Causal machine learning for predicting treatment outcomes
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Abstract (arXiv)
Causal machine learning (ML) offers flexible, data-driven methods for predicting treatment outcomes including efficacy and toxicity, thereby supporting the assessment and safety of drugs. A key benefit of causal ML is that it allows for estimating individualized treatment effects, so that clinical decision-making can be personalized to individual patient profiles. Causal ML can be used in combination with both clinical trial data and real-world data, such as clinical registries and electronic health records, but caution is needed to avoid biased or incorrect predictions. In this Perspective, we discuss the benefits of causal ML (relative to traditional statistical or ML approaches) and outline the key components and steps. Finally, we provide recommendations for the reliable use of causal ML and effective translation into the clinic.
Key facts. Cited by 73 papers in the map (data through 7 Oct 2026). Builds on 44 of its 102 references that are in the map. First posted on arXiv 11 Oct 2024.
Builds on
44 of 102 references.
References · 44
- 2015Estimation and Inference of Heterogeneous Treatment Effects using Random Forestscited by 597
- 2017Meta-learners for Estimating Heterogeneous Treatment Effects using Machine Learningcited by 475
- 2017Quasi-Oracle Estimation of Heterogeneous Treatment Effectscited by 328
- 2016Generalized Random Forestscited by 309
- 2019Adapting Neural Networks for the Estimation of Treatment Effectscited by 285
- 2015Recursive Partitioning for Heterogeneous Causal Effectscited by 254
- 2017Bayesian Inference of Individualized Treatment Effects using Multi-task Gaussian Processescited by 156
- 2020Estimating Counterfactual Treatment Outcomes over Time Through Adversarially Balanced Representationscited by 154
- 2019Orthogonal Statistical Learningcited by 150
- 2021Nonparametric Estimation of Heterogeneous Treatment Effects: From Theory to Learning Algorithmscited by 144
- 2022Causal Transformer for Estimating Counterfactual Outcomescited by 121
- 2022Causal Machine Learning: A Survey and Open Problemscited by 107
32 more
- 2019Learning Counterfactual Representations for Estimating Individual Dose-Response Curvescited by 90
- 2020Estimating the Effects of Continuous-valued Interventions using Generative Adversarial Networkscited by 78
- 2018Removing Hidden Confounding by Experimental Groundingcited by 64
- 2015Nonparametric methods for doubly robust estimation of continuous treatment effectscited by 62
- 2021Sensitivity Analysis of Individual Treatment Effects: A Robust Conformal Inference Approachcited by 62
- 2021VCNet and Functional Targeted Regularization For Learning Causal Effects of Continuous Treatmentscited by 59
- 2022Continuous-Time Modeling of Counterfactual Outcomes Using Neural Controlled Differential Equationscited by 59
- 2020Identifying Causal-Effect Inference Failure with Uncertainty-Aware Modelscited by 57
- 2018Interval Estimation of Individual-Level Causal Effects Under Unobserved Confoundingcited by 51
- 2020Causal inference methods for combining randomized trials and observational studies: a reviewcited by 50
- 2021Sharp Sensitivity Analysis for Inverse Propensity Weighting via Quantile Balancingcited by 32
- 2022Combining Observational and Randomized Data for Estimating Heterogeneous Treatment Effectscited by 32
- 2019Machine Learning Estimation of Heterogeneous Treatment Effects with Instrumentscited by 29
- 2022Estimating average causal effects from patient trajectoriescited by 29
- 2023In Search of Insights, Not Magic Bullets: Towards Demystification of the Model Selection Dilemma in Heterogeneous Treatment Effect Estimationcited by 29
- 2023B-Learner: Quasi-Oracle Bounds on Heterogeneous Causal Effects Under Hidden Confoundingcited by 28
- 2023Bayesian Neural Controlled Differential Equations for Treatment Effect Estimationcited by 26
- 2023Sharp Bounds for Generalized Causal Sensitivity Analysiscited by 24
- 2021DoubleML -- An Object-Oriented Implementation of Double Machine Learning in Pythoncited by 23
- 2023Conformal Meta-learners for Predictive Inference of Individual Treatment Effectscited by 23
- 2020Estimating Individual Treatment Effects with Time-Varying Confounderscited by 21
- 2022Normalizing Flows for Interventional Density Estimationcited by 21
- 2021DoWhy: Addressing Challenges in Expressing and Validating Causal Assumptionscited by 20
- 2022Estimating individual treatment effects under unobserved confounding using binary instrumentscited by 17
- 2021Generalizing Off-Policy Learning under Sample Selection Biascited by 16
- 2023Reliable Off-Policy Learning for Dosage Combinationscited by 15
- 2023Accounting For Informative Sampling When Learning to Forecast Treatment Outcomes Over Timecited by 14
- 2020G-Net: A Deep Learning Approach to G-computation for Counterfactual Outcome Prediction Under Dynamic Treatment Regimescited by 13
- 2022Multi-disciplinary fairness considerations in machine learning for clinical trialscited by 8
- 2023Prediction under interventions: evaluation of counterfactual performance using longitudinal observational datacited by 8
- 2020Prediction meets causal inference: the role of treatment in clinical prediction modelscited by 7
- 2023A Causal Roadmap for Generating High-Quality Real-World Evidencecited by 6
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Citing papers per year: 2024: 3; 2025: 44; 2026: 26.
