Haemophilia prophylaxis: How can we justify the costs?

Haemophilia

Volumn 18, Issue 5, 2012, Pages 680-684

  Feldman, B M ^a   Berger, K ^b   Bohn, R ^c   Carcao, M ^a   Fischer, K ^d   Gringeri, A ^e   Hoots, K ^f   Mantovani, L ^g   Willan, A R ^h   Schramm, W ⁱ  

^a HOSPITAL FOR SICK CHILDREN   (Canada)

^b LUDWIG MAXIMILIANS UNIVERSITY   (Germany)

^c Rhonda L Bohn LLC   (United States)

^d UNIVERSITY MEDICAL CENTER UTRECHT   (Netherlands)

^e UNIVERSITY OF MILAN   (Italy)

^f NATIONAL HEART LUNG AND BLOOD INSTITUTE   (United States)

^g UNIVERSITY OF NAPLES FEDERICO II   (Italy)

^h HOSPITAL FOR SICK CHILDREN RESEARCH INSTITUTE   (Canada)

ⁱ UNIVERSITY OF MUNICH   (Germany)

Author keywords

Cost; Haemophilia; Prophylaxis; Treatment; Willingness to pay

Indexed keywords

RECOMBINANT BLOOD CLOTTING FACTOR 8; RECOMBINANT BLOOD CLOTTING FACTOR 9;

DISEASE SEVERITY; DRUG COST; DRUG SAFETY; HEALTH CARE COST; HEMOPHILIA; HEMOPHILIA PROPHYLAXIS; HUMAN IMMUNODEFICIENCY VIRUS INFECTION; ITALY; MEDICAL DECISION MAKING; NOTE; OUTCOME ASSESSMENT; PRIORITY JOURNAL; PROPHYLAXIS; SWEDEN; VIRUS INACTIVATION;

BLOOD COAGULATION FACTORS; DRUG COSTS; HEMOPHILIA A; HEMORRHAGE; HUMANS; MALE; PREVENTIVE MEDICINE;

EID: 84865571022     PISSN: 13518216     EISSN: 13652516     Source Type: Journal    
DOI: 10.1111/j.1365-2516.2012.02790.x     Document Type: Note

References (64)

