N-of-1 trials: an opportunity to tailor treatment in individual patients.
Year: 2005
Type: article
Abstract: During a consultation, a situation may arise in which the benefits or harms associated with a particular treatment are uncertain. These uncertainties may occur for a number of reasons: well conducted randomised controlled trials (RCTs) may not have been performed; the patient may possess certain characteristics, for example multiple chronic diseases, that might attenuate the benefit of the therapy or increase the likelihood of harm; or there may be a disagreement between the patient and the healthcare professional about the potential benefits or harms of alternative therapies, particularly as RCTs only give ‘average’ estimates of benefit and harm, with some patients benefiting while others do not.1 When these situations occur, subjective forms of decision making, such as ‘trials of therapy’, have often been relied upon. In these situations of trials of therapy, benefit or harm from treatment is decided on loose criteria and weak methodology, and the individual patient's response to treatment provides little rigorous support concerning the actual efficacy of treatment.2
N -of-1 studies are randomised, double blinded, multiple crossover comparisons of an active drug against a placebo or an alternative treatment in a single patient.1,3,4 They limit biases of standard practice such as trials of therapy. More than 50 n -of-1 trials have been reported, each one designed to improve the care …
Source: Europe PMC (PubMed Central)
Authors: Vishnu Madhok, Tom Fahey
Institution: University of Dundee
Language: English
Subfield: Statistics and Probability
Field: Mathematics
Domain: Physical Sciences
Open Access status: green
OpenAlex ID: W1540072722