Pub Date : 2025-11-27eCollection Date: 2025-01-01DOI: 10.1515/jci-2024-0051
Stijn Vansteelandt, Paweł Morzywołek
Orthogonal meta-learners, such as DR-learner (Kennedy EH. Towards optimal doubly robust estimation of heterogeneous causal effects. arXiv preprint arXiv:2004.14497 2020), R-learner (Nie X, Wager S. Quasi-oracle estimation of heterogeneous treatment effects. Biometrika 2021;108:299-319) and IF-learner (Curth A, Alaa AM, van der Schaar M. Estimating structural target functions using machine learning and influence functions. arXiv preprint arXiv:2008.06461 2020), are increasingly used to estimate conditional average treatment effects. They are hoped to improve convergence rates relative to naïve meta-learners (e.g., T-, S- and X-learner (Künzel SR, Sekhon JS, Bickel PJ, Yu B. Metalearners for estimating heterogeneous treatment effects using machine learning. Proc Natl Acad Sci 2019;116:4156-65)) through de-biasing procedures that involve applying standard learners to specifically transformed outcome data. This leads them to disregard the possibly constrained outcome space, which can be particularly problematic for dichotomous outcomes: these typically get transformed to values that are no longer constrained to the unit interval, which may cause instability and makes it difficult for standard learners to guarantee predictions within the unit interval. To address this, we construct a non-orthogonal imputation-learner and an orthogonal 'i-learner' for the prediction of counterfactual outcomes, which respect the outcome space. These are more generally expected to outperform existing learners, even when the outcome is unconstrained, as we confirm empirically in simulation studies and an analysis of critical care data. Our development also sheds broader light onto the construction of orthogonal learners for other estimands.
{"title":"Orthogonal prediction of counterfactual outcomes.","authors":"Stijn Vansteelandt, Paweł Morzywołek","doi":"10.1515/jci-2024-0051","DOIUrl":"10.1515/jci-2024-0051","url":null,"abstract":"<p><p>Orthogonal meta-learners, such as DR-learner (Kennedy EH. Towards optimal doubly robust estimation of heterogeneous causal effects. arXiv preprint arXiv:2004.14497 2020), R-learner (Nie X, Wager S. Quasi-oracle estimation of heterogeneous treatment effects. Biometrika 2021;108:299-319) and IF-learner (Curth A, Alaa AM, van der Schaar M. Estimating structural target functions using machine learning and influence functions. arXiv preprint arXiv:2008.06461 2020), are increasingly used to estimate conditional average treatment effects. They are hoped to improve convergence rates relative to naïve meta-learners (e.g., T-, S- and X-learner (Künzel SR, Sekhon JS, Bickel PJ, Yu B. Metalearners for estimating heterogeneous treatment effects using machine learning. Proc Natl Acad Sci 2019;116:4156-65)) through de-biasing procedures that involve applying standard learners to specifically transformed outcome data. This leads them to disregard the possibly constrained outcome space, which can be particularly problematic for dichotomous outcomes: these typically get transformed to values that are no longer constrained to the unit interval, which may cause instability and makes it difficult for standard learners to guarantee predictions within the unit interval. To address this, we construct a non-orthogonal imputation-learner and an orthogonal 'i-learner' for the prediction of counterfactual outcomes, which respect the outcome space. These are more generally expected to outperform existing learners, even when the outcome is unconstrained, as we confirm empirically in simulation studies and an analysis of critical care data. Our development also sheds broader light onto the construction of orthogonal learners for other estimands.</p>","PeriodicalId":48576,"journal":{"name":"Journal of Causal Inference","volume":"13 1","pages":"20240051"},"PeriodicalIF":1.8,"publicationDate":"2025-11-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12658738/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145649827","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-11-17eCollection Date: 2026-01-01DOI: 10.1515/jci-2025-0002
Debashis Ghosh, Lei Wang
There has been widespread use of causal inference methods for the rigorous analysis of observational studies and to identify policy evaluations. In this article, we consider a class of generalized coarsened procedures for confounding. At a high level, these procedures can be viewed as performing a clustering of confounding variables, followed by treatment effect and attendant variance estimation using the confounder strata. In addition, we propose two new algorithms for generalized coarsened confounding. While previous authors have developed some statistical properties for one special case in our class of procedures, we instead develop a general asymptotic framework. We provide asymptotic results for the average causal effect estimator as well as providing conditions for consistency. In addition, we provide an asymptotic justification for the variance formulae for coarsened exact matching. A bias correction technique is proposed, and we apply the proposed methodology to data from two well-known observational studies.
