Krishnamoorthy Yuvaraj, Govindan Dhanajayan, Kannan Narasimhapriyan, Majella Marie Gilbert, Hariharan Vishnu Shankar, Valliappan Vivek
PROPUL Evidence Synthesis Unit (PROPUL ESU), PROPUL (Partnership for Research Opportunity Planning Upskilling and Leadership) Evidence, Chennai, Tamilnadu, India.
Department of Hematology, All India Institute of Medical Sciences (AIIMS), New Delhi, India.
Heliyon. 2024 Mar 1;10(5):e27089. doi: 10.1016/j.heliyon.2024.e27089. eCollection 2024 Mar 15.
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.
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.
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.
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]).
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.
患有抑制物的重度甲型血友病患者目前正在接受旁路制剂治疗，如活化凝血酶原复合物浓缩剂（aPCC）和重组凝血因子VIIa。艾美赛珠单抗是一种重组人源化单克隆抗体，旨在减少出血事件、提高治疗依从性和改善生活质量。然而，在印度这样的低收入和中等收入环境中，该干预措施的成本效益和长期可持续性尚未得到研究。
本研究的主要目的是评估在印度，与传统旁路制剂相比，艾美赛珠单抗治疗患有抑制物的重度甲型血友病患者的成本效用。次要目的是从印度公共卫生系统的角度分析引入艾美赛珠单抗对该患者群体的预算影响。
创建马尔可夫模型，比较针对印度10岁青少年的假设队列，预防性使用艾美赛珠单抗疗法与旁路制剂的效果。时间跨度为10年，模型基于卫生系统视角构建。成本效用以每获得一个质量调整生命年（QALY）的成本表示。所有成本均以2021年美元表示。进行概率敏感性分析以检验估计值的稳健性。
预防性使用艾美赛珠单抗是一种节省成本的干预措施，与重组凝血因子VIIa相比，增量成本效用比（ICUR）为负，为-853,573美元（-63,109,773印度卢比），与aPCC相比，ICUR为负，为-211,675美元（-15,650,403印度卢比）。在10年的时间跨度内，印度治疗所有患有抑制物的重度甲型血友病患者的估计总预算为59,042,000美元（4,365,329,312印度卢比）（每位患者成本为295,210美元[21,826,646.56印度卢比]）。
与两种旁路制剂相比，预防性使用艾美赛珠单抗疗法是一种节省成本的干预措施，因为它对印度患有抑制物的重度甲型血友病患者成本更低且更有效。
