Pharmacoeconomic analysis of the causal therapy of cerebral adrenoleukodystrophy

[M. Z. Vapljanin Muratović]

90 listova

Sažetak: Uvod: Cerebralna adrenoleukodistrofija je težak oblik X vezane adrenoleukodistrofije, retke bolesti koja nastaje usled poremećaja razgradnje masnih kiselina veoma dugačkog lanca u peroksizomima. Za lečenje ove bolesti danas na raspolaganju imamo tri terapijske opcije: simptomatsku terapiju, alogenu transplantaciju hematopoetskih matičnih ćelija (HSCT) i gensku terapiju u vidu elivaldogen autotemcela. Cilj ovog istraživanja je bio da se utvrdi koja od ovih terapijska opcija ima najbolji farmakoekonomski profil. Materijal i metod: Studija je sprovedena konstruisanjem farmakoekonomskih modela simulacije diskretnih događaja. Sprovedena je iz perspektive Republičkog fonda za zdravstveno osiguranje Republike Srbije kao institucije, tako da su uračunavani samo direktni medicinski troškovi. Efekti su izražavani kroz broj dobijenih godina života prilagođenih za kvalitet. Rezultati: Elivaldogen autotemcel nije farmakoekonomski isplativa opcija za lečenje cerebralne adrenoleukodistrofije u odnosu na alogenu HSCT i simptomatsku terapiju koja se takođe koristi za lečenje ove retke metaboličke bolesti, budući da je njegova primena praćena neprihvatljivo visokim vrednostima ICER-a i negativnim neto novčanim benefitom. Alogena HSCT je farmakoekonomski isplativija terapijska opcija za lečenje pacijenata sa cerebralnom adrenoleukodistrofijom u poređenju sa simptomatskom terapijom koja se koristi za lečenje ove retke metaboličke bolesti. Zaključak: Rezultati ove farmakoekonomske analize ukazuju da alogena HSCT predstavlja farmakoekonomski najisplativiju opciju za lečenje pacijenata obolelih od cerebralne adrenoleukodistrofije.

Abstract: Introduction: Cerebral adrenoleukodystrophy is a severe form of X-linked adrenoleukodystrophy, a rare disease that results from a disorder in the breakdown of very long-chain fatty acids in peroxisomes. For the treatment of this disease, we have three therapeutic options available today: symptomatic therapy, allogeneic hematopoietic stem cell transplantation (HSCT) and gene therapy in the form of elivaldogen autotemcel. The aim of this research was to determine which of these therapeutic options has the best pharmacoeconomic profile. Material and methods: The study was conducted by constructing discrete event simulation pharmacoeconomic models. It was conducted from the perspective of the Republic Health Insurance Fund of the Republic of Serbia as an institution, so only direct medical costs were included. Effects are expressed in terms of quality-adjusted life years gained. Results: Elivaldogen autotemcel is not a cost-effective option for the treatment of cerebral adrenoleukodystrophy compared to allogeneic HSCT and symptomatic therapy that is also used for the treatment of this rare metabolic disease, since its use is accompanied by unacceptably high ICER values and a negative net monetary benefit. Allogeneic HSCT is a more cost-effective therapeutic option for treating patients with cerebral adrenoleukodystrophy compared with symptomatic therapy used to treat this rare metabolic disease. Conclusion: The results of this pharmacoeconomic analysis indicate that allogeneic HSCT represents the most cost-effective option for the treatment of patients with cerebral adrenoleukodystrophy.

cerebralna adrenoleukodistrofija, elivaldogen autotemcel, alogena transplantacija hematopoetskih matičnih ćelija, farmakoekonomija

cerebral adrenoleukodystrophy, elivaldogen autotemcel, allogeneic hematopoietic stem cell transplantation, pharmacoeconomics.

616.45-008-08(043.3) 615.3:33(043.3)

Metabolički poremećaji - Lečenje - Farmakoekonomija

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