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. Author manuscript; available in PMC: 2023 Mar 1.
Published in final edited form as: Contemp Clin Trials. 2022 Feb 2;114:106702. doi: 10.1016/j.cct.2022.106702

Table 3.

Simulation scenarios and results for continuous outcomes

Scenarioa Parameters Results Methods
IEEb IEEWc EEEd EEEWe

Homogeneous treatment effect

1 Mean Estimate 2.0025 2.0009 2.0014 2.0003
βl = 2 Mean Variance of Estimate 0.0101 0.0100 0.0089 0.0140
βs = 2 Mean Bias 0.0025 0.0009 0.0014 0.0003
σb = 0.2 Mean Relative Bias (%) 0.1274 0.0439 0.0725 0.0159
σe = 1 Monte-Carlo Variance 0.0117 0.0105 0.0099 0.0163
ρ = 0.038 Empirical Coverage 0.9200 0.9430 0.9340 0.9210
Mean ICC - - 0.0338 -

2 Mean Estimate 2.0047 2.0032 2.0040 2.0030
βl = 2 Mean Variance of Estimate 0.0170 0.0230 0.0164 0.0289
βs = 2 Mean Bias 0.0047 0.0032 0.0040 0.0030
σb = 0.2 Mean Relative Bias (%) 0.2341 0.1621 0.2006 0.1517
σe = 2 Monte-Carlo Variance 0.0195 0.0237 0.0187 0.0333
ρ = 0.010 Empirical Coverage 0.9210 0.9460 0.9230 0.9320
Mean ICC - - 0.0075 -

Heterogeneous treatment effects

3 Mean Estimate 3.6505 2.9963 3.0363 2.3495
βl = 4 Mean Variance of Estimate 0.0381 0.0839 0.0830 0.0449
βs = 2 Mean Bias 0.6505 −0.0037 0.0363 −0.6505
σb = 0.2 Mean Relative Bias (%) 21.6832 −0.1246 1.2101 −21.6828
σe = 1 Monte-Carlo Variance 0.0278 0.0517 0.0553 0.0360
ρ = 0.038 Empirical Coverage 0.1360 0.9860 0.9800 0.1740
Mean ICC - - 0.3212 -

4 Mean Estimate 3.6526 2.9986 3.1603 2.3786
βl = 4 Mean Variance of Estimate 0.0450 0.0968 0.0889 0.0752
βs = 2 Mean Bias 0.6526 −0.0014 0.1603 −0.6214
σbs = 0.2 Mean Relative Bias (%) 21.7544 −0.0458 5.3440 −20.7122
σe = 2 Monte-Carlo Variance 0.0362 0.0655 0.0785 0.0703
ρ = 0.010 Empirical Coverage 0.1670 0.9750 0.9030 0.3910
Mean ICC - - 0.0878 -
a

Scenarios 1 and 3 have same values of σb and σe, but Scenario 1 has homogenous treatment effect and 3 has heterogeneous treatment effects, resulting in the difference in the mean empirical ICCs. Similar patterns for Scenarios 2 and 4. The mean UATE estimand of Scenarios 1 and 2 (homogenous treatment effect) is 2; the mean UATE estimand of Scenarios 3 and 4 (heterogeneous treatment effects) is 3. The true ICC for homogeneous treatment effect (Scenarios 1 and 2) are estimated directly using ρ=σb2σb2+σe2, yielding values of 0.0385 and 0.0099, respectively. In contrast, the true ICC for heterogeneous treatment effects (Scenarios 3 and 4) are obtained via a weighted average of the ICC for the large and small clusters.

b

IEE: Independence working correlation matrix with no cluster size weighting.

c

IEEW: Independence working correlation matrix with inverse cluster size weighting.

d

EEE: Exchangeable working correlation matrix with no cluster size weighting.

e

EEEW: Exchangeable working correlation matrix with inverse cluster size weighting.