Methodological considerations for N-of-1 trials

Carriere, Keumhee C., Li, Yin, Mitchell, Geoffrey and Senior, Hugh (2015). Methodological considerations for N-of-1 trials. In Jane Nikles and Geoffrey Mitchell (Ed.), The essential guide to N-of-1 trials in health (pp. 67-80) Dordrecht, Netherlands: Springer. doi:10.1007/978-94-017-7200-6_6

Author Carriere, Keumhee C.
Li, Yin
Mitchell, Geoffrey
Senior, Hugh
Title of chapter Methodological considerations for N-of-1 trials
Title of book The essential guide to N-of-1 trials in health
Place of Publication Dordrecht, Netherlands
Publisher Springer
Publication Year 2015
Sub-type Research book chapter (original research)
DOI 10.1007/978-94-017-7200-6_6
ISBN 9789401771993
Editor Jane Nikles
Geoffrey Mitchell
Chapter number 6
Start page 67
End page 80
Total pages 14
Total chapters 17
Language eng
Abstract/Summary N-of-1 trials are extremely useful in subject-focused investigations, for example, medical experiments. As far as we are aware, no guidelines are available in the literature on how to plan such a trial optimally. In this chapter, we discuss the considerations when choosing a particular N-of-1 trial design. We assume that the outcome of interest is measured on a continuous scale. Our discussion will be limited to comparisons of two treatments, without implying that the designs constructed can apply to non-continuous or binary outcomes. We construct optimal N-of-1 trials under various models depending upon how we accommodate the carryover effects and the error structures for the repeated measurements. Overall, we conclude that alternating between AB and BA pairs in subsequent cycles will result in practically optimal N-of-1 trials for a single patient, under all the models we considered without the need to guess at the correlation structure or conduct a pilot study. Alternating between AB and BA pairs in a single trial is nearly robust to misspecification of the error structure of the repeated measurements.
Keyword N-of-1 Trials
Essential Guide
Optimal design
Clinical trials
Crossover design
Residual effects
Direct treatment effect
Q-Index Code B1
Q-Index Status Provisional Code
Institutional Status UQ

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Created: Sat, 21 Nov 2015, 00:40:18 EST by Patricia Speechly on behalf of Discipline of General Practice