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Integrating Heterogeneous Information in Randomized Experiments: A Unified Calibration Framework
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{\bf Calibration for Treatment Effect Estimation in Randomized Controlled Trials: a Unified Approach for Covariates Adjustment and Information Borrowing}
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bibunit\begin{abstract}
In modern randomized experiments, large-scale data collection increasingly yields rich baseline covariates and auxiliary information from multiple sources. Such information offers opportunities for more precise treatment effect estimation, but it also raises the challenge of integrating heterogeneous information coherently without compromising validity. Covariate-adaptive randomization (CAR) is widely used to improve covariate balance at the design stage, but it typically balances only a small set of covariates used to form strata, making covariate adjustment at the analysis stage essential for more efficient estimation of treatment effects. Beyond standard covariate adjustment, it is often desirable to incorporate auxiliary information, including cross-stratum information, predictions from various machine learning models, and external data from historical trials or real-world sources. While this auxiliary information is widely available, existing covariate adjustment methods under CAR primarily exploit within-stratum covariates and do not provide a coherent mechanism for integrating it. We propose a unified calibration framework that integrates such information through an information proxy vector and calibration weights defined by a convex optimization problem. The resulting estimator recovers many recent covariate adjustment procedures as special cases while providing a systematic mechanism for both internal and external information borrowing within a single framework. We establish large-sample validity and a no-harm efficiency guarantee, showing that incorporating additional information sources cannot increase asymptotic variance, and we extend the theory to settings in which both the number of strata and the number of information sources grow with the sample size. Simulation studies and an empirical analysis of a field experiment on savings behavior in Uganda and Malawi demonstrate the strong finite-sample performance and practical utility of our method.
\end{abstract}
{\it Keywords:} Calibration weights; Covariate-adaptive randomization; Covariate adjustment; Information integration; Machine learning
\doublespacing
\section{Introduction}
In modern randomized experiments, ensuring balance in baseline covariates
across treatment groups is critical for reducing bias and improving
the credibility of the trial results. Covariate-adaptive randomization
(CAR) methods, such as stratified biased coin randomization (efron1971Forcing),
stratified block randomization (zelen1974Randomization),
and minimization (pocock1975Sequential,taves1974Minimization),
are widely used to achieve this balance during the design stage. In practice, however, CAR is typically implemented using only a small set of covariates to form strata, so balance is not directly enforced for many other baseline covariates. Moreover, some important pre-treatment covariates may only be observed after randomization, for instance when they are collected together with the outcome variable (bai2024Covariate).
Consequently, covariate adjustment at the statistical analysis stage
plays a crucial complementary role. By incorporating baseline covariates
into the statistical analysis, covariate adjustment methods correct
for residual imbalances, thereby improving the precision and efficiency
of treatment effect estimates and strengthening the validity of the
trial.
The adjustment of baseline covariates in the statistical analysis
stage has been studied for a long time (see, e.g., tsiatis2008Covariate,zhang2008Improving,lin2013Agnostic
and the references therein). Under the CAR design, ma2022Regression,ye2022Inference,gu2023RegressionBased
adjusted for additional covariates using linear regression, while
liu2023Lassoadjusted applied Lasso (tibshirani1996Regression).
These approaches resulted in average treatment effect (ATE) estimators
that are more efficient than the naive difference-in-means estimator
and the ordinary least squares estimator (regressing outcomes on strata
indicators) proposed by bugni2018Inference and bugni2019Inference.
This holds true even in the absence of strong evidence for a linear
relationship between covariates and outcomes. To further enhance efficiency,
recent work by rafi2023Efficient, tu2024Unified
and bannick2025General introduced unified frameworks for
nonlinear covariate adjustment. Relying on the augmented inverse probability
weighting (AIPW, robins1994Estimation), these frameworks
incorporate machine learning techniques, such as random forests and
deep neural networks, to adjust for covariates. These machine learning
methods can be effective in capturing complex nonlinear relationships
between covariates and outcomes.
However, much of the existing covariate adjustment literature can
be interpreted as focusing on a particular form of internal information
borrowing, namely using baseline covariates from the current trial,
typically within each stratum, to improve efficiency. This focus leaves
relatively little room for other practically important forms of information
borrowing, and it limits the extent to which standard adjustment methods
can integrate heterogeneous information in a systematic way. Internally,
efficiency can often be improved by borrowing information across strata
when the outcome--covariate relationship is stable, and by combining
predictions from multiple machine learning methods when no single
learner is uniformly reliable. Externally, there is increasing interest
in leveraging historical trials and real-world data to support analyses
of concurrent trials, especially when sample sizes are constrained
by cost, ethical considerations, or recruitment challenges FDA2019RareDiseases,gu2024incorporatingexternaldataanalyzing.
Existing AIPW-based nonlinear adjustment frameworks (tu2024Unified,rafi2023Efficient,bannick2025General),
however, are not designed to integrate these heterogeneous information
sources, as they typically rely on a single nuisance estimate and
lack a systematic mechanism for combining multiple internal predictors
or external information sources. To fill this gap, we propose a unified
calibration framework for integrating heterogeneous information that
accommodates both internal and external borrowing, thereby enabling
estimators that jointly exploit covariates, cross-stratum information,
and auxiliary data sources.
A central methodological feature of our approach is the use of calibration
weights under CAR designs. Although calibration has been studied in
survey sampling (deville1992Calibration,kwon2025Debiased),
missing data problems (qin2007EmpiricalLikelihoodBased,tan2014Secondorder),
and observational studies (chan2016Globally), its analysis under CAR designs raises distinct theoretical issues. Unlike the aforementioned
settings where samples are typically independent and identically distributed
(i.i.d.), CAR designs induce complex dependence structures among treatment
assignments within strata. We address this dependence through a conditional
asymptotic argument. Specifically, we condition on the realized stratum
indicators and treatment assignments, treat them as fixed, and establish
large-sample results using conditional laws of large numbers and conditional
central limit theorems. This approach provides a tractable framework
for inference under CAR and yields proof techniques that extend to
regimes with a growing number of strata and an increasing number of
information sources, which may be of independent interest. We summarize
the main contributions as follows.
\begin{itemize}
• A unified calibration framework. We introduce a calibration-based
framework for estimation and inference under CAR. This framework is
unified in three aspects. First, it provides a common formulation
that recovers many recent covariate adjustment procedures
as special cases (e.g., bugni2019Inference,cohen2024Noharm,ma2022Regression,tu2024Unified,bannick2025General,ye2022Inference,ye2023Better,liu2023Lassoadjusted).
Second, it places internal and external information borrowing within
a single architecture, yielding a systematic approach to integrating
heterogeneous information sources. Third, it applies broadly across
CAR schemes satisfying Assumption (ref),
so that the resulting inference procedure is not tied to a particular
randomization method.
• Flexible and robust information borrowing. We develop practical
constructions of the information proxy vector that accommodate a wide
range of internal and external information sources. Internally, the
framework can borrow across strata and aggregate heterogeneous machine
learning predictions. Externally, it can incorporate information from
historical trials and real-world data. Importantly, our framework
is model-agnostic regarding information sources. We refer to this
property as robustness, meaning that the validity of our
statistical inference holds even if the utilized information is biased
or generated by inaccurate models.
• General inference theory under CAR. We provide a rigorous
theoretical foundation, proving that our estimator is asymptotically
normal with a consistently estimable variance. We establish a no-harm
efficiency guarantee, ensuring that utilizing additional information
sources improves, or at worst maintains, estimation efficiency. Furthermore,
we develop proof techniques tailored to CAR-induced dependence, distinct
from existing arguments (e.g., bugni2018Inference,bugni2019Inference,ma2022Regression,liu2023Lassoadjusted).
These techniques accommodate a growing number of strata and an increasing
number of information sources, offering tools that may be of independent
interest in related problems.
\end{itemize}
The paper is organized as follows. Figure (ref) provides
an overview. Section (ref) introduces the
setting and our unified calibration framework. Section (ref)
discusses practical strategies for constructing the information proxy
vector $\bs{\xi}_{n}$, including approaches that borrow auxiliary
information from both internal and external sources. Section (ref)
establishes the large-sample properties of the proposed estimator,
including asymptotic normality, consistent variance estimation, and
efficiency comparisons. Section (ref) presents theoretical
extensions, including settings with diverging numbers of strata and
a growing dimension of $\bs{\xi}_{n}$, as well as general discrepancy
measures. Finally, Sections (ref) and (ref)
assess finite-sample performance via simulation and illustrate the
method using experimental data from dupas2018Bankinga. All
proofs are provided in the Appendix (Supplementary Material, ma2026Integrating).
\begin{figure}[!tbh]
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\node (sec2) [topnode] {Sec 2: A Unified Calibration Framework};
\node (sec4) [midnode, below=2.5cm of sec2] {Sec 4: Asymptotics\\ Establish normality & efficiency comparison};
\node (sec3) [midnode, left=of sec4] {\textbf{Sec 3: Construction of $\bs{\xi}_{n}$}\\ Leverage internal & external information};
\node (sec5) [midnode, right=of sec4] {\textbf{Sec 5: Extensions}\\ Two extensions of the asymptotic results};
\node (sub3a) [subnode, below=1cm of sec3, xshift=-2cm] {\textbf{Internal:} \\ cross-stratum information & various machine learning & cross-fitting};
\node (sub3b) [subnode, below=1cm of sec3, xshift=2cm] {\textbf{External:} \\ historical trials & real-world data};
\node (sub4a) [subnode, below=1cm of sec4, xshift=-2cm] {asymptotic normality & valid inference};
\node (sub4b) [subnode, below=1cm of sec4, xshift=2cm] {guaranteed \\ efficiency gain};
\node (sub5a) [subnode, below=1cm of sec5, xshift=-2cm] {diverging number of strata and information sources};
\node (sub5b) [subnode, below=1cm of sec5, xshift=2cm] {general discrepancy measure $D(v)$ & second-order bias};
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\caption{Roadmap of the proposed unified calibration framework and theoretical
analysis. }
\end{figure}
\textit{Notation.} We maintain the following notation conventions
throughout the paper. For any column vector $\bs x=\left(x_{1},x_{2},\ldots,x_{d}\right)^{\top}\in\mathbb{R}^{d}$,
where $\mathbb{R}^{d}$ is the $d$-dimensional Euclidean space, $\left\Vert \bs x\right\Vert =\left(\bs x^{\top}\bs x\right)^{1/2}$
denotes its Euclidean norm. For any matrix $A=\left(a_{ij}\right)_{n\times m}$,
$\left\Vert A\right\Vert $ denotes its maximum singular value, i.e.,
the operator norm, $\left\Vert A\right\Vert _{F}=\sqrt{\mathrm{tr}\left(AA^{\top}\right)}$
denotes it Frobenius norm, and $A^{+}$ denotes its Moore-Penrose
inverse. For two positive non-random sequence $a_{n},b_{n}$ and random
vector sequence $X_{n}$, $X_{n}=o_{P}(a_{n})$ means $P\left(\left\Vert X_{n}\right\Vert >a_{n}\epsilon\right)\to0$
as $n\to\infty$ for any $\epsilon>0$ and $X_{n}=O_{P}(a_{n})$ means
for any $\epsilon>0$, there exists a constant $M>0$ such that $\limsup_{n\to\infty}P\left(\left\Vert X_{n}\right\Vert \geq a_{n}M\right)<\epsilon$.
The notation $\1(\cdot)$ denotes the indicator function, which takes
the value 1 if the condition inside the parentheses is true and 0
otherwise.
\section{A unified calibration framework}
\subsection{Preliminaries}
Let $A_{i}\in\{0,1\}$ ($i=1,\ldots,n$) denote the treatment assignment
indicator, where $A_{i}=1$ indicates that the $i$-th unit is assigned
to the treatment group and $A_{i}=0$ otherwise. We assume the assignments
$\{A_{i}\}_{i=1}^{n}$ are generated via a CAR design satisfying Assumption (ref).
Consequently, the variables $A_{i}$ are typically not i.i.d. Adopting
the potential outcomes framework (imbens2015Causal), we
define $Y_{i}(1)$ and $Y_{i}(0)$ as the potential outcomes under
treatment and control, respectively. The observed outcome $Y_{i}$
is determined by $Y_{i}=A_{i}Y_{i}(1)+(1-A_{i})Y_{i}(0)$.
The experimental design stratifies units into $K$ strata, with $B_{i}\in\{1,\ldots,K\}$
indicating the stratum of unit $i$. For notational convenience, let
$[k]=\{i:B_{i}=k\}$ denote the set of indices for units belonging
to stratum $k$. To ensure non-empty strata, we assume positive assignment
probabilities: $p_{[k]}=P(B_{i}=k)>0$ for all $k\in\{1,\ldots,K\}$
and $i\in\{1,\ldots,n\}$. The target treatment allocation proportion
stratum $k$ is $\pi_{[k]}=P(A_{i}=1\mid B_{i}=k)\in(0,1)$. Each
unit has a $p$-dimensional baseline covariate vector $\bs X_{i}=(X_{i1},\ldots,X_{ip})^{\top}\in\mathcal{X}\subset\mathbb{R}^{p}$,
which may be either low- or high-dimensional. We assume that the covariate
vector $\bs X_{i}$ contains the stratum indicator $B_{i}$, but to
highlight the stratum indicator $B_{i}$, we sometimes use the notation
$(\bs X_{i},B_{i})$.
Let subscripts 1 and 0 denote treatment and control groups, respectively.
The treatment group contains $n_{1}=\sum_{i=1}^{n}A_{i}$ units and
the control group contains $n_{0}=\sum_{i=1}^{n}(1-A_{i})$ units.
For stratum-specific quantities, we use subscript $[k]$: let $n_{[k]}=\sum_{i\in[k]}1$
denote the stratum size, with $n_{1[k]}=\sum_{i\in[k]}A_{i}$ and
$n_{0[k]}=\sum_{i\in[k]}(1-A_{i})$ representing treated and control
units in stratum $k$, respectively. The stratum proportion and treatment
allocation proportion are defined as $p_{n[k]}=n_{[k]}/n$ and $\pi_{n[k]}=n_{1[k]}/n_{[k]}$,
correspondingly.
Our parameter of interest is the population average treatment effect
(ATE):
\[
\tau=\mathbb{E}[Y_{i}(1)-Y_{i}(0)].
