Pub Date : 2025-03-01Epub Date: 2025-03-10DOI: 10.1017/rsm.2025.6
Marianne A Jonker, Hassan Pazira, Anthony C C Coolen
To estimate accurately the parameters of a regression model, the sample size must be large enough relative to the number of possible predictors for the model. In practice, sufficient data is often lacking, which can lead to overfitting of the model and, as a consequence, unreliable predictions of the outcome of new patients. Pooling data from different data sets collected in different (medical) centers would alleviate this problem, but is often not feasible due to privacy regulation or logistic problems. An alternative route would be to analyze the local data in the centers separately and combine the statistical inference results with the Bayesian Federated Inference (BFI) methodology. The aim of this approach is to compute from the inference results in separate centers what would have been found if the statistical analysis was performed on the combined data. We explain the methodology under homogeneity and heterogeneity across the populations in the separate centers, and give real life examples for better understanding. Excellent performance of the proposed methodology is shown. An R-package to do all the calculations has been developed and is illustrated in this article. The mathematical details are given in the Appendix.
{"title":"Bayesian Federated Inference for regression models based on non-shared medical center data.","authors":"Marianne A Jonker, Hassan Pazira, Anthony C C Coolen","doi":"10.1017/rsm.2025.6","DOIUrl":"10.1017/rsm.2025.6","url":null,"abstract":"<p><p>To estimate accurately the parameters of a regression model, the sample size must be large enough relative to the number of possible predictors for the model. In practice, sufficient data is often lacking, which can lead to overfitting of the model and, as a consequence, unreliable predictions of the outcome of new patients. Pooling data from different data sets collected in different (medical) centers would alleviate this problem, but is often not feasible due to privacy regulation or logistic problems. An alternative route would be to analyze the local data in the centers separately and combine the statistical inference results with the Bayesian Federated Inference (BFI) methodology. The aim of this approach is to compute from the inference results in separate centers what would have been found if the statistical analysis was performed on the combined data. We explain the methodology under homogeneity and heterogeneity across the populations in the separate centers, and give real life examples for better understanding. Excellent performance of the proposed methodology is shown. An R-package to do all the calculations has been developed and is illustrated in this article. The mathematical details are given in the Appendix.</p>","PeriodicalId":226,"journal":{"name":"Research Synthesis Methods","volume":"16 2","pages":"383-423"},"PeriodicalIF":6.1,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12527543/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146103163","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-03-01Epub Date: 2025-03-07DOI: 10.1017/rsm.2025.1
Gary C K Chan, Estrid He, Janni Leung, Karin Verspoor
When conducting a systematic review, screening the vast body of literature to identify the small set of relevant studies is a labour-intensive and error-prone process. Although there is an increasing number of fully automated tools for screening, their performance is suboptimal and varies substantially across review topic areas. Many of these tools are only trained on small datasets, and most are not tested on a wide range of review topic areas. This study presents two systematic review datasets compiled from more than 8600 systematic reviews and more than 540000 abstracts covering 51 research topic areas in health and medical research. These datasets are the largest of their kinds to date. We demonstrate their utility in training and evaluating language models for title and abstract screening. Our dataset includes detailed metadata of each review, including title, background, objectives and selection criteria. We demonstrated that a small language model trained on this dataset with additional metadata has excellent performance with an average recall above 95% and specificity over 70% across a wide range of review topic areas. Future research can build on our dataset to further improve the performance of fully automated tools for systematic review title and abstract screening.
