Cost-utility Analysis of Dornase alfa in comparison with Tobramycin for Managing Iranian Patients with Cystic Fibrosis
Abstract
Background: Cystic fibrosis (CF) is a progressive, life-threatening, and autosomal disease, which is most prevalent in Europe, North America, and Australia. This genetic disorder does not have a curative treatment, and current medications to manage CF including Tobramycin, are associated with high economic costs to the healthcare system, and noticeable out-of-pocket and time costs to patients and their families. Dornase alfa, is a recombinant human DNAse, and is an advanced therapeutic intervention. It has been indicated in clinical trials that Dornase alfa elevates FEV1 in CF patients, and enhances lung function. Therefore, this study was designed to evaluate the cost-utility of Dornase alfa compared with Tobramycin in the management of CF patients’ symptoms. Methods: A Markov model was developed based on previous similar studies to determine the cost-utility of Dornase alfa in comparison with Tobramycin. The time horizon of the model and the length of the cycles were assumed 10 years and 28 days, respectively. Results: Based on the cost-utility analysis, from the health system's perspective, Dornase alfa indicated 0.174 higher effectiveness in patients’ QALY than Tobramycin, and resulted in 27,517,260 Rials reduction in costs in the time horizon of 10 years. Conclusion: It is concluded that Dornase alfa could be considered as a cost-effective strategy in the treatment of CF patients associated with increased benefits compared to Tobramycin.Lieu, T.A., et al., The cost of medical care for patients with cystic fibrosis in a health maintenance organization. Pediatrics, 1999. 103(6): p. e72.
Elborn, J.S., Cystic fibrosis. Lancet, 2016. 388(10059): p. 2519-2531.
Ratjen, F. and G. Döring, Cystic fibrosis. Lancet, 2003. 361(9358): p. 681-9.
Modaresi, M.R., et al., Cystic fibrosis prevalence among a group of high-risk children in the main referral children hospital in Iran. J Educ Health Promot, 2017. 6: p. 54.
Elborn, J.S., et al., Comparison of Inhaled Antibiotics for the Treatment of Chronic Pseudomonas aeruginosa Lung Infection in Patients With Cystic Fibrosis: Systematic Literature Review and Network Meta-analysis. Clin Ther, 2016. 38(10): p. 2204-2226.
Guerriere, D.N., et al., Economic burden of ambulatory and home-based care for adults with cystic fibrosis. Treat Respir Med, 2006. 5(5): p. 351-9.
Armeni, P., et al., Reflections on the Importance of Cost of Illness Analysis in Rare Diseases: A Proposal. Int J Environ Res Public Health, 2021. 18(3).
Huot, L., et al., Evolution of costs of care for cystic fibrosis patients after clinical guidelines implementation in a French network. J Cyst Fibros, 2008. 7(5): p. 403-8.
Castellani, C., et al., ECFS best practice guidelines: the 2018 revision. J Cyst Fibros, 2018. 17(2): p. 153-178.
Mogayzel, P.J., Jr., et al., Cystic fibrosis pulmonary guidelines. Chronic medications for maintenance of lung health. Am J Respir Crit Care Med, 2013. 187(7): p. 680-9.
Narayanan, S., et al., Adherence to therapies in cystic fibrosis: a targeted literature review. Expert Rev Respir Med, 2017. 11(2): p. 129-145.
Smyth, A.R., et al., European Cystic Fibrosis Society Standards of Care: Best Practice guidelines. J Cyst Fibros, 2014. 13 Suppl 1: p. S23-42.
Amin, R., et al., The effect of dornase alfa on ventilation inhomogeneity in patients with cystic fibrosis. Eur Respir J, 2011. 37(4): p. 806-12.
Konstan, M.W., et al., Clinical use of dornase alpha is associated with a slower rate of FEV1 decline in cystic fibrosis. Pediatr Pulmonol, 2011. 46(6): p. 545-53.
McKenzie, S.G., et al., Dornase alfa is well tolerated: data from the epidemiologic registry of cystic fibrosis. Pediatr Pulmonol, 2007. 42(10): p. 928-37.
Najafi, B., et al., Cost effectiveness analysis of Avonex and CinnoVex in Relapsing Remitting MS. Glob J Health Sci, 2014. 7(2): p. 139-47.
Tappenden, P., et al., The cost effectiveness of dry powder antibiotics for the treatment of Pseudomonas aeruginosa in patients with cystic fibrosis. Pharmacoeconomics, 2014. 32(2): p. 159-72.
Schechter, M.S., et al., Inhaled aztreonam lysine versus inhaled tobramycin in cystic fibrosis. An economic evaluation. Ann Am Thorac Soc, 2015. 12(7): p. 1030-8.
Panguluri, S., et al., Economic Evaluation of Tobramycin Inhalation Powder for the Treatment of Chronic Pulmonary Pseudomonas aeruginosa Infection in Patients with Cystic Fibrosis. Clin Drug Investig, 2017. 37(8): p. 795-805.
Abdoli, G., Estimation of social discount rate for Iran. JOER, 2009. 9(34): p. 135-156.
Yang, C.L., et al., Dornase alfa for cystic fibrosis. Paediatr Respir Rev, 2017. 21: p. 65-67
| Files | ||
| Issue | Vol 9 No 1 (2023) | |
| Section | Original Article(s) | |
| DOI | https://doi.org/10.18502/jppm.v9i1.12882 | |
| Keywords | ||
| Cystic Fibrosis Dornase alfa Tobramycin Economic evaluation | ||
| Rights and permissions | |
|
This work is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License. |
