Pierre Côté, Stefano Negrini, Sabrina Donzelli, Carlotte Kiekens, Chiara Arienti, Maria G Ceravolo, Douglas P Gross, Irene Battel, Giorgio Ferriero, Stefano G Lazzarini, Bernard Dan, Heather M Shearer, Jessica J Wong
{"title":"康复目标试验模拟简介:利用观察数据模拟随机对照试验的系统方法。","authors":"Pierre Côté, Stefano Negrini, Sabrina Donzelli, Carlotte Kiekens, Chiara Arienti, Maria G Ceravolo, Douglas P Gross, Irene Battel, Giorgio Ferriero, Stefano G Lazzarini, Bernard Dan, Heather M Shearer, Jessica J Wong","doi":"10.23736/S1973-9087.24.08435-1","DOIUrl":null,"url":null,"abstract":"<p><p>Rehabilitation providers and policymakers need valid evidence to make informed decisions about the healthcare needs of the population. Whenever possible, these decisions should be informed by randomized controlled trials (RCTs). However, there are circumstances when evidence needs to be generated rapidly, or when RCTs are not ethical or feasible. These situations apply to studying the effects of complex interventions, including rehabilitation as defined by Cochrane Rehabilitation. Therefore, we explore using the target trial emulation framework by Hernán and colleagues to obtain valid estimates of the causal effects of rehabilitation when RCTs cannot be conducted. Target trial emulation is a framework guiding the design and analysis of non-randomized comparative effectiveness studies using observational data, by emulating a hypothetical RCT. In the context of rehabilitation, we outline steps for applying the target trial emulation framework using real world data, highlighting methodological considerations, limitations, potential mitigating strategies, and causal inference and counterfactual theory as foundational principles to estimating causal effects. Overall, we aim to strengthen methodological approaches used to estimate causal effects of rehabilitation when RCTs cannot be conducted.</p>","PeriodicalId":12044,"journal":{"name":"European journal of physical and rehabilitation medicine","volume":"60 1","pages":"145-153"},"PeriodicalIF":3.3000,"publicationDate":"2024-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10938938/pdf/","citationCount":"0","resultStr":"{\"title\":\"Introduction to target trial emulation in rehabilitation: a systematic approach to emulate a randomized controlled trial using observational data.\",\"authors\":\"Pierre Côté, Stefano Negrini, Sabrina Donzelli, Carlotte Kiekens, Chiara Arienti, Maria G Ceravolo, Douglas P Gross, Irene Battel, Giorgio Ferriero, Stefano G Lazzarini, Bernard Dan, Heather M Shearer, Jessica J Wong\",\"doi\":\"10.23736/S1973-9087.24.08435-1\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>Rehabilitation providers and policymakers need valid evidence to make informed decisions about the healthcare needs of the population. Whenever possible, these decisions should be informed by randomized controlled trials (RCTs). However, there are circumstances when evidence needs to be generated rapidly, or when RCTs are not ethical or feasible. These situations apply to studying the effects of complex interventions, including rehabilitation as defined by Cochrane Rehabilitation. Therefore, we explore using the target trial emulation framework by Hernán and colleagues to obtain valid estimates of the causal effects of rehabilitation when RCTs cannot be conducted. Target trial emulation is a framework guiding the design and analysis of non-randomized comparative effectiveness studies using observational data, by emulating a hypothetical RCT. In the context of rehabilitation, we outline steps for applying the target trial emulation framework using real world data, highlighting methodological considerations, limitations, potential mitigating strategies, and causal inference and counterfactual theory as foundational principles to estimating causal effects. Overall, we aim to strengthen methodological approaches used to estimate causal effects of rehabilitation when RCTs cannot be conducted.</p>\",\"PeriodicalId\":12044,\"journal\":{\"name\":\"European journal of physical and rehabilitation medicine\",\"volume\":\"60 1\",\"pages\":\"145-153\"},\"PeriodicalIF\":3.3000,\"publicationDate\":\"2024-02-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10938938/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"European journal of physical and rehabilitation medicine\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.23736/S1973-9087.24.08435-1\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"REHABILITATION\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"European journal of physical and rehabilitation medicine","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.23736/S1973-9087.24.08435-1","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"REHABILITATION","Score":null,"Total":0}
Introduction to target trial emulation in rehabilitation: a systematic approach to emulate a randomized controlled trial using observational data.
Rehabilitation providers and policymakers need valid evidence to make informed decisions about the healthcare needs of the population. Whenever possible, these decisions should be informed by randomized controlled trials (RCTs). However, there are circumstances when evidence needs to be generated rapidly, or when RCTs are not ethical or feasible. These situations apply to studying the effects of complex interventions, including rehabilitation as defined by Cochrane Rehabilitation. Therefore, we explore using the target trial emulation framework by Hernán and colleagues to obtain valid estimates of the causal effects of rehabilitation when RCTs cannot be conducted. Target trial emulation is a framework guiding the design and analysis of non-randomized comparative effectiveness studies using observational data, by emulating a hypothetical RCT. In the context of rehabilitation, we outline steps for applying the target trial emulation framework using real world data, highlighting methodological considerations, limitations, potential mitigating strategies, and causal inference and counterfactual theory as foundational principles to estimating causal effects. Overall, we aim to strengthen methodological approaches used to estimate causal effects of rehabilitation when RCTs cannot be conducted.