Washington, DC – With so much at stake for the 1 in 10 Americans living with a rare disease, the FDA’s Rare Disease Innovation Hub could spur the development of new innovations for the 95 percent of rare diseases with no FDA-approved treatment. It has incredible potential to increase collaboration with patients, leverage cross-agency expertise and advance regulatory science on behalf of individuals living with rare diseases; prioritizing the right activities will ensure that happens.
The comment letter below presents our feedback on the Hub’s priorities. We are confident that with these recommendations in mind, the FDA’s new model for rare disease collaboration can fulfill its mission and promise.
We look forward to continuing to work with FDA to support the Hub and our shared commitment to improving the lives of Americans living with rare diseases.
Read the full letter here.