Pharmacoeconomic studies on coagulation factor Ⅷ for the treatment of hemophilia A: a systematic review_Chinese Journal of Evidence-Based Medicine

Authors：

LI Yan ¹ , ZHANG Yao ¹ , LUO Mengjie ¹ , CHEN Pingyu ^1,2 ,  MA Aixia ^1,2

1. School of International Pharmaceutical Business, China Pharmaceutical University, Nanjing 211198, P. R. China;
2. Center for Pharmacoeconomics and Outcomes Research, China Pharmaceutical University, Nanjing 211198, P. R. China;

Corresponding author：

MA Aixia, Email: ma86128@sina.com

Keywords：

Hemophilia A; Coagulation factor Ⅷ; Pharmacoeconomics; Systematic review

DOI：

10.7507/1672-2531.202208070

Video：

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Objective To systematically review the pharmacoeconomics research of coagulation factor Ⅷ for the treatment of hemophilia A. Methods PubMed, EMbase, Web of Science, The Cochrane Library, CNKI, VIP and WanFang Data databases were electronically searched to collect pharmacoeconomic studies of coagulation factor Ⅷ for the treatment of hemophilia A from inception to February 2022. Two reviewers independently screened literature, extracted data and assessed the risk of bias of included studies; then, qualitative systematic review was carried out from the aspects of research model, research parameters and uncertainty analysis. Results A total of 17 pharmacoeconomic studies were included. The overall quality of the included literature was relatively high, and most of them conformed to the basic framework of pharmacoeconomic research; however, there were still differences and deficiencies in model setting and parameter selection. Most results of the study evaluation showed that prophylaxis of coagulation factor Ⅷ had cost-effectiveness advantages over on-demand treatment. Conclusion Current evidence shows that the preventive treatment of coagulation factor Ⅷ may have certain cost-effectiveness advantages compared with on-demand treatment; however, the adaptability of this conclusion to China still needs to be analyzed.

Citation： LI Yan, ZHANG Yao, LUO Mengjie, CHEN Pingyu, MA Aixia. Pharmacoeconomic studies on coagulation factor Ⅷ for the treatment of hemophilia A: a systematic review. Chinese Journal of Evidence-Based Medicine, 2022, 22(12): 1419-1427. doi: 10.7507/1672-2531.202208070 Copy

