Institute for Disability and Rehabilitation Research, Faculty of Health Sciences, Ontario Tech University, Oshawa, ON, Canada.
Department of Biomedical, Surgical and Dentals Sciences, University "La Statale", Milan, Italy.
Eur J Phys Rehabil Med. 2024 Feb;60(1):145-153. doi: 10.23736/S1973-9087.24.08435-1.
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.
康复提供者和政策制定者需要有效的证据来为人口的医疗需求做出明智的决策。只要有可能，这些决策就应该基于随机对照试验（RCT）。然而，在某些情况下，需要快速生成证据，或者当 RCT 不道德或不可行时。这些情况适用于研究复杂干预措施的效果，包括 Cochrane Rehabilitation 定义的康复。因此，我们探讨了使用 Hernán 及其同事提出的目标试验模拟框架来获得无法进行 RCT 时康复的因果效应的有效估计。目标试验模拟是一种通过模拟假设的 RCT 来指导使用观察数据设计和分析非随机比较有效性研究的框架。在康复的背景下，我们概述了使用真实世界数据应用目标试验模拟框架的步骤，强调了方法学考虑因素、局限性、潜在的缓解策略以及因果推理和反事实理论作为估计因果效应的基本原理。总的来说，我们旨在加强在无法进行 RCT 时估计康复因果效应的方法学方法。
