"Me's me and you's you": Exploring patients' perspectives of single patient (n-of-1) trials in the UK

Brookes, Sara; Biddle, Lucy; Paterson, Charlotte; Woolhead, Gillian; Dieppe, Paul

doi:10.1186/1745-6215-8-10

Cited by 18 publications

(14 citation statements)

References 12 publications

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“…Brookes et al [29] conducted a pilot acceptability study among nine patients with osteoarthritis of the knee participating in two series of single-patient trials. One series ( n = 5) compared a standard knee support with a heat-retaining support; the other series ( n = 4) compared a nonsteroidal anti-inflammatory drug, diclofenac, with an analgesic, paracetamol.…”

Section: Acceptabilitymentioning

confidence: 99%

Single-patient (n-of-1) trials: a pragmatic clinical decision methodology for patient-centered comparative effectiveness research

Duan

Kravitz

Schmid

2013

Journal of Clinical Epidemiology

202

197

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Objective To raise awareness among clinicians and epidemiologists that single-patient (n-of-1) trials are potentially useful for informing personalized treatment decisions for patients with chronic conditions. Study Design and Setting We reviewed the clinical and statistical literature on methods and applications of single-patient trials and then critically evaluated the needs for further methodological developments. Results Existing literature reports application of 2,154 single-patient trials in 108 studies for diverse clinical conditions; various recent commentaries advocate for wider application of such trials in clinical decision making. Preliminary evidence from several recent pilot acceptability studies suggests that single-patient trials have the potential for widespread acceptance by patients and clinicians as an effective modality for increasing the therapeutic precision. Bayesian and adaptive statistical methods hold promise for increasing the informational yield of single-patient trials while reducing participant burden, but are not widely used. Personalized applications of single-patient trials can be enhanced through further development and application of methodologies on adaptive trial design, stopping rules, network meta-analysis, washout methods, and methods for communicating trial findings to patients and clinicians. Conclusions Single-patient trials may be poised to emerge as an important part of the methodological armamentarium for comparative effectiveness research and patient-centered outcomes research. By permitting direct estimation of individual treatment effects, they can facilitate finely graded individualized care, enhance therapeutic precision, improve patient outcomes, and reduce costs.

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Section: Acceptabilitymentioning

confidence: 99%

Single-patient (n-of-1) trials: a pragmatic clinical decision methodology for patient-centered comparative effectiveness research

Duan

Kravitz

Schmid

2013

Journal of Clinical Epidemiology

202

197

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“…To further reduce the observer-induced bias, the observer and the patient may be blinded to the intervention. However, it is not essential to the design of N of 1 trials, as long as key variables and outcomes are recognised and predefined [18]. The main characteristic that differentiates N of 1 trials from other trials, and may raise doubts about their clinical relevance and their scientific value, is their application to a single patient.…”

Section: Is An N Of 1 Randomised Trial Indicated For This Patient?mentioning

confidence: 99%

N of 1 trials in diabetes: making individual therapeutic decisions

Τσάπας

Matthews

2008

Diabetologia

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N of 1 trials are single-subject, randomised, crossover studies, in which the patient serves as their own control to compare the efficacy of a treatment. They offer an alternative to large, randomised clinical trials, which have limited applicability, and empirical testing, which is arbitrary in nature. N of 1 trials are regarded as providing the highest strength of evidence for individual subject decisions. They rely upon the patient expressing preferences and making shared decisions for determining the future therapy, and may therefore lead to the successful implementation of lifestyle interventions. N of 1 trials are an optimal approach for making therapeutic decisions in chronic diseases like diabetes mellitus, where decision-making is often reliant upon arbitrary criteria and clinical judgement.

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“…Kravitz comments that limited evidence suggests that patients may be receptive to n-of-1 trials once they understand the benefits (12). Two pilot studies examining patient perspectives suggest that patients do perceive n-of-1 trials as an acceptable approach to the individualisation of treatment (5,13). Indeed, a patient-focussed approach to the roll-out of any widespread use of n-of-1 trials is likely to be essential.…”

Section: Models For a General Uptake Of N-of-1 Trialsmentioning

confidence: 99%

Stakeholders' views on the routine use of n-of-1 trials to improve clinical care and to make resource allocation decisions for drug use

Nikles

Mitchell

Clavarino

et al. 2010

Aust. Health Review

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"Me's me and you's you": Exploring patients' perspectives of single patient (n-of-1) trials in the UK

Cited by 18 publications

References 12 publications

Single-patient (n-of-1) trials: a pragmatic clinical decision methodology for patient-centered comparative effectiveness research

Single-patient (n-of-1) trials: a pragmatic clinical decision methodology for patient-centered comparative effectiveness research

N of 1 trials in diabetes: making individual therapeutic decisions

Stakeholders' views on the routine use of n-of-1 trials to improve clinical care and to make resource allocation decisions for drug use

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