Presentation (CTI)

OBJECTIVES: Cost-effectiveness (CE) analyses are receiving increased attention in the United States with the implementation of Medicare drug price negotiations. Historically, quality-adjusted life years (QALYs) have been standard in CE assessments; however, the Centers for Medicare and Medicaid Services (CMS) are prohibited from considering adjusted life year (LY) measures, including QALYs, in the assessment of a therapy’s value during pricing negotiations. Motivated by this policy shift and need for additional non-QALY measures, we reviewed recent Institute for Clinical and Economic Review (ICER) assessments to characterize previously employed alternative CE measures.
METHODS: 54 ICER reports published between 2019-2025 were extracted using a standardized grid. Model structure, analytic perspective, and incremental CE ratios were recorded. For each therapy, all cost inputs and CE ratio denominators (QALYs, LYs, and equal value LYs [evLYs]) were catalogued along with any alternative CE outcomes. Summary statistics were calculated for the frequency and values of each CE measure.
RESULTS: 52/54 reports published incremental CE ratios. Of those, all included $/QALY gained, 46/52 included $/LY gained, and 40/52 included $/evLY gained. 34/52 included at least one measure beyond $/QALY, LY, or evLY, including: Survival in improved health states: 17/52 (ex: $/progression-free survival month [multiple myeloma] or year without diabetes); events or resource utilization avoided: 12/52 (ex: $/myocardial infarction, stroke, or blood transfusion avoided); symptom management: 5/52 (ex: $/depression- or menopause symptom- free day).
CONCLUSIONS: While QALYs remain foundational in ICER’s cost-effectiveness modeling, evLYs and a variety of alternative, patient- and condition-specific measures are also reported. As CMS are prohibited from considering QALYs in pricing negotiations, understanding the range of alternative CE metrics used in ICER reports may inform and strengthen the evidence base for determining the fair value of a therapy. Further standardization of alternative CE measures could enhance their clarity and practical utility in future CE assessments and value negotiations.