Researchers Propose New Off-Label Drug Regulations

System would provide more treatment options for patients along with evidence of drug safety and efficacy for regulators.

Researchers at UCLA have developed a new system for prescribing drugs off-label that would provide patients with increased treatment options while offering enhanced evidence of safety and efficacy for pharmaceutical manufacturers.

The FDA currently allows off-label prescriptions of drugs and devices similarly to how they are prescribed for approved uses. The FDA is unable to require approval for each off-label application due to the fact that the burden for approval would be so great that a minimal amount of off-label uses would be granted. This would block patients from receiving effective treatments from drugs for purposes which they weren't originally intended for.

Due to this hurdle, health care providers must make decisions regarding the use of off-label drugs with insufficient and uncertain evidence.

To overcome this issue, researchers have proposed a system that combines reporting, testing, and enforcement regulations. The system would permit interim periods of off-label drug prescription to offer regulators with better evidence for the safety and efficacy of off-label drug uses.

While much of the off-label prescribing of medications and medical devices is beneficial, a lack of rigorous study makes it difficult to determine the optimum applications.

"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," said study co-author Ryan Abbott, MD, in a press release. "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."

The new proposed regulations would combine several factors. It would provide improved off-label usage reporting through the disclosure of diagnostic codes in reports to the FDA. The system would also provide data that pharmaceutical manufacturers obtain on the prescribing habits of physicians, and data on FDA and Medicare/Medicaid reimbursement requests, while omitting details that could identify individual patients.

This data could subsequently be shared with academics and pharmaceutical companies to use for research.

The proposal also includes expanded post-market testing requirements for off-label uses of drugs and medical devices; a tiered labeling system for drugs with warnings prohibiting specific off-label uses; informed consent from patients who receive prescriptions for off-label use of drugs that currently carry warnings that identify drugs with a significant risk of serious or life-threatening adverse effects; and a new warning label that blocks Medicare Part D and Medicaid reimbursements by the Centers for Medicare and Medicaid Services.

"The improved reporting, testing and enforcement regulation would work together to produce a more layered range of regulatory responses," the study authors wrote. "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 fail to provide sufficient, timely evidence of safety and efficacy in that particular extrapolation."