USA – Statement from FDA Commissioner Scott Gottlieb, M.D., on new efforts to strengthen FDA’s expanded access program

Since the 1970s, the FDA has helped to facilitate access to promising investigational medical products for patients with serious or immediately life-threatening diseases who are unable to access products through clinical trials. As a cancer survivor, I understand, on a very personal level, that patients who are fighting serious or life-threatening diseases want the flexibility to try new therapeutic approaches, including investigational medical products. This is especially relevant when there’s no other FDA-approved treatment option available to a patient.

FDA is deeply committed to facilitating this access, while also protecting patients and helping them to be able to make informed decisions with their physicians. We also take steps to help make sure that such access doesn’t interfere or jeopardize investigational trials that could support a medical product’s development or timely approval for the treatment indication.

This is the mission of our Expanded Access (EA) program. EA provides a pathway for patients to gain access to investigational drugs, biologics and medical devices for serious diseases and immediately life-threatening conditions outside of clinical trials when no comparable or satisfactory approved alternative therapy options are available. We’re taking new steps to improve this framework.

Over the last five years, FDA has authorized more than 9,000 applications across drugs, biologics and devices through the agency’s expanded access program. Furthermore, we’ve authorized approximately 99% of all the requests we have received, across all application types.

FDA staff is deeply committed to this program and ensuring that it works quickly and effectively for patients and their physicians. Emergency requests for individual patients are usually granted immediately by phone. Non-emergency requests are generally processed within a few days.

Despite the success of the EA program, we recognize that there are opportunities for improvement. We’ve taken steps to expand and update the program over the last year. Many of these changes were made in response to feedback the agency received from stakeholders, as well as input from Congress, on how we can make the program more effective.

One improvement FDA made was streamlining the required supporting documentation for expanded access requests submitted by a physician for access to a drug or biological for the treatment of an individual patient. These changes reduced the administrative burden for these physicians. Following these changes, we estimate that it takes about 45 minutes, on average, to complete a patient application form. That form typically requires just one attachment…