and 3) advance regulatory science with dedicated workstreams for consideration of novel endpoints, biomarker development and assays, innovative trial design, real world evidence, and statistical methods.  We look forward to the implementation of this new Hub for rare disease and the opportunity to leverage the FDA’s extensive experience in collaborating with patients, health care professionals, and companies developing therapies for rare diseases.  This new effort should lead to new and innovative approaches to product development for rare diseases which will ultimately provide much needed therapies for patients who are living with one of the 10,000+ rare diseases in the U.S.