1.	中华医学会血液学分会血栓与止血学组, 中国血友病协作组. 血友病诊断与治疗中国专家共识(2017年版). 中华血液学杂志, 2017, 38(5): 364-370.
2.	Srivastava A, Santagostino E, Dougall A, et al. WFH guidelines for the management of hemophilia, 3rd edition. Haemophilia, 2020, 26(Suppl 6): 1-158.
3.	Fischer K, van der Bom JG, Molho P, et al. Prophylactic versus on-demand treatment strategies for severe haemophilia: a comparison of costs and long-term outcome. Haemophilia, 2002, 8(6): 745-752.
4.	Wu R, Luke KH. The benefit of low dose prophylaxis in the treatment of hemophilia: a focus on China. Expert Rev Hematol, 2017, 10(11): 995-1004.
5.	Husereau D, Drummond M, Augustovski F, et al. Consolidated health economic evaluation reporting standards 2022 (CHEERS 2022) statement: updated reporting guidance for health economic evaluations. Clin Ther, 2022, 44(2): 158-168.
6.	Husereau D, Drummond M, Augustovski F, et al. Consolidated health economic evaluation reporting standards (CHEERS) 2022 explanation and elaboration: a report of the ISPOR CHEERS Ⅱ good practices task force. Value Health, 2022, 25(1): 10-31.
7.	Willke RJ, Pizzi LT. CHEERS to Updated guidelines for reporting health economic evaluations! Value Health, 2022, 25(1): 1-2.
8.	Degeling K, Vu M, Koffijberg H, et al. Health economic models for metastatic colorectal cancer: a methodological review. Pharmacoeconomics, 2020, 38(7): 683-713.
9.	Smith PS, Teutsch SM, Shaffer PA, et al. Episodic versus prophylactic infusions for hemophilia A: a cost-effectiveness analysis. J Pediatr, 1996, 129(3): 424-431.
10.	Miners AH, Sabin CA, Tolley KH, et al. Cost-utility analysis of primary prophylaxis versus treatment on-demand for individuals with severe haemophilia. Pharmacoeconomics, 2002, 20(11): 759-774.
11.	Risebrough N, Oh P, Blanchette V, et al. Cost-utility analysis of Canadian tailored prophylaxis, primary prophylaxis and on-demand therapy in young children with severe haemophilia A. Haemophilia, 2008, 14(4): 743-752.
12.	Daliri AA, Haghparast H, Mamikhani J. Cost-effectiveness of prophylaxis against on-demand treatment in boys with severe hemophilia A in Iran. Int J Technol Assess Health Care, 2009, 25(4): 584-587.
13.	Miners A. Revisiting the cost-effectiveness of primary prophylaxis with clotting factor for the treatment of severe haemophilia A. Haemophilia, 2009, 15(4): 881-887.
14.	Colombo GL, Di Matteo S, Mancuso ME, et al. Cost-utility analysis of prophylaxis versus treatment on demand in severe hemophilia A. Clinicoecon Outcomes Res, 2011, 3: 55-61.
15.	Farrugia A, Cassar J, Kimber MC, et al. Treatment for life for severe haemophilia A- A cost-utility model for prophylaxis vs. on-demand treatment. Haemophilia, 2013, 19(4): e228-e238.
16.	Petrova G, Tachkov K, Georgieva S, et al. Humanistic and economic aspects of haemophilia treatment in Bulgaria. Comparison between two therapeutic approaches:prophylactic vs. on-demand treatment. Biotechnol Biotechnol Equip, 2014, 28(3): 576-582.
17.	Castro JHE, Moreno VM, Mejia AE. Cost-utility analysis of primary prophylaxis, compared with on-demand treatment, for patients with severe hemophilia type a in colombia. Int J Technol Assess Health Care, 2016, 32(5): 337-347.
18.	Iannazzo S, Cortesi PA, Crea R, et al. Cost-effectiveness analysis of pharmacokinetic-driven prophylaxis vs. standard prophylaxis in patients with severe haemophilia A. Blood Coagul Fibrinolysis, 2017, 28(6): 425-430.
19.	Coppola A, D'Ausilio A, Aiello A, et al. Cost-effectiveness analysis of late prophylaxis vs. on-demand treatment for severe haemophilia A in Italy. Haemophilia, 2017, 23(3): 422-429.
20.	Zahedi Z, Karimi M, Keshavarz K, et al. A cost-effectiveness analysis of the prophylaxis versus on-demand regimens in severe hemophilia A patients under 12 years old in southern Iran. Hematology, 2021, 26(1): 240-248.
21.	张瑶, 马爱霞. 重组人凝血因子Ⅷ的小剂量预防方案对比按需治疗方案用于中国A型血友病患者的成本-效用分析. 中国药物经济学, 2021, 16: 62-66.
22.	Henry N, Jovanović J, Schlueter M, et al. Cost-utility analysis of life-long prophylaxis with recombinant factor ⅧFc vs recombinant factor Ⅷ for the management of severe hemophilia A in Sweden. J Med Econ, 2018, 21(4): 318-325.
23.	Bullement A, McMordie ST, Hatswell AJ, et al. Cost-effectiveness analysis of recombinant factor Ⅷ Fc-fusion protein (rFⅧFc) for the treatment of severe hemophilia A in Italy incorporating real-world dosing and joint health data. Pharmacoecon Open, 2020, 4(1): 133-142.
24.	Lotfi F, Talebianpour H, Keshavarz K, et al. Cost-utility analysis of factor Ⅷ diet therapies prepared using blood plasma vs. recombinant technique for patients with hemophilia A. Daru, 2020, 28(1): 287-293.
25.	Bullement A, Knowles ES, DasMahapatra P, et al. Cost-effectiveness analysis of rFⅧFc versus contemporary rFⅧ treatments for patients with severe hemophilia A without inhibitors in the United States. Pharmacoecon Open, 2021, 5(4): 625-633.
26.	孙利华, 吴晶, 左根永, 等. 药物经济学第4版. 北京: 中国医药科技出版社, 2019: 124-125.
27.	刘国恩, 胡善联, 吴久鸿, 等. 中国药物经济学评价指南2020: 中英双语版. 北京: 中国市场出版社, 2020: 5-55.
28.	DF372. 我国首个国产重组凝血八因子产品获批. 北京: 中国证券报, 2021-07-24. Available at: https://finance.eastmoney.com/a/202107242012743283.html.
29.	宣建伟, 孙巧. 中国罕见病药物经济学评估适用模型与支付阈值参考标准探讨. 国际药学研究杂志, 2019, 46(9): 659-665.

