Health and Disability

FDA is committed to expanding access to safe and effective treatment options for patients with rare, debilitating, and sometimes fatal diseases. These patients face unique medical challenges. Sometimes there isn’t an FDA-approved drug to adequately address the needs of a patient with a rare disease. Therefore, the agency needs to take new steps to enable more patients with unmet needs to get access to promising treatments prior to full FDA approval.

Two examples of the recent steps FDA has taken in pursuit of these goals are improvements we made to our Expanded Access Program and our Orphan Drug Program. These programs are high priorities of mine. They address the needs of patients with some of the most challenging conditions. Making sure there’s a close relationship with the efforts we take to expand pre-approval access to promising treatments, and the work of our orphan drugs program, is a key step toward maximizing opportunities for patients. To further achieve these goals, we’re announcing that FDA is widening the scope of the new Expanded Access Navigator tool, a comprehensive online information resource maintained by the nonprofit Reagan-Udall Foundation to facilitate pre-approval access to drugs. Previously this tool was rolled out for drugs that treat cancer. It will now apply to drugs that treat orphan diseases.