arXiv subjects

Catherine R. Lesko

Publications and source records attributed to Catherine R. Lesko.

2 recordsLinked to original sources

arXiv2026

Comparing causal estimands from sequential nested versus single point target trials: A simulation study

Sequential nested trial (SNT) emulation is a powerful approach for maximizing precision and avoiding time-related biases. However, there exists little discussion about the implied causal estimands in comparison to a real-world single point trial. We used Monte Carlo simulation to compare treatment effect estimates from an SNT emulation that re-indexed patients annually and a SNT emulation with a treatment decision design to the estimates from a single point trial. We generated 5,000 cohorts of 5,000 people with 3 years of follow-up. For the single point trial, patients were randomized to initiate or not initiate treatment at Visit 1. For the SNT emulations, simulated patients could contribute up to two index dates. When disease severity did not modify the treatment effect, both SNT approaches returned treatment effect estimates identical to the single point trial. In the presence of treatment effect modification by disease severity, both SNT approaches returned treatment effect estimates that diverged from the single point trial even after confounding-adjustment. These findings underscore the difficulties of interpreting causal estimands from a SNT emulation: the target population does not correspond to a single time point trial. Such implications are important for communicating study results for evidence-based decision-making.

stat.AP↗

arXiv2020

Generalizing Randomized Trial Findings to a Target Population using Complex Survey Population Data

Randomized trials are considered the gold standard for estimating causal effects. Trial findings are often used to inform policy and programming efforts, yet their results may not generalize well to a relevant target population due to potential differences in effect moderators between the trial and population. Statistical methods have been developed to improve generalizability by combining trials and population data, and weighting the trial to resemble the population on baseline covariates.Large-scale surveys in fields such as health and education with complex survey designs are a logical source for population data; however, there is currently no best practice for incorporating survey weights when generalizing trial findings to a complex survey. We propose and investigate ways to incorporate survey weights in this context. We examine the performance of our proposed estimator in simulations by comparing its performance to estimators that ignore the complex survey design.We then apply the methods to generalize findings from two trials - a lifestyle intervention for blood pressure reduction and a web-based intervention to treat substance use disorders - to their respective target populations using population data from complex surveys. The work highlights the importance in properly accounting for the complex survey design when generalizing trial findings to a population represented by a complex survey sample.

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