{"title":"Generalized coarsened confounding for causal effects: a large-sample framework.","authors":"Debashis Ghosh, Lei Wang","doi":"10.1515/jci-2025-0002","DOIUrl":"10.1515/jci-2025-0002","url":null,"abstract":"<p><p>There has been widespread use of causal inference methods for the rigorous analysis of observational studies and to identify policy evaluations. In this article, we consider a class of generalized coarsened procedures for confounding. At a high level, these procedures can be viewed as performing a clustering of confounding variables, followed by treatment effect and attendant variance estimation using the confounder strata. In addition, we propose two new algorithms for generalized coarsened confounding. While previous authors have developed some statistical properties for one special case in our class of procedures, we instead develop a general asymptotic framework. We provide asymptotic results for the average causal effect estimator as well as providing conditions for consistency. In addition, we provide an asymptotic justification for the variance formulae for coarsened exact matching. A bias correction technique is proposed, and we apply the proposed methodology to data from two well-known observational studies.</p>","PeriodicalId":48576,"journal":{"name":"Journal of Causal Inference","volume":"14 1","pages":"20250002"},"PeriodicalIF":1.8,"publicationDate":"2025-11-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12777950/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145935745","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-01-01Epub Date: 2025-03-05DOI: 10.1515/jci-2023-0020
Ting Ye, Qijia He, Shuxiao Chen, Bo Zhang
In an observational study, it is common to leverage known null effects to detect bias. One such strategy is to set aside a placebo sample - a subset of data immune from the hypothesized cause-and-effect relationship. Existence of an effect in the placebo sample raises concerns about unmeasured confounding bias while absence of it helps corroborate the causal conclusion. This paper describes a framework for using a placebo sample to detect and remove bias. We state the identification assumptions and develop estimation and inference methods based on outcome regression, inverse probability weighting, and doubly-robust approaches. Simulation studies investigate the finite-sample performance of the proposed methods. We illustrate the methods using an empirical study of the effect of the earned income tax credit on infant health.
{"title":"Role of placebo samples in observational studies.","authors":"Ting Ye, Qijia He, Shuxiao Chen, Bo Zhang","doi":"10.1515/jci-2023-0020","DOIUrl":"10.1515/jci-2023-0020","url":null,"abstract":"<p><p>In an observational study, it is common to leverage known null effects to detect bias. One such strategy is to set aside a placebo sample - a subset of data immune from the hypothesized cause-and-effect relationship. Existence of an effect in the placebo sample raises concerns about unmeasured confounding bias while absence of it helps corroborate the causal conclusion. This paper describes a framework for using a placebo sample to detect and remove bias. We state the identification assumptions and develop estimation and inference methods based on outcome regression, inverse probability weighting, and doubly-robust approaches. Simulation studies investigate the finite-sample performance of the proposed methods. We illustrate the methods using an empirical study of the effect of the earned income tax credit on infant health.</p>","PeriodicalId":48576,"journal":{"name":"Journal of Causal Inference","volume":"13 1","pages":""},"PeriodicalIF":1.8,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12345972/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144849417","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Abstract Configurational Comparative Methods (CCMs) aim to learn causal structures from datasets by exploiting Boolean sufficiency and necessity relationships. One important challenge for these methods is that such Boolean relationships are often not satisfied in real-life datasets, as these datasets usually contain noise. Hence, CCMs infer models that only approximately fit the data, introducing a risk of inferring incorrect or incomplete models, especially when data are also fragmented (have limited empirical diversity). To minimize this risk, evaluation measures for sufficiency and necessity should be sensitive to all relevant evidence. This article points out that the standard evaluation measures in CCMs, consistency and coverage, neglect certain evidence for these Boolean relationships. Correspondingly, two new measures, contrapositive consistency and contrapositive coverage, which are equivalent to the binary classification measures specificity and negative predictive value, respectively, are introduced to the CCM context as additions to consistency and coverage. A simulation experiment demonstrates that the introduced contrapositive measures indeed help to identify correct CCM models.
