Forecasting the Long-Term Clinical and Economic Outcomes of Lumacaftor/Ivacaftor in Cystic Fibrosis Patients with Homozygous phe508del Mutation

Dec 1, 2017, 00:00 AM
Section Order : 11
First Page : 1329


To forecast lifetime outcomes and cost of lumacaftor/ivacaftor combination therapy in patients with cystic fibrosis (CF) with homozygous phe508del mutation from the US payer perspective.


A lifetime Markov model was developed from a US payer perspective. The model included five health states: 1) mild lung disease (percent predicted forced expiratory volume in 1 second [FEV ] >70%), 2) moderate lung disease (40% ≤ FEV ≤ 70%), 3) severe lung disease (FEV 40%), 4) lung transplantation, and 5) death. All inputs were derived from published literature. We estimated lumacaftor/ivacaftor’s improvement in outcomes compared with a non-CF referent population as well as CF-specific mortality estimates.


Lumacaftor/ivacaftor was associated with additional 2.91 life-years (95% credible interval 2.55–3.56) and additional 2.42 quality-adjusted life-years (QALYs) (95% credible interval 2.10–2.98). Lumacaftor/ivacaftor was associated with improvements in survival and QALYs equivalent to 27.6% and 20.7%, respectively, for the survival and QALY gaps between CF usual care and their non-CF peers. The incremental lifetime cost was $2,632,249.


Lumacaftor/ivacaftor increased life-years and QALYs in CF patients with the homozygous phe508del mutation and moved morbidity and mortality closer to that of their non-CF peers but it came with higher cost.
HEOR Topics :
Tags :
  • cystic fibrosis
  • ivacaftor
  • lumacaftor
  • phe508del mutation
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