Biostatistics PhD student Yuhan Qian has won first place in the 2025 American Statistical Association (ASA) Student Paper Competition in the Biopharmaceutical Section for his paper, “From Estimands to Robust Inference of Treatment Effects in Platform Trials.”
A platform trial is an innovative clinical trial design that uses a master protocol to evaluate multiple treatments in an ongoing manner, accelerating the evaluation of new treatments.
Two key challenges that occur with platform trials which are not present in traditional trials are the focus of the paper. These challenges include precisely defining treatment effects and ensuring robust, efficient inference on these effects.
In the paper, Qian and paper co-authors present a clear framework for constructing a clinically meaningful estimand with precise specification of the population of interest and develop methods for robustly estimating treatment effects with minimal assumptions.
“Our proposed entire concurrently eligible (ECE) population is critical for addressing key issues in future statistical research, such as interim analyses and the use of nonconcurrent controls, as it provides a clear reference point for evaluating both efficiency gains and potential bias. The ECE population not only preserves the integrity of randomized comparisons but also remains invariant to the randomization ratio and trial type. This work enhances the fundamental understanding of platform trials and lays the groundwork for future design and statistical research,” said Qian.
Qian was lead author on the paper and has been invited to present his paper at ASA’s Joint Statistical Meetings in Nashville in August. Study co-authors include: Yifan Yi, Jun Shao, Yanyao Yi, Gregory Levin, Nicole Mayer-Hamblett, Patrick Heagerty, and Ting Ye.
