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This research proposes a novel framework using AI predictions as surrogates to estimate causal treatment effects in medical settings, addressing the challenge of costly or long-term outcome measurements. The method leverages paired before-and-after data with pretrained models to identify average treatment effects, even when direct clinical outcomes are unobserved for treated individuals. By incorporating prediction-powered inference, the approach corrects for bias when standard assumptions fail, validated through cardio-oncology experiments.
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Artificial intelligence surrogates for treatment effect estimation with before-and-after data

arXiv2 min read

arXiv:2609.27180v1 Announce Type: cross Abstract: Estimating the causal effects of medical treatments is difficult when clinically important outcomes are costly to measure or require long follow-up. Short-term or inexpensive surrogate outcomes offer a potential alternative, but surrogate biomarkers may be unavailable or difficult to identify. Advances in artificial intelligence (AI) have enabled increasingly accurate prediction of clinical outcomes from inexpensive, high-dimensional measurements, which creates an opportunity to use AI predictions themselves as surrogates. To this end, we develop a framework for estimating treatment effects from paired measurements obtained before and after treatment for each treated individual. A pretrained AI model is applied to the before and after measurements, and our estimator compares the resulting outcome predictions. We characterize the technical assumptions under which this within-person contrast identifies the average treatment effect on the treated, even when clinical outcomes are never observed for treated individuals. When these assumptions cannot be justified, we use prediction-powered inference to correct bias using a small number of observed clinical outcomes and obtain valid inference. Synthetic and real-world cardio-oncology experiments demonstrate the validity and accuracy of the approach.

Abstract:Estimating the causal effects of medical treatments is difficult when clinically important outcomes are costly to measure or require long follow-up. Short-term or inexpensive surrogate outcomes offer a potential alternative, but surrogate biomarkers may be unavailable or difficult to identify. Advances in artificial intelligence (AI) have enabled increasingly accurate prediction of clinical outcomes from inexpensive, high-dimensional measurements, which creates an opportunity to use AI predictions themselves as surrogates. To this end, we develop a framework for estimating treatment effects from paired measurements obtained before and after treatment for each treated individual. A pretrained AI model is applied to the before and after measurements, and our estimator compares the resulting outcome predictions. We characterize the technical assumptions under which this within-person contrast identifies the average treatment effect on the treated, even when clinical outcomes are never observed for treated individuals. When these assumptions cannot be justified, we use prediction-powered inference to correct bias using a small number of observed clinical outcomes and obtain valid inference. Synthetic and real-world cardio-oncology experiments demonstrate the validity and accuracy of the approach.

Machine Learning (stat.ML); Machine Learning (cs.LG)

arXiv:2609.27180 [stat.ML]

(or arXiv:2609.27180v1 [stat.ML] for this version)

https://doi.org/10.48550/arXiv.2609.27180 Focus to learn more arXiv-issued DOI via DataCite (pending registration)

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Full text under the publisher's open licence · 274 words

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