Cost-effectiveness of treatment strategies for BRAF-mutated metastatic melanoma

PLoS One. 2014 Sep 8;9(9):e107255. doi: 10.1371/journal.pone.0107255. eCollection 2014.


Purpose: Genetically-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.

Methods: We 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.

Results: In 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.

Conclusion: The 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.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Antineoplastic Agents / economics*
  • Antineoplastic Agents / therapeutic use*
  • Cost-Benefit Analysis*
  • Humans
  • Melanoma / diagnosis
  • Melanoma / drug therapy*
  • Melanoma / genetics
  • Melanoma / pathology*
  • Models, Statistical
  • Mutation*
  • Neoplasm Metastasis
  • Prognosis
  • Proto-Oncogene Proteins B-raf / genetics*
  • Quality of Life
  • Treatment Outcome


  • Antineoplastic Agents
  • BRAF protein, human
  • Proto-Oncogene Proteins B-raf