arXiv subjects

Lauren D. Liao

Publications and source records attributed to Lauren D. Liao.

3 recordsLinked to original sources

arXiv2024

JOINTVIP: Prioritizing variables in observational study design with joint variable importance plot in R

Credible causal effect estimation requires treated subjects and controls to be otherwise similar. In observational settings, such as analysis of electronic health records, this is not guaranteed. Investigators must balance background variables so they are similar in treated and control groups. Common approaches include matching (grouping individuals into small homogeneous sets) or weighting (upweighting or downweighting individuals) to create similar profiles. However, creating identical distributions may be impossible if many variables are measured, and not all variables are of equal importance to the outcome. The joint variable importance plot (jointVIP) package to guides decisions about which variables to prioritize for adjustment by quantifying and visualizing each variable's relationship to both treatment and outcome.

stat.ME↗

arXiv2024

Prioritizing Variables for Observational Study Design using the Joint Variable Importance Plot

Observational studies of treatment effects require adjustment for confounding variables. However, causal inference methods typically cannot deliver perfect adjustment on all measured baseline variables, and there is often ambiguity about which variables should be prioritized. Standard prioritization methods based on treatment imbalance alone neglect variables' relationships with the outcome. We propose the joint variable importance plot to guide variable prioritization for observational studies. Since not all variables are equally relevant to the outcome, the plot adds outcome associations to quantify the potential confounding jointly with the standardized mean difference. To enhance comparisons on the plot between variables with different confounding relationships, we also derive and plot bias curves. Variable prioritization using the plot can produce recommended values for tuning parameters in many existing matching and weighting methods. We showcase the use of the joint variable importance plots in the design of a balance-constrained matched study to evaluate whether taking an antidiabetic medication, glyburide, increases the incidence of C-section delivery among pregnant individuals with gestational diabetes.

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arXiv2023

Prognostic Adjustment with Efficient Estimators to Unbiasedly Leverage Historical Data in Randomized Trials

Although randomized controlled trials (RCTs) are a cornerstone of comparative effectiveness, they typically have much smaller sample size than observational studies because of financial and ethical considerations. Therefore there is interest in using plentiful historical data (either observational data or prior trials) to reduce trial sizes. Previous estimators developed for this purpose rely on unrealistic assumptions, without which the added data can bias the treatment effect estimate. Recent work proposed an alternative method (prognostic covariate adjustment) that imposes no additional assumptions and increases efficiency in trial analyses. The idea is to use historical data to learn a prognostic model: a regression of the outcome onto the covariates. The predictions from this model, generated from the RCT subjects' baseline variables, are then used as a covariate in a linear regression analysis of the trial data. In this work, we extend prognostic adjustment to trial analyses with nonparametric efficient estimators, which are more powerful than linear regression. We provide theory that explains why prognostic adjustment improves small-sample point estimation and inference without any possibility of bias. Simulations corroborate the theory: efficient estimators using prognostic adjustment compared to without provides greater power (i.e., smaller standard errors) when the trial is small. Population shifts between historical and trial data attenuate benefits but do not introduce bias. We showcase our estimator using clinical trial data provided by Novo Nordisk A/S that evaluates insulin therapy for individuals with type II diabetes.

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