Researchers Propose Changes to FDA’s Regulation of Off-Label Medical Device Use

University of California-Los Angeles and Yale University researchers are proposing changes to the U.S. Food and Drug Administration’s (FDA) role in the use of pre-approved drugs and medical devices. According to researchers, new regulatory responses would make the current system more dynamic and effective for both doctors and developers.

The FDA prohibits medical device manufacturers from publishing literature or promotional material for an intended use not preapproved by the agency. However, no regulations exist to ensure that doctors prescribe a device for patient use in exact accordance with the device’s labeling.

Ryan Abbot, visiting assistant professor of medicine in UCLA’s division of general internal medicine and health services research, explains to UCLA Newsroom: “Even though a drug or device has been approved for one indication, physicians can prescribe them for other uses as well—it’s been part of medical practice for a long time.”

The UCLA Newsroom article explains that the FDA cannot require approval for every off-label use of a product because to do so would create an enormous regulatory burden. Instead, the FDA elects to leave these decisions to the discretion of the prescribing physician.

Abbot, who also is an attorney, collaborated with Ian Ayres from Yale Law School to write an article in the Duke Law Journal. The authors contend that the freedom physicians currently possess in prescribing is not fully utilized to maximize benefits for both the patient’s wellbeing and the product’s development.

Abbot remarked to UCLA Newsroom that both he and Ayres sought to establish a balance between safety and progress. “Our proposals are important because there is a tension between providing access to the drugs and devices that could benefit patients in untested ways and the need to prevent harmful uses,” he said.

In their article, Abbot and Ayres proposed three new approaches.

First, they support a system that would require physicians to report the off-label use of a product to the FDA (with personal patient information redacted), so the data might be shared with both academic institutions and pharmaceutical companies for their research.

Secondly, the authors recommend that the FDA should expand its current post-market testing requirements.

Lastly, the authors propose a new labeling system that would allow off-label information to appear in a product’s labeling literature with important limitations. Certain off-label uses could be completely prohibited, and others could require doctors to obtain informed consent. Under this system, manufacturers could make off-label suggestions conditional on a commitment to further post-market research.

“The FDA, armed with better information about the extent of off-label use and adverse effects, would be in a better position to require post-market testing and to discourage off-label use with new types of warnings if manufacturers fall to provide sufficient, timely evidence of safety and efficacy in that particular extrapolation,” the authors wrote, according to the Duke Law Journal.

Last February, the FDA issued a draft guidance (a clarified revision of a 2009 guidance) recognizing the need for manufacturers to distribute newly discovered, peer-reviewed evidence for off-label uses of their products, but the guidance reinforced the supremacy of the FDA’s pre-market review process.

According to the agency, the existing process allows the “FDA to be proactive, rather than reactive, in protecting the public from unsafe or ineffective medical products.”