\]
Under CAR, the ATE parameter $\tau$ can be consistently estimated
by aggregating the treatment effect estimates from each stratum. A
simple estimator for this is the stratified difference-in-means estimator
(bugni2019Inference,ma2022Regression):
\[
\widehat{\tau}_{\mathrm{sdim}}:=\sum_{k=1}^{K}p_{n[k]}\left(\overline{Y}_{1[k]}-\overline{Y}_{0[k]}\right),
\]
where $\overline{Y}_{a[k]}:=\frac{1}{n_{a[k]}}\sum_{i\in[k]}\1(A_{i}=a)Y_{i}$
for $a\in\{0,1\}$ and $k=1,\ldots,K$. To improve estimation efficiency,
recent literature has proposed nonlinear covariate adjustment methods
(tu2024Unified,rafi2023Efficient,bannick2025General). These
methods typically use the augmented inverse probability weighting
(AIPW, robins1994Estimation) estimator and rely on estimating
the conditional mean function $h_{a[k]}^{*}(\bs X_{i})=\mathbb{E}\left[Y_{i}(a)\mid\bs X_{i},B_{i}=k\right]$.
However, AIPW-based adjustment methods are limited in that they cannot
combine different estimates of $h_{a[k]}^{*}(\bs X_{i})$. For instance,
these estimates may leverage internal information via cross-stratum
borrowing and heterogeneous machine learning predictions, or incorporate
external information from historical trials, real-world data, and
functional forms suggested by domain experts (see Section (ref)
for a detailed discussion). In the following, we will introduce a
unified adjustment framework that can combine these diverse estimates
of $h_{a[k]}^{*}(\bs X_{i})$, leading to more efficient and robust
ATE estimators.
\subsection{Construction of the calibration estimator}
Let $D(v):\mathbb{R}\to\mathbb{R}$ be a twice continuously differentiable
and strictly convex function that measures the discrepancy from $v$
to 1, e.g., $D(v)=(v-1)^{2}/2$ and $D(v)=v-\log v$. Suppose $\bs{\xi}_{n}:\mathcal{X}\to\mathbb{R}^{d}$
($n\geq1$) is a sequence of $\mathbb{R}^{d}$-valued (possibly) random functions
of $\bs X_{i}$, which we refer to as the \textit{information proxy
vector}. Typically, the elements of $\bs{\xi}_{n}$ may be different
estimates of the conditional mean function $h_{a[k]}^{*}(\bs X_{i})$.
Based on $\bs{\xi}_{n}$, we propose the calibration estimator:
\begin{equation}
\widehat{\tau}_{\mathrm{cal}}:=\widehat{\tau}_{\mathrm{sdim}}+\frac{1}{n}\sum_{i=1}^{n}\widehat{w}_{i}r_{i},
\end{equation}
where $r_{i}:=\sum_{k=1}^{K}\bigl\{\frac{A_{i}}{\pi_{n[k]}}(Y_{i}-\overline{Y}_{1[k]})-\frac{1-A_{i}}{1-\pi_{n[k]}}(Y_{i}-\overline{Y}_{0[k]})\bigr\}\1(B_{i}=k)$
and the calibration weights $\widehat{w}_{i}$'s ($i=1,\ldots,n$)
solve the calibration problem:
\begin{equation}
\begin{cases}
(\widehat{w}_{1},\ldots,\widehat{w}_{n})=\min_{w_{i},1\leq i\leq n}\sum_{i=1}^{n}D(w_{i})\ \text{subject to }\\
\frac{1}{n}\sum_{i=1}^{n}w_{i}\left\{ A_{i}-\pi_{n[k]}\right\} \1(B_{i}=k)\left\{ \bs{\xi}_{n}(\bs X_{i})-\overline{\bs{\xi}}_{n[k]}\right\} =0, & \forall k=1,\ldots,K.
\end{cases}
\end{equation}
In ((ref)), $\overline{\bs{\xi}}_{n[k]}:=\frac{1}{n_{[k]}}\sum_{i\in[k]}\bs{\xi}_{n}(\bs X_{i})$
is defined as the stratum-specific sample mean for $\bs{\xi}_{n}(\bs X_{i})$.
The calibration problem ((ref)) is a convex optimization
problem involving $dK$ linear constraints. This can be efficiently
solved using standard convex optimization software via its dual formulation
(see boyd2004Convex). The calibration estimator ((ref))
consists of two components: (i) the stratified difference-in-means
estimator $\widehat{\tau}_{\mathrm{sdim}}$ and (ii) a correction
term constructed from weighted residuals, where the weights are determined
by the calibration problem ((ref)). Analogous
to linear regression analysis, the residuals $r_{i}=\sum_{k=1}^{K}\bigl\{\frac{A_{i}}{\pi_{n[k]}}(Y_{i}-\overline{Y}_{1[k]})-\frac{1-A_{i}}{1-\pi_{n[k]}}(Y_{i}-\overline{Y}_{0[k]})\bigr\}\1(B_{i}=k)$,
$i=1,\ldots,n$, represent the part of $\sum_{k=1}^{K}\bigl\{\frac{A_{i}}{\pi_{n[k]}}Y_{i}-\frac{1-A_{i}}{1-\pi_{n[k]}}Y_{i}\bigr\}\1(B_{i}=k)$
that remains unexplained by the stratum mean. Geometrically, the residuals
$r_{i}$ arise from projecting $(\sum_{k=1}^{K}\bigl\{\frac{A_{i}}{\pi_{n[k]}}Y_{i}-\frac{1-A_{i}}{1-\pi_{n[k]}}Y_{i}\bigr\}\1(B_{i}=k):1\leq i\leq n)$
onto the orthogonal complement of the space spanned by $(1,...,1)$,
which inherently implies $\sum_{i=1}^{n}r_{i}=0$. The calibration
problem ((ref)) enforces the balance of $\bs{\xi}_{n}(\bs X_{i})-\overline{\bs{\xi}}_{n[k]}$
across different treatment groups within strata when the observed
samples are weighted by $\widehat{w}_{i}$, $i=1,\ldots,n$, thereby
naturally incorporating the information contained in $\bs{\xi}_{n}$
into the calibration weights $\widehat{w}_{i}$'s. This incorporation
of information allows the calibration weights $\widehat{w}_{i}$ to
further explain the variability in $\sum_{k=1}^{K}\bigl\{\frac{A_{i}}{\pi_{n[k]}}Y_{i}-\frac{1-A_{i}}{1-\pi_{n[k]}}Y_{i}\bigr\}\1(B_{i}=k)$.
Consequently, the weighted average of the residuals, $\frac{1}{n}\sum_{i=1}^{n}\widehat{w}_{i}r_{i}$,
acts as a correction term representing the part explained by the calibration
weights. If $\widehat{w}_{i}=1$ for all $i=1,\ldots,n$, which occurs,
for instance, when $\bs{\xi}_{n}(\bs X)-\overline{\bs{\xi}}_{n[k]}$
is already balanced by the randomization procedure, then $\widehat{\tau}_{\mathrm{cal}}$
reduces to $\widehat{\tau}_{\mathrm{sdim}}$.
Our calibration estimator ((ref)) provides a unified
information integration framework. With an appropriate choice of the
information proxy vector $\boldsymbol{\xi}_{n}$, it recovers many
existing covariate adjustment methods, which can be viewed as a special
case of internal information borrowing (e.g., liu2023Lassoadjusted,cohen2024Noharm,tu2024Unified,bannick2025General,ye2022Inference,ye2023Better,gu2024incorporatingexternaldataanalyzing,bugni2019Inference,ma2022Regression).
For instance, when $\boldsymbol{\xi}_{n}$ is specified as a Lasso-based
estimate, ((ref)) becomes analogous to the Lasso-adjusted
estimator of liu2023Lassoadjusted. Beyond internal adjustment,
((ref)) also accommodates external information proxies,
in parallel to external-borrowing approaches such as gu2024incorporatingexternaldataanalyzing,
and thus enables new estimators that jointly leverage internal and
external information.
A key property of our calibration estimator ((ref))
is its invariance under affine transformations of $\bs{\xi}_{n}(\bs X)$.
Specifically, replacing $\bs{\xi}_{n}(\bs X)$ with $\mathbf{Q}\bs{\xi}_{n}(\bs X)+\bs q$
in the optimization problem ((ref)) results in
the same calibration estimator $\widehat{\tau}_{\mathrm{cal}}$, where
$\mathbf{Q}\in\mathbb{R}^{d\times d}$ is any invertible $d\times d$
matrix and $\bs q\in\mathbb{R}^{d}$ is any $d$-dimensional vector.
If $\bs{\xi}_{n}(\bs X)$ serves as an estimate of the conditional
mean function $h_{a[k]}^{*}(\bs X_{i})$, this property allows for
misspecification up to an affine transformation, offering a flexibility
that is absent in standard AIPW-based covariate adjustment methods.
\section{Strategies for constructing the information proxy $\protect\bs{\xi}_{n}$}
The efficacy and flexibility of the proposed unified framework relies
on the construction of $\bs{\xi}_{n}$, which serves as a proxy for
auxiliary information. Accordingly, we categorize the strategies for
determining $\bs{\xi}_{n}$ into internal and external information
borrowing. Sections (ref)--(ref)
focus on internal strategies, encompassing cross-stratum information
borrowing, the aggregation of different machine learning predictions,
and cross-fitting techniques, whereas Section (ref)
discusses the incorporation of external information such as historical
trials and real-world data.
\subsection{Cross-stratum information borrowing}
Suppose $\widehat{h}_{a[k]}(\cdot)$, where $a\in\{0,1\}$ and $1\leq k\leq K$,
are the estimators for $h_{a[k]}^{*}(\cdot)$. According to Theorem (ref)
(to be established in Section (ref)),
if $\widehat{h}_{a[k]}(\cdot)$ is consistent to $h_{a[k]}^{*}(\cdot)$,
then taking $\bs{\xi}_{n}(\bs X)=\left(\sum_{k=1}^{K}\widehat{h}_{1[k]}(\bs X)\1(B=k),\sum_{k=1}^{K}\widehat{h}_{0[k]}(\bs X)\1(B=k)\right)^{\top}$
in ((ref)) will lead to a semiparametrically efficient
estimator for $\tau$. However, it is important to note that this
approach ensures each stratum performs covariate adjustment using
only the information available within that stratum itself.\textcolor{red}{
}However, borrowing information across strata is often advantageous,
particularly when the relationship between covariates $\bs X$ and
potential outcomes $(Y(1),Y(0))$ is stable across different strata.
Our framework accommodates this naturally by taking $\bs{\xi}_{n}(\bs X)=\left(\widehat{h}_{1[k]}(\bs X),\widehat{h}_{0[k]}(\bs X):1\leq k\le K\right)^{\top}$
in ((ref)). This specification allows each stratum
to use information from all strata, leading to a more efficient calibration
estimator.
\subsection{Integration of heterogeneous machine learning predictions}
In practice, a variety of machine learning methods can be used to
estimate the conditional mean function $h_{a[k]}^{*}(\bs X)$, such
as deep neural networks (lecun2015deep,jiao2023Deep,farrell2021Deepa),
random forests (breiman2001random,wager2018estimation),
Lasso (tibshirani1996Regression), and others. Our framework
provides a natural approach to combine these machine learning estimates.
Suppose $\widehat{h}_{a[k]}^{\text{rf}}(\bs X)$ and $\widehat{h}_{a[k]}^{\text{nn}}(\bs X)$
are the estimates of $h_{a[k]}^{*}(\bs X)$ based on random forests
and deep neural networks, respectively. We can then let $\bs{\xi}_{n}(\bs X)=\left(\widehat{h}_{a[k]}^{\text{rf}}(\bs X),\widehat{h}_{a[k]}^{\text{nn}}(\bs X):a\in\{0,1\},1\leq k\le K\right)^{\top}$
in ((ref)) to derive a calibration estimator for
$\tau$. According to Theorem (ref)
(to be established in Section (ref)),
this estimator will be more efficient than those based solely on random
forests or neural networks when a single machine learning method fails
to fully capture the conditional mean function $h_{a[k]}^{*}(\bs X)$.
This improvement enhances the efficiency of covariate adjustment methods.
\subsection{Implementation via cross-fitting and sample splitting}
When estimating $\bs{\xi}_{n}$ via machine learning methods, it is
often desirable to rely on an independent sample in order to mitigate
overfitting and to make Assumption (ref)
more plausible. Such independence can be achieved through sample-splitting.
However, sample-splitting typically reduces efficiency. To recover
efficiency, we adopt the cross-fitting technique (see chernozhukov2018Double,tu2024Unified,bannick2025General,rafi2023Efficient).
Specifically, we follow the sample-splitting procedure outlined in
rafi2023Efficient, which ensures that each fold
contains data from every stratum and treatment arm. Suppose the units
$\{1,\ldots,n\}$ are partitioned into two equal folds, $I_{0}$ and
$I_{1}$. The cross-fitted calibration estimator is then defined as
$\widehat{\tau}_{\mathrm{cal}}^{\mathrm{CF}}:=\frac{1}{2}\widehat{\tau}_{\mathrm{cal}}^{(0)}+\frac{1}{2}\widehat{\tau}_{\mathrm{cal}}^{(1)}$,
where
\begin{align*}
\widehat{\tau}_{\mathrm{cal}}^{(\iota)} & =\sum_{k=1}^{K}p_{n[k]}^{(\iota)}\left(\overline{Y}_{1[k]}^{(\iota)}-\overline{Y}_{0[k]}^{(\iota)}\right)\\
& \quad+\frac{1}{\left|I_{\iota}\right|}\sum_{i\in I_{\iota}}\widehat{w}_{i}\sum_{k=1}^{K}\left\{ \frac{A_{i}}{\pi_{n[k]}^{(\iota)}}\left(Y_{i}-\overline{Y}_{1[k]}^{(\iota)}\right)-\frac{1-A_{i}}{1-\pi_{n[k]}^{(\iota)}}\left(Y_{i}-\overline{Y}_{0[k]}^{(\iota)}\right)\right\} \1(B_{i}=k)
\end{align*}
with $\left|I_{\iota}\right|=\sum_{i\in I_{\iota}}1$, $p_{n[k]}^{(\iota)}=\sum_{i\in I_{\iota}}\1(B_{i}=k)/\left|I_{\iota}\right|$,
$\overline{Y}_{a[k]}^{(\iota)}=\sum_{i\in I_{\iota}}\1(A_{i}=a,B_{i}=k)Y_{i}/\sum_{i\in I_{\iota}}\1(A_{i}=a,B_{i}=k)$,
$\pi_{n[k]}^{(\iota)}=\sum_{i\in I_{\iota}}\1(A_{i}=a,B_{i}=k)/\sum_{i\in I_{\iota}}\1(B_{i}=k)$
for $\iota\in\{0,1\}$. The weights $\widehat{w}_{i}$'s ($i\in I_{\iota}$)
are obtained by solving the following calibration problem:
\[
\begin{cases}
(\widehat{w}_{i})_{i\in I_{\iota}}=\min_{w_{i}:i\in I_{\iota}}\sum_{i\in I_{\iota}}D(w_{i})\ \text{subject to }\\
\sum_{i\in I_{\iota}}w_{i}\left\{ A_{i}-\pi_{n[k]}^{(\iota)}\right\} \1(B_{i}=k)\left\{ \bs{\xi}_{n}^{(1-\iota)}(\bs X_{i})-\overline{\bs{\xi}}_{n[k]}^{(1-\iota)}\right\} =0, & \forall k=1,\ldots,K,
\end{cases}
\]
where $\bs{\xi}_{n}^{(1-\iota)}$ is measurable with respect to $\{(Y_{i},\bs X_{i},A_{i}):i\in I_{\iota}\}$
and $\overline{\bs{\xi}}_{n[k]}^{(1-\iota)}:=\sum_{i\in I_{\iota}}\bs{\xi}_{n}(\bs X_{i})\1(B_{i}=k)/\sum_{i\in I_{\iota}}\1(B_{i}=k)$
denotes the stratum-specific sample mean for $\bs{\xi}_{n}^{(1-\iota)}(\bs X)$.
