Cost-effectiveness of ambroxol in the treatment of Gaucher disease type 2.

Autor:	Milosavljević MN; Department of Pharmacology and Toxicology, Faculty of Medical Sciences, University of Kragujevac, 34000, Kragujevac, Serbia., Gutić M; Department of Pharmacology and Toxicology, Faculty of Medical Sciences, University of Kragujevac, 34000, Kragujevac, Serbia., Janjić V; Department of Psychiatry, Faculty of Medical Sciences, University of Kragujevac, 34000, Kragujevac, Serbia., Veselinović S; Department of Communication Skills, Ethics and Psychology, Faculty of Medical Sciences, University of Kragujevac, 3400, Kragujevac, Serbia., Djordjić M; Department of Communication Skills, Ethics and Psychology, Faculty of Medical Sciences, University of Kragujevac, 3400, Kragujevac, Serbia., Ivanović R; University Hospital Foča, 73300, Foča, Republic of Srpska, Bosnia and Herzegovina.; Faculty of Medicine in Foča, University of East Sarajevo, 73300, Foča, Republic of Srpska, Bosnia and Herzegovina., Milosavljević J; Department of Anatomy, Faculty of Medical Sciences, University of Kragujevac, 34000, Kragujevac, Serbia., Janković SM; Department of Pharmacology and Toxicology, Faculty of Medical Sciences, University of Kragujevac, 34000, Kragujevac, Serbia.
Jazyk:	angličtina
Zdroj:	Open medicine (Warsaw, Poland) [Open Med (Wars)] 2024 May 21; Vol. 19 (1), pp. 20240970. Date of Electronic Publication: 2024 May 21 (Print Publication: 2024).
DOI:	10.1515/med-2024-0970
Abstrakt:	Objective: Our aim was to compare the costs and efficacy of ambroxol in combination with imiglucerase with the costs and efficacy of imiglucerase only in the treatment of Gaucher disease type 2 (GD2) in the socio-economic settings of the Republic of Serbia, an upper-middle-income European economy. Methods: The perspective of the Serbian Republic Health Insurance Fund was chosen for this study, and the time horizon was 6 years. The main outcomes of the study were quality-adjusted life years gained with ambroxol + imiglucerase and comparator, and direct costs of treatment. The study was conducted through the generation and simulation of the Markov chain model. The model results were obtained after Monte Carlo microsimulation of a sample with 1,000 virtual patients. Results: Treatment with ambroxol in combination with imiglucerase was cost-effective when compared with imiglucerase only and was associated with positive values of net monetary benefit regardless of the onset of the disease. Such beneficial result for ambroxol and imiglucerase combination is primarily driven by the low cost of ambroxol and its considerable clinical effectiveness in slowing the progression of neural complications of GD2. Conclusion: If ambroxol and imiglucerase are used in combination for the treatment of GD2, it is more cost-effective than using imiglucerase alone. Competing Interests: Conflict of interest: MNM serves as Editor for Open Medicine, but it did not affected peer-review process. There is no other conflict of interest. (© 2024 the author(s), published by De Gruyter.)
Databáze:	MEDLINE
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