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20142024
most citedA Causal Roadmap for Generating High-Quality Real-World Evidence

10 citations · 17 across the 11 of their papers we have counts for

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9 papers · 1 filter

stat.ME2024

Non-parametric efficient estimation of marginal structural models with multi-valued time-varying treatments

Axel Martin, Michele Santacatterina, Iván Díaz

Marginal structural models are a popular method for estimating causal effects in the presence of time-varying exposures. In spite of their popularity, no scalable non-parametric es…

stat.ME2024

A Surrogate Endpoint Based Provisional Approval Causal Roadmap

Peter B. Gilbert, James Peng, Larry Han +12

For many rare diseases with no approved preventive interventions, promising interventions exist, yet it has been difficult to conduct a pivotal phase 3 trial that could provide dir…

stat.ME20241 cited

Recanting twins: addressing intermediate confounding in mediation analysis

Tat-Thang Vo, Nicholas Williams, Richard Liu +2

The presence of intermediate confounders, also called recanting witnesses, is a fundamental challenge to the investigation of causal mechanisms in mediation analysis, preventing th…

stat.ME2024

Two-Step Targeted Minimum-Loss Based Estimation for Non-Negative Two-Part Outcomes

Nicholas T. Williams, Richard Liu, Katherine L. Hoffman +3

Non-negative two-part outcomes are defined as outcomes with a density function that have a zero point mass but are otherwise positive. Examples, such as healthcare expenditure and…

stat.ME202310 cited

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

Lauren E Dang, Susan Gruber, Hana Lee +23

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…

stat.ME20224 cited

Efficient estimation of modified treatment policy effects based on the generalized propensity score

Nima S. Hejazi, David Benkeser, Iván Díaz +1

Continuous treatments have posed a significant challenge for causal inference, both in the formulation and identification of scientifically meaningful effects and in their robust e…