The asymptotic properties of $\widehat{\tau}_{\mathrm{cal}}^{\mathrm{CF}}$
can be established by combining the proof of Theorem (ref)
with the arguments from the proof of rafi2023Efficient
and we omit the details here. We will evaluate the finite-sample performance
of this cross-fitted estimator $\widehat{\tau}_{\mathrm{cal}}^{\mathrm{CF}}$
in our simulation studies.
\subsection{Leveraging historical and real-world data}
Beyond internal strategies, constructing $\bs{\xi}_{n}$ using external
information offers a powerful way to enhance efficiency. In this subsection,
we focus on two primary sources: historical clinical trials and real-world
data. While these sources provide valuable information, they often
differ distributionally from the current trial. A key distinction
of our framework is its ability to leverage such heterogeneous external
data without requiring restrictive similarity assumptions.
First, historical clinical trials often provide readily available
data from settings similar to the current study. Since treatments
often vary between trials, existing literature has predominantly focused
on leveraging historical control data to enhance the precision of
estimates in the current trial (pocock1975Sequential,callegaro2023Historical).
These methods usually rely on the assumption that the historical and
current control arms are comparable. In contrast, a distinct advantage
of our unified framework is its assumption-lean nature: we impose
no assumptions on the validity or direct transferability of the borrowed
information. Consequently, we are not restricted to borrowing solely
from historical control arms; we can flexibly incorporate information
from historical studies involving treatments that may differ from
those in the current trial.
Second, beyond historical clinical trials, real-world data represents
another vast information source. Real-world data encompasses data
generated from routine healthcare delivery and patient monitoring,
including electronic health records (EHRs), disease registries, and
increasingly, data from wearable devices. Unlike tightly controlled
clinical trials, observational studies based on real-world data typically
involve much larger and more diverse patient populations, which can
significantly strengthen the statistical power of the analysis.
Crucially, both historical trial data and real-world data may exhibit
a “covariate shift”, i.e., the distribution of covariates $\bs X$
in these external sources often differs from that of the current trial.
However, a common phenomenon is that while the marginal distribution
of $\bs X$ changes, the conditional distribution of the potential
outcomes $(Y(1),Y(0))$ given the covariates $\bs X$ remains comparable.
This stability is important because the optimal choice of $\bs{\xi}_{n}(\bs X)$,
which ensures that the resulting estimator $\widehat{\tau}_{\mathrm{cal}}$
is semiparametrically efficient, depends solely on the conditional
distribution of $(Y(1),Y(0))$ given $\bs X$ (see Theorem (ref)).
In practice, we can leverage this by estimating the conditional mean
functions $h_{1[k]}^{*}(\bs X)$ and $h_{0[k]}^{*}(\bs X)$ ($1\leq k\leq K$)
from these external datasets and setting $\bs{\xi}_{n}(\bs X)$ as
the vector of these estimates. Our Theorem (ref)
guarantees that incorporating these estimates from the external datasets
will result in a calibration estimator with no greater asymptotic
variance. Consequently, our framework provides a robust strategy for
improving estimation efficiency without the risk of “negative transfer”.
Unlike existing methods that rely on Bayesian frameworks (hobbs2011Hierarchical,ibrahim2015Power)
or Trans-Lasso (gu2024incorporatingexternaldataanalyzing),
where the transfer of information depends on the prior distributions
and/or model assumptions, our approach is entirely model-free. Notably,
it imposes no similarity constraints between the external data and
the target data-generating process, thereby enhancing flexibility
and robustness against real-world data complexities.
\section{Asymptotic properties}
We make the following assumptions.
\begin{assumption}
$\bs W_{i}=(Y_{i}(1),Y_{i}(0),\bs X_{i}^{\top},B_{i})^{\top},$
$i=1,\ldots,n$, are i.i.d. samples from the population distribution
of $\bs W=(Y(1),Y(0),\bs X^{\top},B)^{\top}$, and we denote $\bs W^{(n)}=\{\bs W_{1},\ldots,\bs W_{n}\}$.
Besides, $\sup_{1\leq k\leq K}\left[\left|Y_{i}(a)\right|^{2+\epsilon}\mid B_{i}=k\right]<\infty$
for both $a=0,1$, where $0<\epsilon\leq1$ is a constant.
\end{assumption}
\begin{assumption}
[Treatment assignment]Let $A^{(n)}=\{A_{1},\ldots,A_{n}\}$
and $B^{(n)}=\{B_{1},\ldots,B_{n}\}$. The treatment assignment mechanism
satisfies the following conditions:
\begin{enumerate}[label=(\arabic*)]
• conditional on the stratum indicators $B^{(n)}$, the treatment assignments
$A^{(n)}$ are independent of $\bs W^{(n)}$, i.e., $\bs W^{(n)}\perp A^{(n)}\mid B^{(n)}$;
• $\sup_{1\leq k\leq K}\left|\pi_{n[k]}-\pi_{[k]}\right|\overset{p}{\to}0$ as $n\to\infty$,
where $\pi_{[k]}$, $k=1,\ldots,K$, satisfy
\[
0<\inf_{n\geq1}\inf_{1\leq k\leq K}\pi_{[k]}\leq\sup_{n\geq1}\sup_{1\leq k\leq K}\pi_{[k]}<1.
\]
Moreover, $\lim_{n\to\infty}P(\inf_{1\leq k\leq K}n_{[k]}\geq d+2)=1$,
where $d$ denotes the dimension of $\bs{\xi}_{n}$.
\end{enumerate}
\end{assumption}
\begin{assumption}
$d$ and $K$ are fixed numbers.
The sequence of (possibly) random functions $\bs{\xi}_{n}$ satisfies
the following conditions:
\begin{enumerate}[label=(\arabic*)]
• There exists a sequence of non-stochastic functions $\bs{\xi}_{n}^{*}:\mathcal{X}\to\mathbb{R}^{d}$
($n\geq1$) such that
\begin{enumerate}
• $\sup_{n\geq1}\mathbb{E}\left[\left\Vert \bs{\xi}_{n}^{*}(\bs X_{i})\right\Vert ^{2+\epsilon}\right]<\infty$,
where $\epsilon$ is defined in Assumption (ref);
• the minimal non-zero singular value of $\mathbb{E}\left[\widetilde{\bs{\xi}}_{n}^{*}(\bs X_{i})\widetilde{\bs{\xi}}_{n}^{*}(\bs X_{i})^{\top}\mid B_{i}=k\right]$
is larger than some constant $c>0$ uniformly over $n\geq1$ and $k=1,\ldots,K$;
• for every $k=1,\ldots,K$,
\[
\frac{1}{n_{1[k]}}\sum_{i\in[k]}A_{i}\left\{ \bs{\xi}_{n}(\bs X_{i})-\bs{\xi}_{n}^{*}(\bs X_{i})\right\} -\frac{1}{n_{0[k]}}\sum_{i\in[k]}\left(1-A_{i}\right)\left\{ \bs{\xi}_{n}(\bs X_{i})-\bs{\xi}_{n}^{*}(\bs X_{i})\right\} =o_{P}(n^{-1/2})
\]
and $\frac{1}{n_{[k]}}\sum_{i\in[k]}\left\Vert \bs{\xi}_{n}(\bs X_{i})-\bs{\xi}_{n}^{*}(\bs X_{i})\right\Vert ^{2}=o_{P}(1)$\textup{;}
• $\liminf_{n\to\infty}(\varsigma_{\widetilde{Y}}^{2}-\varsigma_{\widetilde{Y}\mid\widetilde{\bs{\xi}}_{n}^{*}}^{2})>0$,
where $\varsigma_{\widetilde{Y}}^{2}$ and $\varsigma_{\widetilde{Y}\mid\widetilde{\bs{\xi}}_{n}^{*}}^{2}$
are defined in Appendix (ref);
\end{enumerate}
• For every $k=1,\ldots,K$,
\[
\left\Vert \left\{ \frac{1}{n}\sum_{i\in[k]}(A_{i}-\pi_{n[k]})^{2}\left\{ \bs{\xi}_{n}(\bs X_{i})-\overline{\bs{\xi}}_{n[k]}\right\} \left\{ \bs{\xi}_{n}(\bs X_{i})-\overline{\bs{\xi}}_{n[k]}\right\} ^{\top}\right\} ^{+}\right\Vert =O_{P}(1).
\]
\end{enumerate}
\end{assumption}
In this section, we focus on the case where the number of strata,
$K$, is fixed, and leave the case where $K$ is diverging to Section (ref).
Assumptions (ref) and (ref)
are standard in the literature on statistical inference under CAR
(see, e.g., bugni2018Inference,bannick2025General,bugni2019Inference,ma2022Regression,tu2024Unified,jiang2023Regressionadjusted
and references therein). Assumption (ref)
is satisfied for many randomization methods, such as stratified block
randomization zelen1974Randomization, and Pocock and Simon’s
minimization pocock1975Sequential. Assumption (ref)(1)(a)
is a mild moment assumption on $\bs{\xi}_{n}^{*}$, the probability
limit of $\bs{\xi}_{n}$. Assumption (ref)(1)(b)
restricts that the minimal non-zero singular value of $\mathbb{E}\left[\widetilde{\bs{\xi}}_{n}^{*}(\bs X_{i})\widetilde{\bs{\xi}}_{n}^{*}(\bs X_{i})^{\top}\mid B_{i}=k\right]$
is bounded away from zero. Importantly, this assumption allows the
matrix $\mathbb{E}\left[\widetilde{\bs{\xi}}_{n}^{*}(\bs X_{i})\widetilde{\bs{\xi}}_{n}^{*}(\bs X_{i})^{\top}\mid B_{i}=k\right]$
to be singular. This is reasonable, as in many applications, the components
of $\bs{\xi}_{n}$ may share common information, leading to cases
where the matrix $\mathbb{E}\left[\widetilde{\bs{\xi}}_{n}^{*}(\bs X_{i})\widetilde{\bs{\xi}}_{n}^{*}(\bs X_{i})^{\top}\mid B_{i}=k\right]$
is indeed singular. Assumption (ref)(1)(c)
requires that $\bs{\xi}_{n}$ converges to its probability limit $\bs{\xi}_{n}^{*}$.
This assumption is similar to tu2024Unified,
bannick2025General and jiang2023Regressionadjusted.
When $\bs{\xi}_{n}$ is derived from linear regression, it simplifies
to liu2023Lassoadjusted. When $\bs{\xi}_{n}$
is derived from local linear kernel regression, this assumption can
be verified by tu2024Unified. If $\bs{\xi}_{n}$
is a general machine learning estimator that depends on the entire
observed dataset, then verifying Assumption (ref)(1)(c)
usually requires that the function class containing $\bs{\xi}_{n}$
satisfies the Donsker's condition (see bannick2025General),
which may be violated if $\bs{\xi}_{n}$ is derived from complex machine
learning algorithms. To bypass the Donsker's condition, one can use
sample-splitting and/or cross-fitting (rafi2023Efficient,tu2024Unified,chernozhukov2018Double)
techniques as discussed in Section (ref). By
using cross-fitting, Assumption (ref)(1)(c)
can be replaced by a mean square error convergence condition (see
rafi2023Efficient and tu2024Unified),
which is satisfied for many machine learning methods (see, e.g., farrell2021Deepa,chi2022Asymptotic,wager2018estimation,jiao2023Deep).
Assumption (ref)(1)(d) ensures that
the outcome $Y$ cannot be fully explained by $\bs{\xi}(\bs X)$.
Assumption (ref)(2) is a technical
requirement that ensures the estimated Moore--Penrose inverse remains
well-behaved and does not diverge. This condition is necessary to
address the discontinuity of the Moore--Penrose inverse operation
(see e.g., stewart1977Perturbation), as Assumptions (ref)--(ref)
and (ref)(1)(a)--(c) alone do not guarantee
the convergence of the inverse. However, in the standard case where
the limiting matrix $\mathbb{E}\left[\widetilde{\bs{\xi}}_{n}^{*}(\bs X_{i})\widetilde{\bs{\xi}}_{n}^{*}(\bs X_{i})^{\top}\mid B_{i}=k\right]$
is non-singular, the Moore--Penrose inverse coincides with the standard
matrix inverse. Since standard inversion is a continuous operation,
Assumption (ref)(2) is then automatically
implied by the preceding assumptions.
Recall that $h_{a[k]}^{*}(\bs X)=\mathbb{E}\left[Y(a)\mid\bs X,B=k\right]$
and let $\widetilde{h}_{a[k]}^{*}(\bs X):=h_{a[k]}^{*}(\bs X)-\mathbb{E}\left[h_{a[k]}^{*}(\bs X)\mid B=k\right]$
and $\widetilde{\bs{\xi}}_{n}^{*}(\bs X):=\bs{\xi}_{n}^{*}(\bs X)-\mathbb{E}\left[\bs{\xi}_{n}^{*}(\bs X)\mid B=k\right]$.
