目的:使用适应性健康技术评估(aHTA)方法,评估在印度背景下使用抑制剂预防艾美珠单抗预防A型血友病患者的成本效益。
方法:使用多种方法进行经济评估,旨在根据(1)仅价格差异(\'简单\')和(2)成本差异和预期治疗持续时间(\'中等\')以及成本差异来调整先前产生的成本效益结果,通货膨胀和预期寿命(“复杂”)。
方法:印度典型的血友病护理。
方法:A型血友病和抑制剂患者。
方法:与未预防相比,使用两个小瓶强度(30或150mg/mL)的Emicizumab预防。
方法:调整后的增量成本效益比(ICERa),从卫生系统和社会角度来看,与emicizumab预防相关的增量成本和增量质量调整生命年.
结果:使用简单的ICER调整方法,在年龄≥12岁和<12岁的患者中,从支付者的角度来看,emicizumab预防可节省两种药瓶强度的潜在成本.然而,从社会的角度来看,emicizumab预防并不具有成本效益.采用适度调整的方法,从卫生系统的角度来看,埃米珠单抗预防显示出潜在的成本节约.复杂的调整方法还揭示了不同年龄段的卫生系统和社会观点对emicizumab预防的成本节省。
结论:我们发现,在印度实施对A型血友病患者和抑制剂的emicizumab预防有可能导致成本节约。这项研究强调了在印度背景下使用扩展的aHTA方法进行快速证据生成的可行性。然而,解决某些研究差距至关重要,包括数据限制,将国际证据转化为印度背景和相关不确定性方面的挑战。此外,进行全面预算影响分析是必要的。这些发现对通过印度联邦或/和州政府资助的计划和机构进行潜在的emicizumab预防的决策具有重要意义。
OBJECTIVE: To assess the cost-effectiveness of emicizumab prophylaxis for patients having haemophilia A with inhibitors in the Indian context using an adaptive health technology assessment (aHTA) methodology.
METHODS: Economic evaluation using multiple approaches aimed at adjusting previously generated cost-effectiveness results based on (1) price differences only (\'simple\') and (2) differences in cost and expected treatment duration (\'moderate\') and differences in cost, inflation and life expectancy (\'complex\').
METHODS: Typical haemophilia care in India.
METHODS: Patients with haemophilia A and inhibitors.
METHODS: Emicizumab prophylaxis using two vial strengths (30 or 150 mg/mL) in comparison to no prophylaxis.
METHODS: Adjusted incremental cost-effectiveness ratio (ICERa), incremental costs and incremental quality-adjusted life years associated with emicizumab prophylaxis from both the health system and societal perspectives.
RESULTS: Using the simple ICER adjustment method, emicizumab prophylaxis resulted in potential cost savings from the payers\' perspective for both vial strengths in patients aged ≥12 and <12 years. However, from a societal perspective, emicizumab prophylaxis was not cost-effective. Using the moderate adjustment method, emicizumab prophylaxis showed potential cost saving from the health system perspective. The complex adjustment method also revealed cost savings for emicizumab prophylaxis from the health system and societal perspectives across different age groups.
CONCLUSIONS: We found that implementing emicizumab prophylaxis for patients with haemophilia A and inhibitors in India has the potential to result in cost savings. This study highlights the feasibility of using the expanded aHTA methodology for rapid evidence generation in the Indian context. However, it is crucial to address certain research gaps, including data limitations, challenges in translating international evidence to Indian context and associated uncertainties. Additionally, conducting a comprehensive budget impact analysis is necessary. These findings hold significant implications for decision-making regarding the potential provision of emicizumab prophylaxis through federal or/and state government-funded programmes and institutions in India.