Budget impact and cost-utility analysis of prophylactic emicizumab versus on-demand bypassing agents for adolescent severe haemophilia A patients with inhibitors in India

Introduction: Severe Haemophilia A patients with inhibitors are currently being treated with bypassing agents like activated prothrombin complex concentrates (aPCC) and recombinant factor VIIa. Emicizumab is a recombinant humanized monoclonal antibody, introduced to reduce the bleeding events, impro...

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Main Authors: Yuvaraj Krishnamoorthy, Dhanajayan Govindan, Narasimhapriyan Kannan, Marie Gilbert Majella, Vishnu Shankar Hariharan, Vivek Valliappan
Format: Article
Language:English
Published: Elsevier 2024-03-01
Series:Heliyon
Subjects:
Online Access:http://www.sciencedirect.com/science/article/pii/S2405844024031207
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author Yuvaraj Krishnamoorthy
Dhanajayan Govindan
Narasimhapriyan Kannan
Marie Gilbert Majella
Vishnu Shankar Hariharan
Vivek Valliappan
author_facet Yuvaraj Krishnamoorthy
Dhanajayan Govindan
Narasimhapriyan Kannan
Marie Gilbert Majella
Vishnu Shankar Hariharan
Vivek Valliappan
author_sort Yuvaraj Krishnamoorthy
collection DOAJ
description Introduction: Severe Haemophilia A patients with inhibitors are currently being treated with bypassing agents like activated prothrombin complex concentrates (aPCC) and recombinant factor VIIa. Emicizumab is a recombinant humanized monoclonal antibody, introduced to reduce the bleeding events, improve treatment adherence, and quality of life. However, cost-effectiveness and long-term sustainability of the intervention is not studied in a low middle income setting like India. Aim: The primary objective of this study was to evaluate the cost-utility of Emicizumab compared to traditional bypassing agents in the treatment of severe haemophilia A patients with inhibitors in India. Secondary objective was to analyze the budgetary impact of introducing Emicizumab for this patient population from the perspective of public health system in India. Methods: Markov model was created to compare the prophylactic emicizumab therapy against bypassing agents for a hypothetical cohort of 10-year-old adolescents in India. The time horizon was 10 years and model built based on health system perspective. Cost utility was expressed as costs per quality-adjusted life-years (QALYs) gained. All costs were expressed as 2021 US dollars. Probabilistic sensitivity analysis was performed to check the robustness of the estimates. Results: Prophylactic emicizumab was a cost saving intervention with negative Incremental Cost Utility Ratio (ICUR) against recombinant factor VIIa of −853,573 USD (INR -63,109,773), and negative ICUR of −211,675 USD (INR -15,650,403) against APCC. The estimated total budget for treating all the severe Haemophilia A patients with inhibitors in India was USD 59,042,000 (INR 4,365,329,312) for 10 years’ time horizon (per patient cost of USD 295,210 [INR 21,826,646.56]). Conclusion: Prophylactic emicizumab therapy is a cost saving intervention when compared to both the bypassing agents as it is less costly and more effective for severe Haemophilia A patients with inhibitors in India.