1. 中华医学会血液学分会血栓与止血学组, 中国血友病协作组. 血友病诊断与治疗中国专家共识(2017年版). 中华血液学杂志, 2017, 38(5): 364-370.
2. Srivastava A, Santagostino E, Dougall A, et al. WFH guidelines for the management of hemophilia, 3rd edition. Haemophilia, 2020, 26(Suppl 6): 1-158.
3. Fischer K, van der Bom JG, Molho P, et al. Prophylactic versus on-demand treatment strategies for severe haemophilia: a comparison of costs and long-term outcome. Haemophilia, 2002, 8(6): 745-752.
4. Wu R, Luke KH. The benefit of low dose prophylaxis in the treatment of hemophilia: a focus on China. Expert Rev Hematol, 2017, 10(11): 995-1004.
5. Husereau D, Drummond M, Augustovski F, et al. Consolidated health economic evaluation reporting standards 2022 (CHEERS 2022) statement: updated reporting guidance for health economic evaluations. Clin Ther, 2022, 44(2): 158-168.
6. Husereau D, Drummond M, Augustovski F, et al. Consolidated health economic evaluation reporting standards (CHEERS) 2022 explanation and elaboration: a report of the ISPOR CHEERS Ⅱ good practices task force. Value Health, 2022, 25(1): 10-31.
7. Willke RJ, Pizzi LT. CHEERS to Updated guidelines for reporting health economic evaluations! Value Health, 2022, 25(1): 1-2.
8. Degeling K, Vu M, Koffijberg H, et al. Health economic models for metastatic colorectal cancer: a methodological review. Pharmacoeconomics, 2020, 38(7): 683-713.
9. Smith PS, Teutsch SM, Shaffer PA, et al. Episodic versus prophylactic infusions for hemophilia A: a cost-effectiveness analysis. J Pediatr, 1996, 129(3): 424-431.
10. Miners AH, Sabin CA, Tolley KH, et al. Cost-utility analysis of primary prophylaxis versus treatment on-demand for individuals with severe haemophilia. Pharmacoeconomics, 2002, 20(11): 759-774.
11. Risebrough N, Oh P, Blanchette V, et al. Cost-utility analysis of Canadian tailored prophylaxis, primary prophylaxis and on-demand therapy in young children with severe haemophilia A. Haemophilia, 2008, 14(4): 743-752.
12. Daliri AA, Haghparast H, Mamikhani J. Cost-effectiveness of prophylaxis against on-demand treatment in boys with severe hemophilia A in Iran. Int J Technol Assess Health Care, 2009, 25(4): 584-587.
13. Miners A. Revisiting the cost-effectiveness of primary prophylaxis with clotting factor for the treatment of severe haemophilia A. Haemophilia, 2009, 15(4): 881-887.
14. Colombo GL, Di Matteo S, Mancuso ME, et al. Cost-utility analysis of prophylaxis versus treatment on demand in severe hemophilia A. Clinicoecon Outcomes Res, 2011, 3: 55-61.
15. Farrugia A, Cassar J, Kimber MC, et al. Treatment for life for severe haemophilia A- A cost-utility model for prophylaxis vs. on-demand treatment. Haemophilia, 2013, 19(4): e228-e238.