{"title":"A comprehensive systematic review dataset is a rich resource for training and evaluation of AI systems for title and abstract screening.","authors":"Gary C K Chan, Estrid He, Janni Leung, Karin Verspoor","doi":"10.1017/rsm.2025.1","DOIUrl":"10.1017/rsm.2025.1","url":null,"abstract":"<p><p>When conducting a systematic review, screening the vast body of literature to identify the small set of relevant studies is a labour-intensive and error-prone process. Although there is an increasing number of fully automated tools for screening, their performance is suboptimal and varies substantially across review topic areas. Many of these tools are only trained on small datasets, and most are not tested on a wide range of review topic areas. This study presents two systematic review datasets compiled from more than 8600 systematic reviews and more than 540000 abstracts covering 51 research topic areas in health and medical research. These datasets are the largest of their kinds to date. We demonstrate their utility in training and evaluating language models for title and abstract screening. Our dataset includes detailed metadata of each review, including title, background, objectives and selection criteria. We demonstrated that a small language model trained on this dataset with additional metadata has excellent performance with an average recall above 95% and specificity over 70% across a wide range of review topic areas. Future research can build on our dataset to further improve the performance of fully automated tools for systematic review title and abstract screening.</p>","PeriodicalId":226,"journal":{"name":"Research Synthesis Methods","volume":"16 2","pages":"308-322"},"PeriodicalIF":6.1,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12527522/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146103190","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Marianne A Jonker, Hassan Pazira, Anthony C C Coolen
{"title":"Bayesian Federated Inference for regression models based on non-shared medical center data - ERRATUM.","authors":"Marianne A Jonker, Hassan Pazira, Anthony C C Coolen","doi":"10.1017/rsm.2025.23","DOIUrl":"10.1017/rsm.2025.23","url":null,"abstract":"","PeriodicalId":226,"journal":{"name":"Research Synthesis Methods","volume":"16 2","pages":"424"},"PeriodicalIF":6.1,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12527526/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146103183","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-03-01Epub Date: 2025-03-17DOI: 10.1017/rsm.2024.12
Yu-Lun Liu, Bingyu Zhang, Haitao Chu, Yong Chen
Network meta-analysis (NMA), also known as mixed treatment comparison meta-analysis or multiple treatments meta-analysis, extends conventional pairwise meta-analysis by simultaneously synthesizing multiple interventions in a single integrated analysis. Despite the growing popularity of NMA within comparative effectiveness research, it comes with potential challenges. For example, within-study correlations among treatment comparisons are rarely reported in the published literature. Yet, these correlations are pivotal for valid statistical inference. As demonstrated in earlier studies, ignoring these correlations can inflate mean squared errors of the resulting point estimates and lead to inaccurate standard error estimates. This article introduces a composite likelihood-based approach that ensures accurate statistical inference without requiring knowledge of the within-study correlations. The proposed method is computationally robust and efficient, with substantially reduced computational time compared to the state-of-the-science methods implemented in R packages. The proposed method was evaluated through extensive simulations and applied to two important applications including an NMA comparing interventions for primary open-angle glaucoma, and another comparing treatments for chronic prostatitis and chronic pelvic pain syndrome.
{"title":"Network meta-analysis made simple: A composite likelihood approach.","authors":"Yu-Lun Liu, Bingyu Zhang, Haitao Chu, Yong Chen","doi":"10.1017/rsm.2024.12","DOIUrl":"10.1017/rsm.2024.12","url":null,"abstract":"<p><p>Network meta-analysis (NMA), also known as mixed treatment comparison meta-analysis or multiple treatments meta-analysis, extends conventional pairwise meta-analysis by simultaneously synthesizing multiple interventions in a single integrated analysis. Despite the growing popularity of NMA within comparative effectiveness research, it comes with potential challenges. For example, within-study correlations among treatment comparisons are rarely reported in the published literature. Yet, these correlations are pivotal for valid statistical inference. As demonstrated in earlier studies, ignoring these correlations can inflate mean squared errors of the resulting point estimates and lead to inaccurate standard error estimates. This article introduces a composite likelihood-based approach that ensures accurate statistical inference without requiring knowledge of the within-study correlations. The proposed method is computationally robust and efficient, with substantially reduced computational time compared to the state-of-the-science methods implemented in R packages. The proposed method was evaluated through extensive simulations and applied to two important applications including an NMA comparing interventions for primary open-angle glaucoma, and another comparing treatments for chronic prostatitis and chronic pelvic pain syndrome.</p>","PeriodicalId":226,"journal":{"name":"Research Synthesis Methods","volume":"16 2","pages":"272-290"},"PeriodicalIF":6.1,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12527528/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146103200","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"CausalMetaR: An R package for performing causally interpretable meta-analyses - ERRATUM.","authors":"Guanbo Wang, Sean McGrath, Yi Lian","doi":"10.1017/rsm.2025.22","DOIUrl":"10.1017/rsm.2025.22","url":null,"abstract":"","PeriodicalId":226,"journal":{"name":"Research Synthesis Methods","volume":"16 2","pages":"441"},"PeriodicalIF":6.1,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12527490/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146103177","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-03-01Epub Date: 2025-03-21DOI: 10.1017/rsm.2024.4
Lu Li, Lifeng Lin, Joseph C Cappelleri, Haitao Chu, Yong Chen
Double-zero-event studies (DZS) pose a challenge for accurately estimating the overall treatment effect in meta-analysis (MA). Current approaches, such as continuity correction or omission of DZS, are commonly employed, yet these ad hoc methods can yield biased conclusions. Although the standard bivariate generalized linear mixed model (BGLMM) can accommodate DZS, it fails to address the potential systemic differences between DZS and other studies. In this article, we propose a zero-inflated bivariate generalized linear mixed model (ZIBGLMM) to tackle this issue. This two-component finite mixture model includes zero inflation for a subpopulation with negligible or extremely low risk. We develop both frequentist and Bayesian versions of ZIBGLMM and examine its performance in estimating risk ratios against the BGLMM and conventional two-stage MA that excludes DZS. Through extensive simulation studies and real-world MA case studies, we demonstrate that ZIBGLMM outperforms the BGLMM and conventional two-stage MA that excludes DZS in estimating the true effect size with substantially less bias and comparable coverage probability.
