The cost of hemophilia treatment: the importance of minimizing it without detriment to its quality

References

• Farrugia A, Noone D, Schlenkrich U, et al. Issues in assessing products for the treatment of hemophilia - the intersection between efficacy, economics, and ethics. J Blood Med. 2015;6:185–195.
   PubMed Web of Science ®Google Scholar
• Rocino A, Cortesi PA, Scalone L, et al. Immune tolerance induction in patients with haemophilia a and inhibitors: effectiveness and cost analysis in an European cohort (the ITER study). Haemophilia. 2016;22:96–102.
   PubMed Web of Science ®Google Scholar
• Rocha P, Carvalho M, Lopes M, et al. Costs and utilization of treatment in patients with hemophilia. BMC Health Serv Res. 2015;15:484.
   PubMed Web of Science ®Google Scholar
• Chen SL. Economic costs of hemophilia and the impact of prophylactic treatment on patient management. Am J Manag Care. 2016;22(5 Suppl):s126–33.
   PubMed Web of Science ®Google Scholar
• Golestani M, Eshghi P, Rasekh HR, et al. Cost-effectiveness analysis of biogeneric recombinant activated factor VII (AryoSeven™) and activated prothrombin complex concentrates (FEIBA™) to treat hemophilia A patients with inhibitors in Iran. Iran J Pharm Res. 2016;15:669–677.
   PubMed Web of Science ®Google Scholar
• 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;28:425–430.
   PubMed Web of Science ®Google Scholar
• Drummond M, Houwing N, Slothuus U, et al. Making economic evaluations more helpful for treatment choices in haemophilia. Haemophilia. 2017;23:e58–e66.
   PubMed Web of Science ®Google Scholar
• 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;23:422–429.
   PubMed Web of Science ®Google Scholar
• McMullen S, Buckley B, Hall E 2nd, et al. Budget impact analysis of prolonged half-life recombinant FVIII therapy for hemophilia in the United States. Value Health. 2017;20:93–99.
   PubMed Web of Science ®Google Scholar
• Carneiro JDA, Blanchette V, Ozelo MC, et al. Comparing the burden of illness of haemophilia between resource-constrained and unconstrained countries: the São Paulo-Toronto hemophilia study. Haemophilia. 2017;23:682–688.
   PubMed Web of Science ®Google Scholar
• O’Hara J, Hughes D, Camp C, et al. The cost of severe haemophilia in Europe: the CHESS study. Orphanet J Rare Dis. 2017;12:106.
   PubMed Web of Science ®Google Scholar
• Shrestha A, Eldar-Lissai A, Hou N, et al. Real-world resource use and costs of haemophilia A-related bleeding. Haemophilia. 2017;23:e267–e275.
   PubMed Web of Science ®Google Scholar
• Kenet G, Oladapo A, Epstein JD, et al. Estimating the potential cost of a high dose immune tolerance induction (ITI) therapy relative to the cost of a combined therapy of a low dose ITI therapy with bypassing agent prophylaxis. Haemophilia. 2017;23:e394–e402.
   PubMed Web of Science ®Google Scholar
• Singh P, Mukherjee K. Cost-benefit analysis and assessment of quality of care in patients with hemophilia undergoing treatment at National Rural Health Mission in Maharashtra, India. Value Health Reg Issues. 2017;12:101–106.
   PubMedGoogle Scholar
• Chen CX, Baker JR, Nichol MB. Economic burden of illness among persons with hemophilia B from HUGS Vb: examining the association of severity and treatment regimens with costs and annual bleed rates. Value Health. 2017;20:1074–1082.
   PubMed Web of Science ®Google Scholar
• Nugent D, O’Mahony B, Dolan G. International Haemophilia Access Strategy Council. Value of prophylaxis vs on-demand treatment: application of a value framework in hemophilia. Haemophilia. 2018;24:755–765.
   PubMed Web of Science ®Google Scholar
• Thorat T, Neumann PJ, Chambers JD. Hemophilia burden of disease: A systematic review of the cost-utility literature for hemophilia. J Manag Care Spec Pharm. 2018;24:632–642.
   PubMed Web of Science ®Google Scholar
• D’Angiolella LS, Cortesi PA, Rocino A, et al. The socioeconomic burden of patients affected by hemophilia with inhibitors. Eur J Haematol. 2018;101:435–456.
