Using a surrogate marker for early testing of a treatment effect

The development of methods to identify, validate, and use surrogate markers to test for a treatment effect has been an area of intense research interest given the potential for valid surrogate markers to reduce the required costs and follow-up times of future studies. Several quantities and procedur...

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Bibliographic Details
Published in:Biometrics 2019-12, Vol.75 (4), p.1253-1263
Main Authors: Parast, Layla, Cai, Tianxi, Tian, Lu
Format: Article
Language:English
Subjects:
Antiretroviral drugs
BIOMETRIC METHODOLOGY
biometry
diabetes
Diabetes mellitus
disease control programs
Identification methods
kernel smoothing
Markers
nonparametric method
Nonparametric statistics
resampling
surrogate
survival analysis
testing
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Summary:The development of methods to identify, validate, and use surrogate markers to test for a treatment effect has been an area of intense research interest given the potential for valid surrogate markers to reduce the required costs and follow-up times of future studies. Several quantities and procedures have been proposed to assess the utility of a surrogate marker. However, few methods have been proposed to address how one might use the surrogate marker information to test for a treatment effect at an earlier time point, especially in settings where the primary outcome and the surrogate marker are subject to censoring. In this paper, we propose a novel test statistic to test for a treatment effect using surrogate marker information measured prior to the end of the study in a timeto-event outcome setting. We propose a robust nonparametric estimation procedure and propose inference procedures. In addition, we evaluate the power for the design of a future study based on surrogate marker information. We illustrate the proposed procedure and relative power of the proposed test compared to a test performed at the end of the study using simulation studies and an application to data from the Diabetes Prevention Program.
ISSN:0006-341X
1541-0420
DOI:10.1111/biom.13067