Research map / Probabilistic and scientific modeling / Treatment effects
Quantifying Treatment Effects: Estimating Risk Ratios via Observational Studies
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Proposes and analyzes estimators of the risk ratio (RR) in observational studies (IPW, G-formula, one-step, and AIPW-based RR-AIPW), proving asymptotic normality and CIs, and recommends the doubly-robust RR-AIPW.
Problem
Estimating the causal risk ratio (relative treatment effect) from observational data, where no non-parametric or doubly-robust estimators with theoretical guarantees existed, and confounding must be handled.
Approach
Adapts IPW, G-formula, and AIPW-style efficient influence-function estimators to the nonlinear risk ratio: the RR-AIPW is a ratio of two AIPW estimates of E[Y(1)] and E[Y(0)], with asymptotic normality and CIs derived via M-estimation and influence functions, and shown to be efficient under weaker conditions than the one-step RR-OS.
Key results
- RR-AIPW and RR-OS (linear nuisance) are unbiased even at small sample sizes and converge quickly to the true RR in the linear/logistic DGP.
- In the linear/logistic DGP with n=1000, only the Linear RR-AIPW achieves coverage above 95% among the compared CIs, while RR-N has nearly zero coverage in observational settings.
- On the Traumabase real-world data, most estimators give RR above one (suggesting harmful effect of TXA on mortality), with Forest RR-IPW the only one indicating benefit.
Measured effects reported in the paper
| What was changed | Where | Measured | Effect |
|---|---|---|---|
| Use linear nuisance models for RR-AIPW instead of forest-based nuisance models | RR-AIPW · Linear/Logistic DGP | bias | Helped |
| Use linear nuisance models for RR-OS instead of forest-based nuisance models | RR-OS · Linear/Logistic DGP | bias | Helped |
| Use linear nuisance models for RR-G instead of forest-based nuisance models | RR-G · Linear/Logistic DGP | bias | Helped |
| Use random forests to estimate nuisance functions rather than parametric models | RR estimators · Linear/Logistic DGP | convergence rate | Hurt |
| Use linear outcome models for RR-G when the baseline response is nonlinear | RR-G · Non-Linear/Logistic DGP | bias | Hurt |
| Use forest-based nuisance models for RR-AIPW rather than linear models | RR-AIPW · Non-Linear/Logistic DGP | bias | Hurt |
| Use linear nuisance models for RR-AIPW when at least one model is well specified | RR-AIPW · Non-Linear/Logistic DGP | bias | Helped |
| Use linear nuisance models for RR-OS when at least one model is well specified | RR-OS · Non-Linear/Logistic DGP | bias | Helped |
| Use RR-N confidence intervals in observational data | RR-N · Linear/Logistic DGP | coverage | Hurt |
| Use linear rather than forest-based nuisance models for RR-AIPW confidence intervals | RR-AIPW · Linear/Logistic DGP | coverage | Helped |
| Use forest-based nuisance models for RR-AIPW confidence intervals | RR-AIPW · Linear/Logistic DGP | coverage | Hurt |
| Use linear nuisance models for RR-AIPW confidence intervals | RR-AIPW · Non-Linear/Logistic DGP | coverage | Helped |
Surprising findings
- RR-AIPW, a simple ratio of two AIPW estimators, is efficient and requires weaker assumptions than the one-step RR-OS, challenging expectations about the ratio of two one-step estimators.
- Estimating the propensity score reduces the asymptotic variance of RR-IPW compared with the oracle propensity score.
Limitations
- Relies on standard causal assumptions (unconfoundedness, overlap) that are hard to verify in practice.
- Non-parametric nuisance estimation (random forests) converges slowly and yields poor coverage for n=1000.
- Estimators for the non-RCT and generalization settings remain open; guidelines for choosing linear vs non-parametric nuisance models are lacking.
Experimental setup
Datasets: Traumabase (8,270 TBI patients, Mayer et al. 2020) semi-synthetic and real subsamples Simulated linear/logistic and non-linear DGPs
Benchmarks: Traumabase TXA 28-day mortality (real-world subsample)
Metrics: bias variance/efficiency coverage of 95% confidence intervals CI length convergence to true risk ratio
Baselines: RR-N (Neyman risk ratio) RR-IPW RR-G RR-OS Forest RR-G Forest RR-AIPW Linear RR-AIPW
Builds on: AIPW Inverse Propensity Weighting Katz et al. 1978 risk ratio CI Kennedy 2022 influence function estimators cross-fitting (double machine learning)
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