   PubMed Web of Science ®Google Scholar
• Buckley B, Dreyfus J, Prasad M, et al. Burden of illness and costs among paediatric haemophilia patients with and without central venous access devices treated in US hospitals. Haemophilia. 2018;24:e93–e102.
   PubMed Web of Science ®Google Scholar
• 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;21:318–325.
   PubMed Web of Science ®Google Scholar
• Peyvandi F, Tavakkoli F, Frame D, et al. Burden of mild haemophilia A: systematic literature review. Haemophilia. 2019;25:755–763.
   PubMed Web of Science ®Google Scholar
• Rankin PJ, Bell GK, Wilsdon T. Chapter 2 of the pharmaceutical pricing compendium: A practical guide to the pricing and reimbursement of medicines. In: Global Pricing Strategies for Pharmaceutical Product Launches. Available from: www.pharmaceuticalpricing.com.
   Google Scholar
• Escobar MA. Health economics in haemophilia: a review from the clinician’s perspective. Haemophilia. 2010;16(Suppl 3):29–34.
   PubMed Web of Science ®Google Scholar
• Jimenez-Yuste V, Núñez R, Romero JA, et al. Cost-effectiveness of recombinant activated factor VII vs. plasma-derived activated prothrombin complex concentrate in the treatment of mild-to-moderate bleeding episodes in patients with severe haemophilia A and inhibitors in Spain. Haemophilia. 2013;19:841–846.
   PubMed Web of Science ®Google Scholar
• Dargaud Y, Delavenne X, Hart DP, et al. Individualized PK-based prophylaxis in severe haemophilia. Haemophilia. 2018;24(Suppl 2):3–17.
   PubMedGoogle Scholar
• Johnson KA, Zhou ZY. Costs of care in hemophilia and possible implications of health care reform. Hematology Am Soc Hematol Educ Program. 2011;2011:413–418.
   PubMedGoogle Scholar
• Angelis A, Tordrup D, Kanavos P. Socio-economic burden of rare diseases: a systematic review of cost of illness evidence. Health Policy. 2015;119:964–979.
   PubMed Web of Science ®Google Scholar
• Dalton DR. Hemophilia in the managed care setting. Am J Manag Care. 2015 Mar;21(6 Suppl):S123–30.
   PubMed Web of Science ®Google Scholar
• Cortesi PA, D’Angiolella LS, Lafranconi A, et al. Modern treatments of haemophilia: review of cost-effectiveness analyses and future directions. Pharmacoeconomics. 2018;36:263–284.
   PubMed Web of Science ®Google Scholar
• Vepsäläinen K, Riikonen P, Lassila R, et al. Long-term clinical and economic outcomes in previously untreated paediatric patients with severe haemophilia A: a nationwide real-world study with 700 person-years. Haemophilia. 2018;24:436–444.
   PubMed Web of Science ®Google Scholar
• Café A, Carvalho M, Crato M, et al. Haemophilia A: health and economic burden of a rare disease in Portugal. Orphanet J Rare Dis. 2019;14:211.
   PubMed Web of Science ®Google Scholar
• Duncan N, Roberson C, Lail A, et al. A haemophilia disease management programme targeting cost and utilization of specialty pharmaceuticals. Haemophilia. 2014;20:519–526.
   PubMed Web of Science ®Google Scholar
• Disease management programs: improving health while reducing costs? Available from: hpi.georgetown.edu/management
   Google Scholar
• Malouin RA, Mckernan L, Forsberg A, et al. Impact of the 340B pharmacy program on services and supports for persons served by Hemophilia Treatment centers in the United States. Matern Child Health J. 2018;22:1240–1246.
   PubMed Web of Science ®Google Scholar
• Khleif AA, Rodriguez N, Brown D, et al. Utilization patterns and associated costs of factor assistance programmes among persons with haemophilia: a single institution review. Haemophilia. 2012;18:e95–e100.
   PubMed Web of Science ®Google Scholar
• Li Z, Wu J, Zhao Y, et al. Influence of medical insurance schemes and charity assistance projects on regular prophylaxis treatment of the boys with severe haemophilia A in China. Haemophilia. 2018;24:126–133.
   PubMed Web of Science ®Google Scholar
• Hemophilia patients to get free medication. The New Times. Rwanda´s leading daily. Available from: www.newtimes.co.rw/section/read/90655
   Google Scholar
• Hay JW, Zhou ZY. Economical comparison of APCC vs. rFVIIa for mild-to-moderate bleeding episodes in haemophilia patients with inhibitors. Haemophilia. 2011;17:e969–974.
