Title (srp)

Farmakoekonomska analiza kauzalne terapije neuroloških manifestacija bolesti usled poremećaja razgradnje i deponovanja makromolekula u lizozomima

Author

Gutić, Medo, 1992-

Contributor

Milosavljević, Miloš, 1991-
Milovanović, Dragan, 1967-
Stevanović, Dejan, 1979-
Stašević-Karličić, Ivana, 1973-

Description (eng)

Introduction: Lysosomal storage diseases (LSD) form a heterogeneous group of rarehereditary diseases with about 50 members. The aim of this study was to compare the costsand effectiveness of new causal therapy for LSD with the costs and effectiveness ofsupportive or old causal therapy.Material and methods: This study was conducted using the construction and analysis ofpharmacoeconomic models for three diseases of the BRDL group: Niemann-Pick disease typeC (NP-C), late infantile ceroid neuronal lipofuscinosis type 2 (CLN2), and Gaucher diseasetype 2 (GD2).Two types of models were used: the Markov model and the Descrete EventSimulation model. The models were simulated using Monte Carlo simulations for cohorts of1,000 virtual individuals each.Results: Treatment of NP-C patients with miglustat as a causal therapy is not cost-effectivecompared to treating this disease with symptomatic therapy.Treatment of CLN2 patients withcerliponase alfa as a causal therapy is not cost-effective compared to treating CLN2 withsymptomatic therapy.When compared to imiglucerase monotherapy, combined therapy withambroxol has been found to be pharmacoeconomically more cost-effective for treatingindividuals with GD2.Conclusion: Miglustat and cerliponase alfa are not pharmacoeconomically cost-effectiveoptions for the treatment of NP-C, i.e., CLN2, when traditional principles ofpharmacoeconomic evaluation are applied, so it is necessary for pharmaceutical companies toapply a differential pricing model that should be lower in underdeveloped and developingcountries.Ambroxol is an affordable medication whose use in treatment GD2 should be givenconsiderable thought.

Description (srp)

Uvod: Bolesti usled poremećaja razgradnje i deponovanja makromolekula u lizozomima(BRDL) čine heterogenu grupu rijetkih naslednih oboljenja sa oko 50 članova. Cilj ove studijeje bio da uporedi troškove i efikasnost nove kauzalne terapije BRDL sa troškovima iefikasnošću suportivne ili stare kauzalne terapije.Materijal i metod: Ova studija je sprovedena pomoću izgradnje i analizefarmakoekonomskih modela za tri bolesti iz grupe BRDL: Niman-Pikova bolest tip C (NP-C),kasna infantilna neuronska ceroidna lipofuscinoza tip 2 (CLN2) i Gošeova bolest tip 2 (GD2).Korišćene su dvije vrste modela: Markovljev model i model simulacije diskretnih dogaĎaja.Modeli su simulirani pomoću Monte Karlo simulacije, za kohorte od po 1,000 virtuelnihosoba.Rezultati: Liječenje pacijenata oboljelih od NP-C miglustatom kao kauzalnom terapijom nijeisplativo u odnosu na liječenje ove bolesti simptomatskom terapijom. Liječenje pacijenataoboljelih od CLN2 cerliponazom alfa kao kauzalnom terapijom nije isplativo u odnosu naliječenje CLN2 simptomatskom terapijom. Kombinovana terapija ambroksola i imiglucerazese pokazala kao farmakoekonomski isplativija opcija za liječenje pacijenata oboljelih od GD2u odnosu na monoterapiju imiglucerazom.Zaključak: Miglustat i cerliponaza alfa nisu farmakoekonomski isplative opcije za liječenjeNP-C, odnosno CLN2, kada se primjene tradicionalni principi farmakoekonomske evaluacije,pa je zato neophodno da farmaceutske kompanije primjene model diferencijalnog odreĎivanjacijena koje treba da budu niže u nerazvijenim i zemljama u razvoju. Ambroksol je jeftin lijekčiju primjenu u liječenju GD2 treba podrobnije razmotriti.

Description (srp)

-

Object languages

Serbian

Date

2023

Rights

Creative Commons License
This work is licensed under a
CC BY-NC-ND 3.0 AT - Creative Commons Attribution-NonCommercial-NoDerivs 3.0 Austria License.

http://creativecommons.org/licenses/by-nc-nd/3.0/at/legalcode

Identifiers