By: Wendy Tate, PSM, CIP

The February 2011 issue of Health Affairs published an article criticizing the Food and Drug Administration (FDA)’s humanitarian device exemption (HDE) for deep-brain stimulation in patients with treatment-resistant obsessive-compulsive disorder (OCD). According to FDA regulations, HDEs are granted to devices that are intended for conditions affecting fewer than 4,000 patients annually.

This article, using information from the National Institute of Mental Health (NIMH), claims that there are approximately a half-million people suffering from this form of OCD, more than are allowed if a treatment device is to qualify for an HDE, and enough to do the appropriate clinical trials to approve the device through the premarket approval (PMA) process.

The information in the article is compelling, and the FDA will need to evaluate the allegation and decide whether any modifications need to be made. In the meantime, though, what are the institutional review boards (IRBs) supposed to do?

Multiple IRBs have reviewed and approved this device as a humanitarian use device (HUD). Patients (remember, this isn’t research) have gone through invasive surgery of the brain to receive this device. If an IRB determines that the approval needs to be revisited, what are their options? The IRB could determine that the HDE does not apply to the condition and disapprove the device as an HUD.

However, there are no open clinical trials for this device in treatment-resistant OCD, and it is unclear if Medtronic (the device’s manufacturer) will open one. Disapproval will effectively eliminate the ability to use the device at an institution. An IRB could approve the device again under the HDE regulations, but is that sound in terms of regulations and ethics? As long as the FDA continues allowing deep-brain stimulation, an IRB can state that the determination concurs with the regulations. A more subjective conclusion surrounds the ethics.

One argument to continue providing the device under the HDE regulations is that it provides treatment to the patient. However, rigorous testing to meet FDA approval criteria for effectiveness has not been done. Is it ethical to say that a device that has little or no effectiveness testing behind it can treat a person? You might as well say that you can be cured by a drug that looks good on paper. Should the manufacturer be forced to withdraw access to the device until structured clinical trials can be performed? Can the information obtained from patients who have already received the device be used to fast-track the PMA application?

Lucky for me, I don’t have to answer these questions. That is left to the FDA and the IRBs. The article’s authors have presented a thorough and well thought out argument that the FDA cannot brush aside. IRBs should not dismiss this paper either, as it raises ethical issues surrounding these invasive devices that have, at the most, minimal effectiveness testing behind them.