Article
Study design for non-recurring, time to event outcomes in the presence of error-prone diagnostic tests or self-reports
Statistics in Medicine
(2016)
Abstract
Sequentially administered, laboratory-based diagnostic tests or self-reported questionnaires are often used to
determine the occurrence of a silent event. In this paper, we consider issues relevant in design of studies aimed at
estimating the association of one or more covariates with a non-recurring, time to event outcome that is observed
using a repeatedly administered, error-prone diagnostic procedure. The problem is motivated by the Women’s
Health Initiative, in which diabetes incidence among the approximately 160,000 women is obtained from annually
collected self-reported data. For settings of imperfect diagnostic tests or self-reports with known sensitivity and
specificity, we evaluate the effects of various factors on resulting power and sample size calculations and compare
the relative efficiency of different study designs. The methods illustrated in this paper are readily implemented
using our freely available R software package icensmis [1], which is available at the Comprehensive R Archive
Network (CRAN) website. An important special case is that when diagnostic procedures are perfect, resulting in
interval-censored, time to event outcomes. The proposed methods are applicable for the design of studies in which
a time to event outcome is interval-censored.
Keywords
- imperfect diagnostic tests,
- interval censoring,
- self-reports,
- study design,
- time to event outcomes
Disciplines
Publication Date
Spring March 18, 2016
Citation Information
Xiangdong Gu and Raji Balasubramanian. "Study design for non-recurring, time to event outcomes in the presence of error-prone diagnostic tests or self-reports" Statistics in Medicine (2016) Available at: http://works.bepress.com/raji_balasubramanian/33/