arXiv subjects

Brian D Williamson

Publications and source records attributed to Brian D Williamson.

3 recordsLinked to original sources

arXiv2026

Marginal generalized raking with parametric working models

Generalized raking (GR) was originally developed in the survey statistics literature to incorporate auxiliary information in estimation. Recently, it has been used in the biostatistical and epidemiological literature to estimate regression coefficients in parametric models in cases with missing data, including missing data by design (e.g., two-phase studies). In the regression parameter context, the optimal GR estimator has been shown to be equivalent to the optimal augmented inverse probability weighted estimator. In this paper, we generalize the influence function-based theory for GR to marginal estimands; we call our approach \textit{marginal generalized raking}. We compare our approach to a naive procedure that marginalizes a conditional GR estimator of regression parameters in both fully-synthetic simulations and in an application using data from an observational cohort of persons living with HIV.

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arXiv2023

An Application of the Causal Roadmap in Two Safety Monitoring Case Studies: Covariate-Adjustment and Outcome Prediction using Electronic Health Record Data

Real-world data, such as administrative claims and electronic health records, are increasingly used for safety monitoring and to help guide regulatory decision-making. In these settings, it is important to document analytic decisions transparently and objectively to ensure that analyses meet their intended goals. The Causal Roadmap is an established framework that can guide and document analytic decisions through each step of the analytic pipeline, which will help investigators generate high-quality real-world evidence. In this paper, we illustrate the utility of the Causal Roadmap using two case studies previously led by workgroups sponsored by the Sentinel Initiative -- a program for actively monitoring the safety of regulated medical products. Each case example focuses on different aspects of the analytic pipeline for drug safety monitoring. The first case study shows how the Causal Roadmap encourages transparency, reproducibility, and objective decision-making for causal analyses. The second case study highlights how this framework can guide analytic decisions beyond inference on causal parameters, improving outcome ascertainment in clinical phenotyping. These examples provide a structured framework for implementing the Causal Roadmap in safety surveillance and guide transparent, reproducible, and objective analysis.

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arXiv2023

A Causal Roadmap for Generating High-Quality Real-World Evidence

Increasing emphasis on the use of real-world evidence (RWE) to support clinical policy and regulatory decision-making has led to a proliferation of guidance, advice, and frameworks from regulatory agencies, academia, professional societies, and industry. A broad spectrum of studies use real-world data (RWD) to produce RWE, ranging from randomized controlled trials with outcomes assessed using RWD to fully observational studies. Yet many RWE study proposals lack sufficient detail to evaluate adequacy, and many analyses of RWD suffer from implausible assumptions, other methodological flaws, or inappropriate interpretations. The Causal Roadmap is an explicit, itemized, iterative process that guides investigators to pre-specify analytic study designs; it addresses a wide range of guidance within a single framework. By requiring transparent evaluation of causal assumptions and facilitating objective comparisons of design and analysis choices based on pre-specified criteria, the Roadmap can help investigators to evaluate the quality of evidence that a given study is likely to produce, specify a study to generate high-quality RWE, and communicate effectively with regulatory agencies and other stakeholders. This paper aims to disseminate and extend the Causal Roadmap framework for use by clinical and translational researchers, with companion papers demonstrating application of the Causal Roadmap for specific use cases.

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