![]() 4-6 Many of the pioneers of the movement established active n-of-1 trial units in academic centers, only to abandon them once funding was exhausted. N-of-1 trials were introduced to clinicians by Hogben and Sim as early as 1953, 3 but it took 30 years for the movement to find an effective evangelist in the person of Gordon Guyatt at McMaster University. ![]() 2 N-of-1 trials are a specific form of randomized or balanced designs characterized by periodic switching from active treatment to placebo or between active treatments ("withdrawal-reversal" designs). In the schema of Perdices et al., the Single Case Designs family includes case descriptions, nonrandomized designs, and randomized designs. As such, n-of-1 trials are part of a family of Single Case Designs that have been widely used in psychology, education, and social work. N-of-1 trials in clinical medicine are multiple crossover trials, usually randomized and often blinded, conducted in a single patient. In this chapter, we introduce n-of-1 trials by providing definitions and a rationale, delineating indications for use, describing key design elements, and addressing major opportunities and challenges. Of these, n-of-1 trials provide the most direct route to estimating the effect of a treatment on the individual. Patients want to know: which treatment is likely to work better for me? To generate individual treatment effects (ITEs), clinical investigators have taken several tacks, including subgroup analysis, matched pairs designs, and n-of-1 trials. ![]() 1 In addition, these clinical experiments generate average treatment effects, which may not apply to the individual patient some patients may derive greater benefit than average from a particular treatment, others less. However, the majority of patients may be ineligible for or unable to access such trials. The foundational, gold-standard research design in EBM is the randomized, parallel group clinical trial. The goal of evidence-based medicine (EBM) is to integrate research evidence, clinical judgment, and patient preferences in a way that maximizes benefits and minimizes harms to the individual patient.
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