We have the following theorem.
\begin{thm}
Suppose that Assumptions
(ref)--(ref)
hold and $D(v)=(v-1)^{2}/2$. Then
\[
\frac{\sqrt{n}\left(\widehat{\tau}_{\mathrm{cal}}-\tau\right)}{\sqrt{\varsigma_{H}^{2}+\varsigma_{\widetilde{Y}}^{2}-\varsigma_{\widetilde{Y}\mid\widetilde{\bs{\xi}}_{n}^{*}}^{2}}}\overset{d}{\to} N(0,1)\text{ and }\widehat{\varsigma}_{H}^{2}+\widehat{\varsigma}_{\widetilde{Y}}^{2}-\widehat{\varsigma}_{\widetilde{Y}\mid\widetilde{\bs{\xi}}_{n}^{*}}^{2}=\varsigma_{H}^{2}+\varsigma_{\widetilde{Y}}^{2}-\varsigma_{\widetilde{Y}\mid\widetilde{\bs{\xi}}_{n}^{*}}^{2}+o_{P}(1),
\]
where the definitions of the asymptotic variances $\varsigma_{H}^{2}$,
$\varsigma_{\widetilde{Y}}^{2}$, $\varsigma_{\widetilde{Y}\mid\widetilde{\bs{\xi}}_{n}^{*}}^{2}$
and their estimates $\widehat{\varsigma}_{H}^{2},\widehat{\varsigma}_{\widetilde{Y}}^{2}$
and $\widehat{\varsigma}_{\widetilde{Y}\mid\widetilde{\bs{\xi}}_{n}^{*}}^{2}$
can be found in Appendix (ref). Moreover, if
for every $k=1,\ldots,K$, there exists a non-stochastic vector $\bs{\alpha}_{k}\in\mathbb{R}^{d}$
such that
\[
\left\{ \sqrt{\frac{1-\pi_{[k]}}{\pi_{[k]}}}\widetilde{h}_{1[k]}^{*}(\bs X)+\sqrt{\frac{\pi_{[k]}}{1-\pi_{[k]}}}\widetilde{h}_{0[k]}^{*}(\bs X)\right\} \1(B=k)=\bs{\alpha}_{k}^{\top}\widetilde{\bs{\xi}}_{n}^{*}(\bs X)\1(B=k),
\]
then
\[
\frac{\sqrt{n}\left(\widehat{\tau}_{\mathrm{cal}}-\tau\right)}{\sqrt{\varsigma_{H}^{2}+\varsigma_{\widetilde{Y}}^{2}-\varsigma_{\widetilde{Y}\mid\widetilde{h}^{*}}^{2}}}\overset{d}{\to} N(0,1),
\]
and the asymptotic variance $\varsigma_{H}^{2}+\varsigma_{\widetilde{Y}}^{2}-\varsigma_{\widetilde{Y}\mid\widetilde{h}^{*}}^{2}$
matches the semiparametric efficiency bound developed in rafi2023Efficient.
\end{thm}
In Theorem (ref), we focus on
the case that $D(v)=(v-1)^{2}/2$; the general $D(v)$ will be handled
in Section (ref). Theorem (ref)
shows that our calibration estimator is asymptotically normally distributed
and its asymptotic variance can be consistently estimated. Moreover,
as long as there exists a linear combination of $\bs{\xi}_{n}^{*}(\bs X)\1(B=k)$
that equals $\bigl\{\sqrt{\frac{1-\pi_{[k]}}{\pi_{[k]}}}h_{1[k]}^{*}(\bs X)+\sqrt{\frac{\pi_{[k]}}{1-\pi_{[k]}}}h_{0[k]}^{*}(\bs X)\bigr\}\1(B=k)$,
our calibration estimator is semiparametric efficient. To the best
of our knowledge, this is a new condition for achieving the efficiency
bound in the literature. Existing work (e.g., tu2024Unified)
requires that both $h_{1[k]}^{*}(\bs X)$ and $h_{0[k]}^{*}(\bs X)$
be consistently estimated. In contrast, we only require that the linear
combination of these functions be consistently estimated, which is
weaker. This condition aligns with bai2022Optimality,
which states that optimal stratification can be achieved by knowing
the function $\left\{ \sqrt{\frac{1-\pi_{[k]}}{\pi_{[k]}}}h_{1[k]}^{*}(\bs X)+\sqrt{\frac{\pi_{[k]}}{1-\pi_{[k]}}}h_{0[k]}^{*}(\bs X)\right\} $.
In this context, the optimal stratification refers to the stratification
method under which the difference-in-means estimator has the smallest
mean squared error. Since the components of $\bs{\xi}_{n}(\bs X)$
can be chosen as different estimates of $\bigl\{\sqrt{\frac{1-\pi_{[k]}}{\pi_{[k]}}}h_{1[k]}^{*}(\bs X)+\sqrt{\frac{\pi_{[k]}}{1-\pi_{[k]}}}h_{0[k]}^{*}(\bs X)\bigr\}$
from various pre-specified models, the calibration estimator is \textit{multiply
efficient}: it remains semiparametric efficient as long as at least
one of these models is correctly specified. In contrast, AIPW-based
covariate adjustment methods lack this kind of multiple efficiency.
Theorem (ref) implies that the
calibration estimator ((ref)) possesses two theoretical
advantages. First, Assumption (ref) imposes
no restriction on the choice of randomization scheme, which may include
simple randomization, stratified block randomization, or minimization.
Since the asymptotic distribution of the calibration estimator is
invariant to the randomization method, it satisfies the property referred
to in the literature as “universal applicability” (bannick2025General,ye2023Better).
Second, because $\varsigma_{\widetilde{Y}\mid\widetilde{\bs{\xi}}_{n}^{*}}^{2}\geq0$,
Theorem (ref), together with
ma2022Regression, implies that $\widehat{\tau}_{\mathrm{cal}}$
is always at least as efficient as the stratified difference-in-means
estimator $\widehat{\tau}_{\mathrm{sdim}}$, which corresponds to
the special case where $\widehat{w}_{i}=1$ for all $i=1,\ldots,n$
in ((ref)). Hence, the calibration estimator guarantees
efficiency gains. More generally, we have the following efficiency
comparison result.
\begin{thm}
Rewrite the estimator $\widehat{\tau}_{\mathrm{cal}}$
in ((ref)) as $\widehat{\tau}_{\mathrm{cal}}(\bs{\xi}_{n})$.
Let $\mathbf{\Lambda}$ be a $\widetilde{d}\times d$ non-stochastic
matrix, where $\widetilde{d}\geq1$ is fixed. Suppose that Assumptions
(ref)--(ref)
hold for both $\bs{\xi}_{n}$ and $\mathbf{\Lambda}\bs{\xi}_{n}$.
Then $\widehat{\tau}_{\mathrm{cal}}(\bs{\xi}_{n})$ and $\widehat{\tau}_{\mathrm{cal}}(\mathbf{\Lambda}\bs{\xi}_{n})$
satisfy
\[
\frac{\sqrt{n}\left(\widehat{\tau}_{\mathrm{cal}}(\bs{\xi}_{n})-\tau\right)}{\sqrt{\varsigma_{H}^{2}+\varsigma_{\widetilde{Y}}^{2}-\varsigma_{\widetilde{Y}\mid\widetilde{\bs{\xi}}_{n}^{*}}^{2}}}\overset{d}{\to} N(0,1)\text{ and }\frac{\sqrt{n}\left(\widehat{\tau}_{\mathrm{cal}}(\mathbf{\Lambda}\bs{\xi}_{n})-\tau\right)}{\sqrt{\varsigma_{H}^{2}+\varsigma_{\widetilde{Y}}^{2}-\varsigma_{\widetilde{Y}\mid\mathbf{\Lambda}\widetilde{\bs{\xi}}_{n}^{*}}^{2}}}\overset{d}{\to} N(0,1),
\]
respectively. Furthermore, the asymptotic variances satisfy $\varsigma_{H}^{2}+\varsigma_{\widetilde{Y}}^{2}-\varsigma_{\widetilde{Y}\mid\widetilde{\bs{\xi}}_{n}^{*}}^{2}\leq\varsigma_{H}^{2}+\varsigma_{\widetilde{Y}}^{2}-\varsigma_{\widetilde{Y}\mid\mathbf{\Lambda}\widetilde{\bs{\xi}}_{n}^{*}}^{2}.$
\end{thm}
Theorem (ref) implies that incorporating
additional elements into $\bs{\xi}_{n}$ satisfies a no-harm property;
i.e., it is guaranteed to improve or at least maintain efficiency.
Consequently, from a theoretical perspective, it appears optimal to
include as many elements as possible in $\bs{\xi}_{n}$. However,
as the dimension of $\bs{\xi}_{n}$ becomes large, it is no longer
appropriate to treat the dimension $d$ as fixed. We will address
the scenario where the dimension of $\bs{\xi}_{n}$ diverges in Section (ref).
\section{Extensions}
\subsection{The dimension of $\protect\bs{\xi}_{n}(\cdot)$ and the number of
strata are diverging}
In this section, we allow the dimension of $\bs{\xi}_{n}(\cdot)$
to grow with $n$, that is, $d=d_{n}\to\infty$ as $n\to\infty$.
Additionally, we also allow the number of strata to increase with
$n$, i.e., $K=K_{n}\to\infty$ as $n\to\infty$, which is commonly
encountered in many applications. We begin by stating an assumption.
\begin{assumption}
The sequence of
random functions $\bs{\xi}_{n}(\cdot)$ satisfies the following conditions.
\begin{enumerate}[label=(\arabic*)]
• There exists a sequence of non-stochastic functions $\bs{\xi}_{n}^{*}(\cdot):\mathcal{X}\to\mathbb{R}^{d}$
($n\geq1$) such that
\begin{enumerate}
• $K^{2}r_{n}^{2}/n\to0$, $K^{2}\zeta_{n}^{2}r_{n}/n\to0$ and $\max\left\{ \zeta_{n}^{2}\log(2Kd),r_{n}\log^{2}(2Kd)\right\} /(n\inf_{1\leq k\leq K}p_{[k]})\to0$
as $n\to\infty$, where $r_{n}:=\max_{1\leq k\leq K}\mathrm{rank}\left\{ \mathbb{E}\left[\bs{\xi}_{n}^{*}(\bs X_{i})\bs{\xi}_{n}^{*}(\bs X_{i})^{\top}\mid B_{i}=k\right]\right\} $
and $\zeta_{n}:=\sup_{\bs x\in\mathcal{X}}\left\Vert \bs{\xi}_{n}^{*}(\bs x)\right\Vert $;
• the maximal singular value of $\mathbb{E}\left[\bs{\xi}_{n}^{*}(\bs X_{i})\bs{\xi}_{n}^{*}(\bs X_{i})^{\top}\mid B_{i}=k\right]$
is smaller than some finite constant $C>0$ uniformly over $n\geq1$
and $k=1,\ldots,K$; the minimal non-zero singular value of $\mathbb{E}\left[\widetilde{\bs{\xi}}_{n}^{*}(\bs X_{i})\widetilde{\bs{\xi}}_{n}^{*}(\bs X_{i})^{\top}\mid B_{i}=k\right]$
is larger than some constant $c>0$ uniformly over $n\geq1$ and $k=1,\ldots,K$;
• it holds that
\begin{align*}
& \sup_{1\leq k\leq K}\left\Vert \frac{1}{n_{1[k]}}\sum_{i\in[k]}A_{i}\left\{ \bs{\xi}_{n}(\bs X_{i})-\bs{\xi}_{n}^{*}(\bs X_{i})\right\} -\frac{1}{n_{0[k]}}\sum_{i\in[k]}\left(1-A_{i}\right)\left\{ \bs{\xi}_{n}(\bs X_{i})-\bs{\xi}_{n}^{*}(\bs X_{i})\right\} \right\Vert \\
= & o_{P}(n^{-1/2})
\end{align*}
and $\sup_{1\leq k\leq K}\frac{1}{n_{[k]}}\sum_{i\in[k]}\left\Vert \bs{\xi}_{n}(\bs X_{i})-\bs{\xi}_{n}^{*}(\bs X_{i})\right\Vert ^{2}=O_{P}(n^{-1/2})$\textup{;}
• $\liminf_{n\to\infty}\left(\varsigma_{\widetilde{Y}}^{2}-\varsigma_{\widetilde{Y}\mid\widetilde{\bs{\xi}}_{n}^{*}}^{2}\right)>0$.
\end{enumerate}
• $\sup_{1\leq k\leq K}\left\Vert \left\{ \frac{1}{n_{[k]}}\sum_{i\in[k]}(A_{i}-\pi_{n[k]})^{2}\left\{ \bs{\xi}_{n}(\bs X_{i})-\overline{\bs{\xi}}_{n[k]}\right\} \left\{ \bs{\xi}_{n}(\bs X_{i})-\overline{\bs{\xi}}_{n[k]}\right\} ^{\top}\right\} ^{+}\right\Vert =O_{P}(1)$.
\end{enumerate}
\end{assumption}
Assumption (ref) is very
similar to Assumption (ref), except
that we allow both the dimension of $\bs{\xi}_{n}$ and the number
of strata to diverge. Assumption (ref)(1)(a)
restricts the growth rate of $d$ and $K$. Suppose that the matrix
$\mathbb{E}\left[\bs{\xi}_{n}^{*}(\bs X_{i})\bs{\xi}_{n}^{*}(\bs X_{i})^{\top}\mid B_{i}=k\right]$
is non-singular and the elements in $\bs{\xi}_{n}^{*}(\bs x)$ are
uniformly bounded, we have $r_{n}=d$ and $\zeta_{n}=\sqrt{d}$. Consider
the case that the strata have equal size, i.e., $p_{[k]}=1/K$. Then
a sufficient condition for Assumption (ref)(1)(a)
is $Kd=o(\sqrt{n})$, meaning that we allow the product of the dimension
of $\bs{\xi}_{n}$ and the number of strata to grow, but at a rate
slower than $\sqrt{n}$. When $K$ is fixed, this condition is consistent
with a similar result for the regression-adjusted ATE estimator presented
by lei2021Regression under complete randomization and finite-population
asymptotics. To further relax the growth rate constraint on $d$, lei2021Regression, lu2025Debiased, and gu2025assumptionleancovariateadjustmentcovariate proposed debiased regression-adjusted estimators that allow $d$ to grow faster than $\sqrt{n}$. In contrast, jiang2025Adjustments achieved the same goal by assuming a correctly specified linear relationship between the potential outcomes and covariates. It is also possible
to debias the calibration estimator ((ref)) in a
way that would relax Assumption (ref)(1)(a).
However, addressing this is beyond the scope of the present paper
and will be left for future research. Assumption (ref)(1)(b)
requires that largest and smallest non-zero singular values of $\mathbb{E}\left[\widetilde{\bs{\xi}}_{n}^{*}(\bs X_{i})\widetilde{\bs{\xi}}_{n}^{*}(\bs X_{i})^{\top}\mid B_{i}=k\right]$
are bounded away from zero and infinity, while still allowing this
matrix to be singular. Assumption (ref)(1)(c)
strengthens Assumption (ref)(1)(c).
