Abstract
PurposeGenetically-targeted therapies are both promising and costly advances in the field of oncology. Several treatments for metastatic melanoma with a mutation in the BRAF gene have been approved. They extend life but are more expensive than the previous standard of care (dacarbazine). Vemurafenib, the first drug in this class, costs $13,000 per month ($207,000 for a patient with median survival). Patients failing vemurafenib are often given ipilimumab, an immunomodulator, at $150,000 per course. Assessment of cost-effectiveness is a valuable tool to help navigate the transition toward targeted cancer therapy.MethodsWe performed a cost-utility analysis to compare three strategies for patients with BRAF+ metastatic melanoma using a deterministic expected-value decision tree model to calculate the present value of lifetime costs and quality-adjusted life years (QALYs) for each strategy. We performed sensitivity analyses on all variables.ResultsIn the base case, the incremental cost-effectiveness ratio (ICER) for vemurafenib compared with dacarbazine was $353,993 per QALY gained (0.42 QALYs added, $156,831 added). The ICER for vemurafenib followed by ipilimumab compared with vemurafenib alone was $158,139. In sensitivity analysis, treatment cost had the largest influence on results: the ICER for vemurafenib versus dacarbazine dropped to $100,000 per QALY gained with a treatment cost of $3600 per month.ConclusionThe cost per QALY gained for treatment of BRAF+ metastatic melanoma with vemurafenib alone or in combination exceeds widely-cited thresholds for cost-effectiveness. These strategies may become cost-effective with lower drug prices or confirmation of a durable response without continued treatment.
Highlights
Following advances in understanding of the genetics of tumor growth, several genetic testing-guided metastatic melanoma treatments have entered the market in recent years
In the base case, the incremental cost-effectiveness ratio (ICER) for vemurafenib compared with dacarbazine was $353,993 per qualityadjusted life years (QALYs) gained (0.42 QALYs added, $156,831 added)
Treatment cost had the largest influence on results: the ICER for vemurafenib versus dacarbazine dropped to $100,000 per QALY gained with a treatment cost of $3600 per month
Summary
Following advances in understanding of the genetics of tumor growth, several genetic testing-guided metastatic melanoma treatments have entered the market in recent years. Vemurafenib, the first drug targeting BRAF-mutated melanoma, has become increasingly popular, and others have been developed in the wake of its success [1] These therapies improve outcomes, but are expensive. Vemurafenib was approved for the treatment of BRAF-mutated melanoma in 2012 after a phase III trial that reported a 0.37 hazard ratio for death in patients with BRAF mutations taking vemurafenib compared with dacarbazine, and a phase II trial showing 15.9 month median survival [8,9]. The current cost of vemurafenib is $13,000 per month (for a total of $207,000 for a patient with median survival), and the cobas 4800 BRAF V600 Mutation costs $150 [5,9] Both dabrafenib ($9100 per month) and trametinib ($10,400 per month) were approved in 2013 based on improvement in progression-free survival compared to chemotherapy [5,10,11]
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