Evaluating treatment benefit predictors using observational data: contending with identification and confounding bias
Bibliographic record
Abstract
A treatment benefit predictor (TBP) is a function that maps patient characteristics to a putative treatment benefit for that patient. Such predictors support optimizing individualized treatment decisions, which are central to precision medicine. However, evaluating the predictive performance of a TBP is challenging, as this often must be conducted in a sample where treatment assignment is not random. After briefly reviewing several metrics for evaluating TBPs, we show conceptually how to evaluate a prespecified TBP using observational data from the target population, for a binary treatment decision at a single time point. We exemplify with a particular measure of discrimination (the concentration of benefit index) and a particular measure of calibration (the moderate calibration curve). The population-level definitions of these metrics involve the latent treatment benefit variable, but we show identification by re-expressing the respective estimands in terms of the distribution of observable data only. We also show that in the absence of full confounding control, bias propagates in a more complex manner than when targeting more commonly encountered estimands. We find the patterns of biases are often unpredictable, and general intuition about the direction of bias in causal effect estimates does not hold in the present context.
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How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.212 | 0.541 |
| Meta-epidemiology (narrow) | 0.001 | 0.001 |
| Meta-epidemiology (broad) | 0.003 | 0.003 |
| Bibliometrics | 0.003 | 0.004 |
| Science and technology studies | 0.001 | 0.008 |
| Scholarly communication | 0.004 | 0.008 |
| Open science | 0.003 | 0.006 |
| Research integrity | 0.004 | 0.006 |
| Insufficient payload (model declined to judge) | 0.001 | 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; the direct Gemma label and the distilled Codex classifier agree on what is shown here.
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".