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7.1. Case Study #1

7.1.1. Part 1

You are a neurologist and neuroscience researcher at a busy teaching hospital. For the past five years you and your team have been working on a deep brain stimulation (DBS) project. Patients with movement disorders are referred to you by their treating physicians after their doctors have exhausted all available conventional methods of treatment. When providing the referrals, the treating doctors explain to their patients that they have essentially run out of options, and the patient's best chance for treatment now rests with more experimental therapy.

You recognize that some of the patients are too sick to give proper consent, so you also discuss the treatment with their primary caregivers. You explain that DBS could successfully control extraneous movements, making the patients less dependent on their caregivers and more capable of caring for themselves.

In your discussions with the patient and the family caregivers, you mention the risks of the procedure: the surgery, electrode placement, and the neurostimulator. At the same time, you describe the potential benefits both for the patient and for the caregiver. You successfully enroll several more patients into your study.

You run a very profitable laboratory, and the news of your achievements with DBS has spread to your colleagues. At your patients' follow-up visits to adjust their devices, you conduct in-depth interviews with them, providing information that you heuristically plug back into your practice. These patient meetings provide regular feedback on both the devices and the lab, and as a result you have very high patient satisfaction statistics, and these satisfied patients recommend you to their friends and colleagues. Your program serves as a model for the rest of the hospital and neurology interns clamor for assignments to you and your team.

7.1.2. Part 2

While your lab deals almost exclusively in movement disorders, particularly Parkinson's disease, patients with psychiatric disorders, such as intractable depression, obsessive- compulsive disorder, anxiety, and addiction are being considered for experimental protocols using deep brain stimulation (DBS). Based on some preliminary data of DBS, there have been several case reports indicating modest positive effects on patients with certain psychiatric disorders, so several other neuropsychiatric practices want to explore DBS protocols for psychiatric diseases.

You consult with the hospital IRB before you begin new collaborative studies with your colleagues. You describe the program as a pilot study to determine the effectiveness of DBS in controlling distressing and disabling symptoms of refractory psychiatric disorders including depression, anxiety, panic, and addiction.

At the IRB meeting, you describe the informed consent procedure in which you will detail the risks of the procedure, as well as the benefits to the individual patients, society as a whole, and future sufferers of this same condition. You will tell the patients that the rationale for this exploratory study is the success of DBS in movement disorders. You will go on to explain that this procedure is safer and more conservative than the brain surgeries for mental disorders in the past, and the effects of the procedure are reversible by shutting off the pulsing device if the patient is unhappy with the outcomes.

DBS is a more controlled procedure than the "ice-pick" lobotomy, and the electrode can be removed surgically or the stimulator turned off if necessary. In neither procedure, however, does one know the actual residual and unintended effects produced by interfering with neural circuitry. After lobotomies were performed on an ad hoc basis for over forty years, they were found not to be as useful or therapeutic as once assumed. As we are now in the nascent era of using DBS for this indication, we should ascertain at the start that such a procedure is indeed warranted, safe, and efficacious. Preliminary evidence appears promising.

In reviewing your application, the IRB is concerned that you have no way of determining the efficacy of this program.

The IRB asks you how you intend to assess the placebo effect of having the surgery (for the cases).

You tell the IRB that all the patients who volunteer for the trial will have the surgery to implant the electrode and the pulse-making device, but some patients will by chance (random assignment) have the device turned on where as the other patients who have been assigned to the control arm will not have the device turned on.

Before voting on your proposal, the IRB asks you if anecdotal evidence is sufficient for evaluating the success of DBS in mental disorders. You answer that anecdotal evidence is not sufficient to determine the success of a novel therapy. The value of the randomized controlled trial is that you will control for confounders and determine if the perceived success is quantifiable and statistically significant. Additionally, the RCT controls for factors like the Hawthorne Effect, where individuals may show improvement -- not because of the new intervention, but simply because they are being observed and made to feel special.

The IRB votes to approve your proposal as long as you provide a full informed consent process and include numerous safeguards to enhance the safety and promote the welfare of the participants who have agreed to accept the potential risks as well as the benefits of your DBS trial.