Skip to main content

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.

This is a preview of subscription content, log in via an institution to check access.

Access this chapter

Subscribe and save

Springer+ Basic
$34.99 /Month
  • Get 10 units per month
  • Download Article/Chapter or eBook
  • 1 Unit = 1 Article or 1 Chapter
  • Cancel anytime
Subscribe now

Buy Now

Chapter
USD 29.95
Price excludes VAT (USA)
  • Available as PDF
  • Read on any device
  • Instant download
  • Own it forever
eBook
USD 119.00
Price excludes VAT (USA)
  • Available as EPUB and PDF
  • Read on any device
  • Instant download
  • Own it forever
Softcover Book
USD 159.99
Price excludes VAT (USA)
  • Compact, lightweight edition
  • Dispatched in 3 to 5 business days
  • Free shipping worldwide - see info
Hardcover Book
USD 159.99
Price excludes VAT (USA)
  • Durable hardcover edition
  • Dispatched in 3 to 5 business days
  • Free shipping worldwide - see info

Tax calculation will be finalised at checkout

Purchases are for personal use only

Institutional subscriptions

Similar content being viewed by others

References

  • Bland M (1995) An introduction to medical statistics. Oxford University Press, New York

    Google Scholar 

  • Davis MP, Mitchell GK (2012) Topics in research: structuring studies in palliative care. Curr Opin Support Palliat Care 6:483–489

    Article  PubMed  Google Scholar 

  • 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

    Article  CAS  PubMed  Google Scholar 

  • 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

    Article  PubMed  Google Scholar 

  • 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

    Article  PubMed  Google Scholar 

Download references

Author information

Authors and Affiliations

Authors

Corresponding author

Correspondence to Geoffrey Mitchell .

Editor information

Editors and Affiliations

Rights and permissions

Reprints and permissions

Copyright information

© 2015 Springer Science+Business Media Dordrecht

About this chapter

Cite this chapter

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

Download citation

Publish with us

Policies and ethics