Under cross-fitting, Assumption (ref)(1)(c)
requires that $\bs{\xi}_{n}$ converges to its probability limit $\bs{\xi}_{n}^{*}$
at rate $n^{-1/4}$ uniformly over $k=1,\ldots,K$. Such a rate is
achievable for many modern machine learning methods (see, e.g., farrell2021Deepa,jiao2023Deep).
Assumption (ref)(2) parallels
Assumption (ref)(2), except that it
is required to hold uniformly over $k=1,\ldots,K$. We are now ready
to state the following theorem.
\begin{thm}
Suppose
that Assumptions (ref)--(ref)
and (ref) hold and $D(v)=(v-1)^{2}/2$.
Then
\[
\frac{\sqrt{n}\left(\widehat{\tau}_{\mathrm{cal}}-\tau\right)}{\sqrt{\varsigma_{H}^{2}+\varsigma_{\widetilde{Y}}^{2}-\varsigma_{\widetilde{Y}\mid\widetilde{\bs{\xi}}_{n}^{*}}^{2}}}\overset{d}{\to} N(0,1)\text{ and }\widehat{\varsigma}_{H}^{2}+\widehat{\varsigma}_{\widetilde{Y}}^{2}-\widehat{\varsigma}_{\widetilde{Y}\mid\widetilde{\bs{\xi}}_{n}^{*}}^{2}=\varsigma_{H}^{2}+\varsigma_{\widetilde{Y}}^{2}-\varsigma_{\widetilde{Y}\mid\widetilde{\bs{\xi}}_{n}^{*}}^{2}+o_{P}(1).
\]
Moreover, when $K$ is a fixed number, if for every $k=1,\ldots,K$,
there exists a non-stochastic vector $\bs{\alpha}_{k}\in\mathbb{R}^{d}$ such
that
\[
\left\{ \sqrt{\frac{1-\pi_{[k]}}{\pi_{[k]}}}\widetilde{h}_{1[k]}^{*}(\bs X)+\sqrt{\frac{\pi_{[k]}}{1-\pi_{[k]}}}\widetilde{h}_{0[k]}^{*}(\bs X)\right\} \1(B=k)=\bs{\alpha}_{k}^{\top}\widetilde{\bs{\xi}}_{n}^{*}(\bs X)\1(B=k),
\]
then
\[
\frac{\sqrt{n}\left(\widehat{\tau}_{\mathrm{cal}}-\tau\right)}{\sqrt{\varsigma_{H}^{2}+\varsigma_{\widetilde{Y}}^{2}-\varsigma_{\widetilde{Y}\mid\widetilde{h}^{*}}^{2}}}\overset{d}{\to} N(0,1),
\]
and the asymptotic variance $\varsigma_{H}^{2}+\varsigma_{\widetilde{Y}}^{2}-\varsigma_{\widetilde{Y}\mid\widetilde{h}^{*}}^{2}$
matches the semiparametric efficiency bound developed in rafi2023Efficient.
\end{thm}
Theorem (ref) shows
that, even when both the dimension of $\bs{\xi}_{n}$ and the number
of strata diverge, the calibration estimator ((ref))
remains asymptotically normal. It is important to note that, because
we allow the number of strata to increase with the sample size, Assumption
(ref) becomes non-trivial. xin2024inferencecovariateadaptiverandomizationstrata
studied inference under CAR with a growing number of strata, and our
Assumption (ref) corresponds directly to
their Assumptions (B1)--(B3). Moreover, xin2024inferencecovariateadaptiverandomizationstrata
derived more primitive conditions under which Assumption (ref)
holds in the cases of simple randomization, stratified adaptive biased-coin
randomization and stratified block randomization. For further discussion
of Assumption (ref), we refer readers to
xin2024inferencecovariateadaptiverandomizationstrata.
\subsection{General discrepancy measure $D(v)$}
In Theorem (ref), our analysis
was limited to the quadratic discrepancy measure $D(v)=(v-1)^{2}/2$.
In this section, we extend the analysis to a general class of discrepancy
measures $D(v)$, which can be theoretically shown to exhibit smaller
second-order bias. To proceed, we impose the following mild regularity
condition on $D(v)$.
\begin{assumption}
Let $D^{\prime}$ be the derivative
of $D$ and $(D^{\prime})^{-1}$ is the inverse function of $D^{\prime}$.
Define $\rho(v):=D\left\{ (D^{\prime})^{-1}(-v)\right\} +v\cdot(D^{\prime})^{-1}(-v)$.
We assume $\rho(v)$ is concave and three times continuously differentiable,
$\rho^{\prime}(0)=1$, $-\infty<\rho^{\prime\prime}(0)<0$ and there
exist constants $\delta>0$ and $0\leq C_{\rho}<\infty$ such that
$\left|\rho^{\prime\prime\prime}(v)-\rho^{\prime\prime\prime}(0)\right|\leq C_{\rho}\left|v\right|$
for all $\left|v\right|\leq\delta$.
\end{assumption}
Assumption (ref) holds for a wide range of
commonly used discrepancy measures, and Table (ref)
provides several popular examples. The case that $D(v)=v\log v-v$
is related to the exponential tilting estimator studied by kitamura1997Informationtheoretic,
and $D(v)=v-\log v$ is related to empirical likelihood estimator
studied by qin1994Empirical.
\begin{table}[!tbh]
\caption{Different choices of $D(v)$
and their corresponding $\rho(v)$.}
\begin{tabular}{ccccc}
\toprule
$D(v)$ & $\rho(v)$ & $\rho^{\prime}(v)$ & $\rho^{\prime\prime}(0)$ & $\rho^{\prime\prime\prime}(0)$\tabularnewline
\midrule
\midrule
$(v-1)^{2}/2$ & $-v^{2}/2+v$ & $-v+1$ & $-1$ & $0$\tabularnewline
\midrule
$v\log v-v$ & $-e^{-v}$ & $e^{-v}$ & $-1$ & $1$\tabularnewline
\midrule
$v-\log v$ & $1+\log(1+v)$ & $\frac{1}{1+v}$ & $-1$ & $2$\tabularnewline
\bottomrule
\end{tabular}
\end{table}
Since our goal is to characterize the second-order bias of the calibration
estimator, we require a strengthened version of Assumption (ref).
\begin{assumption}
\begin{enumerate}[label=(\arabic*)]
• The sequence of random functions $\bs{\xi}_{n}(\cdot)$ satisfies
$\frac{1}{n}\sum_{i=1}^{n}\left\Vert \bs{\xi}_{n}(\bs X_{i})\right\Vert ^{4}=O_{P}(1)$;
• The non-stochastic functions $\bs{\xi}_{n}^{*}(\cdot):\mathcal{X}\to\mathbb{R}^{d}$
($n\geq1$) in Assumption (ref) satisfy
the following conditions: $\sup_{n\geq1}\mathbb{E}\left[\left\Vert \bs{\xi}_{n}^{*}(\bs X_{i})\right\Vert ^{4}\right]<\infty$,
and for each $k=1,\ldots,K$,
\[
\left\Vert \frac{1}{n_{1[k]}}\sum_{i\in[k]}A_{i}\left\{ \bs{\xi}_{n}(\bs X_{i})-\bs{\xi}_{n}^{*}(\bs X_{i})\right\} -\frac{1}{n_{0[k]}}\sum_{i\in[k]}\left(1-A_{i}\right)\left\{ \bs{\xi}_{n}(\bs X_{i})-\bs{\xi}_{n}^{*}(\bs X_{i})\right\} \right\Vert =o_{P}(n^{-1}),
\]
and $\frac{1}{n_{[k]}}\sum_{i\in[k]}\left\Vert \bs{\xi}_{n}(\bs X_{i})-\bs{\xi}_{n}^{*}(\bs X_{i})\right\Vert ^{2}=O_{P}(\Delta_{n}^{2})$\textup{,}
where $\Delta_{n}\to0$ as $n\to\infty$ is a sequence of real numbers.
\end{enumerate}
\end{assumption}
We have the following theorem.
\begin{thm}
Suppose
that Assumptions (ref)--(ref)
and (ref) hold. If $\sup_{n\geq1}\sup_{1\leq i\leq n}\mathbb{E}\left[\left\Vert \bs{\xi}_{n}(\bs X_{i})\right\Vert ^{2+\epsilon}\right]<\infty$
for some $\epsilon>0$ and $\mathbb{E}\left[\widetilde{\bs{\xi}}_{n}^{*}(\bs X_{i})\widetilde{\bs{\xi}}_{n}^{*}(\bs X_{i})^{\top}\mid B_{i}=k\right]$
is non-singular for every $k=1,\ldots,K$, then the conclusion of
Theorem (ref) holds.
If, in addition, Assumption (ref)
holds and $\mathbb{E}\left[Y_{i}(a)^{4}\right]<\infty$ for all $a=0,1$,
then
\[
\widehat{\tau}_{\mathrm{cal}}-\tau=\frac{1}{n}\sum_{i=1}^{n}\psi_{1,i}+\frac{1}{n}\psi_{2}+o_{P}(n^{-1})+O_{P}(\Delta_{n}n^{-1/2}),
\]
where $\psi_{1,i}=\sum_{k=1}^{K}\left\{ \left(\frac{A_{i}}{\pi_{n[k]}}-\frac{1-A_{i}}{1-\pi_{n[k]}}\right)\1(B_{i}=k)\cdot Y_{i}-\tau-\bs{\beta}_{[k],\mathcal{C}_{n}}^{\top}\bs{\Xi}_{i,[k]}^{*}\right\} $
with $\mathbb{E}\left[\frac{1}{n}\sum_{i=1}^{n}\psi_{1,i}\right]=0$ and $\mathbb{E}\left[\psi_{2}\right]=\left\{ \frac{\rho^{\prime\prime\prime}(0)}{2\rho^{\prime\prime}(0)^{2}}-1\right\} \sum_{k=1}^{K}\mathrm{tr}\left\{ \mathbb{E}\left[\left(\mathbf{\Sigma}_{[k]}^{\mathcal{C}_{n}}\right)^{-1}\mathbf{\Sigma}_{\bs{\Xi}\bs{\Xi}\epsilon[k]}^{\mathcal{C}_{n}}\right]\right\} $\textup{.}
The definitions of $\bs{\beta}_{[k],\mathcal{C}_{n}}^{\top}\bs{\Xi}_{i,[k]}^{*}$
and $\left(\mathbf{\Sigma}_{[k]}^{\mathcal{C}_{n}}\right)^{-1}\mathbf{\Sigma}_{\bs{\Xi}\bs{\Xi}\epsilon[k]}^{\mathcal{C}_{n}}$
are provided in Appendix (ref).
\end{thm}
The first conclusion of Theorem (ref)
implies that, provided that Assumption (ref)
holds, different choices of $D(v)$ lead to calibration estimators
with the same asymptotic distribution. We also note that this conclusion
requires the non-singularity of $\mathbb{E}\left[\widetilde{\bs{\xi}}_{n}^{*}(\bs X_{i})\widetilde{\bs{\xi}}_{n}^{*}(\bs X_{i})^{\top}\mid B_{i}=k\right]$
in order to ensure that ((ref)) admits a unique
solution asymptotically. In the special case where $D(v)=(v-1)^{2}/2$,
such a condition is not necessary. This is because, under the quadratic
discrepancy, the weights $\widehat{w}_{i},i=1,\ldots,n$, admit a
closed-form expression. Even if (ref) admits
infinitely many solutions, one can always select the solution obtained
via the Moore-Penrose inverse (see ((ref)) in
the proof of Theorem (ref)
in Appendix (ref)). In contrast, when $D(v)$
is a general discrepancy measure, the weights $\widehat{w}_{i}$ no
longer have an explicit form. In this case, if ((ref))
admits infinitely many solutions, it is unclear which solution an
optimization algorithm would converge to. In practice, a regularization
term could be introduced to enforce the uniqueness of the solution,
although this is beyond the scope of the present paper. Additionally,
if $\mathbb{E}\left[\widetilde{\bs{\xi}}_{n}^{*}(\bs X_{i})\widetilde{\bs{\xi}}_{n}^{*}(\bs X_{i})^{\top}\mid B_{i}=k\right]$
is singular, one could apply singular value decomposition (SVD) to
eliminate the redundant components of $\bs{\xi}_{n}$ and use the
reduced version of $\bs{\xi}_{n}$ to compute the calibration estimator
((ref)). We leave the rigorous theoretical development
of this strategy for future investigation.
The second conclusion of Theorem (ref)
provides a characterization of the second-order bias under the CAR
design, which, to the best of our knowledge, is a novel contribution
to the literature. Unlike existing studies on second-order bias (newey2004Higher,tan2014Secondorder),
the observed samples under the CAR design are not i.i.d. The proof
of this result relies on a conditional argument: we first examine
the asymptotic expansion of the calibration estimator conditional
on the treatment and stratum indicators, and then apply the law of
iterated expectation. From Theorem (ref),
we see that when $\Delta_{n}=o(n^{-1/2})$, taking $D(v)=v-\log v$
gives the calibration estimator with zero second-order bias $\mathbb{E}\left[\psi_{2}\right]$.
This aligns with the findings in i.i.d. settings, where empirical
likelihood-based estimators exhibit smaller second-order bias (newey2004Higher,tan2014Secondorder).
\section{Simulation studies}
We evaluate the performance of our calibration estimators using Monte
Carlo simulations. For $a\in\{0,1\}$ and $1\leq i\leq n$, the potential
outcomes are generated as follows:
\[
Y_{i}(a)=g_{a}(\bs X_{i})+\epsilon_{a,i}\ \ i=1,\ldots,n,\ a\in\{0,1\},
\]
where $\bs X_{i}$, $\epsilon_{a,i}$ and $g_{a}(\cdot)$ will be
specified in each model and the triplet $(\bs X_{i},\epsilon_{0,i},\epsilon_{1,i})$
for $1\leq i\leq n$ is i.i.d.
We present simulation results for the estimators under three randomization
methods: simple randomization, stratified block randomization, and
minimization (pocock1975Sequential). The sample size $n$
varies across 500, 1000, and 2000, and the number of covariates is
set to be $p=30$. For stratified block randomization, we use a block
size of 6. In the minimization method, a biased-coin probability of
0.75 and equal weights are employed. The cross-fitting technique is
applied with two folds as demonstrated in Section (ref).
