arXiv subjects

Birol Emir

Publications and source records attributed to Birol Emir.

3 recordsLinked to original sources

arXiv2026

From Cumulative Weights to Marginal Density Ratios: Per-Protocol Estimation in Sequential Target Trial Emulation

Sequential target trial emulation evaluates eligibility at multiple baseline times to emulate a sequence of randomized trials using observational data. Estimating per-protocol effects in this setting is challenging because treatment deviations and loss to follow-up induce selection among individuals who remain observed and adherent over time. Conventional inverse-probability methods address this selection using cumulative weights constructed from estimated adherence and censoring probabilities, but these weights can be highly variable, leading to unstable and imprecise effect estimates. We propose a different approach based on marginal density ratios (MDRs). The MDR directly compares the state distribution among individuals who would remain event-free under a target treatment strategy with the corresponding distribution among observed-adherent individuals. We use longitudinal g-computation to generate the target risk sets and a probabilistic classifier to estimate density ratios for reweighting the observed outcomes. Building on this approach, we also develop a doubly robust extension. Favorable performance across the simulation study suggests that MDR weighting is a promising alternative to cumulative longitudinal weights when its identification assumptions are plausible.

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arXiv2026

Externally Controlled Trials: A Review of Design and Borrowing Through a Causal Lens

Externally controlled trials (ECTs) are increasingly used when randomized controls are infeasible, unethical, or insufficient, including applications in rare diseases, oncology, pediatrics, and post-approval effectiveness research. Although methodological work has expanded rapidly across causal inference, Bayesian dynamic borrowing, and hybrid trial designs, the literature remains fragmented. We adopt a six-step scientific roadmap to organize modern ECT methodology in two primary settings: (i) single-arm trials that evaluate efficacy through comparison with external controls, and (ii) hybrid controlled trials that augment the internal control arm with external controls drawn from real-world data or historical studies. The roadmap clarifies causal estimands, identifiability assumptions, and how statistical parameters arise from identification, and shows how modeling and borrowing strategies trade off efficiency and robustness, especially under covariate shift and outcome drift. Within this framework, we synthesize and evaluate recent Bayesian and frequentist developments, compare their strengths, limitations, operating characteristics, and available software, and emphasize the role of sensitivity analysis. By re-framing ECT methodology through a causal lens, this work establishes a coherent foundation for integrating external data into regulatory and clinical decision-making and highlights core challenges and opportunities for future research.

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arXiv2023

A New Projection Pursuit Index for Big Data

Visualization of extremely large datasets in static or dynamic form is a huge challenge because most traditional methods cannot deal with big data problems. A new visualization method for big data is proposed based on Projection Pursuit, Guided Tour and Data Nuggets methods, that will help display interesting hidden structures such as clusters, outliers, and other nonlinear structures in big data. The Guided Tour is a dynamic graphical tool for high-dimensional data combining Projection Pursuit and Grand Tour methods. It displays a dynamic sequence of low-dimensional projections obtained by using Projection Pursuit (PP) index functions to navigate the data space. Different PP indices have been developed to detect interesting structures of multivariate data but there are computational problems for big data using the original guided tour with these indices. A new PP index is developed to be computable for big data, with the help of a data compression method called Data Nuggets that reduces large datasets while maintaining the original data structure. Simulation studies are conducted and a real large dataset is used to illustrate the proposed methodology. Static and dynamic graphical tools for big data can be developed based on the proposed PP index to detect nonlinear structures.

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