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Commentary on ``Nonparametric identification is not enough, but randomized controlled trials are'': Statistical considerations for generating reliable evidence across a spectrum of studies that increasingly involve real-world elements. 对“非参数识别是不够的,但随机对照试验是”的评论:在越来越多地涉及现实世界元素的一系列研究中产生可靠证据的统计考虑。
Pub Date : 2025-04-11 eCollection Date: 2025-01-01 DOI: 10.1353/obs.2025.a956842
Rachael Phillips, Mark van der Laan

Judea Pearl, quoted in Pearl and Mackenzie (2008), stated that "once we have understood why [randomized controlled trials] RCTs work, there is no need to put them on a pedestal and treat them as the gold standard of causal analysis, which all other methods should emulate." In Aronow et al. (2024), this claim is refuted, drawing on results of Robins and Ritov (1997). The argument is made that statistical estimation and inference tend to be fundamentally more difficult in observational studies than in randomized controlled trials, even when all confounders are observed and measured without error. We congratulate the authors for raising this highly timely, interesting discussion and welcome this opportunity to join this important debate. In this commentary, we focus on what it takes to generate reliable evidence across a spectrum of studies that increasingly involve real-world elements and less control over design. A related question is whether, along this spectrum of studies, the reliability of evidence generated by a statistical analysis decreases. We claim that this is not the case, but that the challenge for the appropriate statistical method increases, requiring sophisticated and careful execution.

Pearl and Mackenzie(2008)引用朱迪亚·珀尔(Judea Pearl)的话说:“一旦我们理解了随机对照试验(rrcts)有效的原因,就没有必要把它们奉为因果分析的黄金标准,所有其他方法都应该效仿。”在Aronow et al.(2024)中,这一说法被反驳,借鉴了Robins和Ritov(1997)的结果。有人认为,在观察性研究中,统计估计和推断往往从根本上比在随机对照试验中更困难,即使所有的混杂因素都被准确地观察和测量。我们祝贺作者提出了这一非常及时、有趣的讨论,并欢迎有机会参加这一重要的辩论。在这篇评论中,我们关注的是如何在一系列研究中产生可靠的证据,这些研究越来越多地涉及现实世界的元素,对设计的控制越来越少。一个相关的问题是,在这一系列研究中,统计分析产生的证据的可靠性是否会降低。我们声称,情况并非如此,而是对适当统计方法的挑战增加了,需要复杂和仔细的执行。
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引用次数: 0
Nonparametric identification is not enough, but randomized controlled trials are. 非参数识别是不够的,但随机对照试验。
Pub Date : 2025-04-11 eCollection Date: 2025-01-01 DOI: 10.1353/obs.2025.a956837
P M Aronow, James M Robins, Theo Saarinen, Fredrik Sävje, Jasjeet S Sekhon

We argue that randomized controlled trials (RCTs) are special even among studies for which a nonparametric unconfoundedness assumption is credible. This claim follows from two results of Robins and Ritov (1997). First, in settings with at least one continuous confounder, there exists no estimator of the average treatment effect that is uniformly consistent unless the propensity score is known or additional assumptions are made on the complexity of the propensity score function. Second, with binary outcomes, knowledge of the propensity score yields a uniformly consistent estimator and finite-sample valid confidence intervals that shrink at a parametric rate, regardless of how complicated the propensity score function might be. We emphasize the latter point, and note that a successfully executed RCT provides knowledge of the propensity score to the researcher. We conclude that statistical estimation and inference tend to be fundamentally more difficult in observational settings than in RCTs, even when all confounders are observed and measured without error.

