Science-based medicine rests on twin pillars that are utterly essential to the development of treatments that are safe and efficacious. Both of these pillars depend on science, but in different ways. The first of these is, of course, the basic science that provides the hypotheses to test about the mechanisms behind the diseases and malfunctions that plague the human body. This basic science suggests ways of either correcting or alleviating these malfunctions in order to alleviate symptoms and prevent morbidity and mortality and how to improve health to increase quality and quantity of life. Another critical aspect of basic science is that it also provides scientists with an estimate of the plausibility of various proposed interventions, treatments and cures designed to treat disease and improve health. For example, if a proposed remedy relies upon ideas that do not jibe with some of the most well-established laws in science, such as homeopathy, the concepts behind which violate multiple laws of physics and chemistry, it’s a very safe bet that that particular treatment will not work and that we should test something else. Of course, the raison d’être of this blog derives from the unfortunate fact that in today’s medicine this is not the case and we are wasting incredible amounts of time, money, and lost opportunities in order to pursue the scientific equivalent of fairy dust as though it represented a promising breakthrough that will save medicine, even though much of it is based on prescientific thinking and mysticism. Examples include homeopathy, reiki, therapeutic touch, acupuncture, and much of traditional Chinese medicine and Ayurveda, all of which have managed to attach themselves to medical academia like kudzu.
Of course, basic science alone is not enough. Humans are incredibly complex organisms, and what we consider to be an adequate understanding of disease won’t always result in an efficacious treatment, no matter how good the science is. Note that this is not the same thing as saying that utter implausibility from a scientific basis (as is the case with homeopathy) doesn’t mean a treatment won’t work. When a proposed treatment relies on claiming “memory” for water that doesn’t exist or postulates the existence of a “life energy” that no scientific instrument can detect and the ability to manipulate that life energy that no scientist can prove, it’s a pretty safe bet that that treatment is a pair of fetid dingo’s kidneys. Outside of these sorts of cases, though, clinical trials and epidemiological studies are the second pillar of science-based medicine, in particular clinical trials, which is where the “rubber hits the road,” so to speak. In clinical trials, we take observations from the laboratory that have led to treatments and test them in humans. The idea is to test for both safety and efficacy and then to begin to figure out which patients are most likely to benefit from the new treatment.