arXiv subjects

Imke Mayer

Publications and source records attributed to Imke Mayer.

7 recordsLinked to original sources

arXiv2025

Treatment Effect Estimation in Causal Survival Analysis: Practical Recommendations

The restricted mean survival time (RMST) difference offers an interpretable causal contrast to estimate the treatment effect for time-to-event outcomes, yet a wide range of available estimators leaves limited guidance for practice. We provide a unified review of RMST estimators for randomized trials and observational studies, establish identification and asymptotic properties, and supply new derivations where needed. Our extensive simulation study compares simple nonparametric methods (such as unweighted Kaplan-Meier estimators) alongside parametric and nonparametric implementations of the G-formula, weighting approaches, Buckley-James transformations, and augmented estimators under diverse censoring mechanisms and model specifications. Across scenarios, classical Kaplan-Meier estimators (weighted when required by the censoring process) and G-formula methods perform well in randomized settings, while in observational data G-formula estimators remain competitive; however, augmented estimators such as AIPTW-AIPCW generally offer robustness to model misspecification and a favorable bias-variance trade-off. Parametric estimators perform best under correct specification, whereas nonparametric methods avoid functional assumptions but require large sample sizes to achieve reliable performance. We offer practical recommendations for estimator choice and provide open-source R code to support reproducibility and application.

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arXiv2023

FedECA: Federated External Control Arms for Causal Inference with Time-To-Event Data in Distributed Settings

External control arms can inform early clinical development of experimental drugs and provide efficacy evidence for regulatory approval. However, accessing sufficient real-world or historical clinical trials data is challenging. Indeed, regulations protecting patients' rights by strictly controlling data processing make pooling data from multiple sources in a central server often difficult. To address these limitations, we develop a method that leverages federated learning to enable inverse probability of treatment weighting for time-to-event outcomes on separate cohorts without needing to pool data. To showcase its potential, we apply it in different settings of increasing complexity, culminating with a real-world use-case in which our method is used to compare the treatment effect of two approved chemotherapy regimens using data from three separate cohorts of patients with metastatic pancreatic cancer. By sharing our code, we hope it will foster the creation of federated research networks and thus accelerate drug development.

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arXiv2023

Generalizing treatment effects with incomplete covariates: identifying assumptions and multiple imputation algorithms

We focus on the problem of generalizing a causal effect estimated on a randomized controlled trial (RCT) to a target population described by a set of covariates from observational data. Available methods such as inverse propensity sampling weighting are not designed to handle missing values, which are however common in both data sources. In addition to coupling the assumptions for causal effect identifiability and for the missing values mechanism and to defining appropriate estimation strategies, one difficulty is to consider the specific structure of the data with two sources and treatment and outcome only available in the RCT. We propose three multiple imputation strategies to handle missing values when generalizing treatment effects, each handling the multi-source structure of the problem differently (separate imputation, joint imputation with fixed effect, joint imputation ignoring source information). As an alternative to multiple imputation, we also propose a direct estimation approach that treats incomplete covariates as semi-discrete variables. The multiple imputation strategies and the latter alternative rely on different sets of assumptions concerning the impact of missing values on identifiability. We discuss these assumptions and assess the methods through an extensive simulation study. This work is motivated by the analysis of a large registry of over 20,000 major trauma patients and an RCT studying the effect of tranexamic acid administration on mortality in major trauma patients admitted to ICU. The analysis illustrates how the missing values handling can impact the conclusion about the effect generalized from the RCT to the target population.

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arXiv2023

Causal inference methods for combining randomized trials and observational studies: a review

With increasing data availability, causal effects can be evaluated across different data sets, both randomized controlled trials (RCTs) and observational studies. RCTs isolate the effect of the treatment from that of unwanted (confounding) co-occurring effects but they may suffer from unrepresentativeness, and thus lack external validity. On the other hand, large observational samples are often more representative of the target population but can conflate confounding effects with the treatment of interest. In this paper, we review the growing literature on methods for causal inference on combined RCTs and observational studies, striving for the best of both worlds. We first discuss identification and estimation methods that improve generalizability of RCTs using the representativeness of observational data. Classical estimators include weighting, difference between conditional outcome models, and doubly robust estimators. We then discuss methods that combine RCTs and observational data to either ensure uncounfoundedness of the observational analysis or to improve (conditional) average treatment effect estimation. We also connect and contrast works developed in both the potential outcomes literature and the structural causal model literature. Finally, we compare the main methods using a simulation study and real world data to analyze the effect of tranexamic acid on the mortality rate in major trauma patients. A review of available codes and new implementations is also provided.

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arXiv2020

Doubly robust treatment effect estimation with missing attributes

Missing attributes are ubiquitous in causal inference, as they are in most applied statistical work. In this paper, we consider various sets of assumptions under which causal inference is possible despite missing attributes and discuss corresponding approaches to average treatment effect estimation, including generalized propensity score methods and multiple imputation. Across an extensive simulation study, we show that no single method systematically out-performs others. We find, however, that doubly robust modifications of standard methods for average treatment effect estimation with missing data repeatedly perform better than their non-doubly robust baselines; for example, doubly robust generalized propensity score methods beat inverse-weighting with the generalized propensity score. This finding is reinforced in an analysis of an observations study on the effect on mortality of tranexamic acid administration among patients with traumatic brain injury in the context of critical care management. Here, doubly robust estimators recover confidence intervals that are consistent with evidence from randomized trials, whereas non-doubly robust estimators do not.

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arXiv2020

MissDeepCausal: Causal Inference from Incomplete Data Using Deep Latent Variable Models

Inferring causal effects of a treatment, intervention or policy from observational data is central to many applications. However, state-of-the-art methods for causal inference seldom consider the possibility that covariates have missing values, which is ubiquitous in many real-world analyses. Missing data greatly complicate causal inference procedures as they require an adapted unconfoundedness hypothesis which can be difficult to justify in practice. We circumvent this issue by considering latent confounders whose distribution is learned through variational autoencoders adapted to missing values. They can be used either as a pre-processing step prior to causal inference but we also suggest to embed them in a multiple imputation strategy to take into account the variability due to missing values. Numerical experiments demonstrate the effectiveness of the proposed methodology especially for non-linear models compared to competitors.

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arXiv2019

R-miss-tastic: a unified platform for missing values methods and workflows

Missing values are unavoidable when working with data. Their occurrence is exacerbated as more data from different sources become available. However, most statistical models and visualization methods require complete data, and improper handling of missing data results in information loss or biased analyses. Since the seminal work of Rubin (1976), a burgeoning literature on missing values has arisen, with heterogeneous aims and motivations. This led to the development of various methods, formalizations, and tools. For practitioners, it remains nevertheless challenging to decide which method is most suited for their problem, partially due to a lack of systematic covering of this topic in statistics or data science curricula. To help address this challenge, we have launched the "R-miss-tastic" platform, which aims to provide an overview of standard missing values problems, methods, and relevant implementations of methodologies. Beyond gathering and organizing a large majority of the material on missing data (bibliography, courses, tutorials, implementations), "R-miss-tastic" covers the development of standardized analysis workflows. Indeed, we have developed several pipelines in R and Python to allow for hands-on illustration of and recommendations on missing values handling in various statistical tasks such as matrix completion, estimation and prediction, while ensuring reproducibility of the analyses. Finally, the platform is dedicated to users who analyze incomplete data, researchers who want to compare their methods and search for an up-to-date bibliography, and also teachers who are looking for didactic materials (notebooks, video, slides).

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