The n-of-1 clinical trial: the ultimate strategy for individualizing medicine?
By E. Lillie, B. Patay, J. Diamant, B. Issell, Eric J. Topol, Nicholas J. Schork. Personalized Medicine, 2011
650 citations recorded by the source corpus.
N-of-1 or single subject clinical trials consider an individual patient as the sole unit of observation in a study investigating the efficacy or side-effect profiles of different interventions. The ultimate goal of an n-of-1 trial is to determine the optimal or best intervention for an individual patient using objective data-driven criteria. Such trials can leverage study design and statistical techniques associated with standard population-based clinical trials, including randomization, washout and crossover periods, as well as placebo controls. Despite their obvious appeal and wide use in educational settings, n-of-1 trials have been used sparingly in medical and general clinical settings. We briefly review the history, motivation and design of n-of-1 trials and emphasize the great utility of modern wireless medical monitoring devices in their execution. We ultimately argue that n-of-1 trials demand serious attention among the health research and clinical care communities given the contemporary focus on individualized medicine.
Subfields: Statistics and Probability
View Paper: https://www.semanticscholar.org/paper/b92b61f7bd5145faddececfe5aab77e6e027e98e
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Citations in the corpus
- The next generation of evidence-based medicine (2023, positive): “The N-of-1 trial is a single-patient clinical trial using the individual person as a unit of investigation to evaluate the efficacy and/or adverse events of different interventions through objective data-driven criteria 54 .”
- Systematic review of smartphone-based passive sensing for health and wellbeing (2017, background): “The use of personal sensing mirrors n-of-1 clinical trials and indeed, some have suggested the use of sensing devices for n-of-1 trials [79].”
- Mobile technology and the digitization of healthcare. (2016, methodology): “The clinician must remain cognisant of noise signals and artefacts observed on mHealth devices that may result in false-positive findings, alarms, and misinterpretations when performed in real-world settings.(55)”
- Personalized medicine: motivation, challenges, and progress. (2018, methodology): “(32) Cross-over based N-of-1 trials are impractical, if not unethical, in settings where an individual is suffering from an acute or life-threatening condition, since switching from one intervention to another may exacerbate that individual's condition.”