{"title":"ZIBGLMM: Zero-inflated bivariate generalized linear mixed model for meta-analysis with double-zero-event studies.","authors":"Lu Li, Lifeng Lin, Joseph C Cappelleri, Haitao Chu, Yong Chen","doi":"10.1017/rsm.2024.4","DOIUrl":"10.1017/rsm.2024.4","url":null,"abstract":"<p><p>Double-zero-event studies (DZS) pose a challenge for accurately estimating the overall treatment effect in meta-analysis (MA). Current approaches, such as continuity correction or omission of DZS, are commonly employed, yet these ad hoc methods can yield biased conclusions. Although the standard bivariate generalized linear mixed model (BGLMM) can accommodate DZS, it fails to address the potential systemic differences between DZS and other studies. In this article, we propose a zero-inflated bivariate generalized linear mixed model (ZIBGLMM) to tackle this issue. This two-component finite mixture model includes zero inflation for a subpopulation with negligible or extremely low risk. We develop both frequentist and Bayesian versions of ZIBGLMM and examine its performance in estimating risk ratios against the BGLMM and conventional two-stage MA that excludes DZS. Through extensive simulation studies and real-world MA case studies, we demonstrate that ZIBGLMM outperforms the BGLMM and conventional two-stage MA that excludes DZS in estimating the true effect size with substantially less bias and comparable coverage probability.</p>","PeriodicalId":226,"journal":{"name":"Research Synthesis Methods","volume":"16 2","pages":"251-271"},"PeriodicalIF":6.1,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12527523/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146103148","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-03-01Epub Date: 2025-03-07DOI: 10.1017/rsm.2024.18
Zahra Premji, Hilary Kraus
Search filters are single-concept systematic search strategies created by experts. Filters are a valuable resource for systematic searchers. Typically, filters are designed for a single database in a single interface. If researchers do not have access to that specific interface, the existing filter will be unusable without translation. Filter translation is a complex process that requires an understanding of information retrieval concepts, as well as the unique indexing and search functionality of databases and interfaces. The authors undertook a project to translate an APA PsycInfo search filter for Randomized Controlled Trials/Clinical Controlled Trials (RCT/CCT), developed by Canada's Drug Agency, from the Wolters Kluwer Health Ovid interface to the EBSCO Information Services EBSCOhost interface. We present here a guide for translation, from the first principles of systematic searching to fine details of the relevant database and interfaces, based on our experience and illustrated by a worked example. We discuss each element of a systematic search in a stepwise process, addressing both the underlying information retrieval concepts and the technical strategies for effective translation between the two interfaces. We end with a discussion on translation challenges, with some guidance on how to mitigate potential impacts on sensitivity. While we have endeavored to explain the workings of this process accessibly for researchers who are not experts in systematic searching, anyone undertaking a search translation project should work with a trained information specialist if they lack information retrieval expertise or are unfamiliar with the inner workings of the database, the original interface, and the destination interface.
