Machine learning (ML) models are increasingly used as decision-support tools in high-risk domains. Evaluating the causal impact of deploying such models can be done with a randomized controlled trial (RCT) that randomizes users to ML vs. control groups and assesses the effect on relevant outcomes. However, ML models are inevitably updated over time, and we often lack evidence for the causal impact of these updates. While the causal effect could be repeatedly validated with ongoing RCTs, such experiments are expensive and time-consuming to run. In this work, we present an alternative solution: using only data from a prior RCT, we give conditions under which the causal impact of a new ML model can be precisely bounded or estimated, even if it was not included in the RCT. Our assumptions incorporate two realistic constraints: ML predictions are often deterministic, and their impacts depend on user trust in the model. Based on our analysis, we give recommendations for trial designs that maximize our ability to assess future versions of an ML model. Our hope is that our trial design recommendations will save practitioners time and resources while allowing for quicker deployments of updates to ML models.

A fundamental problem in decision-making systems is the presence of inequity along demographic lines. However, inequity can be difficult to quantify, particularly if our notion of equity relies on hard-to-measure notions like risk (e.g., equal access to treatment for those who would die without it). Auditing such inequity requires accurate measurements of individual risk, which is difficult to estimate in the realistic setting of unobserved confounding. In the case that these unobservables “explain” an apparent disparity, we may understate or overstate inequity. In this paper, we show that one can still give informative bounds on allocation rates among high-risk individuals, even while relaxing or (surprisingly) even when eliminating the assumption that all relevant risk factors are observed. We utilize the fact that in many real-world settings (e.g., the introduction of a novel treatment) we have data from a period prior to any allocation, to derive unbiased estimates of risk. We apply our framework to a real-world setting of Paxlovid allocation to COVID-19 patients, finding that observed racial inequity cannot be explained by unobserved confounders of the same strength as important observed covariates.

Drawing causal inferences from observational studies (OS) requires unverifiable validity assumptions; however, one can falsify those assumptions by benchmarking the OS with experimental data from a randomized controlled trial (RCT). A major limitation of existing procedures is not accounting for censoring, despite the abundance of RCTs and OSes that report right-censored time-to-event outcomes. We consider two cases where censoring time (1) is independent of time-to-event and (2) depends on time-to-event the same way in OS and RCT. For the former, we adopt a censoring-doubly-robust signal for the conditional average treatment effect (CATE) to facilitate an equivalence test of CATEs in OS and RCT, which serves as a proxy for testing if the validity assumptions hold. For the latter, we show that the same test can still be used even though unbiased CATE estimation may not be possible. We verify the effectiveness of our censoring-aware tests via semi-synthetic experiments and analyze RCT and OS data from the Women’s Health Initiative study.

Several recent works seek to develop foundation models specifically for medical applications, adapting general-purpose large language models (LLMs) and vision-language models (VLMs) via continued pretraining on publicly available biomedical corpora.These works typically claim that such domain-adaptive pretraining (DAPT) improves performance on downstream medical tasks, such as answering medical licensing exam questions.In this paper, we compare seven public "medical" LLMs and two VLMs against their corresponding base models, arriving at a different conclusion: all medical VLMs and nearly all medical LLMs fail to consistently improve over their base models in the zero-/few-shot prompting regime for medical question-answering (QA) tasks.For instance, across the tasks and model pairs we consider in the 3-shot setting, medical LLMs only outperform their base models in 12.1% of cases, reach a (statistical) tie in 49.8% of cases, and are significantly worse than their base models in the remaining 38.2% of cases.Our conclusions are based on (i) comparing each medical model head-to-head, directly against the corresponding base model; (ii) optimizing the prompts for each model separately; and (iii) accounting for statistical uncertainty in comparisons.While these basic practices are not consistently adopted in the literature, our ablations show that they substantially impact conclusions.Our findings suggest that state-of-the-art generaldomain models may already exhibit strong medical knowledge and reasoning capabilities, and offer recommendations to strengthen the conclusions of future studies.Table 1: Summary of open-source autoregressive VLM and LLM pairs used for evaluation.

Randomized Controlled Trials (RCT)s are relied upon to assess new treatments, but suffer from limited power to guide personalized treatment decisions. On the other hand, observational (i.e., non-experimental) studies have large and diverse populations, but are prone to various biases (e.g. residual confounding). To safely leverage the strengths of observational studies, we focus on the problem of falsification, whereby RCTs are used to validate causal effect estimates learned from observational data. In particular, we show that, given data from both an RCT and an observational study, assumptions on internal and external validity have an observable, testable implication in the form of a set of Conditional Moment Restrictions (CMRs). Further, we show that expressing these CMRs with respect to the causal effect, or “causal contrast”, as opposed to individual counterfactual means, provides a more reliable falsification test. In addition to giving guarantees on the asymptotic properties of our test, we demonstrate superior power and type I error of our approach on semi-synthetic and real world datasets. Our approach is interpretable, allowing a practitioner to visualize which subgroups in the population lead to falsification of an observational study.

