Informed Consent

This week in my ethics class, we'll be discussing informed consent forms (ICFs) and their use in trials involving emergency medicine (EM).  Can someone in an emergency situation provide consent?  Could their next of kin/caretaker?  Personalizing from my recent adventure to the Emergency Room, I can tell you neither I nor My Beloved was in any state of mind to authorize an experimental procedure or to understand the risks associated with a research program that included placebo.

See, the deal with a placebo-controlled study is this:  you might get the placebo.  Imagine you have a disease, say toe fungus.  Fungal infections are *very* hard to treat.  There are no good medicines and those that do exist must be take for a looong time.  Being the beneficent person that you are, when approached by your doctor with a request to participate in an experimental therapy, you generously agree to participate in the hopes that your efforts will further science, spare your fellow man, yada yada.  And then you read the consent form which describes that this study includes a placebo.  Which means there is a chance that you might get exactly ZERO drugs.  Placebo effect (which is not insignificant and can be as high as 30% in some trials) and your humanitarian gesture aside, this sort of bums you out because it means your toe fungus has a change of remaining untreated for another X number of weeks.

Now, imagine you have a more serious disease, like Crohn's or cancer.  You'll get the standard of care (whatever the feds/insurance agencies agree that is), but for as long as you're enrolled in the trial, you can't get any treatments or take any medicines beyond the standard of care plus whichever product (experimental drug or placebo) you were issued through the trial.  Depending on the structure of the trial, your odds of getting the placebo vs. drug could be 50:50, or higher or lower. Of course, the trial is structured so those patients who worsen in symptoms and who were on placebo could be re-randomized (meaning they draw another lot to see if this time they get drug or placebo again).  Patients, also, are free to drop out of the study at any time.

But is it ethical to include a placebo in such a population?

As a scientist, I come down on the side of research.  To meet the FDA criteria for new drug approval and to clearly demonstrate effectiveness, a placebo-controlled arm may be essential.  As someone who has been involved in such trials, I am comfortable with and confident in the patient enrollment and discontinuation process.  One could argue for the use of placebo-controlled studies in smaller trials, only, or only in the animal-versions of such studies.  In much the same way humans screw up the best laid plans in unpredictable ways, I believe there is no animal model or synthetic assay capable of imitating the complex human system, particularly on a large scale such as that involved in clinical trials. These carefully controlled studies are imperative.

As a citizen with a close family member with MS, I believe in placebo-controlled studies almost exclusively as an option of last resort.  For a disease with many proven therapies, I have difficulty in consenting to a treatment plan that includes placebo unless all other options were exhausted.  There are no guinea pigs in my family.  Well, literally, there are, but not figuratively.  You know what I mean.