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. Author manuscript; available in PMC: 2022 Sep 1.
Published in final edited form as: Biometrika. 2020 Sep 4;108(3):507–527. doi: 10.1093/biomet/asaa070

Table 3.

Performance of EMEE, ECE, GEE.ind, and GEE.exch for the treatment effect modification St = f(Ht) = Zt, when the working model is correctly specified, i.e., g(Ht)Tα=α0+α1Zt+α21Zt=2.

Estimator Sample size β 0
β 1
Bias RMSE SD CP (unadj) CP (adj) Bias RMSE SD CP (unadj) CP (adj)
EMEE 30 0.00 0.21 0.21 0.93* 0.94 0.00 0.13 0.13 0.94 0.95
50 0.00 0.16 0.16 0.94 0.95 0.00 0.11 0.11 0.94 0.95
100 0.00 0.11 0.11 0.95 0.95 0.00 0.07 0.07 0.95 0.95
ECE 30 0.01 0.17 0.17 0.94 0.95 −0.01 0.12 0.12 0.93* 0.94
50 0.00 0.13 0.13 0.94 0.95 0.00 0.09 0.09 0.94 0.95
100 0.00 0.10 0.10 0.94 0.95 0.00 0.06 0.06 0.95 0.95
GEE.ind 30 0.01 0.17 0.17 0.94 0.95 −0.01 0.12 0.12 0.93* 0.94
50 0.00 0.13 0.13 0.94 0.95 0.00 0.09 0.09 0.94 0.95
100 0.00 0.10 0.10 0.94 0.94 0.00 0.06 0.06 0.94 0.95
GEE.exch 30 0.01 0.17 0.17 0.94 0.95 −0.01 0.12 0.12 0.93* 0.94
50 0.00 0.13 0.13 0.94 0.95 0.00 0.09 0.09 0.94 0.95
100 0.00 0.10 0.10 0.94 0.94 0.00 0.06 0.06 0.95 0.95

EMEE: the estimator of the marginal excursion effect proposed in Section 5. ECE: the semiparametric, locally efficient estimator of the conditional effect described in Section 4. GEE.ind: GEE with independent working correlation structure. GEE.exch: GEE with exchangeable working correlation structure. SD: standard deviation. RMSE: root mean squared error. CP: 95% confidence interval coverage probability, before (unadj) and after (adj) small sample correction. Numbers with

*

indicate when Bias or CP are significantly different, at the 5% level, from 0 or 0.95, respectively. Sample size refers to the number of individuals in each simulated trial.