Should a placebo group be required for FDA trials?
Click Here to Manage Email Alerts
Click here to read the Cover Story, "Perseverance, personnel needed to begin clinical trials."
Yes. Placebo arms are important
The major purpose of control groups is to allow for discrimination of patient outcomes by the observer.
The control group experience tells us what would have happened to patients if they had not received the test treatment. If the course of a disease was uniform, and we knew what to expect, we would not need control groups. However, most situations are, obviously, not that simple.
One of the benefits of the placebo control is that it is a little easier to set a randomization protocol in place. If there is already a known active treatment that is being used, sometimes things can be known about that treatment. In placebo-based controls, it is easy to randomly assign patients and mask accordingly. A placebo can have the exact same appearance as the drug you are studying.
Additionally, in placebo-based trials, the treatment drug can be titrated to the effect of tolerance and can be given in one or more fixed doses. This allows you to understand the effect of dosing tolerance given that you have eliminated the possibility of a placebo effect through your control group.
Having a placebo control also allows you to study influences, such as spontaneous change or the natural history or regression of the disease. A placebo control shows us the difference between the efficacy and in the safety measure, as well.
It is important to remember that the use of a placebo control group does not necessarily imply that the control group is untreated, especially in the context of an FDA trial. You have to consider the “add-on studies,” in which a placebo and the new treatment are added to a common standard therapy. That way, the control group is not untreated and allows for a multiplicity effect of studying the same set of patients.
Eric D. Rosenberg, DO, is an ophthalmologist at SightMD in New York.
No. They are not the best option for patients
There is considerable ethical debate about the use of placebos in clinical trials, and a lot of that has to do with if the patients perceive that they are receiving no treatment.
If a treatment is available for a disease state and you are studying a new drug for that disease vs. basically nothing — a placebo — ethically, it can be argued that you are not treating the patient, and that can violate the idea of “do no harm.”
In almost every study that we have done, the control group has gotten some form of treatment. That could be a treatment that is known to be top of line or a lesser-known treatment that still provides some relief to the patient. For example, that could mean providing artificial tears vs. using cyclosporine. In glaucoma, it is normal to compare the new treatment to standard of care rather than using a saline eye drop or doing a sham procedure.
You do not want to pose additional health risks by enrolling a patient in a clinical trial and giving them a placebo if a treatment does exist. If a treatment does exist, you are ethically obligated to provide that treatment to the patient. Patients should never feel like they are in harm’s way by having a placebo.
An active control trial can demonstrate efficacy by showing that the new treatment is similar in efficacy to a known effective treatment. If I were to participate in a trial but not getting the experimental drug, I would want to know that I am at least being treated with the standard of care. These active control trials can provide a lot of value.
Alanna Nattis, DO, FAAO, is an ophthalmologist at SightMD in New York.
Collapse
Read more about
Click Here to Manage Email Alerts