Effect Estimation in Point-Exposure Studies with Binary Outcomes and High-Dimensional Covariate Data – A Comparison of Targeted Maximum Likelihood Estimation and Inverse Probability of Treatment Weighting
Bibliographic record
Abstract
Inverse probability of treatment weighting (IPW) and targeted maximum likelihood estimation (TMLE) are relatively new methods proposed for estimating marginal causal effects. TMLE is doubly robust, yielding consistent estimators even under misspecification of either the treatment or the outcome model. While IPW methods are known to be sensitive to near violations of the practical positivity assumption (e. g., in the case of data sparsity), the consequences of this violation in the TMLE framework for binary outcomes have been less widely investigated. As near practical positivity violations are particularly likely in high-dimensional covariate settings, a better understanding of the performance of TMLE is of particular interest for pharmcoepidemiological studies using large databases. Using plasmode and Monte-Carlo simulation studies, we evaluated the performance of TMLE compared to that of IPW estimators based on a point-exposure cohort study of the marginal causal effect of post-myocardial infarction statin use on the 1-year risk of all-cause mortality from the Clinical Practice Research Datalink. A variety of treatment model specifications were considered, inducing different degrees of near practical non-positivity. Our simulation study showed that the performance of the TMLE and IPW estimators were comparable when the dimension of the fitted treatment model was small to moderate; however, they differed when a large number of covariates was considered. When a rich outcome model was included in the TMLE, estimators were unbiased. In some cases, we found irregular bias and large standard errors with both methods even with a correctly specified high-dimensional treatment model. The IPW estimator showed a slightly better root MSE with high-dimensional treatment model specifications in our simulation setting. In conclusion, for estimation of the marginal expectation of the outcome under a fixed treatment, TMLE and IPW estimators employing the same treatment model specification may perform differently due to differential sensitivity to practical positivity violations; however, TMLE, being doubly robust, shows improved performance with richer specifications of the outcome model. Although TMLE is appealing for its double robustness property, such violations in a high-dimensional covariate setting are problematic for both methods.
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How this classification was reachedexpand
Full frame distilled prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. Learned from the 10,348 direct Codex labels and 10,348 direct Gemma labels. Candidate is the union of thresholded teacher heads; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels or direct frontier model labels.
Codex and Gemma teacher scores by category
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.001 | 0.002 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.000 | 0.000 |
| Bibliometrics | 0.000 | 0.000 |
| Science and technology studies | 0.000 | 0.000 |
| Scholarly communication | 0.000 | 0.000 |
| Open science | 0.000 | 0.000 |
| Research integrity | 0.000 | 0.000 |
| Insufficient payload (model declined to judge) | 0.000 | 0.000 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one teacher head, not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".