N-of-1 trials: an opportunity to tailor treatment in individual patients

[摘要] 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

[学科分类] 卫生学