FDA Launches Call Center for Compassionate Use Requests

The U.S. Food and Drug Administration (FDA) has launched a call center to field doctors’ requests for access to experimental cancer drugs for individual patients who have run out of treatment options.

The pilot program, called “Project Facilitate,” was unveiled at the 2019 American Society of Clinical Oncology annual meeting, where agency officials passed out cards providing a phone number and email address for doctors to call with compassionate use requests for drugs that the agency hasn’t approved for marketing.

Compassionate use requests for experimental treatment tend to arise when patients can’t participate in clinical trials, for reasons that range from not meeting eligibility criteria to living too far from a study site. In these cases, the pharmaceutical company decides whether to provide access to the drug, then the FDA review the request. (Between October 2017 and September 2018, the FDA approved 95% of single-patient compassionate use requests.)

The cumbersome nature of the process led 41 states to pass “Right to Try” laws in the past several years that allow patients to bypass the FDA’s expanded-access program. Last year, President Donald Trump signed a federal law to similar effect. ASH Clinical News explored the realities of “Right to Try” in an October 2018 feature story.

Project Facilitate provides a single point of contact, simplifying a process that many doctors, especially those in community oncology clinics, consider “difficult to maneuver,” Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence, told The Wall Street Journal.

The agency is urging doctors to call the new center before seeking expanded-use permission from drug manufacturers so that it can work directly with the company if the request is denied. For approvals, the FDA plans to follow up with doctors regarding treatment results, which will then be studied for the potential additional drug uses.

Dr. Pazdur said that the program is being tested for cancer drugs first, then may be expanded to drugs for other diseases.

Source: The Wall Street Journal, June 3, 2019.