PMU49 Patient Heterogeneity and the Limitations of One-Size-Fits-All Value-Based Pricing: A Systematic Review and Future Directions

Because of heterogeneity treatment effect, decisions based on average measures cost-effectiveness may lead to incorrect recommendations for specific subsets the population. This study examined whether Institute Clinical and Economic Review (ICER), a leading advocate wider use value-based pricing in U.S., accounts patient their assessments. We conducted systematic review 8 ICER reports treatments range conditions (major depressive disorder [MDD], rheumatoid arthritis [RA], ulcerative colitis, high cholesterol, anemia chronic kidney disease [CKD], 3 cancers). Within each report we identified number type subgroups CEA along with factors or mentioned elsewhere but not CEA. While variety potential were report, assessment within was typically limited where high-quality trial evidence supported efficacy parameters, either directly via meta-analysis. Two (MDD RA) did examine any subgroup CEA, 4 (all cancers colitis) presented defined by 1 factor, 2 recent (high cholesterol CKD) 2-3 factors. Examples assessed included cancer mutation grade, biologic-naïve vs. experienced, dialysis-dependent independent. Prices meeting cost-per-QALY thresholds sometimes reported these subgroups, budget-threshold prices not. Given existing data limitations, are unlikely reflect clinically nuanced, patient-centered value assessment. There is urgent need national discussion around limitations when known exist incomplete, as well improving generation integration analysis pricing, important implications equity.