title: Review of US Comparative Economic Evidence for Treatment of Metastatic Renal Cell Carcinoma after Failure of First-Line VEGF Inhibitor Therapy.
abstract: BACKGROUND
  In 2006, the economic burden of metastatic renal cell carcinoma (mRCC) was estimated to be up to $1.6 billion worldwide and has since grown annually. With the continuing increase of the economic burden of this disease in the United States, there is a growing need for economic analyses to guide treatment and policy decisions for this patient population.
  
  
  OBJECTIVE
  To evaluate available comparative economic data on targeted therapies for patients with mRCC who have failed first-line targeted therapies.
  
  
  METHODS
  A broad and comprehensive literature review was conducted of US-based studies between January 1, 2005, and February 11, 2013, evaluating comparative economic evidence for targeted agents that are used as second-line therapy or beyond. Based on the specific search parameters that focused on cost-effectiveness and economic comparisons between vascular endothelial growth factor (VEGF)/VEGF receptor (VEGFr) inhibitors and mammalian target of rapamycin (mTOR) inhibitors, only 7 relevant, US-based economic evaluations were found appropriate for inclusion in the analysis. All authors, who are experts in the health economics and outcomes research field, reviewed the search results. Studies of interest were those with a targeted agent, VEGF/VEGFr or mTOR inhibitor, in at least 1 study arm.
  
  
  CONCLUSIONS
  As a group, targeted therapies were found to be cost-effective options in treating patients with refractory mRCC in the United States. Oral therapies showed an economic advantage over intravenous agents, presumably because oral therapies have a lower impact on outpatient resources. Based on 3 studies, everolimus has been shown to have an economic advantage over temsirolimus and to be cost-effective compared with sorafenib. No economic comparison between everolimus and axitinib, the only 2 drugs with a National Comprehensive Cancer Network category 1 recommendation for use after the failure of VEGFr tyrosine kinase inhibitors, is available.
  
  
  CONCLUSIONS
  The limited and heterogeneous sum of the currently available economic evidence does not allow firm conclusions to be drawn about the most cost-effective targeted treatment option in the second-line setting and beyond in patients with mRCC. It is hoped that ongoing head-to-head therapeutic trials and biomarker studies will help improve the economic efficiency of these expensive agents.
authors.affiliation: Dr Wong is Professor of Medicine, Keck School of Medicine, University of Southern California, Los Angeles, CA.
authors.forename: Michael K
authors.initials: MK
authors.lastname: Wong
authors.affiliation: Dr Wang is Director, Worldwide Health Outcomes, Value & Access, Novartis Pharmaceuticals.
authors.forename: Xufang
authors.initials: X
authors.lastname: Wang
authors.affiliation: Mr Chulikavit is Associate Director, LA-SER Analytica International, New York, NY.
authors.forename: Maruit J
authors.initials: MJ
authors.lastname: Chulikavit
authors.affiliation: Dr Liu is Director, Oncology US Health Economics & Outcomes Research, Novartis Pharmaceuticals, East Hanover, NJ.
authors.forename: Zhimei
authors.initials: Z
authors.lastname: Liu