Search arXiv⌕ Search

arXiv · 2501.11181

Sample size and power calculations for causal inference of observational studies

Abstract

This paper investigates the theoretical foundation and develops analytical formulas for sample size and power calculations for causal inference with observational data. By analyzing the variance of an inverse probability weighting estimator of the average treatment effect, we decompose the power calculation into three components: propensity score distribution, potential outcome distribution, and their correlation. We show that to determine the minimal sample size of an observational study, in addition to the standard inputs in the power calculation of randomized trials, it is sufficient to have two parameters, which quantify the strength of the confounder-treatment and the confounder-outcome association, respectively. For the former, we propose using the Bhattacharyya coefficient, which measures the covariate overlap and, together with the treatment proportion, leads to a uniquely identifiable and easily computable propensity score distribution. For the latter, we propose a sensitivity parameter bounded by the R-squared statistic of the regression of the outcome on covariates. Our procedure relies on a parametric propensity score model and a semiparametric restricted mean outcome model, but does not require distributional assumptions on the multivariate covariates. We develop an associated R package PSpower and an online calculator.

Explore related subjects

Keep this discovery

Explore connections, maps & timelines

BibTeXRIS

Bo Liu, Chengxin Yang, Fan Li. 2026-05-17. Sample size and power calculations for causal inference of observational studies. https://arxiv.org/abs/2501.11181

Cite the original work for its findings. Save a collection to share your selection of sources.

KEEP EXPLORING

Related papers

Bridging Impulse Control of Piecewise Deterministic Markov Processes and Markov Decision Processes: Frameworks, Extensions, and Open Challenges

Control theory plays a pivotal role in understanding and optimizing the behavior of complex dynamical systems across various scientific and engineering disciplines. Two key frameworks that have emerged for modeling and solving control problems in stochastic systems are piecewise deterministic Markov processes (PDMPs) and Markov decision processes (MDPs). Each framework has its unique strengths, and their intersection offers promising opportunities for tackling a broad class of problems, particularly in the context of impulse controls and decision-making in complex systems. The relationship between PDMPs and MDPs is a natural subject of exploration, as embedding impulse control problems for PDMPs into the MDP framework could open new avenues for their analysis and resolution. Specifically, this integration would allow leveraging the computational and theoretical tools developed for MDPs to address the challenges inherent in PDMPs. On the other hand, PDMPs can offer a versatile and simple paradigm to model continuous time problems that are often described as discrete-time MDPs parametrized by complex transition kernels. This transformation has the potential to bridge the gap between the two frameworks, enabling solutions to previously intractable problems and expanding the scope of both fields. This paper presents a comprehensive review of two research domains, illustrated through a recurring medical example. The example is revisited and progressively formalized within the framework of thevarious concepts and objects introduced

stat.ME↗

Decision Theoretic Subgroup Detection With Bayesian Machine Learning

We consider the problem of identifying promising subpopulations in terms of treatment effectiveness or treatment effect heterogeneity, from a Bayesian decision theoretic perspective. We first show that a straight-forward application of Bayesian decision theory to subgroup detection leads to a counter-intuitive risk-seeking (RS) behavior. Motivated by this observation, we introduce the Bayesian Risk-Aware Inference and Detection of Subgroups (BRAIDS) utility and use it to perform subgroup selection and post selection inference. The BRAIDS utility interpolates between risk-seeking (RS) and risk-averse (RA) identifications of subgroups, with a variant of the virtual twins algorithm as its risk-neutral midpoint. We also argue that effective subgroup estimation and inference requires the use of regularization priors to safeguard inferences from the winner's curse. We provide empirical evidence that posterior credible intervals for subgroup effects can still obtain nominal coverage levels, provided that an appropriate prior distribution is chosen. The proposed framework is illustrated on data from clinical trial assessing the efficacy of canagliflozin as a treatment for type 2 diabetes.

stat.ME↗

Modeling cyclostationarity in time series using ASCA

Modern data analysis across diverse disciplines increasingly relies on time series. Many of these datasets exhibit cyclostationarity, where patterns approximately repeat in a regular manner, often across multiple time scales, such as daily, weekly or yearly cycles. In this context, statistical inference is essential to distinguish genuine underlying effects from random variability. While tools like Analysis of Variance (ANOVA) provide such inference, they often lack interpretability and struggle with the complexities of multivariate data. To address these limitations, we propose a unified pipeline for the exploratory analysis of cyclostationary times series using ANOVA Simultaneous Component Analysis (ASCA). ASCA is an extension of ANOVA that is able to work in both univariate and multivariate cases. Combining inference with the visualization capabilities of Principal Component Analysis (PCA), ASCA provides powerful options for interpretability. ASCA's capabilities have been well-established in the analysis of experimental data, but they remain largely unexplored for observational data like time series. Our workflow introduces an algorithmic approach to modeling time-dependent data using ASCA, enabling control over multiple cyclostationary time scales while also accounting for the specific challenges of this type of data, such as autocorrelation. Furthermore, we observed that ASCA provides a better separation of variability across factors than ANOVA in unbalanced designs due to its multivariate nature. We demonstrate the efficacy of this methodology through two real-world case studies: water temperature trends in mountain lakes in Sierra Nevada, Spain, and airborne pollen trends over 30 years recorded in the city of Granada, Spain.

stat.ME↗