OBJECTIVES: Because of heterogeneity of treatment effect, decisions based on average measures of cost-effectiveness may lead to incorrect recommendations for specific subsets of the population. This study examined whether the Institute for Clinical and Economic Review (ICER), a leading advocate for wider use of value-based pricing in the U.S., accounts for patient heterogeneity in their assessments.

METHODS: We conducted a systematic review of 8 ICER reports on treatments for a range of conditions (major depressive disorder [MDD], rheumatoid arthritis [RA], ulcerative colitis, high cholesterol, anemia in chronic kidney disease [CKD], and 3 cancers). Within each report we identified the number and type of subgroups examined in CEA along with heterogeneity factors or subgroups mentioned elsewhere in the report but not examined in the CEA.

RESULTS: While a variety of potential heterogeneity factors were mentioned in each ICER report, assessment of subgroups within the CEA was typically limited to factors where high-quality trial evidence supported efficacy parameters, either directly or via meta-analysis. Two reports (MDD and RA) did not examine any subgroup in their CEA, 4 reports (all cancers and ulcerative colitis) presented CEA for subgroups defined by 1 factor, and 2 recent reports (high cholesterol and CKD) examined subgroups by 2-3 factors. Examples of heterogeneity factors assessed in CEA included cancer mutation or grade, biologic-naïve vs. experienced, and dialysis-dependent vs. independent. Prices meeting cost-per-QALY thresholds were sometimes reported by these subgroups, but budget-threshold prices were not.

CONCLUSIONS: Given existing data limitations, ICER recommendations are unlikely to reflect a clinically nuanced, patient-centered value assessment. There is urgent need of a national discussion around the limitations of value assessment when heterogeneity is known to exist but data are incomplete, as well as improving data generation and integration of patient subgroups in cost-effectiveness analysis and value-based pricing, with important implications for equity.