{"title":"Evaluating Boolean relationships in Configurational Comparative Methods","authors":"Luna De Souter","doi":"10.1515/jci-2023-0014","DOIUrl":"https://doi.org/10.1515/jci-2023-0014","url":null,"abstract":"Abstract Configurational Comparative Methods (CCMs) aim to learn causal structures from datasets by exploiting Boolean sufficiency and necessity relationships. One important challenge for these methods is that such Boolean relationships are often not satisfied in real-life datasets, as these datasets usually contain noise. Hence, CCMs infer models that only approximately fit the data, introducing a risk of inferring incorrect or incomplete models, especially when data are also fragmented (have limited empirical diversity). To minimize this risk, evaluation measures for sufficiency and necessity should be sensitive to all relevant evidence. This article points out that the standard evaluation measures in CCMs, consistency and coverage, neglect certain evidence for these Boolean relationships. Correspondingly, two new measures, contrapositive consistency and contrapositive coverage, which are equivalent to the binary classification measures specificity and negative predictive value, respectively, are introduced to the CCM context as additions to consistency and coverage. A simulation experiment demonstrates that the introduced contrapositive measures indeed help to identify correct CCM models.","PeriodicalId":48576,"journal":{"name":"Journal of Causal Inference","volume":"8 12","pages":""},"PeriodicalIF":1.4,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139457038","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-01-01Epub Date: 2024-01-10DOI: 10.1515/jci-2023-0031
Amy J Pitts, Charlotte R Fowler
Many software packages have been developed to assist researchers in drawing directed acyclic graphs (DAGs), each with unique functionality and usability. We examine five of the most common software to generate DAGs: TikZ, DAGitty, ggdag, dagR, and igraph. For each package, we provide a general description of its background, analysis and visualization capabilities, and user-friendliness. Additionally in order to compare packages, we produce two DAGs in each software, the first featuring a simple confounding structure, while the second includes a more complex structure with three confounders and a mediator. We provide recommendations for when to use each software depending on the user's needs.
为了帮助研究人员绘制有向无环图(DAG),已经开发了许多软件包,每种软件都有独特的功能和可用性。我们研究了五种最常用的生成 DAG 的软件:TikZ、DAGitty、ggdag、dagR 和 igraph。我们对每个软件包的背景、分析和可视化能力以及用户友好性进行了总体描述。此外,为了对软件包进行比较,我们在每个软件中制作了两个 DAG,第一个 DAG 包含一个简单的混杂结构,第二个 DAG 包含一个包含三个混杂因素和一个中介因素的更复杂的结构。我们将根据用户的需求,为何时使用每种软件提供建议。
{"title":"Comparison of open-source software for producing directed acyclic graphs.","authors":"Amy J Pitts, Charlotte R Fowler","doi":"10.1515/jci-2023-0031","DOIUrl":"10.1515/jci-2023-0031","url":null,"abstract":"<p><p>Many software packages have been developed to assist researchers in drawing directed acyclic graphs (DAGs), each with unique functionality and usability. We examine five of the most common software to generate DAGs: Ti<i>k</i>Z, DAGitty, ggdag, dagR, and igraph. For each package, we provide a general description of its background, analysis and visualization capabilities, and user-friendliness. Additionally in order to compare packages, we produce two DAGs in each software, the first featuring a simple confounding structure, while the second includes a more complex structure with three confounders and a mediator. We provide recommendations for when to use each software depending on the user's needs.</p>","PeriodicalId":48576,"journal":{"name":"Journal of Causal Inference","volume":"12 1","pages":""},"PeriodicalIF":1.4,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10869111/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139742392","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-06-17DOI: 10.30998/inference.v5i3.12353
Hilda Zubaidah, Gustaman Saragih
