Abstract
BackgroundThe n-of-1 trial offers a more methodologically sound approach to determining optimum treatment for an individual patient than "trials of therapy" routinely conducted in clinical practice. However, such methodology is rarely used in the UK. This pilot study explores the acceptability of n-of-1 trials to patients in the UK.MethodsPatients with osteoarthritis of the knee were recruited to their own 12-week n-of-1 trial comparing either two knee supports or an NSAID with simple analgesic. Patients were interviewed at the start and completion of their trial to explore reasons for participation, understanding of the trial design and experiences of participation. Daily diaries were completed to inform future treatment.ResultsNine patients participated (5 supports, 4 drugs). Patients were keen to participate, believing that the trial may lead to personal gains such as improved symptom control and quality of life. However, recruitment to the pharmacological comparison was more difficult since this could also entail risk. All patients were eager to complete the trial, even when difficulties were encountered. Completing the daily diary provided some patients with greater insight into their condition, which allowed them to improve their self-management. The n-of-1 trial design was viewed as a 'logical' design offering an efficient method of reaching a personalised treatment decision tailored to suit individual needs and preferences.ConclusionThis pilot study suggests that patients perceive the n-of-1 trial as an acceptable approach to the individualisation of treatment. In addition, further benefits over and above any gained from the interventions can be derived from involvement in such a study.
Highlights
The n-of-1 trial offers a more methodologically sound approach to determining optimum treatment for an individual patient than "trials of therapy" routinely conducted in clinical practice
In routine clinical practice, when optimum treatment for an individual patient is uncertain, clinicians frequently conduct a "trial of therapy", in which the patient is given a treatment and the subsequent clinical course determines whether the treatment is deemed effective and continued
It has been suggested that the n-of-1 trial is the study design with the potential to deliver the highest strength of evidence for making individual treatment decisions[4]
Summary
The n-of-1 trial offers a more methodologically sound approach to determining optimum treatment for an individual patient than "trials of therapy" routinely conducted in clinical practice. Such methodology is rarely used in the UK. It has been suggested that the n-of-1 trial is the study design with the potential to deliver the highest strength of evidence for making individual treatment decisions[4] In such a trial, an individual serves as their own control in assessing the comparative effectiveness of different treatments. An individual serves as their own control in assessing the comparative effectiveness of different treatments This design provides the opportunity to measure the symptoms that matter to the individual concerned. The patient and health care providers are blind to the allocation of treatment within each period
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