Engaging Stakeholders in Building Patient-Centered, N-of-1 Randomized and Other Controlled Trial Methods

Background: The PCORI Methodology Committee report identified the heterogeneity of treatment effect (HTE) as one of the priority methods gaps to be addressed, and we will do so by laying the foundation necessary for conducting N-of-1 randomized controlled trials (RCTs). HTEs are large for many treatments; some treatments have large benefits for some patients but are minimally effective or even harmful for others. N-of-1 RCTs address this gap by providing empirical data on each individual patient’s own treatment effect. Although N-of-1 RCTs have been discussed for decades and used occasionally to great effect, significant barriers and methodological issues currently limit their use for improving outcomes for the majority of patients in the United States.