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

Nikles, J; Mitchell, GK; Schluter, P; Good, P; Hardy, J; Rowett, D; Shelby-James, T; Vohra, S; Currow, D

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

Nikles, J Mitchell, GK Schluter, P Good, P

Hardy, J Rowett, D Shelby-James, T Vohra, S Currow, D

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Publication Type:: Journal Article
Citation:: Journal of Clinical Epidemiology, 2011, 64 (5), pp. 471 - 480
Issue Date:: 2011-05-01

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Field	Value	Language
dc.contributor.author	Nikles, J	en_US
dc.contributor.author	Mitchell, GK	en_US
dc.contributor.author	Schluter, P	en_US
dc.contributor.author	Good, P https://orcid.org/0000-0002-8198-0375	en_US
dc.contributor.author	Hardy, J	en_US
dc.contributor.author	Rowett, D	en_US
dc.contributor.author	Shelby-James, T	en_US
dc.contributor.author	Vohra, S	en_US
dc.contributor.author	Currow, D https://orcid.org/0000-0003-1988-1250	en_US
dc.date.available	2010-05-28	en_US
dc.date.issued	2011-05-01	en_US
dc.identifier.citation	Journal of Clinical Epidemiology, 2011, 64 (5), pp. 471 - 480	en_US
dc.identifier.issn	0895-4356	en_US
dc.identifier.uri	http://hdl.handle.net/10453/105668
dc.description.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. © 2011 Elsevier Inc. All rights reserved.	en_US
dc.relation.ispartof	Journal of Clinical Epidemiology	en_US
dc.relation.isbasedon	10.1016/j.jclinepi.2010.05.009	en_US
dc.subject.classification	Epidemiology	en_US
dc.subject.mesh	Humans	en_US
dc.subject.mesh	Palliative Care	en_US
dc.subject.mesh	Cross-Over Studies	en_US
dc.subject.mesh	Double-Blind Method	en_US
dc.subject.mesh	Patient Selection	en_US
dc.subject.mesh	Female	en_US
dc.subject.mesh	Male	en_US
dc.subject.mesh	Randomized Controlled Trials as Topic	en_US
dc.title	Aggregating single patient (n-of-1) trials in populations where recruitment and retention was difficult: The case of palliative care	en_US
dc.type	Journal Article
utslib.citation.volume	5	en_US
utslib.citation.volume	64	en_US
utslib.for	1110 Nursing	en_US
utslib.for	01 Mathematical Sciences	en_US
utslib.for	11 Medical and Health Sciences	en_US
pubs.embargo.period	Not known	en_US
pubs.organisational-group	/University of Technology Sydney
pubs.organisational-group	/University of Technology Sydney/Faculty of Health
pubs.organisational-group	/University of Technology Sydney/Faculty of Health/IMPACCT
utslib.copyright.status	closed_access
pubs.issue	5	en_US
pubs.publication-status	Published	en_US
pubs.volume	64	en_US

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. © 2011 Elsevier Inc. All rights reserved.

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http://hdl.handle.net/10453/105668