Nonfactor Therapies in Hemophilia A: An Essential Drug Entity.

Rahul U Ramachandran, Vishnu Sharma, Shailendra P Verma
Author Information
  1. Rahul U Ramachandran: Pediatrics, National Health Mission (NHM) Kerala, Trivandrum, IND.
  2. Vishnu Sharma: Clinical Hematology, Sawai Man Singh (SMS) Medical College, Jaipur, IND.
  3. Shailendra P Verma: Hematology and Oncology/ Clinical Hematology, King George's Medical University, Lucknow, IND.

Abstract

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.

Keywords

References

  1. Orphanet J Rare Dis. 2021 Jul 13;16(1):308 [PMID: 34256816]
  2. Res Pract Thromb Haemost. 2022 Aug 26;6(6):e12741 [PMID: 36051543]
  3. Haemophilia. 2022 May;28 Suppl 4:103-110 [PMID: 35521723]
  4. Haemophilia. 2021 Nov;27(6):938-946 [PMID: 34273215]
  5. Blood Adv. 2020 May 12;4(9):2111-2118 [PMID: 32396619]
  6. Indian J Community Med. 2010 Jan;35(1):10-3 [PMID: 20606912]
  7. Indian J Med Res. 2014 Jul;140(1):19-31 [PMID: 25222774]
  8. Blood Transfus. 2013 Sep;11 Suppl 4:s55-63 [PMID: 24333314]
  9. Haemophilia. 2021 Jan;27(1):e1-e11 [PMID: 33210412]
  10. Haemophilia. 2014 May;20(3):382-7 [PMID: 24847522]
  11. Patient Prefer Adherence. 2019 Jun 14;13:941-957 [PMID: 31354248]
  12. BMJ Evid Based Med. 2024 May 6;: [PMID: 38719438]
  13. Res Pract Thromb Haemost. 2023 May 10;7(4):100173 [PMID: 37538493]
  14. Haematologica. 2019 Sep;104(9):1702-1709 [PMID: 31399527]
  15. Indian J Pharmacol. 2023 Jan-Feb;55(1):1-5 [PMID: 36960514]
  16. Haemophilia. 2020 Aug;26 Suppl 6:1-158 [PMID: 32744769]
  17. Value Health Reg Issues. 2017 May;12:101-106 [PMID: 28648306]
  18. Indian J Hematol Blood Transfus. 2020 Jul;36(3):458-463 [PMID: 32647418]
  19. Heliyon. 2024 Mar 01;10(5):e27089 [PMID: 38468938]
  20. Cureus. 2023 May 16;15(5):e39079 [PMID: 37378105]
  21. Cureus. 2024 Apr 24;16(4):e58941 [PMID: 38725780]
  22. Blood. 2015 Mar 26;125(13):2038-44 [PMID: 25712992]
  23. Biomolecules. 2024 Jul 15;14(7): [PMID: 39062568]
  24. PLoS One. 2022 Apr 14;17(4):e0266934 [PMID: 35421181]
  25. Patient. 2019 Dec;12(6):611-619 [PMID: 31313270]
  26. Patient Prefer Adherence. 2021 Apr 28;15:871-883 [PMID: 33953547]
  27. Cureus. 2024 Feb 21;16(2):e54663 [PMID: 38524060]
  28. Hematology Am Soc Hematol Educ Program. 2016 Dec 2;2016(1):657-662 [PMID: 27913543]
  29. Eur J Haematol. 2018 Oct;101(4):435-456 [PMID: 29889317]
  30. J Biomed Sci. 2021 Sep 14;28(1):64 [PMID: 34521404]
  31. Haemophilia. 2024 Mar;30(2):426-436 [PMID: 38147060]

Word Cloud

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