Randomized Controlled Assays and Randomized Controlled Trials: A Category Error With Consequences

Abstract

In 1962, in the wake of the thalidomide crisis, a new Amendment to the Food and Drugs Act introduced Randomized Controlled Trials (RCTs) into the regulations governing the licensing of medicines. It was believed that requiring companies to demonstrate their products were effective through RCTs would contribute to safety. In 1962, RCTs were a little-understood technique. It was thought trials would produce generalizable knowledge with similar outcomes for successive trials. As a result, regulators adopted a criterion of two positive placebo-controlled trials for licensing medicine. For physicians keen to stall therapeutic bandwagons and eliminate ineffective treatments, a negative RCT result was a good outcome. When made a gateway to the market, companies, in contrast, had an interest to transform RCTs from assessments that might throw up unexpected or negative results into Randomized Controlled Assays (RCAs) that efficiently generated approvable results. This article outlines the differences between RCTs and RCAs, the steps companies took to transform RCTs into RCAs, and the consequences of this transformation.