Randomized Controlled Trials (RCTs) represent a gold standard when developing policy guidelines. However, RCTs are often narrow, and lack data on broader populations of interest. Causal effects in these populations are often estimated using observational datasets, which may suffer from unobserved confounding and selection bias. Given a set of observational estimates (e.g., from multiple studies), we propose a meta-algorithm that attempts to reject observational estimates that are biased. We do so using validation effects, causal effects that can be inferred from both RCT and observational data. After rejecting estimators that do not pass this test, we generate conservative confidence intervals on the extrapolated causal effects for subgroups not observed in the RCT. Under the assumption that at least one observational estimator is asymptotically normal and consistent for both the validation and extrapolated effects, we provide guarantees on the coverage probability of the intervals output by our algorithm. To facilitate hypothesis testing in settings where causal effect transportation across datasets is necessary, we give conditions under which a doubly-robust estimator of group average treatment effects is asymptotically normal, even when flexible machine learning methods are used for estimation of nuisance parameters. We illustrate the properties of our approach on semi-synthetic experiments based on the IHDP dataset, and show that it compares favorably to standard meta-analysis techniques.

We give a method for proactively identifying small, plausible shifts in distribution which lead to large differences in model performance. These shifts are defined via parametric changes in the causal mechanisms of observed variables, where constraints on parameters yield a "robustness set" of plausible distributions and a corresponding worst-case loss over the set. While the loss under an individual parametric shift can be estimated via reweighting techniques such as importance sampling, the resulting worst-case optimization problem is non-convex, and the estimate may suffer from large variance. For small shifts, however, we can construct a local second-order approximation to the loss under shift and cast the problem of finding a worst-case shift as a particular non-convex quadratic optimization problem, for which efficient algorithms are available. We demonstrate that this second-order approximation can be estimated directly for shifts in conditional exponential family models, and we bound the approximation error. We apply our approach to a computer vision task (classifying gender from images), revealing sensitivity to shifts in non-causal attributes.

We propose a method for learning linear models whose predictive performance is robust to causal interventions on unobserved variables, when noisy proxies of those variables are available. Our approach takes the form of a regularization term that trades off between in-distribution performance and robustness to interventions. Under the assumption of a linear structural causal model, we show that a single proxy can be used to create estimators that are prediction optimal under interventions of bounded strength. This strength depends on the magnitude of the measurement noise in the proxy, which is, in general, not identifiable. In the case of two proxy variables, we propose a modified estimator that is prediction optimal under interventions up to a known strength. We further show how to extend these estimators to scenarios where additional information about the "test time" intervention is available during training. We evaluate our theoretical findings in synthetic experiments and using real data of hourly pollution levels across several cities in China.

Individuals often make different decisions when faced with the same context, due to personal preferences and background. For instance, judges may vary in their leniency towards certain drug-related offenses, and doctors may vary in their preference for how to start treatment for certain types of patients. With these examples in mind, we present an algorithm for identifying types of contexts (e.g., types of cases or patients) with high inter-decision-maker disagreement. We formalize this as a causal inference problem, seeking a region where the assignment of decision-maker has a large causal effect on the decision. Our algorithm finds such a region by maximizing an empirical objective, and we give a generalization bound for its performance. In a semi-synthetic experiment, we show that our algorithm recovers the correct region of heterogeneity accurately compared to baselines. Finally, we apply our algorithm to real-world healthcare datasets, recovering variation that aligns with existing clinical knowledge.

Overlap between treatment groups is required for non-parametric estimation of causal effects. If a subgroup of subjects always receives the same intervention, we cannot estimate the effect of intervention changes on that subgroup without further assumptions. When overlap does not hold globally, characterizing local regions of overlap can inform the relevance of causal conclusions for new subjects, and can help guide additional data collection. To have impact, these descriptions must be interpretable for downstream users who are not machine learning experts, such as policy makers. We formalize overlap estimation as a problem of finding minimum volume sets subject to coverage constraints and reduce this problem to binary classification with Boolean rule classifiers. We then generalize this method to estimate overlap in off-policy policy evaluation. In several real-world applications, we demonstrate that these rules have comparable accuracy to black-box estimators and provide intuitive and informative explanations that can inform policy making.

In several medical decision-making problems, such as antibiotic prescription, laboratory testing can provide precise indications for how a patient will respond to different treatment options. This enables us to "fully observe" all potential treatment outcomes, but while present in historical data, these results are infeasible to produce in real-time at the point of the initial treatment decision. Moreover, treatment policies in these settings often need to trade off between multiple competing objectives, such as effectiveness of treatment and harmful side effects. We present, compare, and evaluate three approaches for learning individualized treatment policies in this setting: First, we consider two indirect approaches, which use predictive models of treatment response to construct policies optimal for different trade-offs between objectives. Second, we consider a direct approach that constructs such a set of policies without intermediate models of outcomes. Using a medical dataset of Urinary Tract Infection (UTI) patients, we show that all approaches learn policies that achieve strictly better performance on all outcomes than clinicians, while also trading off between different objectives. We demonstrate additional benefits of the direct approach, including flexibly incorporating other goals such as deferral to physicians on simple cases.

We introduce an off-policy evaluation procedure for highlighting episodes where applying a reinforcement learned (RL) policy is likely to have produced a substantially different outcome than the observed policy. In particular, we introduce a class of structural causal models (SCMs) for generating counterfactual trajectories in finite partially observable Markov Decision Processes (POMDPs). We see this as a useful procedure for off-policy “debugging” in high-risk settings (e.g., healthcare); by decomposing the expected difference in reward between the RL and observed policy into specific episodes, we can identify episodes where the counterfactual difference in reward is most dramatic. This in turn can be used to facilitate review of specific episodes by domain experts. We demonstrate the utility of this procedure with a synthetic environment of sepsis management.