Cost/effectiveness of therapeutic alternatives in the treatment of alpha mannosidosis

[A. S. Antanasković]

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Uvod: Postoje tri mogućnosti za lečenje blage do umerene alfa manozidoze: nadoknada enzima velmanaze alfa, transplantacija kostne srži i simptomatska terapija. Cilj: Upoređenje troškova i efekata velmanaze alfa sa troškovima i efektima transplantacije kostne srži i simptomatske terapije u lečenju blage do umerene alfa manozidoze. Metod: Istraživanje je sprovedeno pomoću modela simulacije diskretnih događaja izgrađenog u tabelarnom kalkulativnom softveru. Ulazni parametri u model su dobijeni sistematskom pretragom naučne literature, a ishodi su bili inkrementalni odnos troškova i efekata, neto novčani benefit, verovatnoća prihvatljivosti velmanaze alfa od strane Republičkog fonda zdravstvenog osiguranja i procenjena vrednost savršene informacije. Ishodi su dobijeni pomoću Monte Karlo simulacije na kohorti od 1000 virtuelnih bolesnika sa alfa manozidozom. Rezultati: Nadoknada enzima alfa manozidaze primenom velmanaze alfa nije isplativa u poređenju sa simptomatskom terapijom ili sa transplantacijom kostne srži (neto novčani benefit je iznosio -366,179,427.48 RSD odnosno -358,074,387.57 RSD, po redosledu). Transplantacija kostne srži može biti isplativa opcija lečenja blage do umerene alfamanozidoze u poređenju sa simptomatskom terapijom, ako se spremnosti na plaćanje Fonda zdravstvenog osiguranja u Srbiji poveća na 3 - 9 bruto nacionalnih dohodaka po glavi stanovnika po dodatnoj godini života prilagođenoj za kvalitet (neto novčani benefit: - 6,849,191.70 RSD). Ulaganje u dodatna istraživanja kako bi se ispitala tačnost ovih zaključaka nije isplativo. Zaključak: Farmakoekonomski najpovoljnija strategija lečenja blage do umerene alfa manozidoze je transplantacija kostne srži.

Introduction: There are three treatment options for mild to moderate alpha mannosidosis: replacement of the enzyme velmanase alfa, bone marrow transplantation, and symptomatic therapy. Objective: To compare the costs and effects of velmanase alfa with the costs and effects of bone marrow transplantation and symptomatic therapy in the treatment of mild to moderate alpha mannosidosis. Method: The research was conducted using a discrete event simulation model built in a spreadsheet software. The input parameters to the model were obtained by a systematic search of the scientific literature, and the outcomes were the incremental cost-effectiveness ratio, the net monetary benefit, the probability of acceptance of velmanase alfa by the Republic Health Insurance Fund and the estimated value of perfect information. The results were obtained using Monte Carlo simulation on a cohort of 1000 virtual patients with alpha mannosidosis. Results: Alpha mannosidase enzyme replacement using velmanase alfa is not cost-effective compared to symptomatic therapy or bone marrow transplantation (net monetary benefit was - 366,179,427.48 RSD and -358,074,387.57 RSD, respectively). Bone marrow transplantation may be a cost-effective treatment option for mild to moderate alpha-mannosidosis compared to symptomatic therapy, if the willingness to pay of the Health Insurance Fund in Serbia is increased to 3 - 9 gross domestic products per capita per additional quality-adjusted life year (net monetary benefit: -6,849,191.70 RSD). Investing in additional research to test the accuracy of these conclusions is not worthwhile. Conclusion: The most pharmacoeconomically favorable treatment strategy for mild to moderate alpha mannosidosis is bone marrow transplantation.

alfa manozidaza; alfa manozidoza; bolest lizozomalnih depoa; velmanaza alfa; transplantacija kostne srži; simptomatska terapija; odnos troškova i efekata; neto novčani benefit; probabilistička analiza senzitivnosti; procenjena vrednost savršene informacije.

alpha mannosidase; alpha mannosidosis; lysosomal depot disease; velmanase alfa; bone marrow transplantation; symptomatic therapy; the cost/effectiveness ratio; net monetary benefit; probabilistic sensitivity analysis; the estimated value of perfect information.

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