我们认为,随机对照试验(rct)是特殊的研究,即使是非参数无混杂假设是可信的。这一说法来自罗宾斯和里托夫(1997)的两个结果。首先,在至少有一个连续混杂因素的设置中,除非已知倾向得分或对倾向得分函数的复杂性做出额外假设,否则不存在均匀一致的平均治疗效果估计量。其次,对于二元结果,倾向得分的知识产生一致的估计量和有限样本有效置信区间,该置信区间以参数速率收缩,无论倾向得分函数可能有多复杂。我们强调后一点,并注意到成功执行的RCT为研究人员提供了倾向得分的知识。我们的结论是,在观察环境中,统计估计和推断往往比在随机对照试验中更困难,即使在所有混杂因素都被准确地观察和测量时也是如此。
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引用次数: 0
Why are RCTs the Gold Standard? The Epistemological Difference Between Randomized Experiments and Observational Studies. 为什么随机对照试验是黄金标准?随机实验与观察性研究的认识论差异。
Pub Date : 2025-04-11 eCollection Date: 2025-01-01 DOI: 10.1353/obs.2025.a956840
Christopher Harshaw

In response to Pearl, Aronow et al. (2025) argue that randomized experiments are special among causal inference methods due to their statistical properties. I believe that the key distinction between randomized experiments and observational studies is not statistical, but rather epistemological in nature. In this comment, I aim to articulate this epistemological distinction and argue that it ought to take a more central role in these discussions.

针对Pearl, Aronow等人(2025)认为随机实验因其统计特性而在因果推理方法中是特殊的。我认为,随机实验和观察性研究之间的关键区别不在于统计,而在于本质上的认识论。在这篇评论中,我的目的是阐明这种认识论上的区别,并认为它应该在这些讨论中发挥更重要的作用。
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引用次数: 0
Enough? 足够了吗?
Pub Date : 2025-04-11 eCollection Date: 2025-01-01 DOI: 10.1353/obs.2025.a956838
Drew Dimmery, Kevin Munger

We provide a critical response to Aronow et al. (2021) which argued that randomized controlled trials (RCTs) are "enough," while nonparametric identification in observational studies is not. We first investigate what is meant by "enough," arguing that this is a fundamentally a sociological claim about the relationship between statistical work and relevant institutional processes (here, academic peer review), rather than something that can be decided from within the logic of statistics. For a more complete conception of "enough," we outline all that would need to be known - not just knowledge of propensity scores, but knowledge of many other spatial and temporal characteristics of the social world. Even granting the logic of the critique in Aronow et al. (2021), its practical importance is a question of the contexts under study. We argue that we should not be satisfied by appeals to intuition or experience about the complexity of "naturally occurring" propensity score functions. Instead, we call for more empirical metascience to begin to characterize this complexity. We apply this logic to the case of recommender systems as a demonstration of the weakness of allowing statisticians' intuitions to serve in place of metascientific data. This may be, as Aronow et al. (2021) claim, one of the "few free lunches in statistics"-but like many of the free lunches consumed by statisticians, it is only available to those working at a handful of large tech firms. Rather than implicitly deciding what is "enough" based on statistical applications the social world has determined to be most profitable, we are argue that practicing statisticians should explicitly engage with questions like "for what?" and "for whom?" in order to adequately answer the question of "enough?"

Aronow等人(2021)认为随机对照试验(rct)“足够”,而观察性研究中的非参数识别则不够。我们首先调查了“足够”的含义,认为这基本上是一个关于统计工作与相关制度过程(这里是学术同行评审)之间关系的社会学主张,而不是可以从统计逻辑中决定的东西。为了得到一个更完整的“足够”的概念,我们概述了所有需要知道的东西——不仅仅是倾向得分的知识,还有社会世界的许多其他空间和时间特征的知识。即使承认Aronow等人(2021)的批评逻辑,其实际重要性也是研究背景的问题。我们认为,对于“自然发生的”倾向得分函数的复杂性,我们不应该满足于诉诸直觉或经验。相反,我们需要更多的经验元科学来开始描述这种复杂性。我们将这种逻辑应用到推荐系统的案例中,以证明允许统计学家的直觉代替元科学数据的弱点。正如Aronow等人(2021)所声称的那样,这可能是“统计学中为数不多的免费午餐”之一——但就像许多统计学家享用的免费午餐一样,它只适用于少数几家大型科技公司的员工。我们认为,实践统计学家应该明确地参与诸如“为了什么?”和“为了谁?”这样的问题,以便充分回答“足够”的问题,而不是根据社会确定的最有利可图的统计应用来隐含地决定什么是“足够”。
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引用次数: 0
Rejoinder: Nonparametric identification is not enough, but randomized controlled trials are. 反驳:非参数识别是不够的,但随机对照试验是足够的。
Pub Date : 2025-04-11 eCollection Date: 2025-01-01 DOI: 10.1353/obs.2025.a956844
P M Aronow, James M Robins, Theo Saarinen, Fredrik Sävje, Jasjeet S Sekhon

