Nonfactor Therapies in Hemophilia A: An Essential Drug Entity

Inequity in the access to effective therapies for rare diseases, such as hemophilia A, is a significant concern in resource-limited settings. Including necessary medications in the essential drug list can ensure that populations have access to safe and affordable treatments. Emicizumab, a nonfactor therapy, addresses many of the challenges associated with traditional factor replacement for hemophilia A, including frequent administration and the risk of inhibitor development. Studies in India show that emicizumab is cost-effective and clinically beneficial. The inclusion of emicizumab in the National Health Mission's (NHM) Essential Drug List (India) would improve treatment access and adherence, reduce healthcare burden, and improve outcomes for patients, particularly those with high bleeding rates and poor accessibility to care. Moreover, it will ensure the continuity of therapy for all existing patients who have been on emicizumab supported by the NHM in the last four years. This review aims to consolidate evidence regarding the current status of care and the benefits of emicizumab in India to highlight the importance of the addition of emicizumab to NHM's Essential Drug List.