TY  - JOUR
AU  - Antanasković, Ana
AU  - Stević, Ivana
AU  - Gojak, Refet
AU  - Lakić, Dragana
AU  - Janković, Slobodan
PY  - 2023
UR  - https://farfar.pharmacy.bg.ac.rs/handle/123456789/5269
AB  - 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.
PB  - Taylor and Francis Ltd.
T2  - Biotechnology and Biotechnological Equipment
T1  - Cost-effectiveness of velmanase alfa vs. bone marrow transplantation or no causal therapy in patients with mild to moderate alpha-mannosidosis
VL  - 37
IS  - 1
DO  - 10.1080/13102818.2023.2271574
ER  - 

@article{
author = "Antanasković, Ana and Stević, Ivana and Gojak, Refet and Lakić, Dragana and Janković, Slobodan",
year = "2023",
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.",
publisher = "Taylor and Francis Ltd.",
journal = "Biotechnology and Biotechnological Equipment",
title = "Cost-effectiveness of velmanase alfa vs. bone marrow transplantation or no causal therapy in patients with mild to moderate alpha-mannosidosis",
volume = "37",
number = "1",
doi = "10.1080/13102818.2023.2271574"
}

Antanasković, A., Stević, I., Gojak, R., Lakić, D.,& Janković, S.. (2023). Cost-effectiveness of velmanase alfa vs. bone marrow transplantation or no causal therapy in patients with mild to moderate alpha-mannosidosis. in Biotechnology and Biotechnological Equipment
Taylor and Francis Ltd.., 37(1).
https://doi.org/10.1080/13102818.2023.2271574

Antanasković A, Stević I, Gojak R, Lakić D, Janković S. Cost-effectiveness of velmanase alfa vs. bone marrow transplantation or no causal therapy in patients with mild to moderate alpha-mannosidosis. in Biotechnology and Biotechnological Equipment. 2023;37(1).
doi:10.1080/13102818.2023.2271574 .

Antanasković, Ana, Stević, Ivana, Gojak, Refet, Lakić, Dragana, Janković, Slobodan, "Cost-effectiveness of velmanase alfa vs. bone marrow transplantation or no causal therapy in patients with mild to moderate alpha-mannosidosis" in Biotechnology and Biotechnological Equipment, 37, no. 1 (2023),
https://doi.org/10.1080/13102818.2023.2271574 . .