We thank the editor for organizing a diverse and wide-ranging discussion, and we thank the commentators for their detailed and thoughtful remarks. Most of the commentators provide broader perspectives on randomized experiments and their role in modern empirical practice. We believe this broader perspective is important, and the comments serve as complements to the somewhat narrow points we made in our paper. However, we believe these narrow points are of great consequence, and we find it useful to briefly recapitulate them here. When a practitioner aims to estimate averages of bounded potential outcomes (e.g., the average treatment effect on a binary outcome) in a setting where both ignorability and positivity are known to hold after adjusting for at least one continuous covariate, the following statements are true: • If the propensity score is known, such as in a randomized controlled trial (RCT), there exist simple estimators that are uniformly root-n consistent and asymptotically normal. Confidence intervals based on these estimators are finite-sample valid and their widths shrink at a root-n rate. • If the propensity score is not known, such as in an observational study, there exist neither uniformly consistent estimators nor uniform (i.e., honest) large-sample confidence intervals whose widths are shrinking with the sample size. To achieve these properties, the practitioner must impose untestable assumptions on either the propensity score function or the conditional expectation function of the outcomes.

我们感谢编辑组织了一场多样而广泛的讨论,我们感谢评论员们详细而深思熟虑的评论。大多数评论者对随机实验及其在现代实证实践中的作用提供了更广泛的观点。我们认为这种更广泛的观点是重要的,这些评论是对我们在论文中提出的一些狭隘观点的补充。然而,我们认为这些狭隘的观点具有重大意义,我们认为在这里简要地概括一下是有益的。当从业者的目标是估计有界潜在结果的平均值(例如,在至少一个连续协变量调整后,已知可忽略性和正性都保持不变的情况下,二元结果的平均治疗效果)时,以下陈述是正确的:•如果倾向得分已知,例如在随机对照试验(RCT)中,存在一致的根n一致且渐近正态的简单估计量。基于这些估计的置信区间是有限样本有效的,它们的宽度以根n的速率收缩。•如果倾向得分未知,例如在观察性研究中,则既不存在统一一致的估计量,也不存在统一的(即诚实的)大样本置信区间,其宽度随样本量而缩小。为了实现这些特性,从业者必须对结果的倾向得分函数或条件期望函数施加不可检验的假设。
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引用次数: 0
A Bureaucratic Theory of Statistics. 统计的官僚理论。
Pub Date : 2025-04-11 eCollection Date: 2025-01-01 DOI: 10.1353/obs.2025.a956843
Benjamin Recht

This commentary proposes a framework for understanding the role of statistics in policymaking, regulation, and bureaucratic systems. I introduce the concept of "ex ante policy," describing statistical rules and procedures designed before data collection to govern future actions. Through examining examples, particularly clinical trials, I explore how ex ante policy serves as a calculus of bureaucracy, providing numerical foundations for governance through clear, transparent rules. The ex ante frame obviates heated debates about inferential interpretations of probability and statistical tests, p-values, and rituals. I conclude by calling for a deeper appreciation of statistics' bureaucratic function and suggesting new directions for research in policy-oriented statistical methodology.