搜索过滤器是由专家创建的单概念系统搜索策略。过滤器是系统搜索器的宝贵资源。通常,过滤器是为单个接口中的单个数据库设计的。如果研究人员无法访问特定的接口,那么没有翻译,现有的过滤器将无法使用。过滤器翻译是一个复杂的过程,需要理解信息检索概念,以及数据库和接口的独特索引和搜索功能。作者承担了一个项目,将由加拿大药品管理局开发的APA PsycInfo随机对照试验/临床对照试验(RCT/CCT)搜索过滤器从Wolters Kluwer Health Ovid界面翻译到EBSCO信息服务EBSCOhost界面。在此,我们根据自己的经验,并通过一个工作实例,提供了一个翻译指南,从系统搜索的基本原则到相关数据库和接口的细节。我们在一个逐步的过程中讨论了系统搜索的每个元素,解决了潜在的信息检索概念和在两个接口之间有效转换的技术策略。最后,我们讨论了翻译面临的挑战,并就如何减轻对敏感性的潜在影响提供了一些指导。虽然我们努力为非系统搜索专家的研究人员解释这一过程的工作原理,但任何从事搜索翻译项目的人,如果缺乏信息检索专业知识或不熟悉数据库、原始接口和目标接口的内部工作原理,都应该与受过训练的信息专家合作。
{"title":"Translating systematic searches in the APA PsycInfo database from Ovid to EBSCOhost: A tutorial based on a filter translation.","authors":"Zahra Premji, Hilary Kraus","doi":"10.1017/rsm.2024.18","DOIUrl":"10.1017/rsm.2024.18","url":null,"abstract":"<p><p>Search filters are single-concept systematic search strategies created by experts. Filters are a valuable resource for systematic searchers. Typically, filters are designed for a single database in a single interface. If researchers do not have access to that specific interface, the existing filter will be unusable without translation. Filter translation is a complex process that requires an understanding of information retrieval concepts, as well as the unique indexing and search functionality of databases and interfaces. The authors undertook a project to translate an APA PsycInfo search filter for Randomized Controlled Trials/Clinical Controlled Trials (RCT/CCT), developed by Canada's Drug Agency, from the Wolters Kluwer Health Ovid interface to the EBSCO Information Services EBSCOhost interface. We present here a guide for translation, from the first principles of systematic searching to fine details of the relevant database and interfaces, based on our experience and illustrated by a worked example. We discuss each element of a systematic search in a stepwise process, addressing both the underlying information retrieval concepts and the technical strategies for effective translation between the two interfaces. We end with a discussion on translation challenges, with some guidance on how to mitigate potential impacts on sensitivity. While we have endeavored to explain the workings of this process accessibly for researchers who are not experts in systematic searching, anyone undertaking a search translation project should work with a trained information specialist if they lack information retrieval expertise or are unfamiliar with the inner workings of the database, the original interface, and the destination interface.</p>","PeriodicalId":226,"journal":{"name":"Research Synthesis Methods","volume":"16 2","pages":"228-250"},"PeriodicalIF":6.1,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12527542/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146103161","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-03-01Epub Date: 2025-03-21DOI: 10.1017/rsm.2025.3
Xuan Qin, Minghong Yao, Xiaochao Luo, Jiali Liu, Yu Ma, Yanmei Liu, Hao Li, Ke Deng, Kang Zou, Ling Li, Xin Sun
Machine learning (ML) models have been developed to identify randomised controlled trials (RCTs) to accelerate systematic reviews (SRs). However, their use has been limited due to concerns about their performance and practical benefits. We developed a high-recall ensemble learning model using Cochrane RCT data to enhance the identification of RCTs for rapid title and abstract screening in SRs and evaluated the model externally with our annotated RCT datasets. Additionally, we assessed the practical impact in terms of labour time savings and recall improvement under two scenarios: ML-assisted double screening (where ML and one reviewer screened all citations in parallel) and ML-assisted stepwise screening (where ML flagged all potential RCTs, and at least two reviewers subsequently filtered the flagged citations). Our model achieved twice the precision compared to the existing SVM model while maintaining a recall of 0.99 in both internal and external tests. In a practical evaluation with ML-assisted double screening, our model led to significant labour time savings (average 45.4%) and improved recall (average 0.998 compared to 0.919 for a single reviewer). In ML-assisted stepwise screening, the model performed similarly to standard manual screening but with average labour time savings of 74.4%. In conclusion, compared with existing methods, the proposed model can reduce workload while maintaining comparable recall when identifying RCTs during the title and abstract screening stages, thereby accelerating SRs. We propose practical recommendations to effectively apply ML-assisted manual screening when conducting SRs, depending on reviewer availability (ML-assisted double screening) or time constraints (ML-assisted stepwise screening).
