In this paper we propose methods for estimating heterogeneity in causal effects in experimental and observational studies and for conducting hypothesis tests about the magnitude of differences in treatment effects across subsets of the population. We provide a data-driven approach to partition the data into subpopulations that differ in the magnitude of their treatment effects. The approach enables the construction of valid confidence intervals for treatment effects, even with many covariates relative to the sample size, and without “sparsity” assumptions. We propose an “honest” approach to estimation, whereby one sample is used to construct the partition and another to estimate treatment effects for each subpopulation. Our approach builds on regression tree methods, modified to optimize for goodness of fit in treatment effects and to account for honest estimation. Our model selection criterion anticipates that bias will be eliminated by honest estimation and also accounts for the effect of making additional splits on the variance of treatment effect estimates within each subpopulation. We address the challenge that the “ground truth” for a causal effect is not observed for any individual unit, so that standard approaches to cross-validation must be modified. Through a simulation study, we show that for our preferred method honest estimation results in nominal coverage for 90% confidence intervals, whereas coverage ranges between 74% and 84% for nonhonest approaches. Honest estimation requires estimating the model with a smaller sample size; the cost in terms of mean squared error of treatment effects for our preferred method ranges between 7–22%.
Statistical Models Answer the Fundamental Clinical Question and Provide Clinical Trial Estimands – Statistical Thinking
Specific goals and estimation targets for randomized clinical trials have still not been well defined for general outcome variables. Proponents of causal inference calculus have claimed to define goals and estimands, but they have largely done so in a way that is not concordant with the most popular design, the parallel-group randomized trial. Causal inferential methods require the use of counterfactuals that are not informed by any data (outside of crossover studies) and make assumptions that are unverifiable, e.g., about the correlation structure of potential outcomes. Causal inferential structure also leads practitioners to act as if marginal treatment effect estimates are both helpful in decision making and transport to populations when in fact neither is true. Heterogeneity of participants within a treatment arm dictates heterogeneity of outcomes and heterogeneity of treatment effects when quantified on an absolute scale. Statistical models are best poised for estimation and causal inference that is specific to patient types. The increasing generality and robustness of statistical models bolsters the case. In this article I provide a succinct statement of the clinical goal of a parallel-group trial, and statistical estimands for it in the context of a general family of robust and efficient ordinal models that contain virtually all routinely used statistical models and tests as special cases.
Estimating Causal Effects of Treatments in Randomized and Nonrandomized Studies
Presents a discussion of matching, randomization, random sampling, and other methods of controlling extraneous variation. The objective was to specify the benefits of randomization in estimating causal effects of treatments. It is concluded that randomization should be employed whenever possible but that the use of carefully controlled nonrandomized data to estimate causal effects is a reasonable and necessary procedure in many cases.
Reducing Bias in Observational Studies Using Subclassification on the Propensity Score
The propensity score is the conditional probability of assignment to a particular treatment given a vector of observed covariates. Previous theoretical arguments have shown that subclassification on the propensity score will balance all observed covariates. Subclassification on an estimated propensity score is illustrated, using observational data on treatments for coronary artery disease. Five subclasses defined by the estimated propensity score are constructed that balance 74 covariates, and thereby provide estimates of treatment effects using direct adjustment. These subclasses are applied within subpopulations, and model-based adjustments are then used to provide estimates of treatment effects within these subpopulations. Two appendixes address theoretical issues related to the application: the effectiveness of subclassification on the propensity score in removing bias, and balancing properties of propensity scores with incomplete data.
Machine Learning Who to Nudge: Causal vs Predictive Targeting in a Field Experiment on Student Financial Aid Renewal
In many settings, interventions may be more effective for some individuals than others, so that targeting interventions may be beneficial. We analyze the value of targeting in the context of a large-scale field experiment with over 53,000 college students, where the goal was to use "nudges" to encourage students to renew their financial-aid applications before a non-binding deadline. We begin with baseline approaches to targeting. First, we target based on a causal forest that estimates heterogeneous treatment effects and then assigns students to treatment according to those estimated to have the highest treatment effects. Next, we evaluate two alternative targeting policies, one targeting students with low predicted probability of renewing financial aid in the absence of the treatment, the other targeting those with high probability. The predicted baseline outcome is not the ideal criterion for targeting, nor is it a priori clear whether to prioritize low, high, or intermediate predicted probability. Nonetheless, targeting on low baseline outcomes is common in practice, for example because the relationship between individual characteristics and treatment effects is often difficult or impossible to estimate with historical data. We propose hybrid approaches that incorporate the strengths of both predictive approaches (accurate estimation) and causal approaches (correct criterion); we show that targeting intermediate baseline outcomes is most effective in our specific application, while targeting based on low baseline outcomes is detrimental. In one year of the experiment, nudging all students improved early filing by an average of 6.4 percentage points over a baseline average of 37% filing, and we estimate that targeting half of the students using our preferred policy attains around 75% of this benefit.
