This primer explains the history, purpose, and reauthorization process for PDUFA and why it matters for rare disease therapy development. It also outlines opportunities for patient advocates to engage in shaping future FDA priorities.
New Name, Same Commitment to Rare Diseases
The EveryLife Foundation for Rare Diseases has become The RARE Foundation! Read more about our new brand and how it unifies our advocacy work on behalf of the rare disease community.
