
A robust method for comparing two treatments in a confirmatory clinical trial via multivariate time-to-event methods that jointly incorporate information from longitudinal and time-to-event data.
We consider regulatory clinical trials that require a prespecified method for the comparison of two treatments for chronic diseases (e.g. Chronic Obstructive Pulmonary Disease) in which patients suffer deterioration in a longitudinal process until death occurs. We define a composite endpoint structure that encompasses both the longitudinal data for deterioration and the time-to-event data for death, and use multivariate time-to-event methods to assess treatment differences on both data structures simultaneously, without a need for parametric assumptions or modeling. Our method is straightforward to implement, and simulations show that the method has robust power in situations in which incomplete data could lead to lower than expected power for either the longitudinal or survival data. We illustrate the method on data from a study of chronic lung disease.
Duke Scholars
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Related Subject Headings
- Survival Analysis
- Statistics & Probability
- Randomized Controlled Trials as Topic
- Pulmonary Disease, Chronic Obstructive
- Middle Aged
- Male
- Longitudinal Studies
- Humans
- Forced Expiratory Volume
- Female
Citation

Published In
DOI
EISSN
ISSN
Publication Date
Volume
Issue
Start / End Page
Related Subject Headings
- Survival Analysis
- Statistics & Probability
- Randomized Controlled Trials as Topic
- Pulmonary Disease, Chronic Obstructive
- Middle Aged
- Male
- Longitudinal Studies
- Humans
- Forced Expiratory Volume
- Female