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.

Dr. Scott GottliebTwo 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.

As I discussed in my testimony at a recent hearing of the Energy and Commerce Committee, expanded access programs play an important role for terminal patients who don’t have access to conventional, FDA-approved treatments. Because many orphan diseases don’t have FDA-approved treatment options, patients with a rare disease can benefit from early access programs.

There are numerous challenges to developing treatments for patients with rare diseases. For example, it can be hard to recruit clinical trial participants because of the small patient populations. In addition, there’s no single, consolidated place for patients with rare diseases to find companies that offer drugs on an expanded access basis, and then submit the information to enroll in these opportunities. This leaves a large, unmet need for access to investigational therapies among critically ill patients with rare diseases. Many of the innovative developers of orphan drug products are small companies that may face their own obstacles making this information available. We believe that patients shouldn’t face obstacles finding out which companies offer drugs on an expanded access basis, and then face additional unnecessary hurdles in accessing these programs.

Widening the scope of the Navigator program will help maximize the opportunity for patients to get access to promising treatments through clinical trials when these treatments are otherwise unavailable, and will advance the development of new drugs in a more transparent, accessible, and straightforward manner.