Cost Effectiveness of Inhaled Mannitol (Bronchitol®) in Patients with Cystic Fibrosis

• Published in: PharmacoEconomics 2019-03, Vol.37 (3), p.435-446
• Main Authors: Warren, Emma, Morgan, Kristen, Toward, Toby J., Schwenkglenks, Matthias, Leadbetter, Joanna
• Format: Article
• Language: English
• Subjects: Administration, Inhalation; Adolescent; Adult; Australia; Body mass index; Care and treatment; Child; Clinical medicine; Cost analysis; Cost benefit analysis; Costs; Cystic fibrosis; Cystic Fibrosis - drug therapy; Cystic Fibrosis - economics; Deoxyribonuclease I - administration & dosage; Diagnosis; Disease; Dosage and administration; Female; Forced Expiratory Volume; Health Administration; Health Economics; Health technology assessment; Hospital costs; Humans; Infections; Life expectancy; Lung transplants; Male; Mannitol; Mannitol - administration & dosage; Mannitol - economics; Markov Chains; Medicine; Medicine & Public Health; Methods; Middle Aged; Mortality; Original Research Article; Palliative care; Patients; Pharmacoeconomics and Health Outcomes; Public Health; Quality of life; Quality of Life Research; Quality-Adjusted Life Years; Recombinant Proteins - administration & dosage; Simulated patients; Studies; Transplants & implants; Willingness to pay; Young Adult
• ISSN: 1170-7690; 1179-2027
• DOI: 10.1007/s40273-019-00767-8

Background Inhaled mannitol (Bronchitol ® ) is licensed in Australia as a safe and efficacious addition to best supportive care in patients with cystic fibrosis. Objective The objective of this study was to assess the cost effectiveness of inhaled mannitol (in addition to best supportive care) in the Australian setting from the perspective of a government-funded national healthcare system. Methods A probabilistic patient-level simulation Markov model estimated life-time costs and outcomes of mannitol when added to best supportive care, compared with best supportive care alone in patients aged 6 years and older. We estimated treatment-related inputs (initial change in percentage of predicted forced expiratory volume, relative reduction in severe pulmonary exacerbations, and treatment discontinuations) from two phase III trials. Longer term natural history rates of predicted forced expiratory volume decline over time and severe pulmonary exacerbation rates for best supportive care were taken from Australian CF registries. The utility value for the cystic fibrosis health state was as measured in the trials using the Health Utility Index, whereas the impact of pulmonary exacerbations and lung transplantation on utility was ascertained from the published literature. The underlying cost of managing cystic fibrosis, and the cost associated with pulmonary exacerbations and transplantations was taken from published Australian sources. Results The addition of inhaled mannitol to best supportive care resulted in a discounted cost per quality-adjusted life-year of AU$39,165. The result was robust with 77% of probabilistic sensitivity analysis samples below a willingness-to-pay threshold of AU$45,000/quality-adjusted life-year. Conclusion Benchmarked against an implicit Australian willingness-to-pay threshold for life-threatening diseases, our model suggests inhaled mannitol provides a cost-effective addition to best supportive care in patients with cystic fibrosis, irrespective of concomitant dornase alfa use.