title: Forecasting US ivacaftor outcomes and cost in cystic fibrosis patients with the G551D mutation.
abstract: Ivacaftor, a breakthrough treatment for cystic fibrosis (CF) patients with the G551D genetic mutation, lacks long-term clinical and cost projections. This study forecasted outcomes and cost by comparing ivacaftor plus usual care versus usual care alone.A lifetime Markov model was conducted from a US payer perspective. The model consisted of five health states: 1) forced expiratory volume in 1 s (FEV1) % pred ≥70%, 2) 40%≤ FEV1 % pred <70%, 3) FEV1 % pred <40%, 4) lung transplantation and 5) death. All inputs were extracted from published literature. Budget impact was also estimated. We estimated ivacaftor's improvement in outcomes compared with a non-CF referent population.Ivacaftor was associated with 18.25 (95% credible interval (CrI) 13.71-22.20) additional life-years and 15.03 (95% CrI 11.13-18.73) additional quality-adjusted life-years (QALYs). Ivacaftor was associated with improvements in survival and QALYs equivalent to 68% and 56%, respectively, for the survival and QALY gaps between CF usual care and their non-CF peers. The incremental lifetime cost was $3 374 584. The budget impact was $0.087 per member per month.Ivacaftor increased life-years and QALYs in CF patients with the G551D mutation, and moved morbidity and mortality closer to that of their non-CF peers. Ivacaftor costs much more than usual care, but comes at a relatively limited budget impact.
authors.affiliation: Center of Pharmaceutical Outcomes Research, Dept of Pharmacy Practice, Faculty of Pharmaceutical Sciences, Naresuan University, Muang, Phitsanulok, Thailand Center for Pharmaceutical Outcomes Research, Skaggs School of Pharmacy and Pharmaceutical Sciences, University of Colorado Anschutz Medical Campus, Aurora, CO, USA.
authors.forename: Piyameth
authors.initials: P
authors.lastname: Dilokthornsakul
authors.affiliation: Pharmaceutical Outcomes Research and Policy Program, School of Pharmacy, University of Washington, Seattle, WA, USA.
authors.forename: Ryan N
authors.initials: RN
authors.lastname: Hansen
authors.affiliation: Center for Pharmaceutical Outcomes Research, Skaggs School of Pharmacy and Pharmaceutical Sciences, University of Colorado Anschutz Medical Campus, Aurora, CO, USA Jon.campbell@ucdenver.edu.
authors.forename: Jonathan D
authors.initials: JD
authors.lastname: Campbell
mesh_terms: D000627:Aminophenols / Q000191:economics / Q000627:therapeutic use*; D015331:Cohort Studies; D003198:Computer Simulation; D003362:Cost-Benefit Analysis; D003550:Cystic Fibrosis / Q000188:drug therapy* / Q000191:economics / Q000235:genetics*; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000235:genetics; D004358:Drug Therapy / Q000639:trends; D005541:Forced Expiratory Volume / Q000187:drug effects*; D005544:Forecasting; D006304:Health Status; D006801:Humans; D016040:Lung Transplantation; D008390:Markov Chains; D009010:Monte Carlo Method; D009154:Mutation; D019057:Quality-Adjusted Life Years; D015363:Quinolones / Q000191:economics / Q000627:therapeutic use*; D012129:Respiratory Function Tests; D016896:Treatment Outcome; D014481:United States
chemical_list: D000627:Aminophenols; C505032:CFTR protein, human; D015363:Quinolones; D019005:Cystic Fibrosis Transmembrane Conductance Regulator; C545203:ivacaftor