Patient Expectations of Assigned Treatments Impact Strength of Randomised Control Trials

• Main Authors: Roberto Truzoli, Phil Reed, Lisa A. Osborne
• Format: Article
• Language: English
• Published: Frontiers Media S.A. 2021-06-01
• Series: Frontiers in Medicine
• Subjects: RCT; clinical outcome-effectiveness; patient expectations; patient variables; false negatives; Monte Carlo simulations
• Online Access: https://www.frontiersin.org/articles/10.3389/fmed.2021.648403/full

Patient engagement with treatments potentially poses problems for interpreting the results and meaning of Randomised Control Trials (RCTs). If patients are assigned to treatments that do, or do not, match their expectations, and this impacts their motivation to engage with that treatment, it will affect the distribution of outcomes. In turn, this will impact the obtained power and error rates of RCTs. Simple Monto Carlo simulations demonstrate that these patient variables affect sample variance, and sample kurtosis. These effects reduce the power of RCTs, and may lead to false negatives, even when the randomisation process works, and equally distributes those with positive and negative views about a treatment to a trial arm.