Clinical Trials 101

The last few months have demonstrated that we all need to understand the process to approve medical treatments. The body is a complex organism and it is not always easy to determine why we get better or worse. Is it a "natural" trend? Did I actually recover faster? Did it prevent other symptoms? Did it have any side effects? The list of questions is endless.

A clinical trial is a process that is used to answer these and other related questions. Essentially it is a planned experiment designed to remove biases and determine the efficacy (effectiveness) of a treatment. It requires that we assess the outcomes in a statistically significant number of comparable individuals. Conclusions are driven by statistics, with no room for anecdotal evidence. 

Individuals are selected at random. Control is established by grouping people based on the stage of illness, and ideally similar or no concurrent illness. Additionally, outcomes can be influenced by the patient or the investigator's expectations. This is managed by using a placebo (inert) treatment in a blind study. The blind element ensures that bias is removed from the patient (single-blind), the investigator (double-blind), and data clean-up personnel (triple-blind).

This randomized, double-blind, placebo-controlled trial (RDBPCT) is considered to be the gold standard in determining the efficacy of any treatment. It is expensive and time-consuming. Compromises are made to reduce costs and increase speed. However, as a consumer, it is important to understand the framework so that we can wade through all the information and claims and reach good conclusions.