Abstract
The original purpose of N-of-1 trials is to determine whether a treatment works in a person. However, these trials can be considered as mini-randomized controlled trials (RCTs), with the person providing multiple datasets to the intervention and control groups. Therefore, several people undergoing the same N-of-1 trial can contribute many data sets and this rapidly scales up to the point where the power of the trial can equate to a normal RCT, but with far fewer participants. This characteristic means that RCT-level evidence can be derived from populations that are almost impossible to gather data from, because of low prevalence conditions, or difficulty in recruiting or retaining subjects. This chapter describes the method in detail, along with methodological challenges and limitations of the method.
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References
Bland M (1995) An introduction to medical statistics. Oxford University Press, New York
Davis MP, Mitchell GK (2012) Topics in research: structuring studies in palliative care. Curr Opin Support Palliat Care 6:483–489
Hardy J, Quinn S, Fazekas B, Plummer J, Eckermann S, Agar M, Spruyt O, Rowett D, Currow DC (2012) Randomized, double-blind, placebo-controlled study to assess the efficacy and toxicity of subcutaneous ketamine in the management of cancer pain. J Clin Oncol 30:3611–3617
Mitchell G, Hardy J, Nikles J, Carmont S, Senior H, Schluter P, Good P, Currow D (2015) The effect of methylphenidate on fatigue in advanced cancer: an aggregated n-of-1 trial. J Pain Symptom Manag (in press). doi: 10.1016/jpainsymman.2015.03.009
Nikles J, Mitchell GK, Schluter P, Good P, Hardy J, Rowett D, Shelby-James T, Vohra S, Currow D (2011) Aggregating single patient (n-of-1) trials in populations where recruitment and retention was difficult: the case of palliative care. J Clin Epidemiol 64:471–480
Shelby-James TM, Hardy J, Agar M, Yates P, Mitchell G, Sanderson C, Luckett T, Abernethy AP, Currow DC (2012) Designing and conducting randomized controlled trials in palliative care: a summary of discussions from the 2010 clinical research forum of the Australian Palliative Care Clinical Studies Collaborative. Palliat Med 26:1042–1047
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Mitchell, G. (2015). Aggregated N-of-1 Trials. In: Nikles, J., Mitchell, G. (eds) The Essential Guide to N-of-1 Trials in Health. Springer, Dordrecht. https://doi.org/10.1007/978-94-017-7200-6_5
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DOI: https://doi.org/10.1007/978-94-017-7200-6_5
Publisher Name: Springer, Dordrecht
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