title: Nilotinib versus dasatinib as second-line therapy in patients with Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase who are resistant or intolerant to imatinib: a cost-effectiveness analysis based on real-world data.
abstract: OBJECTIVE
  To evaluate the cost-effectiveness of second-line nilotinib vs dasatinib among patients with Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP) who are resistant or intolerant to imatinib, from a US third-party perspective.
  
  
  METHODS
  A lifetime partitioned survival model was developed to compare the costs and effectiveness of nilotinib vs dasatinib, which included four health states: CP on treatment, CP post-discontinuation, progressive disease (accelerated phase [AP] or blast crisis [BC]), and death. Time on treatment, progression-free survival, and overall survival of nilotinib and dasatinib were estimated using real-world comparative effectiveness data. Parametric survival models were used to extrapolate outcomes beyond the study period. Drug treatment costs, medical costs, and adverse event costs were obtained from the literature and publicly available databases. Utilities of health states were derived from the literature. Incremental cost-effectiveness ratios, including incremental cost per life-year (LY) gained and incremental cost per quality-adjusted life-year (QALY) gained, were estimated comparing nilotinib and dasatinib. Deterministic sensitivity analyses were performed by varying patient characteristics, cost, and utility inputs.
  
  
  RESULTS
  Over a lifetime horizon, nilotinib-treated patients were associated with 11.7 LYs, 9.1 QALYs, and a total cost of $1,409,466, while dasatinib-treated patients were associated with 9.5 LYs, 7.3 QALYs, and a total cost of $1,422,122. In comparison with dasatinib, nilotinib was associated with better health outcomes (by 2.2 LYs and 1.9 QALYs) and lower total costs (by $12,655). Deterministic sensitivity analysis results showed consistent findings in most scenarios.
  
  
  CONCLUSIONS
  In the absence of long-term real-world data, the lifetime projection could not be validated.
  
  
  CONCLUSIONS
  Compared with dasatinib, second-line nilotinib was associated with better life expectancy, better quality-of-life, and lower costs among patients with Ph+ CML-CP who were resistant or intolerant to imatinib.
authors.affiliation: a Analysis Group , Boston , MA , USA.
authors.forename: Nanxin
authors.initials: N
authors.lastname: Li
authors.affiliation: a Analysis Group , Boston , MA , USA.
authors.forename: Xi
authors.initials: X
authors.lastname: Yang
authors.affiliation: a Analysis Group , Boston , MA , USA.
authors.forename: Liangyi
authors.initials: L
authors.lastname: Fan
authors.affiliation: a Analysis Group , Boston , MA , USA.
authors.forename: Todor
authors.initials: T
authors.lastname: Totev
authors.affiliation: b Analysis Group , Montreal , QC , Canada.
authors.forename: Annie
authors.initials: A
authors.lastname: Guerin
authors.affiliation: c Novartis Pharmaceuticals Corporation , East Hanover , NJ , USA.
authors.forename: Lei
authors.initials: L
authors.lastname: Chen
authors.affiliation: d Novartis Healthcare Pvt. Ltd. , Hyderabad , India.
authors.forename: Subrata
authors.initials: S
authors.lastname: Bhattacharyya
authors.affiliation: c Novartis Pharmaceuticals Corporation , East Hanover , NJ , USA.
authors.forename: George
authors.initials: G
authors.lastname: Joseph
mesh_terms: D000368:Aged; D000970:Antineoplastic Agents / Q000009:adverse effects / Q000191:economics / Q000627:therapeutic use*; D003362:Cost-Benefit Analysis; D000069439:Dasatinib / Q000009:adverse effects / Q000191:economics / Q000627:therapeutic use*; D018572:Disease-Free Survival; D005252:Fees, Pharmaceutical; D005260:Female; D005102:Health Expenditures; D006296:Health Services / Q000191:economics / Q000706:statistics & numerical data; D006801:Humans; D000068877:Imatinib Mesylate; D015464:Leukemia, Myelogenous, Chronic, BCR-ABL Positive / Q000188:drug therapy*; D008297:Male; D008875:Middle Aged; D017059:Models, Econometric; D010677:Philadelphia Chromosome; D011743:Pyrimidines / Q000009:adverse effects / Q000191:economics / Q000627:therapeutic use*; D019057:Quality-Adjusted Life Years; D013997:Time Factors
chemical_list: D000970:Antineoplastic Agents; D011743:Pyrimidines; D000068877:Imatinib Mesylate; C498826:nilotinib; D000069439:Dasatinib
keywords: Cost-effectiveness; Philadelphia chromosome-positive; chronic myeloid leukemia; dasatinib; nilotinib; second-line