{"title":"Machine learning for identifying randomised controlled trials when conducting systematic reviews: Development and evaluation of its impact on practice.","authors":"Xuan Qin, Minghong Yao, Xiaochao Luo, Jiali Liu, Yu Ma, Yanmei Liu, Hao Li, Ke Deng, Kang Zou, Ling Li, Xin Sun","doi":"10.1017/rsm.2025.3","DOIUrl":"10.1017/rsm.2025.3","url":null,"abstract":"<p><p>Machine learning (ML) models have been developed to identify randomised controlled trials (RCTs) to accelerate systematic reviews (SRs). However, their use has been limited due to concerns about their performance and practical benefits. We developed a high-recall ensemble learning model using Cochrane RCT data to enhance the identification of RCTs for rapid title and abstract screening in SRs and evaluated the model externally with our annotated RCT datasets. Additionally, we assessed the practical impact in terms of labour time savings and recall improvement under two scenarios: ML-assisted double screening (where ML and one reviewer screened all citations in parallel) and ML-assisted stepwise screening (where ML flagged all potential RCTs, and at least two reviewers subsequently filtered the flagged citations). Our model achieved twice the precision compared to the existing SVM model while maintaining a recall of 0.99 in both internal and external tests. In a practical evaluation with ML-assisted double screening, our model led to significant labour time savings (average 45.4%) and improved recall (average 0.998 compared to 0.919 for a single reviewer). In ML-assisted stepwise screening, the model performed similarly to standard manual screening but with average labour time savings of 74.4%. In conclusion, compared with existing methods, the proposed model can reduce workload while maintaining comparable recall when identifying RCTs during the title and abstract screening stages, thereby accelerating SRs. We propose practical recommendations to effectively apply ML-assisted manual screening when conducting SRs, depending on reviewer availability (ML-assisted double screening) or time constraints (ML-assisted stepwise screening).</p>","PeriodicalId":226,"journal":{"name":"Research Synthesis Methods","volume":"16 2","pages":"350-363"},"PeriodicalIF":6.1,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12527483/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146103188","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-03-01Epub Date: 2025-03-10DOI: 10.1017/rsm.2025.2
David M Phillippo, Antonio Remiro-Azócar, Anna Heath, Gianluca Baio, Sofia Dias, A E Ades, Nicky J Welton
Effect modification occurs when a covariate alters the relative effectiveness of treatment compared to control. It is widely understood that, when effect modification is present, treatment recommendations may vary by population and by subgroups within the population. Population-adjustment methods are increasingly used to adjust for differences in effect modifiers between study populations and to produce population-adjusted estimates in a relevant target population for decision-making. It is also widely understood that marginal and conditional estimands for non-collapsible effect measures, such as odds ratios or hazard ratios, do not in general coincide even without effect modification. However, the consequences of both non-collapsibility and effect modification together are little-discussed in the literature.In this article, we set out the definitions of conditional and marginal estimands, illustrate their properties when effect modification is present, and discuss the implications for decision-making. In particular, we show that effect modification can result in conflicting treatment rankings between conditional and marginal estimates. This is because conditional and marginal estimands correspond to different decision questions that are no longer aligned when effect modification is present. For time-to-event outcomes, the presence of covariates implies that marginal hazard ratios are time-varying, and effect modification can cause marginal hazard curves to cross. We conclude with practical recommendations for decision-making in the presence of effect modification, based on pragmatic comparisons of both conditional and marginal estimates in the decision target population. Currently, multilevel network meta-regression is the only population-adjustment method capable of producing both conditional and marginal estimates, in any decision target population.
