Budget Impact and Cost-utility Analysis of Prophylactic Emicizumab Versus On-demand Bypassing Agents for Adolescent Severe Haemophilia A Patients with Inhibitors in India

Overview

Journal Heliyon

Specialty Social Sciences

Date 2024 Mar 12

PMID 38468938

Authors

Yuvaraj Krishnamoorthy

Dhanajayan Govindan

Narasimhapriyan Kannan

Marie Gilbert Majella

Vishnu Shankar Hariharan

Vivek Valliappan

Affiliations

Soon will be listed here.

Abstract

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.

Citing Articles

Nonfactor Therapies in Hemophilia A: An Essential Drug Entity.

Ramachandran R, Sharma V, Verma S Cureus. 2024; 16(10):e70763.

PMID: 39493198 PMC: 11531249. DOI: 10.7759/cureus.70763.

Emicizumab in Children with Severe Hemophilia A.

Thota U, Martha S, Ravula C, Cherukuri N Indian J Pediatr. 2024; .

PMID: 39320428 DOI: 10.1007/s12098-024-05263-2.

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