Cost‐effectiveness analysis of emicizumab prophylaxis in patients with haemophilia A in India

Abstract

IntroductionEmicizumab is the initial subcutaneously administered bispecific antibody approved as a prophylactic treatment for patients with haemophilia A (PwHA).AimThis study assessed the economic evaluation of emicizumab treatment for non‐inhibitor severe haemophilia A (HA) patients in India.MethodsA Markov model evaluated the cost‐effectiveness of emicizumab prophylaxis compared to on‐demand therapy (ODT), low‐dose prophylaxis (LDP; 1565 IU/kg/year), intermediate‐dose prophylaxis (IDP; 3915 IU/kg/year) and high‐dose prophylaxis (HDP; 7125 IU/kg/year) for HA patients without factor VIII inhibitors. Inputs from HAVEN‐1 and HAVEN‐3 trials included transition probabilities of different bleeding types. Costs and benefits were discounted at a 3.5% annual rate.ResultsIn the base‐case analysis, emicizumab was cost‐effective compared to HDP, with an incremental cost‐effectiveness ratio (ICER) per quality‐adjusted life‐years (QALY) of Indian rupees (INR) 27,869. Compared to IDP, ODT and LDP, emicizumab prophylaxis could be considered a cost‐effective option if the paying threshold is >1 per capita gross domestic product (GDP) with ICER/QALY values of INR 264,592, INR 255,876 and INR 305,398, respectively. One‐way sensitivity analysis (OWSA) highlighted emicizumab cost as the parameter with the greatest impact on ICERs. Probabilistic sensitivity analysis (PSA) indicated that emicizumab had a 94.7% and 49.4% probability of being cost‐effective at willingness‐to‐pay (WTP) thresholds of three and two‐times per capita GDP.ConclusionEmicizumab prophylaxis is cost‐effective compared to HDP and provides value for money compared to ODT, IDP, and LDP for severe non‐inhibitor PwHA in India. Its long‐term humanistic, clinical and economic benefits outweigh alternative options, making it a valuable choice in resource‐constrained settings.