这篇评论提出了一个理解统计在政策制定、监管和官僚体系中的作用的框架。我介绍了“事前政策”的概念,描述了在数据收集之前设计的统计规则和程序,以管理未来的行动。通过研究实例,特别是临床试验,我探讨了事前政策如何作为官僚主义的演算,通过清晰、透明的规则为治理提供数字基础。事前框架避免了关于概率和统计检验、p值和仪式的推理解释的激烈辩论。最后,我呼吁更深入地了解统计的官僚功能,并为政策导向统计方法的研究提出新的方向。
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引用次数: 0
Priors and Propensity Scores in Bayesian Causal Inference. 贝叶斯因果推理中的先验和倾向得分。
Pub Date : 2025-04-11 eCollection Date: 2025-01-01 DOI: 10.1353/obs.2025.a956841
Arman Oganisian, Antonio Linero

Aronow et al. (2025) provide a convincing case for the special status of randomized controlled trials (RCTs) in which the propensity scores are known and can be used to make causal inferences. Here we provide a Bayesian perspective on their work by summarizing recent developments in the Bayesian literature on the topic. Whether the propensity score should play a role in Bayesian causal inference - and what that role(s) should be - has been a controversial topic for some time. We begin by describing Bayesian inference for population-level estimands and show that under commonly made (but not necessarily required) assumptions, the propensity score model has no role to play in Bayesian causal inference from a purist perspective. We discuss recent work on why these assumptions can be problematic - particularly in high-dimensional models - and discuss several Bayesian motivations for relaxing them. We describe out recent approaches for incorporating the propensity score correspond to di erent ways of relaxing these assumptions. Given these considerations, we illustrate how a Bayesian might approach the synethic examples of Aronow et al. (2025).

Aronow等人(2025)为随机对照试验(rct)的特殊地位提供了一个令人信服的案例,在随机对照试验中,倾向得分是已知的,可以用来进行因果推断。在这里,我们通过总结关于该主题的贝叶斯文献的最新发展,提供了他们工作的贝叶斯观点。倾向得分是否应该在贝叶斯因果推理中发挥作用——以及应该发挥什么样的作用——一直是一个有争议的话题。我们首先描述总体水平估计的贝叶斯推断,并表明在通常做出的(但不一定是必需的)假设下,从纯粹主义的角度来看,倾向得分模型在贝叶斯因果推断中没有作用。我们讨论了最近关于为什么这些假设会有问题的研究——特别是在高维模型中——并讨论了放松这些假设的几个贝叶斯动机。我们描述了最近合并倾向得分的方法,这些方法对应于放松这些假设的不同方式。考虑到这些因素,我们说明了贝叶斯如何接近Aronow等人(2025)的综合例子。
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引用次数: 0
What randomization can and cannot guarantee. 随机化能保证什么,不能保证什么。
Pub Date : 2025-04-11 eCollection Date: 2025-01-01 DOI: 10.1353/obs.2025.a956839
Peng Ding

Aronow et al. (2024) provide a great service to the causal inference community by delineating the key results in Robins and Ritov (1997). They show that randomized controlled trials (RCTs) ensure much stronger statistical inference than unconfounded observational studies even though nonparametric identification is identical in both settings. These results are in sharp contrast to the claim in Pearl and Mackenzie (2018) that RCTs are not the gold standard of causal analysis. Pearl and Mackenzie's (2018) claim is false and misleading for empirical researchers who want to infer causal effects based on data with finite sample sizes. I will further review what randomization can and cannot guarantee more broadly. In particular, I will highlight the value of randomization-based inference in RCTs, the limit of randomization alone for more complicated causal inference questions, and the importance of sensitivity analysis in observational studies.

