Cost-effectiveness of emicizumab prophylaxis for haemophilia A with inhibitors: an adaptive health technology assessment for the Indian setting

BMJ Evid Based Med. 2024 May 6:bmjebm-2023-112492. doi: 10.1136/bmjebm-2023-112492. Online ahead of print.


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.

Design: 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').

Setting: Typical haemophilia care in India.

Participants: Patients with haemophilia A and inhibitors.

Intervention: Emicizumab prophylaxis using two vial strengths (30 or 150 mg/mL) in comparison to no prophylaxis.

Main outcome measures: 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.

Conclusion: 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.

Keywords: Blood Coagulation Disorders; Economics; Health Care Economics and Organizations; Hemorrhagic Disorders.