Unless otherwise specified, the calibration estimator is obtained
by setting $D(v)=(v-1)^{2}/2$. We compare nine estimators: \textbf{(i)}
\texttt{cal_rf}: For each stratum $k$, we use random forest to estimate
the conditional mean function $g_{0}(\cdot)$ and $g_{1}(\cdot)$,
denoting their estimates as $\widehat{g}_{0k}^{\text{rf}}(\cdot)$
and $\widehat{g}_{1k}^{\text{rf}}(\cdot)$. Then \texttt{cal_rf }is
obtained by taking $\bs{\xi}_{n}(\bs X_{i})=(\sum_{k=1}^{K}\widehat{g}_{0k}^{\text{rf}}(\bs X_{i})\1(B_{i}=k),\sum_{k=1}^{K}\widehat{g}_{1k}^{\text{rf}}(\bs X_{i})\1(B_{i}=k))^{\top}$
in our calibration estimator ((ref)); \textbf{(ii)}
\texttt{cal_nn}: Defined similarly to \texttt{cal_rf}, but with
the random forest estimates replaced by neural network estimates $\widehat{g}_{0k}^{\text{rf}}(\cdot)$
and $\widehat{g}_{1k}^{\text{rf}}(\cdot)$; \textbf{(iii)} \texttt{cal_rfnn}:
This estimator is obtained by combining the random forest and neural
network estimates. It is obtained by taking $\bs{\xi}_{n}(\bs X_{i})=(\sum_{k=1}^{K}\widehat{g}_{0k}^{\sharp}(\bs X_{i})\1(B_{i}=k),\sum_{k=1}^{K}\widehat{g}_{1k}^{\sharp}(\bs X_{i})\1(B_{i}=k):\sharp\in\{\text{rf},\text{nn}\})^{\top}$
in our calibration estimator ((ref)); \textbf{(iv)}
\texttt{cal_rflin}: Defined similarly to \texttt{cal_rfnn}, but
obtained by combining the random forest and linear regression estimates.
\textbf{(v)}\texttt{ cal_rf_g}: Take $\bs{\xi}_{n}(\bs X_{i})=(\widehat{g}_{0k}^{\text{rf}}(\bs X_{i}),\widehat{g}_{1k}^{\text{rf}}(\bs X_{i}):1\leq k\leq K)^{\top}$
and use it in our calibration estimator ((ref));
\textbf{(vi)} \texttt{cal_nn_g}: Defined similarly to \texttt{cal_rf_g},
but with neural network estimates; \textbf{(vii)} \texttt{cal_lin_EL}:
Defined similarly to \texttt{cal_rf}, but replacing the random forest
estimates with linear regression estimates and taking $D(v)=v-\log v$;\textbf{
(viii)} \texttt{aipw_rf}: We use AIPW-base method (tu2024Unified)
with the random forest estimates $\widehat{g}_{0k}^{\text{rf}}(\bs X_{i})$
and $\widehat{g}_{1k}^{\text{rf}}(\bs X_{i})$ to adjust the covariates;
\textbf{(ix)} \texttt{aipw_nn}: Defined similarly to \texttt{aipw_rf},
but replacing the random forest estimates with neural network estimates
$\widehat{g}_{0k}^{\text{nn}}(\cdot)$ and $\widehat{g}_{1k}^{\text{nn}}(\cdot)$;
\textbf{(x)} \texttt{aipw_lin}: Defined similarly to \texttt{aipw_rf},
but replacing the random forest estimates with linear regression estimates;
\textbf{(xi)} \texttt{sdim}: The stratified difference-in-means estimator
$\widehat{\tau}_{\mathrm{sdim}}$. For each estimator, we report the
following metrics based on 300 replications: absolute bias, empirical
standard deviation (SD), average estimated standard error (SE), and
the empirical coverage probability (CP) of the 95% confidence intervals.
\textbf{Model 1.} Model 1 imposes linear models for the conditional
mean functions $g_{0}(\bs X)$ and $g_{1}(\bs X)$. In this model,
we set
\begin{align*}
g_{0}(\bs X_{i}) & =\mu_{0}+\sum_{j=1}^{4}\beta_{0j}X_{ij}\ \text{ and }\ g_{1}(\bs X_{i})=\mu_{1}+\sum_{j=1}^{4}\beta_{1j}X_{ij},
\end{align*}
with $\mu_{0}=1$, $\mu_{1}=4$, $(\beta_{01},\ldots,\beta_{04})=(75,35,125,80)$,
and $(\beta_{11},\ldots,\beta_{14})=(100,80,60,40)$. Additionally,
the variables are specified as follows: $\epsilon_{0,i}\sim N(0,1)$,
$\epsilon_{1,i}\sim N(0,9)$, $X_{i1}\sim\text{Beta}(3,4)$, $X_{i2}\sim\text{Uniform}(-2,2)$,
$X_{i3}$ takes values in $\{-1,1\}$ with equal probability, $X_{i4}$
takes values in $\{3,5\}$ with probabilities 0.6 and 0.4, respectively.
These variables are independent of one another. The remaining variables
$X_{i5},\dots,X_{ip}$ are independent of $X_{i1},\dots,X_{i4}$ and
follow a multivariate normal distribution with zero mean and a covariance
matrix where all off-diagonal elements are 0.2, while the diagonal
elements are 1. The randomization variable is an additional variable
taking values in $\{1,2,3,4\}$ with probabilities 0.2, 0.3, 0.3,
and 0.2, respectively, and is independent of $X_{ij}$ for $j=1,\dots,p$.
The simulation results for Model 1 are presented in Table (ref).
Since both $g_{0}(\bs X_{i})$ and $g_{1}(\bs X_{i})$ are linear
in the covariates $X_{ij}$, the linear regression-adjusted estimator,
\texttt{aipw_lin}, performs optimally in large samples ($n=1000,2000$),
as expected. The performance of \texttt{cal_rflin} is very close
to that of \texttt{aipw_lin}, which aligns with the conclusion of
Theorem (ref). However, in smaller
samples ($n=500$), the \texttt{aipw_lin} estimator does not perform
the best, as linear regression is sensitive to outliers. In contrast,
\texttt{cal_rflin} maintains stable performance, suggesting that
incorporating different estimates of $g_{0}(\bs X_{i})$ and $g_{1}(\bs X_{i})$
into the calibration estimator enhances its robustness.
\begin{table}[!tbh]
\caption{The comparison of the performance of different
estimators under Model 1.}
\resizebox{\textwidth}{!}{
\begin{threeparttable}
\begin{centering}
\begin{tabular}{clrrrrrrrrrrrr}
\toprule
\multirow{2}{*}{$n$} & \multirow{2}{*}{Estimator} & \multicolumn{4}{c}{Simple Rand.} & \multicolumn{4}{c}{Stratified Block Rand.} & \multicolumn{4}{c}{Minimization}\tabularnewline
\cmidrule{3-14}
& & Bias & SD & SE & CP & Bias & SD & SE & CP & Bias & SD & SE & CP\tabularnewline
\midrule
\multirow{11}{*}{500} & \texttt{cal_rf} & 0.12 & 6.85 & 7.39 & 0.973 & 0.12 & 6.74 & 7.31 & 0.960 & 0.36 & 6.98 & 7.33 & 0.953\tabularnewline
& \texttt{cal_nn} & 0.33 & 11.38 & 11.03 & 0.960 & 0.65 & 11.01 & 11.05 & 0.950 & 0.63 & 11.47 & 11.00 & 0.933\tabularnewline
& \texttt{cal_rfnn} & 0.47 & 6.90 & 7.33 & 0.960 & 0.18 & 6.90 & 7.24 & 0.960 & 0.12 & 7.03 & 7.27 & 0.950\tabularnewline
& \texttt{cal_rflin} & 0.08 & 4.36 & 5.23 & 0.983 & 0.35 & 4.78 & 5.19 & 0.967 & 0.37 & 4.60 & 5.19 & 0.973\tabularnewline
& \texttt{cal_rf_g} & 0.13 & 5.83 & 6.08 & 0.967 & 0.48 & 6.01 & 6.07 & 0.960 & 0.01 & 5.68 & 6.09 & 0.960\tabularnewline
& \texttt{cal_nn_g} & 0.48 & 9.23 & 9.10 & 0.960 & 0.79 & 9.41 & 9.15 & 0.943 & 0.31 & 9.31 & 9.10 & 0.950\tabularnewline
& \texttt{cal_lin_EL} & 0.10 & 4.25 & 5.26 & 0.980 & 0.32 & 4.49 & 5.21 & 0.983 & 0.14 & 4.39 & 5.22 & 0.987\tabularnewline
& \texttt{aipw_rf} & 0.31 & 9.03 & 8.91 & 0.957 & 0.07 & 8.74 & 8.88 & 0.940 & 0.65 & 9.13 & 8.88 & 0.943\tabularnewline
& \texttt{aipw_nn} & 0.29 & 14.17 & 13.55 & 0.923 & 0.74 & 14.45 & 13.54 & 0.910 & 1.22 & 13.98 & 13.52 & 0.950\tabularnewline
& \texttt{aipw_lin} & 3.22 & 50.59 & 14.23 & 0.953 & 0.69 & 25.12 & 9.04 & 0.967 & 1.78 & 19.45 & 7.30 & 0.953\tabularnewline
& \texttt{sdim} & 0.48 & 12.35 & 12.34 & 0.953 & 0.06 & 11.71 & 12.31 & 0.943 & 0.93 & 12.61 & 12.28 & 0.927\tabularnewline
\midrule
\multirow{11}{*}{1000} & \texttt{cal_rf} & 0.21 & 4.00 & 4.13 & 0.957 & 0.06 & 3.70 & 4.12 & 0.960 & 0.09 & 3.85 & 4.12 & 0.963\tabularnewline
& \texttt{cal_nn} & 0.10 & 6.39 & 6.48 & 0.963 & 0.15 & 6.42 & 6.48 & 0.940 & 0.38 & 6.14 & 6.44 & 0.960\tabularnewline
& \texttt{cal_rfnn} & 0.05 & 3.93 & 4.06 & 0.963 & 0.06 & 3.78 & 4.05 & 0.960 & 0.17 & 3.90 & 4.05 & 0.973\tabularnewline
& \texttt{cal_rflin} & 0.17 & 2.94 & 3.28 & 0.973 & 0.24 & 2.84 & 3.27 & 0.973 & 0.05 & 3.14 & 3.27 & 0.957\tabularnewline
& \texttt{cal_rf_g} & 0.24 & 3.68 & 3.66 & 0.940 & 0.05 & 3.45 & 3.67 & 0.963 & 0.08 & 3.67 & 3.66 & 0.950\tabularnewline
& \texttt{cal_nn_g} & 0.40 & 4.48 & 4.73 & 0.967 & 0.18 & 4.83 & 4.76 & 0.950 & 0.55 & 4.74 & 4.74 & 0.950\tabularnewline
& \texttt{cal_lin_EL} & 0.12 & 2.87 & 3.27 & 0.973 & 0.29 & 2.76 & 3.27 & 0.977 & 0.05 & 3.06 & 3.27 & 0.970\tabularnewline
& \texttt{aipw_rf} & 0.07 & 5.11 & 5.24 & 0.967 & 0.04 & 5.25 & 5.23 & 0.947 & 0.16 & 5.27 & 5.24 & 0.950\tabularnewline
& \texttt{aipw_nn} & 0.17 & 7.96 & 7.51 & 0.943 & 0.26 & 7.52 & 7.53 & 0.953 & 0.36 & 7.38 & 7.44 & 0.973\tabularnewline
& \texttt{aipw_lin} & 0.08 & 2.81 & 2.90 & 0.943 & 0.32 & 2.74 & 2.91 & 0.960 & 0.05 & 3.03 & 2.91 & 0.940\tabularnewline
& \texttt{sdim} & 0.11 & 8.27 & 8.71 & 0.953 & 0.10 & 8.70 & 8.67 & 0.950 & 0.27 & 8.61 & 8.67 & 0.953\tabularnewline
\midrule
\multirow{11}{*}{2000} & \texttt{cal_rf} & 0.10 & 2.49 & 2.53 & 0.960 & 0.02 & 2.32 & 2.53 & 0.960 & 0.11 & 2.50 & 2.53 & 0.967\tabularnewline
& \texttt{cal_nn} & 0.27 & 2.50 & 2.68 & 0.960 & 0.01 & 2.62 & 2.68 & 0.963 & 0.05 & 2.64 & 2.67 & 0.950\tabularnewline
& \texttt{cal_rfnn} & 0.17 & 2.23 & 2.33 & 0.960 & 0.04 & 2.17 & 2.33 & 0.963 & 0.04 & 2.32 & 2.33 & 0.953\tabularnewline
& \texttt{cal_rflin} & 0.12 & 2.04 & 2.19 & 0.967 & 0.04 & 2.06 & 2.19 & 0.973 & 0.02 & 2.11 & 2.19 & 0.970\tabularnewline
& \texttt{cal_rf_g} & 0.13 & 2.37 & 2.38 & 0.950 & 0.07 & 2.22 & 2.38 & 0.957 & 0.14 & 2.33 & 2.38 & 0.953\tabularnewline
& \texttt{cal_nn_g} & 0.28 & 2.09 & 2.23 & 0.957 & 0.15 & 2.19 & 2.24 & 0.957 & 0.05 & 2.18 & 2.23 & 0.967\tabularnewline
& \texttt{cal_lin_EL} & 0.16 & 2.01 & 2.19 & 0.970 & 0.00 & 2.03 & 2.19 & 0.970 & 0.02 & 2.08 & 2.19 & 0.973\tabularnewline
& \texttt{aipw_rf} & 0.12 & 3.04 & 3.05 & 0.957 & 0.15 & 3.00 & 3.05 & 0.953 & 0.20 & 3.10 & 3.05 & 0.947\tabularnewline
& \texttt{aipw_nn} & 0.29 & 2.77 & 2.91 & 0.967 & 0.04 & 2.93 & 2.89 & 0.927 & 0.06 & 2.81 & 2.88 & 0.953\tabularnewline
& \texttt{aipw_lin} & 0.16 & 1.99 & 2.06 & 0.967 & 0.00 & 2.02 & 2.06 & 0.957 & 0.03 & 2.06 & 2.05 & 0.963\tabularnewline
& \texttt{sdim} & 0.22 & 5.87 & 6.13 & 0.967 & 0.41 & 6.28 & 6.13 & 0.940 & 0.37 & 6.12 & 6.13 & 0.953\tabularnewline
\bottomrule
\end{tabular}
\end{centering}
\begin{tablenotes}[flushleft]
• \textit{Abbreviations:} Rand., Randomization; SD, standard deviation, SE: standard error; CP, coverage probability.
\end{tablenotes}
\end{threeparttable}
}
\end{table}
\textbf{Model 2.} Model 2 imposes additive but nonlinear models for
the conditional mean functions $g_{0}(\bs X)$ and $g_{1}(\bs X)$.