The textbooks play an important role in teaching and learning activity in language program. Because of the various textbooks provided, textbook analysis is seen as an important thing to be conducted in order to find out how the components of the textbook are served. This study was aimed to investigate to what extent the English textbook entitled “Bahasa Inggris: When English Rings a Bell” for eighth grade students meet the criteria of BSNP (linguistic features and presentation of materias). The linguistic features consist of language appropriateness while the presentation of materials consist of content appropriates, presentation appropriateness, and graphic appropriateness. This study was descriptive qualitative approach. The instrument used to collect the data is document study used in the form of checklist. A checklist was made adopted from BSNP (2011) framework. The results of this study showed that textbook entitled “When English Rings a Bell” for Eight Grade is suitable to be used in teaching learning process. The textbook achieved the fulfilment score of language appropriateness (100%), content appropriateness (81,25%), presentation appropriateness (88,89%), and graphics appropriateness (97,64%). This book is categorized “good” textbook by achieving the score of 95,07%. Thus, it can be concluded that textbook is suitable to be used in order to help the teaching learning process in the classroom with the help of other sources and teacher improvisation.
{"title":"LINGUISTIC FEATURES AND PRESENTATION OF MATERIALS ON ENGLISH TEXTBOOK “WHEN ENGLISH RINGS A BELL” BASED ON BSNP","authors":"Hilda Zubaidah, Gustaman Saragih","doi":"10.30998/inference.v5i3.12353","DOIUrl":"https://doi.org/10.30998/inference.v5i3.12353","url":null,"abstract":"The textbooks play an important role in teaching and learning activity in language program. Because of the various textbooks provided, textbook analysis is seen as an important thing to be conducted in order to find out how the components of the textbook are served. This study was aimed to investigate to what extent the English textbook entitled “Bahasa Inggris: When English Rings a Bell” for eighth grade students meet the criteria of BSNP (linguistic features and presentation of materias). The linguistic features consist of language appropriateness while the presentation of materials consist of content appropriates, presentation appropriateness, and graphic appropriateness. This study was descriptive qualitative approach. The instrument used to collect the data is document study used in the form of checklist. A checklist was made adopted from BSNP (2011) framework. The results of this study showed that textbook entitled “When English Rings a Bell” for Eight Grade is suitable to be used in teaching learning process. The textbook achieved the fulfilment score of language appropriateness (100%), content appropriateness (81,25%), presentation appropriateness (88,89%), and graphics appropriateness (97,64%). This book is categorized “good” textbook by achieving the score of 95,07%. Thus, it can be concluded that textbook is suitable to be used in order to help the teaching learning process in the classroom with the help of other sources and teacher improvisation.","PeriodicalId":48576,"journal":{"name":"Journal of Causal Inference","volume":"17 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-06-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135525979","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Gabriel Danelian, Yohann Foucher, Maxime Léger, Florent Le Borgne, Arthur Chatton
Abstract Background The positivity assumption is crucial when drawing causal inferences from observational studies, but it is often overlooked in practice. A violation of positivity occurs when the sample contains a subgroup of individuals with an extreme relative frequency of experiencing one of the levels of exposure. To correctly estimate the causal effect, we must identify such individuals. For this purpose, we suggest a regression tree-based algorithm. Development Based on a succession of regression trees, the algorithm searches for combinations of covariate levels that result in subgroups of individuals with a low (un)exposed relative frequency. Application We applied the algorithm by reanalyzing four recently published medical studies. We identified the two violations of the positivity reported by the authors. In addition, we identified ten subgroups with a suspicion of violation. Conclusions The PoRT algorithm helps to detect in-sample positivity violations in causal studies. We implemented the algorithm in the R package RISCA to facilitate its use.
