Optimal placebo-treatment comparisons in trials with an incomplete within-subject design and heterogeneous costs and variances

Mirjam Moerbeek

PLOS ONE, 2023, vol. 18, issue 4, 1-16

Abstract: The aim of a clinical trial is to compare placebo to one or more treatments. The within-subject design is known to be more efficient than the between-subject design. However, in some trials that implement a within-subject design it is not possible to evaluate the placebo and all treatments within each subject. The design then becomes an incomplete within-subject design. An important question is how many subjects should be allocated to each combination of placebo and treatments. This paper studies optimal allocations of subjects in trials with a placebo and two treatments under heterogenous costs and variances. Two optimality criteria that consider the placebo-treatment contrasts simultaneously are considered, and the design is derived under a budgetary constraint. More subjects are allocated to those combinations with higher variances and lower costs. The optimal allocation is compared to the uniform allocation, which allocates equal number of subjects to each placebo and treatment combination, and to the complete within-subject design, where placebo and all treatments are available in each subject. The methodology is illustrated on the basis of an example on consultation time in primary care. A Shiny app is available to facilitate use of the methodology.

Date: 2023
References: View complete reference list from CitEc
Citations:

Downloads: (external link)
https://journals.plos.org/plosone/article?id=10.1371/journal.pone.0283382 (text/html)
https://journals.plos.org/plosone/article/file?id= ... 83382&type=printable (application/pdf)

Related works:
This item may be available elsewhere in EconPapers: Search for items with the same title.

Export reference: BibTeX RIS (EndNote, ProCite, RefMan) HTML/Text

Persistent link: https://EconPapers.repec.org/RePEc:plo:pone00:0283382

DOI: 10.1371/journal.pone.0283382

Access Statistics for this article

More articles in PLOS ONE from Public Library of Science
Bibliographic data for series maintained by plosone ().