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Cost-effectiveness of miglustat versus symptomatic therapy of Niemann–Pick disease type C

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Abstract

Background

Niemann–Pick disease type C (NP-C) is a progressive neurodegenerative disorder with early infantile (< 2 years), late infantile (2–6 years), juvenile (7–15 years) and adolescent (> 15 years) onset. The mainstay of therapy for NP-C patients with neurological symptoms is miglustat, a drug that may modify the course of the disease.

Aim

Our aim was to evaluate the cost-effectiveness of miglustat in comparison to symptomatic therapy in patients with NP-C in the socio-economic settings of the Republic of Serbia, an upper-middle-income European economy.

Method

The perspective of the Serbian Republic Health Insurance Fund was chosen for this study, and the time horizon was eighty years. The main outcomes of the study were quality-adjusted life years gained with miglustat and comparator, and direct costs of treatment. The study was conducted through the generation and simulation of the Discrete-Event Simulation model. The model results were obtained after Monte Carlo microsimulation of a sample with 1000 virtual patients.

Results

Treatment with miglustat was not cost-effective when compared with symptomatic therapy and was associated with negative values of net monetary benefit regardless of the onset of neurological manifestations (− 110,447,627.00 ± 701,614.00 RSD, − 343,871,695.00 ± 2,577,441.00 RSD, − 1,397,908,502.00 ± 23,084,235.00 RSD and − 2,953,680,879.00 ± 33,297,412.00 RSD) for early infantile, late infantile, juvenile and adolescent cohorts, respectively).

Conclusion

When traditional pharmacoeconomic evaluation is employed, miglustat is not a cost-effective option in comparison to symptomatic therapy for the treatment of NP-C. However, given the proven efficacy of miglustat, there is a need to find ways to make this drug available to all patients with NP-C.

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Acknowledgements

The authors would like to thank dr Žan Friščić for his assistance with English grammar and syntax editing.

Funding

This study was partially financed by grant No 175007, given by the Serbian Ministry of Education, Science and Technological Development.

Conflicts of interest

The authors have no conflicts of interest to declare.

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Authors and Affiliations

  1. Department of Pharmacology and Toxicology, Faculty of Medical Sciences, University of Kragujevac, Svetozara Markovića 69, 34000, Kragujevac, Serbia

    Medo Gutić, Miloš N. Milosavljević & Slobodan M. Janković

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  1. Medo Gutić
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Correspondence to Miloš N. Milosavljević.

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Gutić, M., Milosavljević, M.N. & Janković, S.M. Cost-effectiveness of miglustat versus symptomatic therapy of Niemann–Pick disease type C. Int J Clin Pharm 44, 1442–1453 (2022). https://doi.org/10.1007/s11096-022-01491-8

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