{"title":"Identification of in-sample positivity violations using regression trees: The PoRT algorithm","authors":"Gabriel Danelian, Yohann Foucher, Maxime Léger, Florent Le Borgne, Arthur Chatton","doi":"10.1515/jci-2022-0032","DOIUrl":"https://doi.org/10.1515/jci-2022-0032","url":null,"abstract":"Abstract Background The positivity assumption is crucial when drawing causal inferences from observational studies, but it is often overlooked in practice. A violation of positivity occurs when the sample contains a subgroup of individuals with an extreme relative frequency of experiencing one of the levels of exposure. To correctly estimate the causal effect, we must identify such individuals. For this purpose, we suggest a regression tree-based algorithm. Development Based on a succession of regression trees, the algorithm searches for combinations of covariate levels that result in subgroups of individuals with a low (un)exposed relative frequency. Application We applied the algorithm by reanalyzing four recently published medical studies. We identified the two violations of the positivity reported by the authors. In addition, we identified ten subgroups with a suspicion of violation. Conclusions The PoRT algorithm helps to detect in-sample positivity violations in causal studies. We implemented the algorithm in the R package RISCA to facilitate its use.","PeriodicalId":48576,"journal":{"name":"Journal of Causal Inference","volume":"259 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135501800","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Abstract Personalized decision making targets the behavior of a specific individual, while population-based decision making concerns a subpopulation resembling that individual. This article clarifies the distinction between the two and explains why the former leads to more informed decisions. We further show that by combining experimental and observational studies, we can obtain valuable information about individual behavior and, consequently, improve decisions over those obtained from experimental studies alone. In particular, we show examples where such a combination discriminates between individuals who can benefit from a treatment and those who cannot – information that would not be revealed by experimental studies alone. We outline areas where this method could be of benefit to both policy makers and individuals involved.
{"title":"Personalized decision making – A conceptual introduction","authors":"Scott Mueller, Judea Pearl","doi":"10.1515/jci-2022-0050","DOIUrl":"https://doi.org/10.1515/jci-2022-0050","url":null,"abstract":"Abstract Personalized decision making targets the behavior of a specific individual, while population-based decision making concerns a subpopulation resembling that individual. This article clarifies the distinction between the two and explains why the former leads to more informed decisions. We further show that by combining experimental and observational studies, we can obtain valuable information about individual behavior and, consequently, improve decisions over those obtained from experimental studies alone. In particular, we show examples where such a combination discriminates between individuals who can benefit from a treatment and those who cannot – information that would not be revealed by experimental studies alone. We outline areas where this method could be of benefit to both policy makers and individuals involved.","PeriodicalId":48576,"journal":{"name":"Journal of Causal Inference","volume":"129 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"136297925","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2023-01-01DOI: 10.48550/arXiv.2303.05396
J. Peña
Abstract We present two methods for bounding the probabilities of benefit (a.k.a. the probability of necessity and sufficiency, i.e., the desired effect occurs if and only if exposed) and harm (i.e., the undesired effect occurs if and only if exposed) under unmeasured confounding. The first method computes the upper or lower bound of either probability as a function of the observed data distribution and two intuitive sensitivity parameters, which can then be presented to the analyst as a 2-D plot to assist in decision-making. The second method assumes the existence of a measured nondifferential proxy for the unmeasured confounder. Using this proxy, tighter bounds than the existing ones can be derived from just the observed data distribution.
