ABSTRACT
In this chapter, the authors review how medical research is done. They look at the value of different types of studies, such as studies on animals and preliminary studies on humans, into the effectiveness of treatments. The gold standard for medical research is the randomized, controlled clinical trial (RCT). An RCT will include at least two groups, namely, a treatment group and a control group. The treatment group receives the new treatment being investigated while the control group receives either a placebo or an already-existing treatment. Ideas for new treatments may come from diverse lines of investigation such as epidemiology, studies of disease mechanisms, from fishing expeditions looking for new chemicals with potentially useful actions in the body, or may be borrowed from herbal treatments. The Food and Drug Administration (FDA) of the USA customarily accepts trial designs that test against a placebo. In fact, the FDA considers this trial design to be the "gold standard".
