Comparison of Statistical Models to Address Ceiling Effect in Utility Scores: A Simulation Study

Author(s)

Pandey S1, Singh B2, Kaur S1, Sharma A3
1Heorlytics Private Limited, Mohali, India, 2Pharmacoevidence Pvt. Ltd., SAS Nagar Mohali, PB, India, 3Pharmacoevidence Pvt. Ltd., SAS Nagar, Mohali, India

Presentation Documents

OBJECTIVES: To assess the preference-based health status, the EQ-5D index is preferable and is a widely used instrument that could serve as the basis for summarizing and comparing health outcomes. Various regression models have been used to generate the utility estimate for the economic models from patient-level utility scores. However, some of the regression models do not perform well in the case of the ceiling effect. The research aimed to compare the different regression models in case of a heavy ceiling effect in the EQ-5D scores.

METHODS: The data for EQ-5D scores and covariates were simulated in R (v4.0.2) with an assumption that 75% of patients had the EQ-5D scores of 1. Different regression models such as Ordinary Least Square (OLS), Tobit/Censored (around mean/median), Beta regression and its extensions (bias correction and bias reduction), Two-part model (TPM) with OLS, TPM with Tobit, Log-transformed TPM with OLS and Log-transformed TPM with Tobit were fitted on EQ-5D scores to generate the coefficient associated with covariates. The predictive accuracy/goodness-of-fit of all the models was compared using absolute and squared errors, that are, R1 and R2.

RESULTS: Log-transformed TPM with OLS performed best on EQ-5D data with a heavy ceiling effect, having predictive accuracy metric R1 (-24.39) and R2 (-444.36) followed by Log-transformed TPM with Tobit (R1=-22.98, R2=-395.88). The performance of the two-part models (with OLS/Tobit) (R2=-73.36) was superior to the Tobit model around mean (R2=-5.71) and median (R2=-3.13), followed by OLS (R2=0.18), whereas the beta regression and its extensions did not perform well.

CONCLUSIONS: The log-transformed TPM model with OLS was superior followed by log-transformed TPM with Tobit to all the other approaches in handling data with heavy ceiling effects.

Conference/Value in Health Info

2022-11, ISPOR Europe 2022, Vienna, Austria

Value in Health, Volume 25, Issue 12S (December 2022)

Code

MSR115

Topic

Methodological & Statistical Research, Patient-Centered Research

Topic Subcategory

Health State Utilities, PRO & Related Methods

Disease

No Additional Disease & Conditions/Specialized Treatment Areas

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