Patients with rare diseases face unique challenges. Often, there may be no FDA approved treatments for them, or the patient population with an orphan disease is very small – it may be difficult to find clinical trials developing medicine for their diseases. For this reason, the FDA is expanding the use of the Reagan-Udall Foundation’s Expanded Access Navigator tool to include rare or orphan diseases. After this expansion, patients and their physicians will have the ability to use the Navigator to explore options for pre-approval access to drugs. For more on this, please read the announcement by FDA Commissioner Scott Gottlieb, MD.

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