The trade-off between impartiality and freedom in the 21st Century Cures Act

Abstract

Randomized controlled trials test new drugs using various debiasing devices to prevent participants from manipulating the trials. But participants often dislike controls, arguing that they impose a paternalist constraint on their legitimate preferences. The 21st Century Cures Act, passed by US Congress in 2016, encourages the Food and Drug Administration to use alternative testing methods, incorporating participants’ preferences, for regulatory purposes. We discuss, from a historical perspective, the trade-off between trial impartiality and participants’ freedom. We argue that the only way out is considering which methods improve upon the performance of conventional trials in keeping dangerous or inefficacious compounds out of pharmaceutical markets