Randomized treatment-belief trials

It is widely recognized that traditional randomized controlled trials (RCTs) have limited generalizability due to the numerous ways in which conditions of RCTs differ from those experienced each day by patients and physicians. As a result, there has been a recent push towards pragmatic trials that better mimic real-world conditions. One way in which RCTs differ from normal everyday experience is that all patients in the trial have uncertainty about what treatment they were assigned. Outside of the RCT setting, if a patient is prescribed a drug then there is no reason for them to wonder if it is a placebo. Uncertainty about treatment assignment could affect both treatment and placebo response. We use a potential outcomes approach to define relevant causal effects based on combinations of treatment assignment and belief about treatment assignment. We show that traditional RCTs are designed to estimate a quantity that is typically not of primary interest. We propose a new study design that has the potential to provide information about a wider range of interesting causal effects.