Target-Weighted Neyman Allocation: Experimental Design for Heterogeneous Treatment Effects under Population Shift

Randomized experiments are often run in one population to guide decisions in another. Allocating by experimental proportions wastes budget on groups that rarely appear in deployment, whereas allocating by deployment proportions under-samples groups that are hard to measure precisely. We propose \textbf{TWNA} (Target-Weighted Neyman Allocation), a two-stage stratified design that uses pilot estimates of group--arm outcome variances to allocate final-stage sample sizes and treatment probabilities for target-weighted group average treatment effect (GATE) precision. The oracle rule has a closed form and balances deployment importance with statistical difficulty; the plug-in rule recovers it as pilot variance estimates stabilize. We also extend TWNA to handle uncertainty about deployment composition, remaining robust whether the target mix is roughly known or entirely unknown. Finally, we distinguish this weight robustness from a pilot-robust variant for skewed, rare-event, or contaminated outcomes. Simulations and real-covariate benchmarks show the largest gains when groups are both deployment-important and difficult to measure.

Bayesian fusion forests for heterogeneous treatment effects on survival from randomised and real-world data

We develop the Bayesian fusion forest, a nonparametric framework to estimate heterogeneous treatment effects on survival outcomes by combining a randomised controlled trial and real-world data. The framework relaxes the unconfoundedness assumption on the real-world data by assuming instead that the treatment effect transports across the two sources. Our method opens up right- and interval-censored outcomes to data fusion. We model the survival time with an accelerated failure time decomposition into a shared baseline prognosis, a source-specific deviation, a treatment effect, and a confounding function. The confounding function absorbs the confounding bias in the real-world data. Each component receives a Bayesian tree ensemble prior. The shared baseline prognosis borrows strength across sources, while the deviation captures between-source heterogeneity. A hierarchical Dirichlet process mixture models the error distribution nonparametrically. A simulation study shows efficiency gains over a trial-only analysis across varying levels of confounding and between-source heterogeneity. We combine the ACTG 175 trial with the Multicenter AIDS Cohort Study to estimate the effect of combination antiretroviral therapy for HIV. The fusion identifies a benefit for nearly every patient whereas the trial alone is inconclusive.

Attenuated Heterogeneity in Fixed-Effects Causal Forests, and a Cross-Fitted Correction

Causal forests that estimate conditional average treatment effects by averaging honest leaf-level effects across trees are widely used in fixed-effects panel settings. We show that this averaging systematically attenuates the estimated heterogeneity: the raw prediction behaves like a + b*tau(x) with slope b < 1, so the spread of the CATEs is compressed toward the average effect, and the additive recentering used to report an unbiased average treatment effect does not fix it. Benchmarking against a similarity-weight generalized random forest on the same within-transformed signal, we find both estimators attenuate but the leaf-averaging construction attenuates materially more. We characterize how b moves with the design, worsening with lower signal-to-noise, smaller panels, and higher dimension; this diagnosis is our main contribution. As a remedy we adapt the best-linear-predictor calibration of Chernozhukov et al., estimating the de-attenuation slope out-of-bag so that it is self-contained within the observational panel and asymptotically inert under a homogeneous effect. In simulations the correction cuts CATE mean-squared error by 25-42% relative to the recentering default; on a standard county minimum-wage panel the attenuation is present but mild and the correction restores the imposed spread. We ship the method in the causalfe Python package.

A Fixed-Effects Causal Forest for Staggered Adoption, with an Application to Medicaid Expansion

Difference-in-differences with staggered adoption identifies group-time average treatment effects ATT(g,t) by comparing each cohort to units not yet treated, which avoids the "forbidden comparisons" that bias two-way fixed-effects estimators when effects are heterogeneous. This paper studies the covariate-conditional version of that object, tau_{g,t}(x), and estimates it with a fixed-effects causal forest. Within each (g,t) comparison block, the outcome and treatment are residualized on unit and period fixed effects inside each tree node, and honest causal trees split on treatment-effect heterogeneity in the covariates. The estimand is not new: Hatamyar, Kreif, Rocha and Huber (2023) introduced it using a doubly-robust R-learner, and Imai, Qin and Yanagi (2023) study it for a single continuous covariate. What we add is a different way to estimate it. Where those methods remove confounding by modeling nuisance functions, we remove it by differencing out unit and period effects within each tree node, following the fixed-effects residualization of Kattenberg, Scheer and Thiel (2023) and Gavrilova, Langorgen and Zoutman (2025) and carrying it into the Callaway-Sant'Anna group-time structure. In Monte Carlo experiments the estimator is the only forest-based method that stays unbiased and correctly covered for the overall effect under staggered timing with cohort-varying effects; two-way fixed effects and a pooled causal forest inherit large forbidden-comparison bias. We apply the method to the Callaway-Sant'Anna minimum-wage panel as a validation and to the staggered county-level rollout of the ACA Medicaid expansion, where it recovers an average 2.25 percentage-point fall in the uninsured rate and a conditional surface on which poorer and lower-income counties gained substantially more coverage -- heterogeneity measured along socioeconomic covariates that are not lags of the outcome.

