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BSU Seminar: "Design and Inference for Enrichment Trials with a Continuous Biomarker"

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If you have a question about this talk, please contact Alison Quenault.

This will be a hybrid seminar. If you would like to attend virtually, please register for free here - https://us02web.zoom.us/meeting/register/tZErduCprzwiEtQhPshDBMXcqhwlvdZtp_IP

Joint work with Nancy Flournoy, Rosamarie Frieri, and Zhantao Lin

We describe the philosophical approaches to two-stage enrichment designs, in which a benefitting subpopulation is targeted in a second stage, after a first stage identifies the threshold of a predictive, continuous biomarker. The design issue we address is sample size estimation for the first and second stages, and the consequences of poorly estimating the threshold. These design issues are established based on an approach where the two stages are conducted and analyzed separately, and stage two is considered a confirmatory trial. Another approach is to combine the data from the two stages, and we demonstrate how to do that by testing two hypotheses simultaneously with test statistics that (we show) have an asymptotic normal distribution. While a bivariate normal model is used to give insights into the predictive nature of the biomarker, and to visualize some closed-form solutions, in principle other models can certainly be used (but perhaps yield fewer insights). As in many ongoing, long-term research projects, our work probably raises more questions than it answers!

This talk is part of the MRC Biostatistics Unit Seminars series.

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