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Date Published: 12/06/2022
Online Published: 12/06/2023
Abstract Views: 237
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Issue
No. 46 (2022)
Section
Article
How to Cite
Huynh, T. N. V., Pham, T. T., Tran, T. D. L., & Huynh, T. P. D. (2023). COST-EFFECTIVENESS OF ORPHAN DRUGS IN THE TREATMENT OF HEMOPHILIA A: A SYSTEMATIC REVIEW. Cantho Journal of Medicine and Pharmacy, 46, 126-135. https://tapchi.ctump.edu.vn/index.php/ctump/article/view/923

COST-EFFECTIVENESS OF ORPHAN DRUGS IN THE TREATMENT OF HEMOPHILIA A: A SYSTEMATIC REVIEW

Thy Nhac Vu Huynh1, Thu Thuy Pham1,, Thi Dien Linh Tran1, Thi Phuong Duyen Huynh2
1 University of Medicine and Pharmacy at Ho Chi Minh City
2 Department of Health of Lam Dong Province

Main Article Content

Abstract

Background: Hemophilia A is a rare genetic disease caused by a deficiency or abnormality in clotting  factor  VIII  (FVIII).  Objectives:  A  systematic  review of  the  cost-effectiveness  studies performed on orphan drugs to treat Hemophilia A was conducted for the purposes of gaining comprehensive information about cost-effectiveness analysis and optimal selection on drugs for disease treatment. Materials and methods: Data were obtained from PubMed (National Center for Biotechnology Information - NCBI) and SpringerLink databases on cost-effectiveness analysis of Hemophilia A drugs in Vietnam's Orphan Drug List. Cost, effectiveness, willingness-to-pay thresholds, and incremental cost-effectiveness ratios of orphan drugs were evaluated in this systematic review. Results: In the period 2016-2021, there were 4 drugs to treat Hemophilia A in Vietnam's Orphan Drugs List, which were assessed on cost-effectiveness, including: Emicizumab; Recombinant Factor VII activated (rFVIIa); Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) and Damoctocog alfa pegol. In the majority of studies included in this systematic review, Emicizumab  and  rFVIIIFc  were  found  to  be  cost-effective  when  analyzed  along  with  their comparators. Conculsions: Treatment of Hemophilia A with orphan drugs was significantly expensive. Financial policies should be considered for patients to access orphan drugs for the treatment of Hemophilia A.

Article Details

Keywords

Hemophilia A, cost-effectiveness, orphan drugs, systematic review

References

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