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Misuse of FDA Exemption Law May Harm Vulnerable Psychiatric Patients

Warning that patients could be at risk, an interdisciplinary group of multinational investigators is calling on Congress and federal regulators to tighten a law that permits use of brain devices to treat rare neuropsychiatric disorders without supporting clinical trials or stringent patient oversight.

In Health Affairs, lead author and Weill Cornell Medical College ethicist Joseph J. Fins, MD, says that the FDA's Humanitarian Device Exemption regulation should be revised so that uses of such devices in neuropsychiatric patients meet the highest standards of clinical research, patient safety, and research integrity.

The authors voice concern that the humanitarian approval allows device manufacturers to seek a "simpler, cheaper, and faster approval process" while depriving patients of "the rigorous trial that the severity and complexity of their condition warrants, as well as hampering scientific discovery."

They make the case for revision by examining how the law was used to approve deep brain stimulation in patients with treatment-resistant obsessive-compulsive disorder, which they argue is not a rare disorder. The researchers say such misuse of the law could open the door to a number of other inappropriate applications of what was intended to be compassionate legislation designed for orphan disorders that affect only a few thousand Americans a year.

"We believe there needs to be more careful regulation of the use of the Humanitarian Device Exemption in psychiatric patients," says Fins, the E. William Davis Jr, MD Professor of Medical Ethics, chief of the division of medical ethics and a professor of medicine, public health, and medicine in psychiatry at Weill Cornell Medical College.

"We want to ensure that only orphan diseases are included in this exemption and that safety information is collected from every patient treated with these devices," adds Fins, who also serves as director of medical ethics at NewYork-Presbyterian Hospital/Weill Cornell Medical Center and chairs its ethics committee.

"Stimulating various areas of the brain in different ways to treat a variety of disorders could produce a wide array of effects, but under the Humanitarian Device Exemption, there is no requirement for clinicians to obtain efficacy data or systematically collect information on adverse events or even to ensure that patients are protected to the fullest extent possible," he says. "That has to change."

The authors are not advocating for elimination of the humanitarian device exemption but are calling for more careful regulation of its use in psychiatric research so that critical safety information is not side stepped.

They suggest that in approving such future applications, the FDA should make sure the disorder in question is indeed rare. But they also suggest that device manufacturers be required to collect safety data and place it in a central repository, as well as develop a plan to monitor safety data. Finally, to protect patients, they suggest review boards enlist the assistance of advocates who can help ensure patients are competent enough to provide informed consent, understanding that the clinical benefit of the device has not been proven.

"Currently the safety and efficacy data collected from researchers who obtain a humanitarian device exemption remains in a silo—no one else sees it," says Fins. "Requiring clinicians to compile data into a central repository can highlight unexpected side effects as well as benefits and possible other uses. This is the least we can do for our patients."

— Source: NewYork-Presbyterian Hospital/Weill Cornell Medical Center/Weill Cornell Medical College