On May 30, President Trump signed federal right-to-try legislation, providing another pathway for desperately ill patients who want to try experimental medicines. The legislation leaves in place the existing Food and Drug Administration (FDA) expanded access/compassionate use program. As a result, patients and physicians now have two options for accessing investigational treatments for single-patient treatment uses.

Clinical Research Pathways joins scientists and more than 75 patient advocacy organizations nationwide in recommending that patients and physicians stay the course—and choose the FDA expanded access program with its built-in protections for patients.

We also remind you that we stand ready, as a resource on expanded access, to help patients, physicians, and institutional review boards (IRBs) navigate the FDA process.

Expanded access vs. right to try

What’s the difference between FDA expanded access and right to try? In a word, safety.

The FDA process includes two reviews—by FDA and an IRB—to help protect patients’ rights and well-being. At most, the reviews could delay the process by about a week, and it is time well spent.

The reviews will make sure the risks to the patient are reasonable, the patient understands those risks, and the patient has given consent. In addition, by taking the FDA expanded access pathway, the patient benefits from knowledge that is unique to FDA. Only FDA has data on the drug’s effectiveness, how it has been used in the past, and possible side effects. Furthermore, the FDA process has proved efficient. From 2010 through 2015, FDA approved 99 percent of single-patient requests for expanded access.

The right-to-try pathway is so new that guidelines are not yet in place. Both physicians and drug manufacturers have expressed uncertainty about how to proceed. For the foreseeable future, the pathway we already know is the pathway of choice. It works, and it provides the greatest possible protections for patients.

Next steps

If you’re a patient interested in trying an experimental medicine, talk with your physician and learn more at the Reagan-Udall Foundation’s Expanded Access Navigator. If you’re a physician seeking expanded access for a patient, check out our Resources for Expanded Access, including our step-by-step guide.

Marjorie A. Speers, Ph.D.

Executive Director, Clinical Research Pathways