# A Deep Subgrouping Framework for Precision Drug Repurposing via Emulating Clinical Trials on Real-world Patient Data

arXiv: https://arxiv.org/abs/2412.20373 · HTML: https://mlsanity.com/paper/2412.20373/

## Key facts

- Cited by 1 papers in the map (data through 14 Oct 2026).
- Builds on 10 of its 54 references that are in the map.
- First posted on arXiv 29 Dec 2024.

## Builds on (most cited first, 10 of 10)

- [Attention Is All You Need](https://mlsanity.com/paper/1706.03762/) (2017)
- [Meta-learners for Estimating Heterogeneous Treatment Effects using Machine Learning](https://mlsanity.com/paper/1706.03461/) (2017)
- [Adapting Neural Networks for the Estimation of Treatment Effects](https://mlsanity.com/paper/1906.02120/) (2019)
- [Bayesian Inference of Individualized Treatment Effects using Multi-task Gaussian Processes](https://mlsanity.com/paper/1704.02801/) (2017)
- [Nonparametric Estimation of Heterogeneous Treatment Effects: From Theory to Learning Algorithms](https://mlsanity.com/paper/2101.10943/) (2021)
- [Learning Counterfactual Representations for Estimating Individual Dose-Response Curves](https://mlsanity.com/paper/1902.00981/) (2019)
- [On Inductive Biases for Heterogeneous Treatment Effect Estimation](https://mlsanity.com/paper/2106.03765/) (2021)
- [An Evaluation Toolkit to Guide Model Selection and Cohort Definition in Causal Inference](https://mlsanity.com/paper/1906.00442/) (2019)
- [Interpretable Subgroup Discovery in Treatment Effect Estimation with Application to Opioid Prescribing Guidelines](https://mlsanity.com/paper/1905.03297/) (2019)
- [Robust Recursive Partitioning for Heterogeneous Treatment Effects with Uncertainty Quantification](https://mlsanity.com/paper/2006.07917/) (2020)

## Built on by (most cited first, 1 of 1)

- [Pretrained Medical Representations for the Practical Screening of Drug Repositioning Candidates](https://mlsanity.com/paper/2609.19865/) (2026)

## Abstract (arXiv)

Drug repurposing identifies new therapeutic uses for existing drugs, reducing the time and costs compared to traditional de novo drug discovery. Most existing drug repurposing studies using real-world patient data often treat the entire population as homogeneous, ignoring the heterogeneity of treatment responses across patient subgroups. This approach may overlook promising drugs that benefit specific subgroups but lack notable treatment effects across the entire population, potentially limiting the number of repurposable candidates identified. To address this, we introduce STEDR, a novel drug repurposing framework that integrates subgroup analysis with treatment effect estimation. Our approach first identifies repurposing candidates by emulating multiple clinical trials on real-world patient data and then characterizes patient subgroups by learning subgroup-specific treatment effects. We deploy model to Alzheimer's Disease (AD), a condition with few approved drugs and known heterogeneity in treatment responses. We emulate trials for over one thousand medications on a large-scale real-world database covering over 8 million patients, identifying 14 drug candidates with beneficial effects to AD in characterized subgroups. Experiments demonstrate STEDR's superior capability in identifying repurposing candidates compared to existing approaches. Additionally, our method can characterize clinically relevant patient subgroups associated with important AD-related risk factors, paving the way for precision drug repurposing.