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The N-of-1 trial design aims to provide a definitive answer as to whether a treatment works in a particular patient. As such, the entire process of testing a treatment is personalized to that patient—from the selection of measurable outcomes to the use of data once the trial is over. The approach therefore differs from most randomized controlled trials (RCTs), which are usually geared toward answering a particular research question. Yet despite their individualized design, N-of-1 trials can also be useful in clinical research. Data collected from multiple N-of-1 trials can be aggregated and—provided that the correct statistical tools are applied—analyzed to generate population-level data about drug response, while capturing far more information about intra- and interindividual heterogeneity than most RCT designs.

TYPICAL N-OF-1 TRIAL

STANDARD RANDOMIZED
CONTROLLED TRIAL (RCT)

A patient works with her physician to develop a study design with the primary goal of...

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