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Activity Number: 315 - SPEED: Biopharmaceutical Applications: Trials, Biomarkers, and Enpoint Validation
Type: Contributed
Date/Time: Tuesday, July 31, 2018 : 9:25 AM to 10:10 AM
Sponsor: Biopharmaceutical Section
Abstract #332996
Title: Statistical Approaches for Assessing the Utility of Urinary Glycosaminoglycans as a Surrogate Endpoint in Clinical Trials
Author(s): Di Xiao* and Yeh-Fong Chen and Min Min
Companies: The Food and Drug Adminstration and US FDA and U.S. Food and Drug Administration, CDER/OTS/OB
Keywords: 6MWD; uGAGs; logistic regression; cross validation; C statistic

When it takes a long period of time for meaningful clinical outcomes to manifest in clinical trials, the use of surrogate endpoints for the accelerated approval of the new therapy can be appealing. However, whether the selected surrogate endpoint is reasonably likely to predict clinical benefit(s) needs thorough evaluations. In this presentation, we will evaluate the potential statistical methodologies that can be used for assessing the concordance of the surrogate endpoints and the clinical outcomes. We will use simulated data based on the pooled information from the Mucopolysaccharidoses trials as an example to illustrate our approach. In particular, we will demonstrate the modeling strategy in assessing the concordance between patients' urinary glycosaminoglycans (uGAGs) and six-minute walk distance (6MWD). Besides the model selection, we will share our idea of pooling trial data and ways of applying cross-validation to assess accuracy of the selected model and propose an appropriate cut-off based on several concordance statistics. We will also discuss methods for dealing with missing data.

Authors who are presenting talks have a * after their name.

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