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Testing Treatment Effect Heterogeneity with Functional Outcomes

Abstract

Assessing treatment-effect heterogeneity is important in causal inference, particularly for functional outcomes where effects may vary over time and the common effect is unknown. We propose a nonparametric procedure that assesses treatment-effect homogeneity through a testable marginal implication. By comparing within-arm difference distributions, the construction removes the unknown common functional effect without estimating it. We measure departures between these distributions through an integrated squared discrepancy of their characteristic functionals. A full-support Gaussian integrating measure yields an explicit bounded-kernel representation and identifies equality of the difference laws without requiring moment conditions on the outcomes. We construct an unbiased generalized U-statistic that accounts for dependence among overlapping differences, derive its null, fixed-alternative, and local-alternative limits, and develop a centered multiplier bootstrap for inference. The framework extends to multiple treatment arms with a shared control. For computation, a random-direction approximation combined with a nine-state recursion reduces the cost to linear order in the sample size for fixed numerical settings. Simulation studies assess the finite-sample performance of the proposed procedure relative to several benchmark methods. An application to the Irish gas smart-metering trial illustrates its use for assessing heterogeneity in functional treatment responses.

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BibTeX
@misc{feng2026testing,
  title = {{Testing Treatment Effect Heterogeneity with Functional Outcomes}},
  author = {Yongzhen Feng and Jie Li and Xiaojun Song},
  howpublished = {arXiv},
  year = {2026},
  month = oct,
  doi = {10.48550/arxiv.2610.23397},
  eprint = {2610.23397},
  archivePrefix = {arXiv},
  url = {https://arxiv.org/abs/2610.23397},
}
RIS
TY  - UNPB
TI  - Testing Treatment Effect Heterogeneity with Functional Outcomes
AU  - Yongzhen Feng
AU  - Jie Li
AU  - Xiaojun Song
PY  - 2026
DA  - 2026/10/16
PB  - arXiv
DO  - 10.48550/arxiv.2610.23397
UR  - https://arxiv.org/abs/2610.23397
AB  - Assessing treatment-effect heterogeneity is important in causal inference, particularly for functional outcomes where effects may vary over time and the common effect is unknown. We propose a nonparametric procedure that assesses treatment-effect homogeneity through a testable marginal implication. By comparing within-arm difference distributions, the construction removes the unknown common functional effect without estimating it. We measure departures between these distributions through an integrated squared discrepancy of their characteristic functionals. A full-support Gaussian integrating measure yields an explicit bounded-kernel representation and identifies equality of the difference laws without requiring moment conditions on the outcomes. We construct an unbiased generalized U-statistic that accounts for dependence among overlapping differences, derive its null, fixed-alternative, and local-alternative limits, and develop a centered multiplier bootstrap for inference. The framework extends to multiple treatment arms with a shared control. For computation, a random-direction approximation combined with a nine-state recursion reduces the cost to linear order in the sample size for fixed numerical settings. Simulation studies assess the finite-sample performance of the proposed procedure relative to several benchmark methods. An application to the Irish gas smart-metering trial illustrates its use for assessing heterogeneity in functional treatment responses.
ER  -