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New Regulations Proposed for Off-label Drugs and Devices

  New Regulations Proposed for Off-label Drugs and Devices

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The off-label use of drugs and medical devices — using approved remedies in unapproved ways — has long been a part of medicine, providing public-health benefits but also presenting some risks to patients. For the most part, the U.S. Food and Drug Administration (FDA) allows physicians to prescribe drugs and devices off-label in the same way they are prescribed for their approved uses, requiring healthcare providers to make their own decisions about using drugs off-label, often in the face of uncertain evidence.

Researchers from the David Geffen School of Medicine at UCLA and Yale University now have proposed a system combining reporting, testing and enforcement regulations and allowing interim periods of off-label drug prescriptions. Their recommendations would give patients more treatment options, while providing regulators with evidence of the drugs’ safety and efficacy.

Much off-label prescribing of medications and medical devices is beneficial, but without rigorous study, it is difficult to know what works and what doesn’t, says Ryan Abbott, MD, JD, visiting assistant professor of medicine. “Even though a drug or device has been approved for one indication, physicians can prescribe it for other uses as well; it’s been part of medical practice for a long time,” Dr. Abbott says. “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 authors’ proposal is comprised of three elements:

• Improved reporting of off-label use through the disclosure of diagnostic codes in reports to the FDA, in detailing data that pharmaceutical companies obtain on physicians’ prescribing habits, and in reports to the FDA and Medicare/Medicaid reimbursement requests.

• Expansion of post-market testing requirements for off-label use of drugs and medical devices.

• A tiered labeling system for drugs consisting of “red-box” warnings that prohibit certain off-label uses; informed consent from patients receiving prescriptions for off-label use of some drugs that currently carry “black-box” warnings, which identify drugs that pose a significant risk of serious or life-threatening adverse effects; and the creation of a new “grey-box” warning 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 authors write. “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.”

“Evidence and Extrapolation: Mechanisms for Regulating Off-Label Uses of Drugs and Devices,” Duke Law Journal, December 2014


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