Erika Augustine, Timothy Yu, Richard Finkel (JAMA Network)
September 25, 2024
Genetically individualized N-of-1 trials have recently emerged as an approach to treating small numbers of individuals with genetically targeted interventions. These studies, involving as few as a single person treated with a novel investigational therapy, are being deployed for individuals with serious genetic conditions for which no other treatment is available.
The term N-of-1 trial dates back at least 35 years, referring to multiple crossover trial designs for single patients. These studies addressed scientific questions for which conventional trials were not ideal and generated evidence for the treatment of a single individual. Such studies involved the use of existing medications or interventions for which safety data from clinical trials already existed in a personalized context.
Augustine EF, Yu TW, Finkel RS. N-of-1 Studies in an Era of Precision Medicine. JAMA. Published online September 25, 2024. doi:10.1001/jama.2024.14637
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