Collaborative Causal Inference with Fair Incentives
Collaborative causal inference (CCI) aims to improve the estimation of the causal effect of treatment variables by utilizing data aggregated from multiple self-interested parties. Since their source data are valuable proprietary assets that can be costly or tedious to obtain, every party has to be incentivized to be willing to contribute to the collaboration, such as with a guaranteed fair and sufficiently valuable reward (than performing causal inference on its own). This paper presents a reward scheme designed using the unique statistical properties that are required by causal inference to guarantee certain desirable incentive criteria (e.g., fairness, benefit) for the parties based on their contributions. To achieve this, we propose a data valuation function to value parties’ data for CCI based on the distributional closeness of its resulting treatment effect estimate to that utilizing the aggregated data from all parties. Then, we show how to value the parties’ rewards fairly based on a modified variant of the Shapley value arising from our proposed data valuation for CCI. Finally, the Shapley fair rewards to the parties are realized in the form of improved, stochastically perturbed treatment effect estimates. We empirically demonstrate the effectiveness of our reward scheme using simulated and real-world datasets.
mediation: R Package for Causal Mediation Analysis
In this paper, we describe the R package mediation for conducting causal mediation analysis in applied empirical research. In many scientific disciplines, the goal of researchers is not only estimating causal effects of a treatment but also understanding the process in which the treatment causally affects the outcome. Causal mediation analysis is frequently used to assess potential causal mechanisms. The mediation package implements a comprehensive suite of statistical tools for conducting such an analysis. The package is organized into two distinct approaches. Using the model-based approach, researchers can estimate causal mediation effects and conduct sensitivity analysis under the standard research design. Furthermore, the design-based approach provides several analysis tools that are applicable under different experimental designs. This approach requires weaker assumptions than the model-based approach. We also implement a statistical method for dealing with multiple (causally dependent) mediators, which are often encountered in practice. Finally, the package also offers a methodology for assessing causal mediation in the presence of treatment noncompliance, a common problem in randomized trials.
A Nonparametric Approach to Practical Identifiability of Nonlinear Mixed Effects Models
Mathematical modelling is a widely used approach to understand and interpret clinical trial data. This modelling typically involves fitting mechanistic mathematical models to data from individual trial participants. Despite the widespread adoption of this individual-based fitting, it is becoming increasingly common to take a hierarchical approach to parameter estimation, where modellers characterize the population parameter distributions, rather than considering each individual independently. This hierarchical parameter estimation is standard in pharmacometric modelling. However, many of the existing techniques for parameter identifiability do not immediately translate from the individual-based fitting to the hierarchical setting. In this work, we propose a nonparametric approach to study practical identifiability within a hierarchical parameter estimation framework. We focus on the commonly used nonlinear mixed effects framework and investigate two well-studied examples from the pharmacometrics and viral dynamics literature to illustrate the potential utility of our approach.
Randomized Controlled Trials without Data Retention
Amidst rising appreciation for privacy and data usage rights, researchers have increasingly acknowledged the principle of data minimization, which holds that the accessibility, collection, and retention of subjects' data should be kept to the bare amount needed to answer focused research questions. Applying this principle to randomized controlled trials (RCTs), this paper presents algorithms for making accurate inferences from RCTs under stringent data retention and anonymization policies. In particular, we show how to use recursive algorithms to construct running estimates of treatment effects in RCTs, which allow individualized records to be deleted or anonymized shortly after collection. Devoting special attention to non-i.i.d. data, we further show how to draw robust inferences from RCTs by combining recursive algorithms with bootstrap and federated strategies.
Identifying heterogeneity using recursive partitioning: evidence from SMS nudges encouraging voluntary retirement savings in Mexico
Abstract Individuals regularly struggle to save for retirement. Using a large-scale field experiment (N=97,149) in Mexico, we test the effectiveness of several behavioral interventions relative to existing policy and each other geared toward improving voluntary retirement savings contributions. We find that an intervention framing savings as a way to secure one’s family future significantly improves contribution rates. We leverage recursive partitioning techniques and identify that the overall positive treatment effect masks subpopulations where the treatment is even more effective and other groups where the treatment has a significant negative effect, decreasing contribution rates. Accounting for this variation is significant for theoretical and policy development as well as firm profitability. Our work also provides a methodological framework for how to better design, scale, and deploy behavioral interventions to maximize their effectiveness.
The central role of the propensity score in observational studies for causal effects
Abstract. The propensity score is the conditional probability of assignment to a particular treatment given a vector of observed covariates. Both large and
Hierarchical Modeling
Hierarchical modeling is a powerful technique for modeling heterogeneity and, consequently, it is becoming increasingly ubiquitous in contemporary applied statistics. Unfortunately that ubiquitous application has not brought with it an equivalently ubiquitous understanding for how awkward these models can be to fit in practice. In this case study we dive deep into hierarchical models, from their theoretical motivations to their inherent degeneracies and the strategies needed to ensure robust computation. We'll learn not only how to use hierarchical models but also how to use them robustly.