{"title":"Bounding the probabilities of benefit and harm through sensitivity parameters and proxies","authors":"J. Peña","doi":"10.48550/arXiv.2303.05396","DOIUrl":"https://doi.org/10.48550/arXiv.2303.05396","url":null,"abstract":"Abstract We present two methods for bounding the probabilities of benefit (a.k.a. the probability of necessity and sufficiency, i.e., the desired effect occurs if and only if exposed) and harm (i.e., the undesired effect occurs if and only if exposed) under unmeasured confounding. The first method computes the upper or lower bound of either probability as a function of the observed data distribution and two intuitive sensitivity parameters, which can then be presented to the analyst as a 2-D plot to assist in decision-making. The second method assumes the existence of a measured nondifferential proxy for the unmeasured confounder. Using this proxy, tighter bounds than the existing ones can be derived from just the observed data distribution.","PeriodicalId":48576,"journal":{"name":"Journal of Causal Inference","volume":"4 1","pages":""},"PeriodicalIF":1.4,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"78561775","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Abstract Understanding the mechanisms of action of interventions is a major general goal of scientific inquiry. The collection of statistical methods that use data to achieve this goal is referred to as mediation analysis. Natural direct and indirect effects provide a definition of mediation that matches scientific intuition, but they are not identified in the presence of time-varying confounding. Interventional effects have been proposed as a solution to this problem, but existing estimation methods are limited to assuming simple (e.g., linear) and unrealistic relations between the mediators, treatments, and confounders. We present an identification result for interventional effects in a general longitudinal data structure that allows flexibility in the specification of treatment-outcome, treatment-mediator, and mediator-outcome relationships. Identification is achieved under the standard no-unmeasured-confounders and positivity assumptions. In this article, we study semi-parametric efficiency theory for the functional identifying the mediation parameter, including the non-parametric efficiency bound, and was used to propose non-parametrically efficient estimators. Implementation of our estimators only relies on the availability of regression algorithms, and the estimators in a general framework that allows the analyst to use arbitrary regression machinery were developed. The estimators are doubly robust, n sqrt{n} -consistent, asymptotically Gaussian, under slow convergence rates for the regression algorithms used. This allows the use of flexible machine learning for regression while permitting uncertainty quantification through confidence intervals and p p -values. A free and open-source R package implementing the methods is available on GitHub. The proposed estimator to a motivating example from a trial of two medications for opioid-use disorder was applied, where we estimate the extent to which differences between the two treatments on risk of opioid use are mediated by craving symptoms.
{"title":"Efficient and flexible mediation analysis with time-varying mediators, treatments, and confounders","authors":"Iván Díaz, Nicholas T Williams, K. Rudolph","doi":"10.1515/jci-2022-0077","DOIUrl":"https://doi.org/10.1515/jci-2022-0077","url":null,"abstract":"Abstract Understanding the mechanisms of action of interventions is a major general goal of scientific inquiry. The collection of statistical methods that use data to achieve this goal is referred to as mediation analysis. Natural direct and indirect effects provide a definition of mediation that matches scientific intuition, but they are not identified in the presence of time-varying confounding. Interventional effects have been proposed as a solution to this problem, but existing estimation methods are limited to assuming simple (e.g., linear) and unrealistic relations between the mediators, treatments, and confounders. We present an identification result for interventional effects in a general longitudinal data structure that allows flexibility in the specification of treatment-outcome, treatment-mediator, and mediator-outcome relationships. Identification is achieved under the standard no-unmeasured-confounders and positivity assumptions. In this article, we study semi-parametric efficiency theory for the functional identifying the mediation parameter, including the non-parametric efficiency bound, and was used to propose non-parametrically efficient estimators. Implementation of our estimators only relies on the availability of regression algorithms, and the estimators in a general framework that allows the analyst to use arbitrary regression machinery were developed. The estimators are doubly robust, n sqrt{n} -consistent, asymptotically Gaussian, under slow convergence rates for the regression algorithms used. This allows the use of flexible machine learning for regression while permitting uncertainty quantification through confidence intervals and p p -values. A free and open-source R package implementing the methods is available on GitHub. The proposed estimator to a motivating example from a trial of two medications for opioid-use disorder was applied, where we estimate the extent to which differences between the two treatments on risk of opioid use are mediated by craving symptoms.","PeriodicalId":48576,"journal":{"name":"Journal of Causal Inference","volume":"10 2 1","pages":""},"PeriodicalIF":1.4,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"88192318","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}