In this model, we set
\begin{align*}
g_{0}(\bs X_{i}) & =\mu_{0}+\beta_{01}\log(X_{i1}+1)+\beta_{02}X_{i2}^{2}+\beta_{03}\exp(X_{i3})+\beta_{04}/(X_{i4}+3)\\
g_{1}(\bs X_{i}) & =\mu_{1}+\beta_{11}\exp(X_{i1}+2)+\beta_{12}/(X_{i1}+1)+\beta_{13}X_{i2}^{2},
\end{align*}
with $\mu_{0}=-3$, $\mu_{1}=0$, $(\beta_{01},\ldots,\beta_{04})=(10,24,15,20)$,
and $(\beta_{11},\beta_{12},\beta_{13})=(20,17,10)$. Additionally,
the variables are specified as follows: $\epsilon_{0,i}\sim N(0,1)$,
$\epsilon_{1,i}\sim N(0,9)$, $X_{i1}\sim\text{Beta}(3,4)$, $X_{i2}\sim\text{Uniform}(-2,2)$
with these two variables being independent of each other. The additional
covariates $X_{i3},\dots,X_{ip}$ are first generated as in Model
1. Then we randomly select $\left\lfloor p/3\right\rfloor $ covariates
from the additional covariates and multiply them by either $X_{i1}$
or $X_{i2}$ with equal probability to form the final additional covariates.
The randomization variable is an additional variable taking values
in $\{1,2,3,4\}$ with probabilities 0.2, 0.3, 0.3, and 0.2, respectively,
and is independent of $X_{ij}$ for $j=1,\dots,p$. The simulation
results for Model 2 are presented in Table (ref). The
results indicate that random forests-based calibration estimators
(\texttt{cal_rf}, \texttt{cal_rfnn}, \texttt{cal_rflm}, \texttt{cal_rf_g})
consistently outperform other methods across different randomization
schemes and sample sizes. The AIPW-based estimators (\texttt{aipw_rf}
and \texttt{aipw_nn}) tend to perform worse than the calibration
estimators (\texttt{cal_rf} and \texttt{cal_nn}), especially when
sample size is small ($n=500$). The empirical coverage probabilities
indicate that, in most cases, the estimators yield reliable 95% confidence
intervals.
\begin{table}[!tbh]
\caption{The comparison of the performance of different
estimators under Model 2.}
\resizebox{\textwidth}{!}{
\begin{threeparttable}
\begin{centering}
\begin{tabular}{clrrrrrrrrrrrr}
\toprule
\multirow{2}{*}{$n$} & \multirow{2}{*}{Estimator} & \multicolumn{4}{c}{Simple Rand.} & \multicolumn{4}{c}{Stratified Block Rand.} & \multicolumn{4}{c}{Minimization}\tabularnewline
\cmidrule{3-14}
& & Bias & SD & SE & CP & Bias & SD & SE & CP & Bias & SD & SE & CP\tabularnewline
\midrule
\multirow{11}{*}{500} & \texttt{cal_rf} & 0.01 & 2.09 & 2.30 & 0.980 & 0.19 & 2.22 & 2.30 & 0.957 & 0.28 & 2.25 & 2.29 & 0.967\tabularnewline
& \texttt{cal_nn} & 0.16 & 3.09 & 3.14 & 0.957 & 0.14 & 3.19 & 3.14 & 0.940 & 0.10 & 3.08 & 3.13 & 0.960\tabularnewline
& \texttt{cal_rfnn} & 0.01 & 2.14 & 2.30 & 0.977 & 0.20 & 2.25 & 2.30 & 0.960 & 0.25 & 2.26 & 2.29 & 0.957\tabularnewline
& \texttt{cal_rflin} & 0.22 & 2.11 & 2.26 & 0.977 & 0.01 & 2.23 & 2.26 & 0.960 & 0.11 & 2.23 & 2.26 & 0.960\tabularnewline
& \texttt{cal_rf_g} & 0.10 & 2.00 & 2.16 & 0.957 & 0.19 & 2.25 & 2.16 & 0.937 & 0.15 & 2.18 & 2.16 & 0.950\tabularnewline
& \texttt{cal_nn_g} & 0.12 & 3.23 & 3.09 & 0.943 & 0.19 & 3.21 & 3.09 & 0.937 & 0.05 & 3.13 & 3.08 & 0.957\tabularnewline
& \texttt{cal_lin_EL} & 0.33 & 2.68 & 2.78 & 0.950 & 0.19 & 2.73 & 2.77 & 0.960 & 0.10 & 2.69 & 2.76 & 0.963\tabularnewline
& \texttt{aipw_rf} & 0.20 & 2.35 & 2.42 & 0.950 & 0.04 & 2.50 & 2.42 & 0.940 & 0.09 & 2.46 & 2.42 & 0.957\tabularnewline
& \texttt{aipw_nn} & 0.87 & 5.98 & 5.99 & 0.963 & 0.47 & 6.02 & 5.92 & 0.957 & 0.25 & 5.94 & 5.87 & 0.937\tabularnewline
& \texttt{aipw_lin} & 2.91 & 196.74 & 43.13 & 0.953 & 3.67 & 142.28 & 37.24 & 0.963 & 6.10 & 95.39 & 33.45 & 0.937\tabularnewline
& \texttt{sdim} & 0.22 & 3.07 & 3.12 & 0.950 & 0.14 & 3.19 & 3.12 & 0.940 & 0.08 & 3.06 & 3.12 & 0.970\tabularnewline
\midrule
\multirow{11}{*}{1000} & \texttt{cal_rf} & 0.22 & 1.47 & 1.51 & 0.957 & 0.15 & 1.48 & 1.50 & 0.950 & 0.29 & 1.54 & 1.50 & 0.943\tabularnewline
& \texttt{cal_nn} & 0.04 & 2.24 & 2.15 & 0.960 & 0.09 & 2.16 & 2.13 & 0.963 & 0.27 & 2.15 & 2.14 & 0.950\tabularnewline
& \texttt{cal_rfnn} & 0.22 & 1.49 & 1.50 & 0.953 & 0.15 & 1.50 & 1.50 & 0.947 & 0.25 & 1.55 & 1.50 & 0.940\tabularnewline
& \texttt{cal_rflin} & 0.08 & 1.44 & 1.47 & 0.960 & 0.08 & 1.43 & 1.47 & 0.957 & 0.15 & 1.53 & 1.46 & 0.937\tabularnewline
& \texttt{cal_rf_g} & 0.17 & 1.46 & 1.45 & 0.940 & 0.12 & 1.46 & 1.45 & 0.947 & 0.25 & 1.50 & 1.45 & 0.937\tabularnewline
& \texttt{cal_nn_g} & 0.02 & 2.17 & 2.02 & 0.950 & 0.11 & 2.08 & 2.00 & 0.937 & 0.30 & 2.04 & 2.02 & 0.943\tabularnewline
& \texttt{cal_lin_EL} & 0.07 & 1.74 & 1.67 & 0.947 & 0.18 & 1.70 & 1.67 & 0.940 & 0.25 & 1.66 & 1.67 & 0.947\tabularnewline
& \texttt{aipw_rf} & 0.07 & 1.62 & 1.59 & 0.947 & 0.06 & 1.60 & 1.59 & 0.943 & 0.26 & 1.63 & 1.59 & 0.943\tabularnewline
& \texttt{aipw_nn} & 0.17 & 3.36 & 3.22 & 0.943 & 0.05 & 3.40 & 3.22 & 0.950 & 0.27 & 3.28 & 3.26 & 0.960\tabularnewline
& \texttt{aipw_lin} & 0.11 & 1.75 & 1.67 & 0.950 & 0.19 & 1.73 & 1.66 & 0.947 & 0.30 & 1.67 & 1.66 & 0.947\tabularnewline
& \texttt{sdim} & 0.01 & 2.25 & 2.20 & 0.950 & 0.07 & 2.27 & 2.20 & 0.933 & 0.31 & 2.20 & 2.20 & 0.960\tabularnewline
\midrule
\multirow{11}{*}{2000} & \texttt{cal_rf} & 0.09 & 1.00 & 1.01 & 0.943 & 0.05 & 1.04 & 1.01 & 0.937 & 0.09 & 0.97 & 1.01 & 0.953\tabularnewline
& \texttt{cal_nn} & 0.03 & 1.26 & 1.27 & 0.950 & 0.13 & 1.31 & 1.27 & 0.950 & 0.05 & 1.25 & 1.27 & 0.950\tabularnewline
& \texttt{cal_rfnn} & 0.07 & 1.01 & 1.01 & 0.943 & 0.07 & 1.04 & 1.01 & 0.923 & 0.07 & 0.97 & 1.01 & 0.953\tabularnewline
& \texttt{cal_rflin} & 0.01 & 1.00 & 0.99 & 0.943 & 0.14 & 1.02 & 0.99 & 0.923 & 0.00 & 0.96 & 0.99 & 0.957\tabularnewline
& \texttt{cal_rf_g} & 0.05 & 1.00 & 0.99 & 0.950 & 0.08 & 1.04 & 0.99 & 0.927 & 0.07 & 0.97 & 0.99 & 0.950\tabularnewline
& \texttt{cal_nn_g} & 0.03 & 1.10 & 1.07 & 0.957 & 0.13 & 1.08 & 1.07 & 0.930 & 0.04 & 1.08 & 1.07 & 0.953\tabularnewline
& \texttt{cal_lin_EL} & 0.01 & 1.15 & 1.13 & 0.943 & 0.12 & 1.14 & 1.13 & 0.943 & 0.03 & 1.13 & 1.13 & 0.963\tabularnewline
& \texttt{aipw_rf} & 0.01 & 1.07 & 1.06 & 0.947 & 0.12 & 1.09 & 1.05 & 0.933 & 0.05 & 1.06 & 1.06 & 0.960\tabularnewline
& \texttt{aipw_nn} & 0.06 & 1.38 & 1.44 & 0.957 & 0.13 & 1.57 & 1.43 & 0.923 & 0.12 & 1.49 & 1.43 & 0.940\tabularnewline
& \texttt{aipw_lin} & 0.00 & 1.14 & 1.12 & 0.930 & 0.11 & 1.14 & 1.12 & 0.937 & 0.02 & 1.11 & 1.12 & 0.960\tabularnewline
& \texttt{sdim} & 0.03 & 1.58 & 1.55 & 0.943 & 0.15 & 1.58 & 1.55 & 0.930 & 0.12 & 1.64 & 1.55 & 0.933\tabularnewline
\bottomrule
\end{tabular}
\end{centering}
\begin{tablenotes}[flushleft]
• \textit{Abbreviations:} Rand., Randomization; SD, standard deviation, SE: standard error; CP, coverage probability.
\end{tablenotes}
\end{threeparttable}
}
\end{table}
\textbf{Model 3.} Model 3 imposes non-additive and nonlinear models
for the conditional mean functions $g_{0}(\bs X)$ and $g_{1}(\bs X)$.
In this model, we set
\begin{align*}
g_{0}(\bs X_{i}) & =\mu_{0}+\beta_{01}X_{i1}X_{i2}/(X_{i1}+X_{i2}+2)+\beta_{02}X_{i1}^{2}(X_{i2}+X_{i3})\\
g_{1}(\bs X_{i}) & =\mu_{1}+\beta_{11}(X_{i2}+X_{i4})+\beta_{12}X_{i2}^{2}/\exp(X_{i1}+2),
\end{align*}
with $\mu_{0}=5$, $\mu_{1}=2$, $(\beta_{01},\beta_{02})=(42,83)$,
and $(\beta_{11},\beta_{12})=(30,75)$. Additionally, the variables
are specified as follows: $\epsilon_{0,i}\sim\text{t}(2)$, $\epsilon_{1,i}\sim3\times\text{t}(2)$,
$X_{i1}\sim\text{Beta}(3,4)$, $X_{i2}\sim\text{Uniform}(-2,2)$,
$X_{i3}\sim N(0,1)$, $X_{i4}\sim\text{Uniform}(0,2)$, respectively.
These variables are independent of one another. The remaining variables
$X_{i5},\dots,X_{ip}$ are independent of $X_{i1},\dots,X_{i4}$ and
follow a multivariate normal distribution with zero mean and a symmetric
Toeplitz covariance matrix where the first row is a geometric sequence
with initial value 1 and common ratio 0.5. The randomization variable
is an additional variable taking values in $\{1,2\}$ with probabilities
0.4, and 0.6, respectively, and is independent of $X_{ij}$ for $j=1,\dots,p$.
The simulation results for Model 3 are presented in Table (ref).
