What are n-of-1 trials, and what are they good for?

Definition of an n-of-1 trial

An n-of-1 trial is a experiment conducted for a single person in which treatment blocks are randomly rotated, symptoms are systematically logged, and results are statistically analyzed. Since many treatments work differently for different individuals, n-of-1 trials help determine a treatment’s efficacy for a specific individual. N-of-1 trials are typically used for chronic conditions and are not considered appropriate for acute illnesses.

Examples of n-of-1 trails

October 2019: A unblinded study of ~200 patients with chronic pain showed that patients using an n-of-1 protocol used significantly less pain medication. The study allowed the n-of-1 users to objectively compare the efficacy of medications, both NSAIDs and opioids, versus non-pharmaceutical interventions like yoga and physical therapy.

2005: Double blinded n-of-1 trials for 71 patients with chronic pain comparing over-the-counter medications found that 65% changed their treatments.

Examples of n-of-1 therapies

In addition to being a term of art of evaluating common treatments for their efficacy on a single individual (as above) “n-of-1” is increasingly used in describing drug or genomic solutions crafted to fit a single patient’s needs. A good example of this is the recent case of Mila, a 6-year-old with a rare fatal genetic condition, was cured by doctors who crafted a drug specific to her genetic mutation.

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