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spelling doaj.art-34b427f19f684f24ac80601a18efa73e2024-03-17T07:57:18ZengElsevierHeliyon2405-84402024-03-01105e27089Budget impact and cost-utility analysis of prophylactic emicizumab versus on-demand bypassing agents for adolescent severe haemophilia A patients with inhibitors in IndiaYuvaraj Krishnamoorthy0Dhanajayan Govindan1Narasimhapriyan Kannan2Marie Gilbert Majella3Vishnu Shankar Hariharan4Vivek Valliappan5PROPUL Evidence Synthesis Unit (PROPUL ESU), PROPUL (Partnership for Research Opportunity Planning Upskilling and Leadership) Evidence, Chennai, Tamilnadu, India; Corresponding author.PROPUL Evidence Synthesis Unit (PROPUL ESU), PROPUL (Partnership for Research Opportunity Planning Upskilling and Leadership) Evidence, Chennai, Tamilnadu, IndiaDepartment of Hematology, All India Institute of Medical Sciences (AIIMS), New Delhi, IndiaPROPUL Evidence Synthesis Unit (PROPUL ESU), PROPUL (Partnership for Research Opportunity Planning Upskilling and Leadership) Evidence, Chennai, Tamilnadu, IndiaDepartment of Internal Medicine, Hindu Mission Hospital, Chengalpattu, IndiaDepartment of Pharmacology, Chettinad Hospital and Research Institute, Chettinad Academy of Research and Education, Kelambakkam, Chengalpattu District, 603103, Tamilnadu, IndiaIntroduction: Severe Haemophilia A patients with inhibitors are currently being treated with bypassing agents like activated prothrombin complex concentrates (aPCC) and recombinant factor VIIa. Emicizumab is a recombinant humanized monoclonal antibody, introduced to reduce the bleeding events, improve treatment adherence, and quality of life. However, cost-effectiveness and long-term sustainability of the intervention is not studied in a low middle income setting like India. Aim: The primary objective of this study was to evaluate the cost-utility of Emicizumab compared to traditional bypassing agents in the treatment of severe haemophilia A patients with inhibitors in India. Secondary objective was to analyze the budgetary impact of introducing Emicizumab for this patient population from the perspective of public health system in India. Methods: Markov model was created to compare the prophylactic emicizumab therapy against bypassing agents for a hypothetical cohort of 10-year-old adolescents in India. The time horizon was 10 years and model built based on health system perspective. Cost utility was expressed as costs per quality-adjusted life-years (QALYs) gained. All costs were expressed as 2021 US dollars. Probabilistic sensitivity analysis was performed to check the robustness of the estimates. Results: Prophylactic emicizumab was a cost saving intervention with negative Incremental Cost Utility Ratio (ICUR) against recombinant factor VIIa of −853,573 USD (INR -63,109,773), and negative ICUR of −211,675 USD (INR -15,650,403) against APCC. The estimated total budget for treating all the severe Haemophilia A patients with inhibitors in India was USD 59,042,000 (INR 4,365,329,312) for 10 years’ time horizon (per patient cost of USD 295,210 [INR 21,826,646.56]). Conclusion: Prophylactic emicizumab therapy is a cost saving intervention when compared to both the bypassing agents as it is less costly and more effective for severe Haemophilia A patients with inhibitors in India.http://www.sciencedirect.com/science/article/pii/S2405844024031207Cost utility analysisEmicizumabHaemophilia AIndia
spellingShingle Yuvaraj Krishnamoorthy
Dhanajayan Govindan
Narasimhapriyan Kannan
Marie Gilbert Majella
Vishnu Shankar Hariharan
Vivek Valliappan
Budget impact and cost-utility analysis of prophylactic emicizumab versus on-demand bypassing agents for adolescent severe haemophilia A patients with inhibitors in India
Heliyon
Cost utility analysis
Emicizumab
Haemophilia A
India
title Budget impact and cost-utility analysis of prophylactic emicizumab versus on-demand bypassing agents for adolescent severe haemophilia A patients with inhibitors in India
title_full Budget impact and cost-utility analysis of prophylactic emicizumab versus on-demand bypassing agents for adolescent severe haemophilia A patients with inhibitors in India
title_fullStr Budget impact and cost-utility analysis of prophylactic emicizumab versus on-demand bypassing agents for adolescent severe haemophilia A patients with inhibitors in India
title_full_unstemmed Budget impact and cost-utility analysis of prophylactic emicizumab versus on-demand bypassing agents for adolescent severe haemophilia A patients with inhibitors in India
title_short Budget impact and cost-utility analysis of prophylactic emicizumab versus on-demand bypassing agents for adolescent severe haemophilia A patients with inhibitors in India
title_sort budget impact and cost utility analysis of prophylactic emicizumab versus on demand bypassing agents for adolescent severe haemophilia a patients with inhibitors in india
topic Cost utility analysis
Emicizumab
Haemophilia A
India
url http://www.sciencedirect.com/science/article/pii/S2405844024031207
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