73 papers.
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- 2025Foundation Models for Causal Inference via Prior-Data Fitted Networkscited by 30
- 2025Multi-modal Time Series Analysis: A Tutorial and Surveycited by 24
- 2024Quantifying Aleatoric Uncertainty of the Treatment Effect: A Novel Orthogonal Learnercited by 10
- 2025Orthogonal Survival Learners for Estimating Heterogeneous Treatment Effects from Time-to-Event Datacited by 10
- 2025DeCaFlow: A deconfounding causal generative modelcited by 8
- 2025Treatment Effect Estimation for Optimal Decision-Makingcited by 7
- 2024Constructing Confidence Intervals for Average Treatment Effects from Multiple Datasetscited by 6
- 2025Position: Causal Machine Learning Requires Rigorous Synthetic Experiments for Broader Adoptioncited by 6
- 2024Continuous Bayesian Model Selection for Multivariate Causal Discoverycited by 5
- 2025Efficient and Sharp Off-Policy Learning under Unobserved Confoundingcited by 5
- 2025Differentially Private Learners for Heterogeneous Treatment Effectscited by 5
- 2025Orthogonal Representation Learning for Estimating Causal Quantitiescited by 4
38 more
- 2025Counterfactual Probabilistic Diffusion with Expert Modelscited by 4
- 2025Improving the Generation and Evaluation of Synthetic Data for Downstream Medical Causal Inferencecited by 4
- 2025AI-Driven Automation Can Become the Foundation of Next-Era Science of Science Researchcited by 3
- 2025A Diffusion-Based Method for Learning the Multi-Outcome Distribution of Medical Treatmentscited by 3
- 2025Overlap-weighted orthogonal meta-learner for treatment effect estimation over timecited by 3
- 2026Amortizing Causal Sensitivity Analysis via Prior Data-Fitted Networkscited by 3
- 2025PrivATE: Differentially Private Confidence Intervals for Average Treatment Effectscited by 2
- 2025LLM-Driven Treatment Effect Estimation Under Inference Time Text Confoundingcited by 2
- 2025Causality-informed Anomaly Detection in Partially Observable Sensor Networks: Moving beyond Correlationscited by 2
- 2025Towards Interpretable and Trustworthy Time Series Reasoning: A BlueSky Visioncited by 2
- 2025Integrating Probabilistic Trees and Causal Networks for Clinical and Epidemiological Datacited by 1
- 2025Your Assumed DAG is Wrong and Here's How To Deal With Itcited by 1
- 2025Flow-based Generative Modeling of Potential Outcomes and Counterfactualscited by 1
- 2025Late Fusion Multi-task Learning for Semiparametric Inference with Nuisance Parameterscited by 1
- 2025Who cuts emissions, who turns up the heat? causal machine learning estimates of energy efficiency interventionscited by 1
- 2025Interpretable Clustering with Adaptive Heterogeneous Causal Structure Learning in Mixed Observational Datacited by 1
- 2025GDR-learners: Orthogonal Learning of Generative Models for Potential Outcomescited by 1
- 2025Overlap-Adaptive Regularization for Conditional Average Treatment Effect Estimationcited by 1
- 2025Causal Graph Neural Networks for Healthcarecited by 1
- 2025DeepBlip: Estimating Conditional Average Treatment Effects Over Timecited by 1
- 2026How Clinicians Think and What AI Can Learn From Itcited by 1
- 2026Generalized Bayes for Causal Inferencecited by 1
- 2026Observationally Informed Adaptive Causal Experimental Designcited by 1
- 2026CAETC: Causal Autoencoding and Treatment Conditioning for Counterfactual Estimation over Timecited by 1
- 2026Causal multi-modal AI for personalized chemosensitivity predictioncited by 1
- 2025Recent Advances, Applications and Open Challenges in Machine Learning for Health: Reflections from Research Roundtables at ML4H 2024 Symposiumcited by 0
- 2025Unsupervised Structural-Counterfactual Generation under Domain Shiftcited by 0
- 2025Sequential Treatment Effect Estimation with Unmeasured Confounderscited by 0
- 2025Conditional Average Treatment Effect Estimation Under Hidden Confounderscited by 0
- 2025Towards Group Fairness with Multiple Sensitive Attributes in Federated Foundation Modelscited by 0
- 2025Annotation-Assisted Learning of Treatment Policies From Multimodal Electronic Health Recordscited by 0
