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

Autor: Jonathan D. Campbell, Piyameth Dilokthornsakul, Mausam Patidar
Rok vydání: 2017
Předmět:
Adult
medicine.medical_specialty
Pediatrics
Cystic Fibrosis
Combination therapy
Aminopyridines
Cystic Fibrosis Transmembrane Conductance Regulator
Quinolones
Aminophenols
Severity of Illness Index
Cystic fibrosis
Ivacaftor
03 medical and health sciences
chemistry.chemical_compound
0302 clinical medicine
Forced Expiratory Volume
medicine
Credible interval
Humans
In patient
Benzodioxoles
030212 general & internal medicine
Intensive care medicine
health care economics and organizations
business.industry
030503 health policy & services
Health Policy
Homozygote
Lumacaftor
Public Health
Environmental and Occupational Health
medicine.disease
Markov Chains
United States
Drug Combinations
Treatment Outcome
chemistry
Lung disease
Mutation
Mutation (genetic algorithm)
Quality-Adjusted Life Years
0305 other medical science
business
medicine.drug
Zdroj: Value in Health. 20:1329-1335
ISSN: 1098-3015
Popis: Objectives 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. Methods 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 1 ] >70%), 2) moderate lung disease (40% ≤ FEV 1 ≤ 70%), 3) severe lung disease (FEV 1 Results 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. Conclusions 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.
Databáze: OpenAIRE
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