Sample Size and Model Prediction Accuracy in EQ-5D-5L Valuations Studies: Expected Out-of-Sample Accuracy Based on Resampling with Different Sample Sizes and Alternative Model Specifications

Background. National valuation studies are costly, with ∼1000 face-to-face interviews recommended, and some countries may deem such studies infeasible. Building on previous studies exploring sample size, we determined the effect of sample size and alternative model specifications on prediction accuracy of modeled coefficients in EQ-5D-5L value set generating regression analyses. Methods. Data sets (n = 50 to ∼1000) were simulated from 3 valuation studies, resampled at the respondent level and randomly drawn 1000 times with replacement. We estimated utilities for each subsample with leave-one-out at the block level using regression models (8 or 20 parameter; with or without a random intercept; time tradeoff [TTO] data only or TTO + discrete choice experiment [DCE] data). Prediction accuracy, root mean square error (RMSE), was calculated by comparing to censored mean predicted values to the left-out block in the full data set. Linear regression was used to estimate the relative effect of changes in sample size and each model specification. Results. Results showed that doubling the sample size decreased RMSE by on average 0.012. Effects of other model specifications were smaller but can when combined compensate for loss in prediction accuracy from a small sample size. For models using TTO data only, 8-parameter models clearly outperformed 20-parameter models. Adding a random intercept, or including DCE responses, also improved mean RMSE, most prominently for variants of the 20-parameter models. Conclusions. The prediction accuracy impact of further increases in sample size after 300 to 500 were smaller than the impact of combining alternative modeling choices. Hybrid modeling, use of constrained models, and inclusion of random intercepts all substantially improve the expected prediction accuracy. Beyond a minimum of 300 to 500 respondents, the sample size may be better informed by other considerations, such as legitimacy and representativeness, than by the technical prediction accuracy achievable. HIGHLIGHTS: Increases in sample size beyond a minimum in the range of 300 to 500 respondents provide smaller gains in expected prediction accuracy than alternative modeling approaches. Constrained, nonlinear models; time tradeoff + discrete choice experiment hybrid modeling; and including a random intercept all improved the prediction accuracy of models estimating values for the EQ-5D-5L based on data from 3 different valuation studies. The tested modeling choices can compensate for smaller sample sizes.