Three families of estimators of μ(a)=E{E[Y∣X,S=1,A=a]∣S=0}: outcome-model (OM), inverse-odds weighting (IOW1 unnormalized, IOW2 normalized), and three doubly robust estimators (DR1-DR3). Each is a solution to a missing-data problem; DR is consistent if either the outcome model or the participation model is right.
Overview
Under consistency the unknown Ya for target individuals is a missing outcome, with missingness indicator I(S=1,A=a). Parametric working models throughout: outcome ga(X;θ)≈E[Y∣X,S=1,A=a]; participation p(X;β)≈Pr[S=1∣X]; trial treatment ea(X;γ)≈Pr[A=a∣X,S=1]. Treatment probabilities are known by design (true values can be used; estimating them can reduce standard errors).
Fit the outcome regression in trial participants (separately by arm, i.e., all treatment-by-covariate interactions) and average predictions over target covariates. Consistent when the outcome model is correct.
The factor (1−p)/p is the inverse of the odds of participation. Interpretation: treat the trial as a survey sample from the target with covariate-dependent, estimated sampling probabilities.
IOW estimators (eqs. 2-3)
Unnormalized: μ^IOW1(a)=(∑i(1−Si))−1∑iw^a(Xi,Si,Ai)Yi.
Normalized (ratio/Hajek form): μ^IOW2(a)=(∑iw^a)−1∑iw^aYi.
IOW2 equals the intercept (and intercept + treatment coefficient) of a saturated weighted least squares regression of Y on A. It always lies in the support of Y (e.g., within [0,1] for binary Y), giving much better finite-sample behaviour with variable weights. Both are consistent if the participation model is right.
where θ^w comes from the outcome regression among trial participants weighted by w^a. DR3 is doubly robust when the outcome is modelled with a linear exponential family quasi-likelihood and canonical link.
Double robustness
DR estimators are consistent for μ(a) and asymptotically normal when either the participation model or the outcome model is correctly specified (formal result in the Appendix, absent from the PDF; refs [36],[40]). In practice DR estimators are usually more precise than IOW and nearly as precise as OM. Caveat: with a misspecified outcome model and highly variable weights, DR can do worse than non-DR estimators using the same misspecified outcome model (refs [41],[34]).
Special cases (paper's argument, Sec. 6.2)
IOW is DR with a grossly misspecified outcome model ga≡0; OM is DR with grossly misspecified participation p≡1 (weights ≡0). So correct IOW or OM performance in simulation already demonstrates the double robustness property.
Inference
All estimators are partial M-estimators, so sandwich variances are available (R package geex is cited); the nonparametric bootstrap is easier and “will often be preferred in practice”.
Examples
Runnable sketch - OM, IOW2 and DR2 on simulated trial + cohort data
import numpy as nprng = np.random.default_rng(0)n = 5000X = rng.standard_normal((n, 3))# trial selection depends on X1 (an effect modifier), X2, X3logit = -2.5 + X @ np.array([1., 1., 1.])p_true = 1 / (1 + np.exp(-logit)); S = rng.random(n) < p_trueA = rng.random(n) < 0.5tau = 1 + 2 * X[:, 0] # effect modification by X1Y = X @ np.array([1., 1., 1.]) + A * tau + rng.standard_normal(n)def fit_logit(X, s): Z = np.c_[np.ones(len(X)), X]; b = np.zeros(Z.shape[1]) for _ in range(50): p = 1 / (1 + np.exp(-Z @ b)); b += np.linalg.solve(Z.T @ (Z * (p * (1 - p))[:, None]), Z.T @ (s - p)) return 1 / (1 + np.exp(-Z @ b))p = fit_logit(X, S.astype(float))def mu(a): sel = S & (A == a) Z = np.c_[np.ones(n), X] beta = np.linalg.lstsq(Z[sel], Y[sel], rcond=None)[0] g = Z @ beta w = np.where(sel, (1 - p) / (p * 0.5), 0.0) # known e_a = 0.5 om = g[~S].mean() iow2 = (w * Y).sum() / w.sum() dr2 = (w * (Y - g)).sum() / w.sum() + g[~S].mean() return om, iow2, dr2est = np.array(mu(1)) - np.array(mu(0))print("target ATE: OM, IOW2, DR2 =", est.round(3), "| truth", tau[~S].mean().round(3), "| trial-only", (Y[S & A].mean() - Y[S & ~A].mean()).round(3))
The trial-only contrast is biased upward (selection over-represents high X1); the three adjusted estimates recover the target ATE (Monte Carlo summaries in Dahabreh 2020 - Simulation Study of Estimators).