- 2025Causal Machine Learning for Patient-Level Intraoperative Opioid Dose Prediction from Electronic Health Recordscited by 0
- 2025Beyond Correlation: Causal Multi-View Unsupervised Feature Selection Learningcited by 0
- 2025Towards Privacy-Aware Bayesian Networks: A Credal Approachcited by 0
- 2025An Orthogonal Learner for Individualized Outcomes in Markov Decision Processescited by 0
- 2025A tutorial on discovering and quantifying the effect of latent causal sources of multimodal EHR datacited by 0
- 2025Estimating Treatment Effects in Networks under Unknown Exposure Mappingscited by 0
- 2025Linear Causal Discovery with Interventional Constraintscited by 0
50 most cited of 73 All 73 (JSON)
- 2026TRACE: Tractable Routing Autoencoder for Clinical ECG20 Sep 2026
- 2026Causal multi-modal AI for personalized chemosensitivity prediction11 Sep 2026
- 2026GoT-CD: Graph-of-Thoughts Causal Discovery and the Fragility of Post-hoc Path-Specific Fairness Audits3 Aug 2026
- 2026Automated Synthesis and Adversarial Validation of Executable Causal Research Pipelines23 Jul 2026
- 2026Beyond travel mode: urban context shapes active mobility's mental health effects over time5 Jul 2026
- 2026Prediction Sets for Counterfactual Decisions: Coverage, Optimality, and Conformal Prediction2 Jul 2026
- 2026Position: Medical AI Neglects Real Treatment Outcomes28 Jun 2026
- 2026A Step Towards Inherently Interpretable Causal Machine Learning Models For Decision Support23 Jun 2026
- 2026AI for Social Good: An Investigation of the Causal Relationship Between Environmental Regulations and Their Effects on Air Pollution in London, UK13 Jun 2026
- 2026Reconciling Causality and Non-Equilibrium Thermodynamics with Hamiltonian Causal Models3 Jun 2026
- 2026Disentangled Double Machine Learning for Accurate Causal Effect Estimation24 May 2026
- 2026Causal Machine Learning Is Not a Panacea: A Roadmap for Observational Causal Inference in Health20 May 2026
38 more
- 2026From Static Risk to Dynamic Trajectories: Toward World-Model-Inspired Clinical Prediction16 May 2026
- 2026Amortizing Causal Sensitivity Analysis via Prior Data-Fitted Networks11 May 2026
- 2026ConfoundingSHAP: Quantifying confounding strength in causal inference11 May 2026
- 2026Real vs. Semi-Simulated: Rethinking Evaluation for Treatment Effect Estimation11 May 2026
- 2026InferenceEvolve: Towards Automated Causal Effect Estimators through Self-Evolving AI5 Apr 2026
- 2026Orthogonal Learner for Estimating Heterogeneous Long-Term Treatment Effects1 Apr 2026
- 2026Integrating Causal Machine Learning into Clinical Decision Support Systems: Insights from Literature and Practice25 Mar 2026
- 2026CAETC: Causal Autoencoding and Treatment Conditioning for Counterfactual Estimation over Time12 Mar 2026
- 2026Observationally Informed Adaptive Causal Experimental Design4 Mar 2026
- 2026Generalized Bayes for Causal Inference3 Mar 2026
- 2026Causal Discovery Should Embrace the Wisdom of the Crowd3 Mar 2026
- 2026Discovering Subgroups with Exceptional Survival Characteristics25 Feb 2026
- 2026An Integrated Causal Inference Framework for Traffic Safety Modeling with Semantic Street-View Visual Features12 Feb 2026
- 2026How Clinicians Think and What AI Can Learn From It18 Jan 2026
- 2025Generating Risky Samples with Conformity Constraints via Diffusion Models21 Dec 2025
- 2025Machine learning for violence prediction: a systematic review and critical appraisal28 Nov 2025
- 2025DeepBlip: Estimating Conditional Average Treatment Effects Over Time18 Nov 2025
- 2025Causal Graph Neural Networks for Healthcare4 Nov 2025
- 2025Assessment of the conditional exchangeability assumption in causal machine learning models: a simulation study30 Oct 2025
- 2025Linear Causal Discovery with Interventional Constraints30 Oct 2025
- 2025Estimating Treatment Effects in Networks under Unknown Exposure Mappings24 Oct 2025
- 2025Overlap-weighted orthogonal meta-learner for treatment effect estimation over time22 Oct 2025
