title: Pharmacoeconomics of ruxolitinib therapy in patients with myelofibrosis.
abstract: BACKGROUND
  Overall survival (OS) and other important clinical trial end-points seem increasingly more elusive in supporting rapid and efficient incorporation of innovative cancer drugs in clinical practice. This study proposes a clinical trial based pharmacoeconomic framework to assess the therapeutic and economic value of ruxolitinib in patients with intermediate-2 or high-risk myelofibrosis.
  
  
  METHODS
  Individual patient level 144 week follow-up data from the COMFORT-II trial was used to account for the crossover effect on overall survival. Lifetime treatment benefits and costs were estimated considering detailed patterns of both ruxolitinib dose adjustments and blood transfusion needs.
  
  
  RESULTS
  The authors estimate a 3.3 years increment in life expectancy (HR = 0.30; 95% CI = 0.17-0.55; p-value <0.001) and an incremental cost-effectiveness ratio of €40,000 per life year gained with the use of ruxolitinib.
  
  
  CONCLUSIONS
  This study also demonstrates how valuable information from clinical trials can be used to support informed decisions about the early incorporation of innovative drugs.
authors.affiliation: a a Exigo Consultores , Lisbon , Portugal.
authors.forename: Björn
authors.initials: B
authors.lastname: Vandewalle
authors.affiliation: a a Exigo Consultores , Lisbon , Portugal.
authors.forename: Valeska
authors.initials: V
authors.lastname: Andreozzi
authors.affiliation: a a Exigo Consultores , Lisbon , Portugal.
authors.forename: João
authors.initials: J
authors.lastname: Almeida
authors.affiliation: a a Exigo Consultores , Lisbon , Portugal.
authors.forename: Jorge
authors.initials: J
authors.lastname: Félix
mesh_terms: D000970:Antineoplastic Agents / Q000191:economics* / Q000627:therapeutic use*; D003362:Cost-Benefit Analysis; D017850:Economics, Pharmaceutical; D006296:Health Services / Q000191:economics / Q000706:statistics & numerical data; D006801:Humans; D017059:Models, Econometric; D009570:Nitriles; D055728:Primary Myelofibrosis / Q000188:drug therapy* / Q000401:mortality; D047428:Protein Kinase Inhibitors / Q000191:economics / Q000627:therapeutic use; D011720:Pyrazoles / Q000191:economics* / Q000627:therapeutic use*; D011743:Pyrimidines; D011788:Quality of Life; D016032:Randomized Controlled Trials as Topic
chemical_list: D000970:Antineoplastic Agents; D009570:Nitriles; D047428:Protein Kinase Inhibitors; D011720:Pyrazoles; D011743:Pyrimidines; C540383:ruxolitinib
keywords: Cost-effectiveness; Individual level data; Myelofibrosis; Ruxolitinib