Aggregating single patient (n-of-1) trials in populations where recruitment and retention was difficult: The case of palliative care

Nikles, Jane, Mitchell, Geoffrey K., Schluter, Philip, Good, Phillip, Hardy, Janet, Rowett, Debra, Shelby-James, Tania, Vohra, Sunita and Currow, David (2011) Aggregating single patient (n-of-1) trials in populations where recruitment and retention was difficult: The case of palliative care. Journal of Clinical Epidemiology, 64 5: 471-480. doi:10.1016/j.jclinepi.2010.05.009

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Author Nikles, Jane
Mitchell, Geoffrey K.
Schluter, Philip
Good, Phillip
Hardy, Janet
Rowett, Debra
Shelby-James, Tania
Vohra, Sunita
Currow, David
Title Aggregating single patient (n-of-1) trials in populations where recruitment and retention was difficult: The case of palliative care
Journal name Journal of Clinical Epidemiology   Check publisher's open access policy
ISSN 0895-4356
1878-5921
Publication date 2011-05
Year available 2010
Sub-type Critical review of research, literature review, critical commentary
DOI 10.1016/j.jclinepi.2010.05.009
Volume 64
Issue 5
Start page 471
End page 480
Total pages 10
Editor Andre J. Knottnerus
Peter Tugwell
Place of publication New York, N.Y. U.S.A.
Publisher Elsevier
Collection year 2011
Language eng
Formatted abstract Randomized controlled trials (RCTs) are the gold standard for evaluating new interventions. Different RCT designs apply depending on the patient population, clinical setting, and intervention being evaluated. A design that may help to generate evidence in some clinical areas where recruitment is a challenge is aggregated n-of-1 trials.

N-of-1 trials are randomized, double-blind, and multiple crossover comparisons of an intervention and a control treatment. Methodologically robust n-of-1 trials provide an objective means of testing the effectiveness of treatments within individual participants. Aggregation of multiple cycle identically conducted n-of-1 trials yield a population estimate of effect, which potentially commensurate with that derived from other RCT designs. Trial participants contribute data for both intervention and control treatments creating matched data sets while using generally smaller sample sizes than conventional RCT trials.

Careful choice of symptoms and medications are required for n-of-1 trials to be feasible. A validated and reliable outcome measure sensitive to change is still required.

This article reviews the utility and limitations of aggregated n-of-1 trials to gather evidence in populations where conducting formal RCTs is difficult because of the low prevalence of the underlying condition or the clinical condition making recruitment and retention difficult. The article examines a proposed palliative care trial as a test case. © 2010 Elsevier Inc. All rights reserved.
Keyword Controlled clinical trials
Hospice care
N-of-1 trials
Palliative care
Randomized controlled trials
Statistical methods
Q-Index Code C1
Q-Index Status Confirmed Code
Institutional Status UQ
Additional Notes Available online 8 October 2010

Document type: Journal Article
Sub-type: Critical review of research, literature review, critical commentary
Collections: Official 2011 Collection
School of Medicine Publications
School of Nursing and Midwifery Publications
 
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Created: Fri, 19 Nov 2010, 21:16:47 EST by Dr Jane Nikles on behalf of Discipline of General Practice