title: Cost-effectiveness of treatment strategies for BRAF-mutated metastatic melanoma.
abstract: OBJECTIVE
  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.
  
  
  CONCLUSIONS
  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.
authors.affiliation: University of California San Francisco, San Francisco, California, United States of America.
authors.forename: Patti
authors.initials: P
authors.lastname: Curl
authors.affiliation: University of California San Francisco, San Francisco, California, United States of America; The Rudolfstiftung Hospital, Vienna, Austria.
authors.forename: Igor
authors.initials: I
authors.lastname: Vujic
authors.affiliation: UCSF Helen Diller Family Comprehensive Cancer Center, San Francisco, California, United States of America.
authors.forename: Laura J
authors.initials: LJ
authors.lastname: van 't Veer
authors.affiliation: UCSF Helen Diller Family Comprehensive Cancer Center, San Francisco, California, United States of America.
authors.forename: Susana
authors.initials: S
authors.lastname: Ortiz-Urda
authors.affiliation: University of California San Francisco, San Francisco, California, United States of America.
authors.forename: James G
authors.initials: JG
authors.lastname: Kahn
mesh_terms: D000970:Antineoplastic Agents / Q000191:economics* / Q000627:therapeutic use*; D003362:Cost-Benefit Analysis; D006801:Humans; D008545:Melanoma / Q000175:diagnosis / Q000188:drug therapy* / Q000235:genetics / Q000473:pathology*; D015233:Models, Statistical; D009154:Mutation; D009362:Neoplasm Metastasis; D011379:Prognosis; D048493:Proto-Oncogene Proteins B-raf / Q000235:genetics*; D011788:Quality of Life; D016896:Treatment Outcome
chemical_list: D000970:Antineoplastic Agents; C482119:BRAF protein, human; D048493:Proto-Oncogene Proteins B-raf