https://orcid.org/0000-0001-8880-7092
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Abstract
Alpha-mannosidosis is an inherited rare disorder of mannose-containing oligosaccharides metabolism that is currently treated by enzyme replacement therapy (ERT), bone marrow transplantation (BMT), or supportive therapy (ST). However, the relative cost-effectiveness of these treatment options is yet unknown. Our study aimed to compare the cost-effectiveness of the treatment options for mild to moderate alpha-mannosidosis. The study is based on a modeling approach using a Discrete-Event Simulation model to generate and simulate the course of the disease under the influence of each of the treatment options: ERT, BMT, and ST. The model had a lifetime horizon and was made from the perspective of the Serbian Health Insurance Fund. Currently, available causal therapy of mild to moderate alpha-mannosidosis with velmanase alpha enzyme replacement is not cost-effective compared with supportive therapy (ICER = 941,587,152 RSD) or bone marrow transplantation (ICER = −398,412,755 RSD). Bone marrow transplantation can be cost-effective compared to supportive therapy (ICER = 6,032,689 RSD), but only if the willingness-to-pay threshold is increased to 9 gross domestic products (GDP) per capita per QALY gained. According to the current threshold, velmanase-alfa is not cost-effective compared to BMT or ST. To make alfa-mannosidosis therapy widely accessible to patients, criteria for assessing the cost-effectiveness of orphan drugs must include not only the absolute value of ICER but other aspects like equity weightings of QALYs, risk-sharing, reimbursement of severe forms of a disease only, or availability of dedicated funding.
Author contributions
Ana Antanasković: conceptualization, data curation, writing—original draft preparation, visualization, investigation, writing—reviewing and editing, resources.
Ivana Stević: conceptualization, methodology, software, formal analysis, data curation, writing—original draft preparation, visualization, investigation, validation, writing—reviewing and editing, funding acquisition.
Refet Gojak: data curation, writing—original draft preparation, visualization, investigation, writing—reviewing and editing.
Dragana Lakić: conceptualization, methodology, data curation, writing—original draft preparation, visualization, investigation, validation, writing—reviewing and editing, funding acquisition.
Slobodan Janković: conceptualization, methodology, software, formal analysis, data curation, writing—original draft preparation, visualization, investigation, supervision, validation, writing—reviewing and editing.
Disclosure statement
No potential conflict of interest was reported by the author(s).
Data availability statement
The study plan is available at the link: https://osf.io/gs756/. The models used in the study and other materials not contained in the Supplementary File are available to interested readers from the corresponding author, at reasonable request.