- 2025Improving the Generation and Evaluation of Synthetic Data for Downstream Medical Causal Inference21 Oct 2025
- 2025Towards Interpretable and Trustworthy Time Series Reasoning: A BlueSky Vision19 Oct 2025
- 2025A tutorial on discovering and quantifying the effect of latent causal sources of multimodal EHR data15 Oct 2025
- 2025An Orthogonal Learner for Individualized Outcomes in Markov Decision Processes30 Sep 2025
- 2025Overlap-Adaptive Regularization for Conditional Average Treatment Effect Estimation29 Sep 2025
- 2025GDR-learners: Orthogonal Learning of Generative Models for Potential Outcomes26 Sep 2025
- 2025Towards Privacy-Aware Bayesian Networks: A Credal Approach23 Sep 2025
- 2025Beyond Correlation: Causal Multi-View Unsupervised Feature Selection Learning17 Sep 2025
- 2025Interpretable Clustering with Adaptive Heterogeneous Causal Structure Learning in Mixed Observational Data4 Sep 2025
- 2025Counterfactual Probabilistic Diffusion with Expert Models18 Aug 2025
- 2025Causal Machine Learning for Patient-Level Intraoperative Opioid Dose Prediction from Electronic Health Records12 Aug 2025
- 2025Position: Causal Machine Learning Requires Rigorous Synthetic Experiments for Broader Adoption12 Aug 2025
- 2025Who cuts emissions, who turns up the heat? causal machine learning estimates of energy efficiency interventions6 Aug 2025
- 2025Annotation-Assisted Learning of Treatment Policies From Multimodal Electronic Health Records28 Jul 2025
- 2025Causality-informed Anomaly Detection in Partially Observable Sensor Networks: Moving beyond Correlations13 Jul 2025
- 2025Late Fusion Multi-task Learning for Semiparametric Inference with Nuisance Parameters10 Jul 2025
50 newest of 73 All 73 (JSON)
- 2025Foundation Models for Causal Inference via Prior-Data Fitted Networkscited by 30
- 2025Multi-modal Time Series Analysis: A Tutorial and Surveycited by 24
- 2024Quantifying Aleatoric Uncertainty of the Treatment Effect: A Novel Orthogonal Learnercited by 10
- 2025Orthogonal Survival Learners for Estimating Heterogeneous Treatment Effects from Time-to-Event Datacited by 10
- 2025DeCaFlow: A deconfounding causal generative modelcited by 8
- 2025Treatment Effect Estimation for Optimal Decision-Makingcited by 7
- 2024Constructing Confidence Intervals for Average Treatment Effects from Multiple Datasetscited by 6
- 2025Position: Causal Machine Learning Requires Rigorous Synthetic Experiments for Broader Adoptioncited by 6
- 2024Continuous Bayesian Model Selection for Multivariate Causal Discoverycited by 5
- 2025Efficient and Sharp Off-Policy Learning under Unobserved Confoundingcited by 5
- 2025Differentially Private Learners for Heterogeneous Treatment Effectscited by 5
- 2025Orthogonal Representation Learning for Estimating Causal Quantitiescited by 4
12 most cited of 70 All 70 (JSON)
- 2026TRACE: Tractable Routing Autoencoder for Clinical ECG20 Sep 2026
- 2026Causal multi-modal AI for personalized chemosensitivity prediction11 Sep 2026
- 2026Automated Synthesis and Adversarial Validation of Executable Causal Research Pipelines23 Jul 2026
- 2026Beyond travel mode: urban context shapes active mobility's mental health effects over time5 Jul 2026
- 2026Prediction Sets for Counterfactual Decisions: Coverage, Optimality, and Conformal Prediction2 Jul 2026
- 2026Position: Medical AI Neglects Real Treatment Outcomes28 Jun 2026
- 2026A Step Towards Inherently Interpretable Causal Machine Learning Models For Decision Support23 Jun 2026
- 2026AI for Social Good: An Investigation of the Causal Relationship Between Environmental Regulations and Their Effects on Air Pollution in London, UK13 Jun 2026
- 2026Reconciling Causality and Non-Equilibrium Thermodynamics with Hamiltonian Causal Models3 Jun 2026
- 2026Disentangled Double Machine Learning for Accurate Causal Effect Estimation24 May 2026
- 2026Causal Machine Learning Is Not a Panacea: A Roadmap for Observational Causal Inference in Health20 May 2026
- 2026From Static Risk to Dynamic Trajectories: Toward World-Model-Inspired Clinical Prediction16 May 2026
12 newest of 70 All 70 (JSON)