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Background: Mapping Patient-Reported Outcomes Measurement Information System-Global Health (PROMIS-GH) to EuroQol 5-dimension, three-level version (EQ-5D-3L) provides a utility score for use in quality-of-life and cost-effectiveness analyses. In 2009, Revicki et al. mapped the PROMIS-GH items to EQ-5D-3L utilities using linear regression (REVReg). More recently, regression was shown to be ill-suited for mapping to preference-based measures due to regression to the mean. Linear and equipercentile equating are alternative mapping methods that avoid the issue of regression to the mean. Another limitation of the prior models is that ordinal predictors were treated as continuous.Methods: Using data collected from the PROMIS Wave 1 sample, we refit REVReg, treating the PROMIS-GH items as categorical variables (CATReg). We applied linear and equipercentile equating to the REVReg model (REVLE, REVequip) and the CATReg model (CATLE, CATequip). We validated and compared the predictive accuracy of these models in a large sample of neurological patients at a single tertiary-care hospital.Results: In the neurological disease patient sample, CATLE produced the strongest correlations between estimated and observed EQ-5D-3L scores and had the lowest mean squared error. The CATequip model had the lowest mean absolute error and had estimated scores that best matched the overall distribution of observed scores.Conclusions: Using linear and equipercentile equating, we created new models mapping PROMIS-GH items to EQ-5D-3L utility scores. EQ-5D-3L utility scores can be more accurately estimated using our models for use in cost-effectiveness studies or studies examining overall health-related quality of life. [ABSTRACT FROM AUTHOR]

Objectives: Reexamine cost-effectiveness of riluzole in the treatment of amyotrophic lateral sclerosis (ALS) in light of recent advances in disease staging and understanding of stage-specific drug effect.Methods: ALS was staged according to the "fine'til 9" (FT9) staging method. Stage-specific health utilities (EQ-5D, US valuation) were estimated from an institutional cohort, whereas literature informed costs and transition probabilities. Costs at 2018 prices were disaggregated into recurring costs (RCs) and "one-off" transition/"tollgate" costs (TCs). Five- and 10-year horizons starting in stage 1 disease were examined from healthcare sector and societal perspectives using Markov models to evaluate riluzole use, at a threshold of $100 000/quality-adjusted life year (QALY). Probabilistic and deterministic sensitivity analyses were conducted.Results: Mean EQ-5D utilities for stages 0 to 4 were 0.79, 0.74, 0.63, 0.54, and 0.46, respectively. From the healthcare sector perspective at the 5-year horizon, riluzole use contributed to 0.182 QALY gained at the cost difference of $12 348 ($5403 riluzole cost, $8870 RC and -$1925 TC differences), translating to an incremental cost-effectiveness ratio (ICER) of $67 658/QALY. Transition probability variation contributed considerably to ICER uncertainty (-30.2% to +90.0%). ICER was sensitive to drug price and RCs, whereas higher TCs modestly reduced ICER due to delayed tollgates.Conclusion: This study provides a framework for health economic studies of ALS treatments using FT9 staging. Prospective stage-specific and disaggregated cost measurement is warranted for accurate future cost-effectiveness analyses. Appropriate separation of TCs from RCs substantially mitigates the high burden of background cost of care on the ICER. [ABSTRACT FROM AUTHOR]