16. Petrova G, Tachkov K, Georgieva S, et al. Humanistic and economic aspects of haemophilia treatment in Bulgaria. Comparison between two therapeutic approaches:prophylactic vs. on-demand treatment. Biotechnol Biotechnol Equip, 2014, 28(3): 576-582.
17. Castro JHE, Moreno VM, Mejia AE. Cost-utility analysis of primary prophylaxis, compared with on-demand treatment, for patients with severe hemophilia type a in colombia. Int J Technol Assess Health Care, 2016, 32(5): 337-347.
18. Iannazzo S, Cortesi PA, Crea R, et al. Cost-effectiveness analysis of pharmacokinetic-driven prophylaxis vs. standard prophylaxis in patients with severe haemophilia A. Blood Coagul Fibrinolysis, 2017, 28(6): 425-430.
19. Coppola A, D'Ausilio A, Aiello A, et al. Cost-effectiveness analysis of late prophylaxis vs. on-demand treatment for severe haemophilia A in Italy. Haemophilia, 2017, 23(3): 422-429.
20. Zahedi Z, Karimi M, Keshavarz K, et al. A cost-effectiveness analysis of the prophylaxis versus on-demand regimens in severe hemophilia A patients under 12 years old in southern Iran. Hematology, 2021, 26(1): 240-248.
21. 张瑶, 马爱霞. 重组人凝血因子Ⅷ的小剂量预防方案对比按需治疗方案用于中国A型血友病患者的成本-效用分析. 中国药物经济学, 2021, 16: 62-66.
22. Henry N, Jovanović J, Schlueter M, et al. Cost-utility analysis of life-long prophylaxis with recombinant factor ⅧFc vs recombinant factor Ⅷ for the management of severe hemophilia A in Sweden. J Med Econ, 2018, 21(4): 318-325.
23. Bullement A, McMordie ST, Hatswell AJ, et al. Cost-effectiveness analysis of recombinant factor Ⅷ Fc-fusion protein (rFⅧFc) for the treatment of severe hemophilia A in Italy incorporating real-world dosing and joint health data. Pharmacoecon Open, 2020, 4(1): 133-142.
24. Lotfi F, Talebianpour H, Keshavarz K, et al. Cost-utility analysis of factor Ⅷ diet therapies prepared using blood plasma vs. recombinant technique for patients with hemophilia A. Daru, 2020, 28(1): 287-293.
25. Bullement A, Knowles ES, DasMahapatra P, et al. Cost-effectiveness analysis of rFⅧFc versus contemporary rFⅧ treatments for patients with severe hemophilia A without inhibitors in the United States. Pharmacoecon Open, 2021, 5(4): 625-633.
26. 孙利华, 吴晶, 左根永, 等. 药物经济学第4版. 北京: 中国医药科技出版社, 2019: 124-125.
27. 刘国恩, 胡善联, 吴久鸿, 等. 中国药物经济学评价指南2020: 中英双语版. 北京: 中国市场出版社, 2020: 5-55.
28. DF372. 我国首个国产重组凝血八因子产品获批. 北京: 中国证券报, 2021-07-24. Available at: https://finance.eastmoney.com/a/202107242012743283.html.
29. 宣建伟, 孙巧. 中国罕见病药物经济学评估适用模型与支付阈值参考标准探讨. 国际药学研究杂志, 2019, 46(9): 659-665.

Chinese Journal of Evidence-Based Medicine

Pharmacoeconomic studies on coagulation factor Ⅷ for the treatment of hemophilia A: a systematic review

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