Often in clinical research it seems like the placebo effect was put on earth solely to cause us headaches. Imagine how simple things would be without it. Controls? Who needs ’em? Just grab a bunch of random dudes with the disease and check back in a month to establish your baseline. Easy peasy.
But here’s another way of looking at it. One of the central tenets of modern EBM is that only treatment benefits above and beyond the effect of placebo (sometimes called “specific” effects) are “real” benefits worth pursuing. In other words, if your drug cured 50% of patients, but a placebo also cured 50%, then your drug had a net effect of zero and did nothing.
This is because we’d like to have medicines that work via the mechanisms we designed them to have, or at least in somewhat predictable and understandable ways. If a sugar pill cures a bunch of people, that’s very nice, but it’s not really medicine, is it? It’s some kind of mind-over-matter trollop that we refuse to accept in our wards.
There are some interesting ethical questions here. For instance, if you’re the one suffering from a painful, debilitating, or deadly disease, do you care if you’re cured by a sugar pill or an exhaustively-developed organic compound? If you feel better, can I really pop from behind a bush and say, “Gotcha! That was the fake stuff! You don’t feel better at all!” And if the placebo benefit hinges on the patient not knowing the truth, would it be ethical to start passing around fake (but effective) drugs?
The current attitude is no. This business leaves a sour taste in our collective mouths, perhaps because medicine spent so many centuries practicing exactly this sort of handwaving mumbo-jumbo. We consider ourselves scientists, and scientists treat using physiological mechanisms, not wishful thinking. Perhaps more importantly, we have an ethical problem with lying to our patients, which seems like a necessary part of intentionally wielding placebos.
But it behooves us to remember that despite all this, the placebo effect can indeed be dramatic and beneficial. That’s the whole reason we try to control for it in trials — because otherwise, every random treatment would look pretty impressive, even if there’s nothing under the hood. No benefit over placebo is often still a big benefit to the patient.
What’s my point? My point is this: in the ivory towers of EBM, we spend a lot of time stroking our beards and worrying that we’re subjecting our patients to drugs, surgeries, and interventions that are not evidence-based. Perhaps there’s even evidence that they’re ineffective, or sometimes even harmful. Until we can banish all such snake oil from our practice, we believe that we’re wasting our patients’ time and money at best and harming them at worst.
But the fact is, this probably isn’t so. Because even our worst snake oil is still convincing, and it probably still carries with it real placebo value. (In fact, this is often why anecdotal reports of treatment success abound even when the controlled trials say “nope.”) When we say that Drug X has no benefit, we mean that it added nothing compared against a placebo — but in clinical practice, we’re not comparing it against anything, we’re just giving it to sick people. In other words, it is the placebo, with all the associated power.
So even without any “specific” effect, odds are that it has some value anyway. It’s not zero, and in fact, the benefit may even outweigh some specific harms.
And so, ladies and gentlemen, we should keep fighting the good fight to rid ourselves of myth and hokum. But in the meanwhile, we shouldn’t lose too much sleep that we’re hurting anyone, because we’d have to hurt them quite a bit to outweigh that sugary placebo goodness.