More careful regulation of FDA's Humanitarian Device Exemption in psychiatric patients needed

Warning that patients could be at risk, an interdisciplinary group of multinational investigators is calling on the U.S. 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 the February issue of the journal Health Affairs, published today, lead author and Weill Cornell Medical College ethicist Dr. Joseph J. Fins says that the FDA's Humanitarian Device Exemption (HDE) 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 (DBS) in patients with treatment-resistant obsessive-compulsive disorder (OCD), 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 Dr. Fins, the E. William Davis Jr., M.D. Professor of Medical Ethics, chief of the Division of Medical Ethics and professor of medicine, professor of public health and professor of 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 Dr. 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 commentary's contributing authors, from the U.S. and Europe, have expertise in psychiatry, neurosurgery, neuropsychology, philosophy, ethics and the law.

Congress established the humanitarian device exemption in the Safe Medical Devices Act of 1990. The law sought to help bring novel devices to the market to treat uncommon conditions that otherwise might not attract the attention of device manufacturers. The act required that in order to receive such an exemption, the device must be intended to diagnose or treat conditions that affect 4,000 or fewer people in the U.S. annually.

Devices considered under this exemption are not subjected to the rigorous study required of other devices intended for use in common disorders. Those devices undergo an Investigational Device Exemption (IDE) review, which subjects the device to clinical trials that gauge both safety and effectiveness. A clinician's use of an IDE has to be approved by the institutional review board (IRB), and further requires informed consent from all patients and clear labeling that the device is considered investigational.

If the FDA's Center for Devices and Radiological Health approves a humanitarian device exemption, as requested by a manufacturer, that device can be marketed without evidence of effectiveness. Clinicians go through their local IRB for approval to use the device, but patients do not sign a research informed consent document and may not clearly understand that the device to be used has not been evaluated for clinical benefit, the authors say. This can confuse patients and foster a therapeutic misconception about an experimental intervention, especially when device manufacturers market the device as a new therapy. Furthermore, studies are never conducted to clarify how the mechanism of action works, which the authors believe is a lost scientific opportunity for discovery.

Specific Recommendations

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 similar to what is required in an IDE application. Finally, to protect patients, they suggest IRBs 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 Dr. 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."

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