Objectives: The introduction of innovative, high-cost oncology treatments, coupled with mounting budgetary pressures, necessitates value trade-offs across cancer types. Defining value is critical to informing decision-making. A cost-value analysis tool was used to assess relative clinical value from a US perspective using multiple outcome metrics for a variety of metastatic cancers.
Methods: Literature published (January 1, 2000–August 31, 2016) was reviewed to identify outcome metrics for approved treatments for metastatic cancers. Data were extracted or derived for median and mean overall survival (OS), landmark survival rates, and other survival metrics, and compared across treatments vs their respective trial comparators, with and without considering costs.
Results: Reported survival metrics varied by agent within cancer type. For treatment of prostate cancer, abiraterone yielded the highest improvement in 1-year survival rate (13.7%, previously treated), whereas enzalutamide yielded the highest median OS improvement (4.8 months, previously treated) and sipuleucel-T, the highest mean OS improvement (3.6 months, previously untreated) vs their respective trial comparators. For treatment of non-small cell lung cancer vs their respective trial comparators, nivolumab yielded the highest improvement in mean OS (11.9 months) and 3-year survival rate (12.6%), each in previously treated squamous disease, whereas afatinib yielded the highest median OS improvement (4.1 months, previously untreated EGFR del19  and L858R  mutants). Cost-value analysis results varied with the applied survival metric.
Conclusions: Although median OS is the traditional gold standard oncology efficacy metric, it fails to capture long-term survival benefits—the ultimate goal of cancer treatment—offered by new treatment modalities. Diverse metrics are needed for comprehensive value assessments of cancer therapies.
Keywords: value framework, value assessment, immuno-oncology, cost-value analysis

摘要视频链接：Relative value assessment