Optimizing Clinical Trial Protocols Using EHR-Derived Heterogeneous Treatment Effects

Traditional randomized trials often obscure clinically meaningful heterogeneity in treatment response by focusing on average effects. Leveraging real-world data to emulate clinical trials and estimate heterogeneous treatment effects (HTEs) offers a promising path toward more precise and efficient trial design. In this study, we emulate the DAPA-HF trial using electronic health records from the Mayo Clinic Cloud (MCC) to investigate whether HTE-guided stratification can identify patient subgroups with distinct treatment responses to dapagliflozin versus placebo in patients with heart failure with reduced ejection fraction. All-cause mortality was evaluated using Cox proportional hazards models, with HTEs estimated using a Meta-S learner and subgroups defined using a decision tree-based thresholding approach. In the overall cohort of the emulation, no significant treatment difference was observed (HR, 1.681; 95% CI, 0.828-3.413; p = 0.1507). However, compared with the overall emulated cohort, in which dapagliflozin showed no statistically significant survival benefit, HTE-driven stratification identified subgroups with significant and directionally distinct treatment effects. The beneficial (low-HTE) subgroup showed a significant survival benefit from dapagliflozin (HR = 0.203, 95% CI, 0.087-0.476, p = 0.0002), whereas the harmful (high-HTE) subgroup showed a significant harmful association with markedly increased mortality risk (HR = 6.680, 95% CI, 2.759-16.171, p < 0.0001). These findings indicate that HTE-guided stratification can uncover clinically meaningful beneficial and harmful treatment-effect patterns that are masked in the full-cohort emulation.

Significance-First Splitting: Aligning Treatment Heterogeneity Detection with Honest Estimation

Estimating heterogeneous treatment effects (CATE) requires simultaneously detecting effect modification and quantifying estimation uncertainty. Existing tree-based methods make an uneasy trade-off: significance-based approaches (Radcliffe and Surry 2011) identify subgroup interactions directly but lack valid inference; honest causal trees (Athey and Imbens 2016) deliver nominal confidence interval coverage but use outcome-agnostic splitting criteria that sacrifice interaction sensitivity. We introduce a hybrid algorithm that fuses significance-based splitting with honest sample-splitting and cross-validation. Our splitting criterion uses the squared $t$-statistic for the treatment $\times$ side interaction ($t^2$), which is shown to be directly aligned with the honest $\text{EMSE}_τ$ criterion when the interaction is strong. Post-hoc honest cross-validation selects the cost-complexity penalty, giving a single principled estimator with nominal CI coverage at the leaf level. For forests, we retain bootstrap count vectors to enable an infinitesimal jackknife (IJ) variance estimate of Monte-Carlo convergence rather than formal pointwise inference. On the three synthetic designs from (Athey and Imbens 2016) the single tree achieves approximately 90% leaf-average CI coverage at the 90% nominal level across all three designs (200 replications each); on the Criteo, Hillstrom and Starbucks uplift datasets we match Qini coefficient performance of S-, T-learner and GRF baselines. An open-source Python package with reproducible seeds, sklearn-compatible API, and full test coverage accompanies this work (https://codeberg.org/hadjipantelis/rattus).

Doubly cross-fit debiased machine learning of heterogeneous treatment effects under principal stratification

Principal stratification provides a foundational framework for causal inference with intermediate outcomes by defining causal effects within subpopulations, yet existing work has largely focused on average effects across strata rather than treatment effect heterogeneity within strata. Such within-stratum heterogeneity informs individualized treatment decisions but the associated methods are sparse. We address this gap by studying the identification and estimation of the conditional principal causal effects under principal ignorability combined with an odds ratio sensitivity parameterization, which relaxes the monotonicity assumption. To efficiently learn these estimands, we propose a novel doubly cross-fit doubly robust machine learner that resolves the nested nuisance structure inherent to principal stratification. Leveraging sequential orthogonal debiased machine learning with regularized least-squares sieves, we derive $\mathcal{L}^2$ and uniform limit theory, establish oracle efficiency, and construct uniform confidence bands for the proposed estimator. We use simulations to demonstrate the finite-sample performance of our estimator, and provide an empirical analysis of a randomized trial in acute lung injury, revealing informative patterns of treatment effect heterogeneity within the always-survivor subpopulation.

From Tokens to Policy: Causal and Interpretable Heterogeneous Treatment Effects Identification

Heterogeneous Treatment Effect (HTE) identification is crucial to explain the impact of an intervention and optimize our policies accordingly. Existing approaches trade expressivity for interpretability, but, if some active heterogeneity drivers are unmeasured, methods at both ends of this spectrum allow for spurious HTE characterization with no causal reading. In this work, we focus on controlled experiments and argue that an oracle HTE causal characterization via the latent interactors is now within reach, thanks to (i) more extensive pre-treatment measurements, i.e., multi-modal and multi-view, and (ii) scalable representations with minimal human supervision. We then re-frame HTE identification as a Markov-blanket discovery problem on a sufficient and aligned pre-treatment representation, and introduce Neural EXposure Interaction Search (NEXIS), an iterative procedure with provable and empirically validated consistent selection. We deploy NEXIS on two anti-poverty programs in Africa, augmenting each with satellite imagery capturing previously unmeasured environmental effect modifiers, leading to novel, interpretable and prescriptive guidelines to optimize the programs' next iterations.