FDA will strengthen expanded access program for non-approved treatments

FDA Commissioner Scott Gottlieb, MD, has signaled that the agency will strengthen its expanded access (EA) program to enable more patients to access investigational medical products and treatments for serious or life-threatening diseases.

Gottlieb’s commitment to the EA program follows the Right to Try (RTT) law, which allows certain patients to access investigational drugs if they have no FDA-approved treatment options. The RTT law circumvents some reporting to the FDA and Institutional Review Board to create a similar pathway to alternative treatment, according to Alison Bateman-House, assistant professor of medical ethics at NYU Langone Health. Under the EA program, the IRB reviews and approves investigational products. 

The EA program provides a pathway for patients to access alternative care treatments beyond clinical trials, including investigational drugs, biologics and medical devices for serious diseases and immediately life-threatening conditions.

The FDA has authorized more than 9,000 applications over the last five years across drugs, biologics and devices through the EA program and about 99 percent of all requests received by the agency, according to Gottlieb.

The FDA has taken steps to expand and update the program, according to Gottlieb, including streamlining the required supporting documentation for EA program requests by a physician. A patient application form for access to a drug or biological for treatment takes about 45 minutes as a result of the changes, Gottlieb said. The agency also changed the IRB process, allowing just one member of the board to review treatment use rather than the entire board.

The FDA will continue rolling out changes to enhance the EA program based on feedback from providers.

“FDA is deeply committed to our Expanded Access program and facilitating access to medical products outside of clinical trials when no alternative therapy options are available to patients,” Gottlieb said in a statement outlining the program changes on Nov. 8. “And we are deeply committed to continuing to enhance this program going forward.”

In response to the statement, industry watchdog group The Goldwater Institute voiced its support for a stronger EA program.

“Today, the FDA has acknowledged that Congress’ intent in passing the federal Right to Try legislation is to protect patient autonomy and safety,” the Institute said in a statement. “FDA Commissioner Scott Gottlieb has previously expressed the FDA’s readiness to implement the legislation according to this intent, and we celebrate the fact that patients with life-threatening illnesses have another avenue to access to the treatments they need to try to save their own lives.”