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Joseph Rigdon

Publications and source records attributed to Joseph Rigdon.

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Randomization inference for treatment effects on survival outcomes

The log-rank test and Kaplan--Meier plot are standard tools for analyzing time-to-event data in randomized clinical trials, yet neither provides a summary of the magnitude of the treatment effect. Practitioners typically fill this gap by reporting a hazard ratio from a Cox proportional-hazards model or an acceleration factor from an accelerated failure time (AFT) model, but both require assumptions beyond those needed for the log-rank test or Kaplan--Meier estimator. We propose two nonparametric confidence intervals for scalar effect-size summaries, an additive shift c and a multiplicative factor $\rho$, obtained by inverting the log-rank test under sharp null hypotheses of constant treatment effects. Building on the randomization-inference framework of Li and Small (2023), both intervals are valid under the randomization distribution alone, requiring no assumptions for the event-time distribution. We evaluate the proposed multiplicative interval via simulation, finding that it maintains nominal coverage across a range of censoring rates and sample sizes, including under data-generating processes that misspecify a parametric AFT model, while incurring only a modest efficiency loss compared to parametric AFT inference under correct specification. We illustrate the approach using data from a randomized trial of rhDNase for cystic fibrosis and provide R code and a Shiny application for ease of implementation.

stat.ME

Predicting Hospitalization from a Whole-Person Health Score with Incomplete Electronic Health Records Data: A Case Study

Embedding a standardized whole-person health measure in electronic health records (EHR) could be instrumental to preventative care. The allostatic load index (ALI), calculated from ten component stressors across three body systems, offers a promising snapshot of holistic health. The ALI can be calculated from EHR data, but many components are missing, since not all patients undergo all tests. Using statistical modeling and machine learning, EHR data for $1000$ patients from a large academic health system were used to predict in-patient hospitalization (as a count or binary) from ALI, controlling for age and sex. Various methods were evaluated to fill in information gaps for patients' missing ALI components, including summary measures combining components or using them separately. Performance was measured using receiver operating characteristic (ROC) curves and corresponding areas under the ROC curve (AUC). Count modeling of hospitalization did not improve upon binary, and logistic regression beat random forest. Overall, summary measures performed similarly, with the complete-case proportion (i.e., the proportion of non-missing components that were "unhealthy") performing best (AUC $= 0.64$) but by $\leq 0.01$. When using components separately, the pattern submodel approach most accurately predicted hospitalization (AUC $= 0.73$) in sample, but did not cross-validate as well (AUC $= 0.63$). All summary measures performed similarly. However, when including the ALI components separately, tailoring models to subsets of patients with the same missing data pattern performed best. Next steps include EHR implementation to enable prediction and support clinician decision-making at scale.

stat.AP

On Using Large Language Models to Enhance Clinically-Driven Missing Data Recovery Algorithms in Electronic Health Records

Objective: Electronic health records (EHR) data are prone to missingness and errors. Previously, we devised an "enriched" chart review protocol where a "roadmap" of auxiliary diagnoses (anchors) was used to recover missing values in EHR data (e.g., a diagnosis of impaired glycemic control might imply that a missing hemoglobin A1c value would be considered unhealthy). Still, chart reviews are expensive and time-intensive, which limits the number of patients whose data can be reviewed. Now, we investigate the accuracy and scalability of a roadmap-driven algorithm, based on ICD-10 codes (International Classification of Diseases, 10th revision), to mimic expert chart reviews and recover missing values. Materials and Methods: In addition to the clinicians' original roadmap from our previous work, we consider new versions that were iteratively refined using large language models (LLM) in conjunction with clinical expertise to expand the list of auxiliary diagnoses. Using chart reviews for 100 patients from the EHR at an extensive learning health system, we examine algorithm performance with different roadmaps. Using the larger study of $1000$ patients, we applied the final algorithm, which used a roadmap with clinician-approved additions from the LLM. Results: The algorithm recovered as much, if not more, missing data as the expert chart reviewers, depending on the roadmap. Discussion: Clinically-driven algorithms (enhanced by LLM) can recover missing EHR data with similar accuracy to chart reviews and can feasibly be applied to large samples. Extending them to monitor other dimensions of data quality (e.g., plausability) is a promising future direction.

cs.LG

Overcoming data challenges through enriched validation and targeted sampling to measure whole-person health in electronic health records

The allostatic load index (ALI) is a 10-component measure of whole-person health. Data from electronic health records (EHR) present a huge opportunity to operationalize the ALI in learning health systems; however, these data are prone to missingness and errors. Validation (e.g., through chart reviews) provides better-quality data, but realistically, only a subset of patients' data can be validated, and most protocols do not recover missing data. Using a representative sample of 1000 patients from the EHR at an extensive learning health system (100 of whom could be validated), we propose methods to design, conduct, and analyze statistically efficient and robust studies of ALI and healthcare utilization. Employing semiparametric maximum likelihood estimation, we robustly incorporate all available patient information into statistical models. Using targeted design strategies, we examine ways to select the most informative patients for validation. Incorporating clinical expertise, we devise a novel validation protocol to promote EHR data quality and completeness. Chart reviews uncovered few errors (99% matched source documents) and recovered some missing data through auxiliary information in patients' charts. On average, validation increased the number of non-missing ALI components per patient from 6 to 7. Through simulations based on preliminary data, residual sampling was identified as the most informative strategy for completing our validation study. Incorporating validation data, statistical models indicated that worse whole-person health (higher ALI) was associated with higher odds of engaging in the healthcare system, adjusting for age.

stat.ME

stratamatch: Prognostic ScoreStratification using a Pilot Design

Optimal propensity score matching has emerged as one of the most ubiquitous approaches for causal inference studies on observational data; However, outstanding critiques of the statistical properties of propensity score matching have cast doubt on the statistical efficiency of this technique, and the poor scalability of optimal matching to large data sets makes this approach inconvenient if not infeasible for sample sizes that are increasingly commonplace in modern observational data. The stratamatch package provides implementation support and diagnostics for `stratified matching designs,' an approach which addresses both of these issues with optimal propensity score matching for large-sample observational studies. First, stratifying the data enables more computationally efficient matching of large data sets. Second, stratamatch implements a `pilot design' approach in order to stratify by a prognostic score, which may increase the precision of the effect estimate and increase power in sensitivity analyses of unmeasured confounding.

stat.CO