{"title":"Effect modification and non-collapsibility together may lead to conflicting treatment decisions: A review of marginal and conditional estimands and recommendations for decision-making.","authors":"David M Phillippo, Antonio Remiro-Azócar, Anna Heath, Gianluca Baio, Sofia Dias, A E Ades, Nicky J Welton","doi":"10.1017/rsm.2025.2","DOIUrl":"10.1017/rsm.2025.2","url":null,"abstract":"<p><p>Effect modification occurs when a covariate alters the relative effectiveness of treatment compared to control. It is widely understood that, when effect modification is present, treatment recommendations may vary by population and by subgroups within the population. Population-adjustment methods are increasingly used to adjust for differences in effect modifiers between study populations and to produce population-adjusted estimates in a relevant target population for decision-making. It is also widely understood that marginal and conditional estimands for non-collapsible effect measures, such as odds ratios or hazard ratios, do not in general coincide even without effect modification. However, the consequences of both non-collapsibility and effect modification together are little-discussed in the literature.In this article, we set out the definitions of conditional and marginal estimands, illustrate their properties when effect modification is present, and discuss the implications for decision-making. In particular, we show that effect modification can result in conflicting treatment rankings between conditional and marginal estimates. This is because conditional and marginal estimands correspond to different decision questions that are no longer aligned when effect modification is present. For time-to-event outcomes, the presence of covariates implies that marginal hazard ratios are time-varying, and effect modification can cause marginal hazard curves to cross. We conclude with practical recommendations for decision-making in the presence of effect modification, based on pragmatic comparisons of both conditional and marginal estimates in the decision target population. Currently, multilevel network meta-regression is the only population-adjustment method capable of producing both conditional and marginal estimates, in any decision target population.</p>","PeriodicalId":226,"journal":{"name":"Research Synthesis Methods","volume":"16 2","pages":"323-349"},"PeriodicalIF":6.1,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12527544/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146103230","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-03-01Epub Date: 2025-03-10DOI: 10.1017/rsm.2025.4
Kaitlyn G Fitzgerald, David Khella, Avery Charles, Elizabeth Tipton
Results of meta-analyses are of interest not only to researchers but often to policy-makers and other decision-makers (e.g., in education and medicine), and visualizations play an important role in communicating data and statistical evidence to the broader public. Therefore, the potential audience of meta-analytic visualizations is broad. However, the most common meta-analytic visualization - the forest plot - uses non-optimal design principles that do not align with data visualization best practices and relies on statistical knowledge and conventions not likely to be familiar to a broad audience. Previously, the Meta-Analytic Rain Cloud (MARC) plot has been shown to be an effective alternative to a forest plot when communicating the results of a small meta-analysis to education practitioners. However, the original MARC plot design was not well-suited for meta-analyses with large numbers of effect sizes as is common across the social sciences. This paper presents an extension of the MARC plot, intended for effective communication of moderate to large meta-analyses (k = 10, 20, 50, 100 studies). We discuss the design principles of the MARC plot, grounded in the data visualization and cognitive science literature. We then present the methods and results of a randomized survey experiment to evaluate the revised MARC plot in comparison to the original MARC plot, the forest plot, and a bar plot. We find that the revised MARC plot is more effective for communicating moderate to large meta-analyses to non-research audiences, offering a 0.30, 0.34, and 1.07 standard deviation improvement in chart users' scores compared to the original MARC plot, forest plot, and bar plot, respectively.
{"title":"Meta-analytic rain cloud plots: Improving evidence communication through data visualization design principles.","authors":"Kaitlyn G Fitzgerald, David Khella, Avery Charles, Elizabeth Tipton","doi":"10.1017/rsm.2025.4","DOIUrl":"10.1017/rsm.2025.4","url":null,"abstract":"<p><p>Results of meta-analyses are of interest not only to researchers but often to policy-makers and other decision-makers (e.g., in education and medicine), and visualizations play an important role in communicating data and statistical evidence to the broader public. Therefore, the potential audience of meta-analytic visualizations is broad. However, the most common meta-analytic visualization - the forest plot - uses non-optimal design principles that do not align with data visualization best practices and relies on statistical knowledge and conventions not likely to be familiar to a broad audience. Previously, the Meta-Analytic Rain Cloud (MARC) plot has been shown to be an effective alternative to a forest plot when communicating the results of a small meta-analysis to education practitioners. However, the original MARC plot design was not well-suited for meta-analyses with large numbers of effect sizes as is common across the social sciences. This paper presents an extension of the MARC plot, intended for effective communication of moderate to large meta-analyses (<i>k</i> = 10, 20, 50, 100 studies). We discuss the design principles of the MARC plot, grounded in the data visualization and cognitive science literature. We then present the methods and results of a randomized survey experiment to evaluate the revised MARC plot in comparison to the original MARC plot, the forest plot, and a bar plot. We find that the revised MARC plot is more effective for communicating moderate to large meta-analyses to non-research audiences, offering a 0.30, 0.34, and 1.07 standard deviation improvement in chart users' scores compared to the original MARC plot, forest plot, and bar plot, respectively.</p>","PeriodicalId":226,"journal":{"name":"Research Synthesis Methods","volume":"16 2","pages":"364-382"},"PeriodicalIF":6.1,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12527513/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146103151","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}