Challenges in Assessing the Cost-effectiveness of Cancer Immunotherapy
Angela K. Green
Abstract
examine the cost-effectiveness of pembrolizumab for the second-line treatment of patients with advanced hepatocellular carcinoma previously treated with sorafenib, on the basis of the results of the pivotal phase 3 trial KEYNOTE-240. This result was consistent even in the optimistic setting, which assumed that patients living beyond 30 months were cured with pembrolizumab. Using a 2-way sensitivity analysis, Chiang et al 1 conclude that to be cost-effective, the median survival benefit vs placebo would need to be longer than 12 months, instead of the benefit of 3.3 months demonstrated in KEYNOTE-240. 2 Alternatively, the list price of pembrolizumab could be reduced by more than half (58%) from $6915 to $2925 per cycle. 1 This work by Chiang et al 1 adds to a growing body of literature demonstrating high cancer drug costs relative to the benefits provided to patients treated on a large scale. There is a concern that such costs, given the widespread use of novel agents, such as immunotherapy, in oncology and an aging US demographic threaten the financial sustainability of our health care system. ior research has shown that the cost-effectiveness of immunotherapy varies widely depending on the therapeutic indication and the use of biomarkers, such as programmed death ligand 1 status. In diseases such as non-small cell lung cancer, where biomarkers are used to refine the patient selection, the number of patients treated is smaller and outcomes are improved. Thus,