   PubMed Web of Science ®Google Scholar
• Hay JW, Zhou ZY. Systematic literature review of economics analysis on treatment of mild-to-moderate bleeds with aPCC versus rFVIIa. J Med Econ. 2011;14:516–525.
   PubMedGoogle Scholar
• Bonnet P, Gringeri A, Gomperts E, et al. Modeling costs and outcomes associated with a treatment algorithm for problem bleeding episodes in patients with severe hemophilia and high-titer inhibitors. Am Health Drug Benefits. 2011;4:219–231.
   PubMedGoogle Scholar
• Rasekh HR, Imani A, Karimi M, et al. Cost-utility analysis of immune tolerance induction therapy versus on-demand treatment with recombinant factor VII for hemophilia A with high titer inhibitors in Iran. Clinicoecon Outcomes Res. 2011;3:207–212.
   PubMedGoogle Scholar
• Salaj P, Penka M, Smejkal P, et al. Economic analysis of recombinant activated factor VII versus plasma-derived activated prothrombin complex concentrate in mild to moderate bleeds: haemophilia registry data from the Czech Republic. Thromb Res. 2012;129:e233–237.
   PubMed Web of Science ®Google Scholar
• Hay JW, Chaugule SC, Young G. Bypass therapy assay testing as a strategy to reduce costs for treatment of haemophilia patients with inhibitors. Haemophilia. 2013;19:711–719.
   PubMed Web of Science ®Google Scholar
• Salaj P, Kubes R, Cetkovsky P, et al. Economic evaluation of rFVIIa high initial dose compared to rFVIIa standard initial dose in patients with haemophilia with inhibitors using the Czech Hemorec Registry. Thromb Res. 2014;133:162–167.
   PubMed Web of Science ®Google Scholar
• Villarrubia R, Oyagüez I, Álvarez-Román MT, et al. Cost analysis of prophylaxis with activated prothrombin complex concentrate vs. on-demand therapy with activated factor VII in severe haemophilia A patients with inhibitors, in Spain. Haemophilia. 2015;21:320–329.
   PubMed Web of Science ®Google Scholar
• Mehta DA, Oladapo AO, Epstein JD, et al. A budget impact model of hemophilia bypassing agent prophylaxis relative to recombinant factor VIIa on-demand. J Manag Care Spec Pharm. 2016;22:149–157.
   PubMed Web of Science ®Google Scholar
• Messori A. Inhibitors in hemophilia A: a pharmacoeconomic perspective. Semin Thromb Hemost. 2018;44:561–567.
   PubMed Web of Science ®Google Scholar
• Sánchez-Vanegas G, Linares A, Sarmiento I, et al. Cost of patients with hemophilia A and high-titer inhibitors in Colombia. Value Health Reg Issues. 2019 Oct 8;20:164–171. Epub ahead of print. .
   PubMedGoogle Scholar
• Patel AM, Corman SL, Chaplin S, et al. Economic impact model of delayed inhibitor development in patients with hemophilia A receiving emicizumab for the prevention of bleeding events. J Med Econ. 2019 Oct 7:1–10. Epub ahead of print. DOI: 10.1080/13696998.2019.1669614.
   Web of Science ®Google Scholar
• Cortesi PA, Castaman G, Trifirò G, et al. Cost-effectiveness and budget impact of emicizumab prophylaxis in haemophilia A patients with inhibitors. Thromb Haemost. 2019 Dec 30 Epub ahead of print. DOI:10.1055/s-0039-3401822.
   PubMed Web of Science ®Google Scholar
• Tortella BJ, Alvir J, McDonald M, et al. Real-world analysis of dispensed IUs of coagulation factor IX and resultant expenditures in hemophilia B patients receiving standard half-life versus extended half-life products and those switching from standard half-life to extended half-life products. J Manag Care Spec Pharm. 2018;24:643–653.
   PubMed Web of Science ®Google Scholar
• 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. 2019 Jul 6 Epub ahead of print. DOI:10.1007/s41669-019-0158-8.
   PubMedGoogle Scholar
• Croteau SE, Cheng D, Cohen AJ, et al. Regional variation and cost implications of prescribed extended half-life factor concentrates among U.S. Haemophilia Treatment Centres for patients with moderate and severe haemophilia. Haemophilia. 2019;25:668–675.
   PubMed Web of Science ®Google Scholar