Aronow等人(2024)通过描述Robins和Ritov(1997)的关键结果,为因果推理界提供了很大的服务。他们表明,随机对照试验(rct)确保比非混杂观察性研究更强的统计推断,即使在两种情况下非参数识别是相同的。这些结果与Pearl和Mackenzie(2018)的说法形成鲜明对比,后者认为随机对照试验不是因果分析的黄金标准。Pearl和Mackenzie(2018)的说法是错误的,对于那些想要根据有限样本量的数据推断因果关系的实证研究人员来说是误导的。我将进一步更广泛地回顾随机化能保证什么和不能保证什么。特别是,我将强调随机化推理在随机对照试验中的价值,随机化在更复杂的因果推理问题中的局限性,以及敏感性分析在观察性研究中的重要性。
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引用次数: 0
Does matching introduce confounding or selection bias into the matched case-control design? 匹配病例对照设计是否会引入混杂或选择偏差?
Pub Date : 2024-06-06 DOI: 10.1353/obs.2024.a929114
Fei Wan, S. Sutcliffe, Jeffrey Zhang, Dylan Small
Abstract:The impact of matching on confounding control in case-control studies remains a subject of ongoing debate, with varying perspectives among researchers. While matching is a well-established method for controlling confounding in cohort studies, its effectiveness in mitigating confounding in case-control studies has long been questioned. Recent studies have determined that matching doesn't eliminate confounding but, instead, introduces a selection bias on top of the initial confounding, as indicated by causal diagram analysis. This conclusion suggests that the control of initial confounding through matching is either only partial or non-existent. However, this conclusion may not be accurate in exactly matched design because causal diagram cannot always reveal precisely the interplay between the initial confounding and the matching induced selection effect. In this paper, we employ analytical results in conjunction with causal diagrams to demonstrate that the cancellation of the initial confounding by the selection effect is complete in exact individually matched case-control studies. Nevertheless, this cancellation results in a residual selection effect that establishes a backdoor connection between the matching factors and the outcome in the matched design. Failure to adjust for this residual selection effect leads to biased estimates of the exposure effect. Furthermore, this backdoor connection causes matching factors to act like confounding factors in the matched case-control design, which complicates the interpretation of the bias introduced by matching in current literature.
摘要:在病例对照研究中,配对对混杂控制的影响一直是一个争论不休的话题,研究人员的观点也不尽相同。在队列研究中,配对是一种行之有效的混杂控制方法,但在病例对照研究中,配对在减少混杂方面的效果却一直受到质疑。最近的研究发现,匹配并不能消除混杂,反而会在初始混杂的基础上引入选择偏倚,因果图分析表明了这一点。这一结论表明,通过配对对初始混杂的控制要么只是部分的,要么根本不存在。然而,这一结论在完全匹配的设计中可能并不准确,因为因果图并不能总是精确地揭示初始混杂和匹配诱导的选择效应之间的相互作用。在本文中,我们将分析结果与因果图结合起来,证明在精确个体匹配的病例对照研究中,选择效应对初始混杂的抵消是完全的。然而,这种抵消会导致残余选择效应,在匹配设计中建立起匹配因素与结果之间的后门联系。如果不对这种残余选择效应进行调整,就会导致对暴露效应的估计出现偏差。此外,这种后门联系会使匹配因素在匹配病例对照设计中起到类似混杂因素的作用,从而使目前文献中对匹配所带来的偏差的解释变得复杂。
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引用次数: 0
Using a difference-in-difference control trial to test an intervention aimed at increasing the take-up of a welfare payment in New Zealand 使用差异中差异对照试验来测试旨在增加新西兰福利支付的干预措施
Pub Date : 2023-09-07 DOI: 10.1353/obs.2023.a906626
David Rea, Dean R. Hyslop
Abstract:This paper describes a difference-in-difference control trial (DDCT) of an intervention designed to increase the take-up of an income support payment in the New Zealand welfare system. The intervention used a microsimulation model to identify potential claimants who were then contacted by either phone, email, or letter. The trial was designed as a DDCT because of ethical concerns associated with a fully randomized approach. The trial provided convincing evidence that the intervention would increase the take-up of the payment and a modified version was then implemented as an ongoing business process by the New Zealand Ministry of Social Development (MSD). The findings from the trial contribute to the literature about how best to increase the take-up of welfare payments. The study also demonstrates the value of using a difference-in-difference control trial.
摘要:本文描述了一项干预措施的差异控制试验(DDCT),旨在提高新西兰福利系统中收入支持支付的接受率。干预使用微观模拟模型来识别潜在的索赔人,然后通过电话、电子邮件或信件联系他们。由于与完全随机方法相关的伦理问题,该试验被设计为DDCT。该试验提供了令人信服的证据,表明干预措施将增加付款的接受率,新西兰社会发展部随后将修改后的版本作为一项持续的业务流程实施。该试验的结果为如何最好地提高福利金的使用率的文献做出了贡献。该研究还证明了使用差异对照试验的价值。
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引用次数: 0
期刊
Observational studies
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