On the Accuracy of Influence Functions for Measuring Group Effects
Influence functions estimate the effect of removing a training point on a model without the need to retrain. They are based on a first-order Taylor approximation that is guaranteed to be accurate for sufficiently small changes to the model, and so are commonly used to study the effect of individual points in large datasets. However, we often want to study the effects of large groups of training points, e.g., to diagnose batch effects or apportion credit between different data sources. Removing such large groups can result in significant changes to the model. Are influence functions still accurate in this setting? In this paper, we find that across many different types of groups and for a range of real-world datasets, the predicted effect (using influence functions) of a group correlates surprisingly well with its actual effect, even if the absolute and relative errors are large. Our theoretical analysis shows that such strong correlation arises only under certain settings and need not hold in general, indicating that real-world datasets have particular properties that allow the influence approximation to be accurate.
Designing Information Provision Experiments
Information provision experiments allow researchers to test economic theories and answer policy-relevant questions by varying the information set available to respondents. We survey the emerging literature using information provision experiments in economics and discuss applications in macroeconomics, finance, political economy, public economics, labor economics, and health economics. We also discuss design considerations and provide best-practice recommendations on how to (i) measure beliefs; (ii) design the information intervention; (iii) measure belief updating; (iv) deal with potential confounds, such as experimenter demand effects; and (v) recruit respondents using online panels. We finally discuss typical effect sizes and provide sample size recommendations.
Picking on the Same Person: Does Algorithmic Monoculture lead to Outcome Homogenization?
As the scope of machine learning broadens, we observe a recurring theme of algorithmic monoculture: the same systems, or systems that share components (e.g. datasets, models), are deployed by multiple decision-makers. While sharing offers advantages like amortizing effort, it also has risks. We introduce and formalize one such risk, outcome homogenization: the extent to which particular individuals or groups experience the same outcomes across different deployments. If the same individuals or groups exclusively experience undesirable outcomes, this may institutionalize systemic exclusion and reinscribe social hierarchy. We relate algorithmic monoculture and outcome homogenization by proposing the component sharing hypothesis: if algorithmic systems are increasingly built on the same data or models, then they will increasingly homogenize outcomes. We test this hypothesis on algorithmic fairness benchmarks, demonstrating that increased data-sharing reliably exacerbates homogenization and individual-level effects generally exceed group-level effects. Further, given the current regime in AI of foundation models, i.e. pretrained models that can be adapted to myriad downstream tasks, we test whether model-sharing homogenizes outcomes across tasks. We observe mixed results: we find that for both vision and language settings, the specific methods for adapting a foundation model significantly influence the degree of outcome homogenization. We also identify societal challenges that inhibit the measurement, diagnosis, and rectification of outcome homogenization in deployed machine learning systems.
Effects of international sanctions on age-specific mortality: a cross-national panel data analysis
Background Previous research has shown a correlation between the imposition of sanctions and worsening health conditions in target countries. However, the direction of causality in this relationship remains unclear. No study has yet examined the effects of sanctions on age-specific mortality rates in cross-country panel data using methods designed to address causal identification in observational data. Methods In this cross-national panel data analysis, we analysed the effect on health of sanctions using a panel dataset of age-specific mortality rates and sanctions episodes for 152 countries between 1971 and 2021. We apply a range of methods designed to address causal questions using observational data, including entropy balancing, Granger causality, event-study representations, and instrumental variables. Findings Our findings showed a significant causal association between sanctions and increased mortality. We found the strongest effects for unilateral, economic, and US sanctions, whereas we found no statistical evidence of an effect for UN sanctions. Mortality effects ranged from 8·4 log points (95% CI 3·9–13·0) for children younger than 5 years to 2·4 log points (0·9–4·0) for individuals aged 60–80 years. We estimated that unilateral sanctions were associated with an annual toll of 564 258 deaths (95% CI 367 838–760 677), similar to the global mortality burden associated with armed conflict. Interpretation Sanctions have substantial adverse effects on public health, with a death toll similar to that of wars. Our findings underscore the need to rethink sanctions as a foreign-policy tool, highlighting the importance of exercising restraint in their use and seriously considering efforts to reform their design. Funding The Center for Economic and Policy Research.
The C-Word: Scientific Euphemisms Do Not Improve Causal Inference From Observational Data
Causal inference is a core task of science. However, authors and editors often refrain from explicitly acknowledging the causal goal of research projects; they refer to causal effect estimates as associational estimates. This commentary argues that using the term “causal” is necessary to improve the quality of observational research. Specifically, being explicit about the causal objective of a study reduces ambiguity in the scientific question, errors in the data analysis, and excesses in the interpretation of the results.
