Open Access
2024 Multivariate tie-breaker designs
Tim P. Morrison, Art B. Owen
Author Affiliations +
Electron. J. Statist. 18(2): 4612-4639 (2024). DOI: 10.1214/24-EJS2312

Abstract

In a tie-breaker design (TBD), subjects with high values of a running variable are given some (usually desirable) treatment, subjects with low values are not, and subjects in the middle are randomized. TBDs are intermediate between regression discontinuity designs (RDDs) and randomized controlled trials (RCTs). TBDs allow a tradeoff between the resource allocation efficiency of an RDD and the statistical efficiency of an RCT. We study a model where the expected response is one multivariate regression for treated subjects and another for control subjects. We propose a prospective D-optimality, analogous to Bayesian optimal design, to understand design tradeoffs without reference to a specific data set. For given covariates, we show how to use convex optimization to choose treatment probabilities that optimize this criterion. We can incorporate a variety of constraints motivated by economic and ethical considerations. In our model, D-optimality for the treatment effect coincides with D-optimality for the whole regression, and, without constraints, an RCT is globally optimal. We show that a monotonicity constraint favoring more deserving subjects induces sparsity in the number of distinct treatment probabilities. We apply the convex optimization solution to a semi-synthetic example involving triage data from the MIMIC-IV-ED database.

Funding Statement

This work was supported by the NSF under grants IIS-1837931 and DMS-2152780. T. M. is supported by a B. C. and E. J. Eaves Stanford Graduate Fellowship.

Acknowledgments

We thank John Cherian, Anav Sood, Harrison Li and Dan Kluger for helpful discussions. We also thank Balasubramanian Narasimhan for helpful input on the convex optimization problem, Michael Baiocchi and Minh Nguyen of the Stanford School of Medicine for discussions about triage to hospital intensive care units, and an anonymous reviewer for helpful comments.

Citation

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Tim P. Morrison. Art B. Owen. "Multivariate tie-breaker designs." Electron. J. Statist. 18 (2) 4612 - 4639, 2024. https://doi.org/10.1214/24-EJS2312

Information

Received: 1 February 2024; Published: 2024
First available in Project Euclid: 19 November 2024

arXiv: 2202.10030
Digital Object Identifier: 10.1214/24-EJS2312

Subjects:
Primary: 62K05
Secondary: 62D20

Keywords: Marginally restricted design , randomized controlled trial , regression discontinuity

Vol.18 • No. 2 • 2024
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