The results indicate that the random forests-based calibration estimators
(\texttt{cal_rf}, \texttt{cal_rfnn}, \texttt{cal_rflm}, \texttt{cal_rf_g})
perform the best across different randomization methods and sample
sizes. As the sample size increases, the calibration estimators converge
toward better performance with lower SD, while maintaining correct
empirical coverage probabilities. However, the \texttt{sdim} estimator
consistently lags behind the calibration estimators in terms of SD,
especially in smaller sample sizes. This finding is consistent with
Theorem (ref), which demonstrates
that the calibration estimator is always more efficient than the \texttt{sdim}
estimator.
\begin{table}[!tbh]
\caption{The comparison of the performance of different
estimators under Model 3.}
\resizebox{\textwidth}{!}{
\begin{threeparttable}
\begin{centering}
\begin{tabular}{clrrrrrrrrrrrr}
\toprule
\multirow{2}{*}{$n$} & \multirow{2}{*}{Estimator} & \multicolumn{4}{c}{Simple Rand.} & \multicolumn{4}{c}{Stratified Block Rand.} & \multicolumn{4}{c}{Minimization}\tabularnewline
\cmidrule{3-14}
& & Bias & SD & SE & CP & Bias & SD & SE & CP & Bias & SD & SE & CP\tabularnewline
\midrule
\multirow{11}{*}{500} & \texttt{cal_rf} & 0.13 & 2.80 & 2.68 & 0.933 & 0.01 & 2.40 & 2.61 & 0.980 & 0.03 & 2.41 & 2.65 & 0.967\tabularnewline
& \texttt{cal_nn} & 0.17 & 3.62 & 3.43 & 0.943 & 0.08 & 3.20 & 3.35 & 0.960 & 0.08 & 3.29 & 3.38 & 0.960\tabularnewline
& \texttt{cal_rfnn} & 0.09 & 2.83 & 2.67 & 0.940 & 0.00 & 2.45 & 2.61 & 0.967 & 0.01 & 2.41 & 2.65 & 0.960\tabularnewline
& \texttt{cal_rflin} & 0.08 & 2.80 & 2.63 & 0.930 & 0.01 & 2.36 & 2.56 & 0.967 & 0.02 & 2.42 & 2.61 & 0.957\tabularnewline
& \texttt{cal_rf_g} & 0.16 & 2.74 & 2.58 & 0.927 & 0.01 & 2.27 & 2.52 & 0.980 & 0.11 & 2.35 & 2.56 & 0.957\tabularnewline
& \texttt{cal_nn_g} & 0.08 & 3.23 & 3.16 & 0.933 & 0.08 & 2.94 & 3.08 & 0.953 & 0.08 & 2.93 & 3.12 & 0.960\tabularnewline
& \texttt{cal_lin_EL} & 0.09 & 3.00 & 2.82 & 0.923 & 0.10 & 2.51 & 2.76 & 0.970 & 0.03 & 2.66 & 2.80 & 0.967\tabularnewline
& \texttt{aipw_rf} & 0.04 & 3.08 & 2.84 & 0.937 & 0.16 & 2.60 & 2.78 & 0.960 & 0.16 & 2.67 & 2.82 & 0.953\tabularnewline
& \texttt{aipw_nn} & 0.11 & 5.09 & 4.88 & 0.943 & 0.12 & 4.72 & 4.62 & 0.947 & 0.23 & 4.93 & 4.79 & 0.947\tabularnewline
& \texttt{aipw_lin} & 0.13 & 3.16 & 3.01 & 0.937 & 0.12 & 2.79 & 2.89 & 0.930 & 0.02 & 2.83 & 2.95 & 0.977\tabularnewline
& \texttt{sdim} & 0.19 & 4.31 & 4.00 & 0.930 & 0.23 & 3.77 & 3.93 & 0.950 & 0.26 & 3.87 & 3.97 & 0.960\tabularnewline
\midrule
\multirow{11}{*}{1000} & \texttt{cal_rf} & 0.03 & 1.87 & 1.79 & 0.947 & 0.06 & 1.82 & 1.80 & 0.940 & 0.07 & 1.66 & 1.78 & 0.957\tabularnewline
& \texttt{cal_nn} & 0.09 & 2.31 & 2.18 & 0.937 & 0.02 & 2.15 & 2.20 & 0.947 & 0.02 & 2.20 & 2.18 & 0.960\tabularnewline
& \texttt{cal_rfnn} & 0.06 & 1.89 & 1.79 & 0.937 & 0.08 & 1.82 & 1.79 & 0.953 & 0.08 & 1.68 & 1.78 & 0.960\tabularnewline
& \texttt{cal_rflin} & 0.06 & 1.88 & 1.76 & 0.943 & 0.05 & 1.82 & 1.77 & 0.943 & 0.08 & 1.69 & 1.75 & 0.953\tabularnewline
& \texttt{cal_rf_g} & 0.01 & 1.87 & 1.75 & 0.933 & 0.06 & 1.76 & 1.75 & 0.957 & 0.11 & 1.67 & 1.74 & 0.953\tabularnewline
& \texttt{cal_nn_g} & 0.04 & 2.12 & 2.01 & 0.950 & 0.04 & 1.94 & 2.02 & 0.963 & 0.15 & 2.03 & 2.01 & 0.963\tabularnewline
& \texttt{cal_lin_EL} & 0.02 & 1.96 & 1.82 & 0.943 & 0.05 & 1.87 & 1.82 & 0.953 & 0.04 & 1.76 & 1.81 & 0.967\tabularnewline
& \texttt{aipw_rf} & 0.00 & 2.04 & 1.87 & 0.943 & 0.01 & 1.91 & 1.88 & 0.947 & 0.01 & 1.81 & 1.86 & 0.957\tabularnewline
& \texttt{aipw_nn} & 0.20 & 2.98 & 2.66 & 0.920 & 0.01 & 2.97 & 2.70 & 0.950 & 0.05 & 2.76 & 2.66 & 0.950\tabularnewline
& \texttt{aipw_lin} & 0.00 & 1.98 & 1.83 & 0.940 & 0.08 & 1.89 & 1.83 & 0.947 & 0.04 & 1.79 & 1.82 & 0.953\tabularnewline
& \texttt{sdim} & 0.07 & 2.69 & 2.81 & 0.967 & 0.05 & 2.77 & 2.81 & 0.950 & 0.00 & 2.77 & 2.80 & 0.967\tabularnewline
\midrule
\multirow{11}{*}{2000} & \texttt{cal_rf} & 0.10 & 1.31 & 1.22 & 0.943 & 0.01 & 1.20 & 1.21 & 0.947 & 0.06 & 1.41 & 1.24 & 0.937\tabularnewline
& \texttt{cal_nn} & 0.08 & 1.42 & 1.36 & 0.947 & 0.08 & 1.37 & 1.36 & 0.957 & 0.11 & 1.59 & 1.39 & 0.943\tabularnewline
& \texttt{cal_rfnn} & 0.10 & 1.29 & 1.21 & 0.943 & 0.03 & 1.21 & 1.21 & 0.947 & 0.09 & 1.43 & 1.24 & 0.940\tabularnewline
& \texttt{cal_rflin} & 0.10 & 1.29 & 1.20 & 0.940 & 0.01 & 1.20 & 1.20 & 0.943 & 0.06 & 1.41 & 1.23 & 0.940\tabularnewline
& \texttt{cal_rf_g} & 0.12 & 1.28 & 1.19 & 0.950 & 0.03 & 1.16 & 1.19 & 0.950 & 0.08 & 1.40 & 1.22 & 0.943\tabularnewline
& \texttt{cal_nn_g} & 0.10 & 1.34 & 1.28 & 0.940 & 0.07 & 1.30 & 1.28 & 0.953 & 0.13 & 1.55 & 1.31 & 0.940\tabularnewline
& \texttt{cal_lin_EL} & 0.07 & 1.34 & 1.24 & 0.930 & 0.04 & 1.24 & 1.23 & 0.957 & 0.02 & 1.46 & 1.26 & 0.953\tabularnewline
& \texttt{aipw_rf} & 0.05 & 1.36 & 1.25 & 0.930 & 0.03 & 1.26 & 1.25 & 0.950 & 0.01 & 1.46 & 1.27 & 0.940\tabularnewline
& \texttt{aipw_nn} & 0.08 & 1.54 & 1.45 & 0.930 & 0.05 & 1.48 & 1.45 & 0.963 & 0.06 & 1.77 & 1.49 & 0.933\tabularnewline
& \texttt{aipw_lin} & 0.07 & 1.31 & 1.23 & 0.933 & 0.03 & 1.25 & 1.23 & 0.953 & 0.04 & 1.57 & 1.26 & 0.930\tabularnewline
& \texttt{sdim} & 0.07 & 2.14 & 1.98 & 0.927 & 0.05 & 2.06 & 1.98 & 0.943 & 0.01 & 2.17 & 2.00 & 0.947\tabularnewline
\bottomrule
\end{tabular}
\end{centering}
\begin{tablenotes}[flushleft]
• \textit{Abbreviations:} Rand., Randomization; SD, standard deviation, SE: standard error; CP, coverage probability.
\end{tablenotes}
\end{threeparttable}
}
\end{table}
Models 1-3 assume the homogeneity of the conditional mean functions
$g_{0}(\bs X)$ and $g_{1}(\bs X)$ across different strata. In Appendix (ref),
we also examine a model that accounts for the heterogeneity of the
conditional mean functions $g_{0}(\bs X)$ and $g_{1}(\bs X)$ across
different strata. Our findings show that the results are very similar
to those obtained under Models 1--3.
\section{Empirical application}
In this section, we apply our calibration method to the experimental
data from dupas2018Bankinga, which conducted covariate-adaptive
randomized experiments to assess the impact of subsidized bank account
access ($A_{i}$) on total savings ($Y_{i}$) for individuals across
three countries: Uganda, Malawi, and Chile. dupas2018Bankinga
estimated both the average treatment effects (ATEs) and the quantile
treatment effects (QTEs) of the subsidy. More recently, jiang2023Regressionadjusted
applied a regression-adjusted estimator to this dataset to analyze
the QTE in Uganda.
In this section, we aim to estimate the ATEs of subsidized access
to bank accounts in Uganda and Malawi. The Uganda sample includes
2,159 observations, stratified into 41 strata based on gender, occupation,
and bank branch, following a stratified block randomization design.
The Malawi sample includes 2,108 observations, stratified into 78
strata based on occupation, gender, marital status, literacy, and
whether the respondent was from the household or market, also following
a stratified block randomization design. To avoid overly small strata,
we exclude those containing fewer than six samples. This results in
a final Uganda sample of 2,115 observations across 37 strata, and
a Malawi sample of 1,987 observations across 67 strata. In both countries,
half of the households were randomly assigned to receive the bank
account subsidy, while the other half served as the control group.
All monetary variables are winsorized at the ninety-ninth percentile
to mitigate the influence of outliers. After the randomization and
the intervention, dupas2018Bankinga conducted 3 rounds of
follow-up surveys in Uganda and Malawi. In line with jiang2023Regressionadjusted,
we focus on the first-round follow-up survey to assess the impact
of the bank account subsidy on total savings.
Following dupas2018Bankinga, we consider one baseline covariate
$X$: the baseline value of total savings. When estimating the ATE
of subsidized bank account access in Uganda, we leverage data from
Malawi to obtain certain components of $\bs{\xi}_{n}(X)$ used in
our calibration estimator, and vice versa when estimating the ATE
for Malawi. For both countries, we consider the following estimators:
(i) \texttt{cal_X}: this estimator is obtained by taking $\bs{\xi}_{n}(X)=X$
in ((ref)); (ii) \texttt{cal_X}$^{\beta}$: this
estimator is obtained by taking $\bs{\xi}_{n}(X)=(X+1)^{\beta}$ in
((ref)); (iii) \texttt{cal_X_X}$^{\beta}$: this
estimator is obtained by taking $\bs{\xi}_{n}(X)=(X,(X+1)^{\beta})^{\top}$
in ((ref)); (iv) \texttt{cal_info_X}: this estimator
is obtained by taking $\bs{\xi}_{n}(X)=(X,\widehat{g}_{\text{rf}}^{\text{info}}(X))^{\top}$
in ((ref)), where $\widehat{g}_{\text{rf}}^{\text{info}}(X)$
is the random forest estimator of $\mathbb{E}\left[Y\mid X\right]$ using
the data from the other country; (v) \texttt{cal_info_X}$^{\beta}$:
this estimator is obtained by taking $\bs{\xi}_{n}(X)=((X+1)^{\beta},\widehat{g}_{\text{rf}}^{\text{info}}(X))^{\top}$
in ((ref)); (vi) \texttt{cal_info_X_X}$^{\beta}$:
this estimator is obtained by taking $\bs{\xi}_{n}(X)=(X,(X+1)^{\beta},\widehat{g}_{\text{rf}}^{\text{info}}(X))^{\top}$
in ((ref)); (vii) \texttt{sdim}: the stratified
difference-in-means estimator $\widehat{\tau}_{\mathrm{sdim}}$. We
include the term $(X+1)^{\beta}$ because an approximately linear
relationship is observed between $\log(Y+1)$ and $\log(X+1)$ in
both countries. We fit linear regressions of the form $\log(Y+1)=\alpha+\beta\log(X+1)+\epsilon$,
yielding an estimated $\beta$ of 0.481 for Uganda and 0.408 for Malawi.
Figure (ref) and Table (ref)
present the 95% confidence intervals and point estimates obtained
using these methods.
\begin{figure}[!tbh]
\begin{centering}
\end{centering}
\begin{centering}
\end{centering}
\caption{The 95% confidence intervals for the ATE
of the bank account subsidy on total household savings in Uganda and
Malawi, respectively. Top panel: Uganda; bottom panel: Malawi. The
estimators whose names contain “\texttt{X}” (or “\texttt{X}$^{\beta}$”)
indicate that we add the component $X$ (or $(X+1)^{\beta}$) to $\protect\bs{\xi}(X)$
to obtain the calibration estimator. The estimators whose names contain “\texttt{info}” indicate that we incorporate information from the
other country to form one component of $\protect\bs{\xi}(X)$.}
\end{figure}
\begin{table}[!tbh]
\caption{The ATE estimates of the bank account
subsidy effect on total household savings in Uganda and Malawi, with
standard errors reported in parentheses.}
\resizebox{\textwidth}{!}{
\begin{threeparttable}
\begin{centering}
\begin{tabular}{cccccccc}
\toprule
& \texttt{sdim} & \texttt{cal_X} & \texttt{cal_X}$^{\beta}$ & \texttt{cal_X_X}$^{\beta}$ & \texttt{cal_info_X} & \texttt{cal_info_X}$^{\beta}$ & \texttt{cal_info_X_X}$^{\beta}$\tabularnewline
\midrule
\midrule
\multirow{2}{*}{Uganda} & 1.289 & 3.687 & 2.979 & 3.459 & 3.604 & 3.313 & 3.426\tabularnewline
& (2.980) & (2.726) & (2.691) & (2.661) & (2.722) & (2.697) & (2.645)\tabularnewline
\multirow{2}{*}{Malawi} & 0.452 & 1.431 & 0.870 & 0.787 & 1.484 & 0.582 & 1.031\tabularnewline
& (1.766) & (1.679) & (1.702) & (1.674) & (1.690) & (1.708) & (1.654)\tabularnewline
\bottomrule
\end{tabular}
\end{centering}
\begin{tablenotes}[flushleft]
• \textit{Note:} The estimators whose names contain “\texttt{X}“ (or “\texttt{X}$^{\beta}$”) indicate that we add the component $X$ (or $(X+1)^{\beta}$) to $\bs{\xi}(X)$ to obtain the calibration estimator. The estimators whose names contain “\texttt{info}” indicate that we incorporate information from the other country to form one component of $\bs{\xi}(X)$.
\end{tablenotes}
\end{threeparttable}
}
\end{table}
The results presented in Figure (ref) and Table (ref)
lead to two key observations. First, in line with the theoretical
results (Theorem (ref)), the standard
errors for the \texttt{cal_info_X_X}$^{\beta}$ estimator are the
lowest among all estimators in both Uganda and Malawi. For example,
the standard errors of \texttt{cal_info_X_X}$^{\beta}$ ATE estimates
are 11.2% and 6.3% smaller than those of the stratified difference-in-means
ATE estimates in Uganda and Malawi, respectively. Second, in both
countries, all ATE estimates are statistically insignificant, suggesting
that expanding access to basic bank accounts does not lead to a significant
increase in total savings on average. This finding is consistent with
the results of dupas2018Bankinga.
\putbib
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This supplementary material includes appendices containing additional simulation results and proofs for the main paper.