1. Larsson SA, Larsson SA. Hemophilia in Sweden. Studies on demography of hemophilia and surgery in hemophilia and von Willebrand's disease. Acta Medica Scandinavica - Supplementum 1984; 684: 1-72.
2. Rosendaal FR, Smit C, Varekamp I et al. Modern haemophilia treatment: medical improvements and quality of life. J Intern Med 1990; 228: 633-40.
3. Ahlberg A. Haemophilia in Sweden. VII. Incidence, treatment and prophylaxis of arthropathy and other musculo-skeletal manifestations of haemophilia A and B. Acta Orthop Scand Suppl 1965; (Suppl 77): 3-132.
4. Plug I, Van der Bom JG, Peters M et al. Mortality and causes of death in patients with hemophilia, 1992-2001: a prospective cohort study. J Thromb Haemost 2006; 4: 510-6.
5. Darby SC, Kan SW, Spooner RJ et al. Mortality rates, life expectancy, and causes of death in people with hemophilia A or B in the United Kingdom who were not infected with HIV. Blood 2007; 110: 815-25.
6. van Genderen FR, Fischer K, Heijnen L et al. Pain and functional limitations in patients with severe haemophilia. Haemophilia 2006; 12: 147-53.
7. Shapiro AD, Donfield SM, Lynn HS et al. Defining the impact of hemophilia: the Academic Achievement in Children with Hemophilia Study. Pediatrics 2001; 108: E105.
8. Miners AH, Sabin CA, Tolley KH, Jenkinson C, Kind P, Lee CA. Assessing health-related quality-of-life in individuals with haemophilia. Haemophilia 1999; 5: 378-85.
9. Naraine VS, Risebrough NA, Oh P et al. Health-related quality-of-life treatments for severe haemophilia: utility measurements using the Standard Gamble technique. Haemophilia 2002; 8: 112-20.
10. Globe DR, Cunningham WE, Andersen R et al. Haemophilia Utilization Group Study: assessment of functional health status in haemophilia. Haemophilia 2002; 8: 121-8.
11. History of Hemophilia. World Federation of Hemophilia; Available at: http://www.wfh.org/2/1/1_1_8_History_Hemophilia.htm. Accessed March 25, 2012.
12. Heemstra HE, Zwaan T, Hemels M et al. Cost of severe haemophilia in Toronto. Haemophilia 2005; 11: 254-60.
13. Globe DR, Curtis RG, Koerper MA, Committee HS. Utilization of care in haemophilia: a resource-based method for cost analysis from the Haemophilia Utilization Group Study (HUGS). Haemophilia 2004; 10(Suppl 1): 63-70.
14. Schramm W, Royal S, Kroner B et al. Clinical outcomes and resource utilization associated with haemophilia care in Europe. Haemophilia 2002; 8: 33-43.
15. Nilsson IM, Berntorp E, Lofqvist T, Pettersson H. Twenty-five years' experience of prophylactic treatment in severe haemophilia A and B. J Intern Med 1992; 232: 25-32.
16. Manco-Johnson MJ, Abshire TC, Brown D et al. Initial results of a randomized, prospective trial of prophylaxis to prevent joint disease in young children with factor VIII (FVIII) deficiency. Blood 2005; 106: 3.
17. Gringeri A, Lundin B, von Mackensen S, Mantovani L, Mannucci PM. A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT Study). J Thromb Haemost 2011; 9: 700-10.
18. Manco-Johnson MJ, Abshire TC, Shapiro AD et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med 2007 9;357: 535-44.
19. Fischer K, Van Den Berg M. Prophylaxis for severe haemophilia: clinical and economical issues. Haemophilia 2003; 9: 376-81.
20. Fischer K, van der Bom JG, Mauser-Bunschoten EP et al. Changes in treatment strategies for severe haemophilia over the last 3 decades: effects on clotting factor consumption and arthropathy. Haemophilia 2001; 7: 446-52.
21. van den Berg HM, Fischer K, Mauser-Bunschoten EP et al. Long-term outcome of individualized prophylactic treatment of children with severe haemophilia. Br J Haematol 2001; 112: 561-5.
22. Feldman BM, Pai M, Rivard GE et al. Tailored prophylaxis in severe hemophilia A: interim results from the first 5years of the Canadian Hemophilia Primary Prophylaxis Study. J Thromb Haemost 2006; 4: 1228-36.
23. Bohn RL, Avorn J, Glynn RJ, Choodnovskiy I, Haschemeyer R, Aledort LM. Prophylactic use of factor VIII: an economic evaluation. Thromb Haemost 1998; 79: 932-7.
24. Lippert B, Berger K, Berntorp E et al. Cost effectiveness of haemophilia treatment: a cross-national assessment. Blood Coagul Fibrinol 2005; 16: 477-85.
25. Miners AH, Sabin CA, Tolley KH, Lee CA. Cost-utility analysis of primary prophylaxis versus treatment on-demand for individuals with severe haemophilia. Pharmacoeconomics 2002; 20: 759-74.
26. Smith PS, Teutsch SM, Shaffer PA, Rolka H, Evatt B. Episodic versus prophylactic infusions for hemophilia A: a cost-effectiveness analysis. J Pediatr 1996; 129: 424-31.
27. Miners A. Revisiting the cost-effectiveness of primary prophylaxis with clotting factor for the treatment of severe haemophilia A. Haemophilia 2009; 15: 881-7.
28. Risebrough N, Oh P, Blanchette V, Curtin J, Hitzler J, Feldman BM. Cost-utility analysis of Canadian tailored prophylaxis, primary prophylaxis and on-demand therapy in young children with severe haemophilia A. Haemophilia 2008; 14: 743-52.
29. Buxton MJ. Economic evaluation and decision making in the UK. Pharmacoeconomics 2006; 24: 1133-42.
30. Devlin N, Parkin D. Does NICE have a cost-effectiveness threshold and what other factors influence its decisions? A binary choice analysis. Health Econ 2004; 13: 437-52.
31. Cook RJ, Sackett DL. The number needed to treat: a clinically useful measure of treatment effect. BMJ 1995; 18: 310.
32. Kasper CK. Products for clotting factor replacement in developing countries. Semin Thromb Hemost 2005; 31: 507-12.
33. Valentino LA, Oza VM. Blood safety and the choice of anti-hemophilic factor concentrate.[see comment]. Pediatr Blood Cancer 2006; 47: 245-54.
34. Kern M, Blanchette V, Stain AM, Einarson TR, Feldman BM. Clinical and cost implications of target joints in Canadian boys with severe hemophilia A. J Pediatr 2004; 145: 628-34.
35. Drummond MF, Sculpher MJ, Torrance GW, O'Brien BJ, Stoddart GL. Cost-benefit Analysis. Methods for the Economic Evaluation of Health Care Programmes, 3 edn. Oxford: Oxford University Press, 2005: 211-45.
36. Blanchette VS, McReady M, Achonu C, Abdolell M, Rivard G, Manco-Johnson MJ. A survey of factor prophylaxis in boys with haemophilia followed in North American haemophilia treatment centres. Haemophilia 2003; 9(Suppl.1): 19-26.
37. Butler RB, McClure W, Wulff K. Practice patterns in haemophilia A therapy - a survey of treatment centres in the United States. Haemophilia 2003; 9: 549-54.
38. Aledort LM, Haschmeyer RH, Pettersson H. A longitudinal study of orthopaedic outcomes for severe factor-VIII-deficient haemophiliacs. J Intern Med 1994; 236: 391-9.
39. Ljung R, Aronis-Vournas S, Kurnik-Auberger K et al. Treatment of children with haemophilia in Europe: a survey of 20 centres in 16 countries. Haemophilia 2000; 6: 619-24.
40. Lockwood M. Humans valuing nature: synthesising insights from philosophy, psychology and economics. Environ Values 1999; 8: 381-401.
41. Kuchler F, Golan E. Assigning values to life: comparing methods for valuing health risks: Economic Research Service, USDA.1999 December.
42. Steen Carlsson K, Hojgard S, Lethagen S, Lindgren A, Berntorp E, Lindgren B. Willingness to pay for on-demand and prophylactic treatment for severe haemophilia in Sweden. Haemophilia 2004; 10: 527-41.
43. Mantovani LG, Monzini MS, Mannucci PM et al. Differences between patients', physicians' and pharmacists' preferences for treatment products in haemophilia: a discrete choice experiment. Haemophilia 2005; 11: 589-97.
44. Cookson R. Willingness to pay methods in health care: a sceptical view. Health Econ 2003; 12: 891-4.
45. Olsen JA, Donaldson C. Helicopters, hearts and hips: using willingness to pay to set priorities for public sector health care programmes. Soc Sci Med 1998; 46: 1-12.
46. West P, West P. Health economics in paediatrics.[comment]. Arch Dis Child 2004; 89: 2-3.
47. Petrou S Should health gains by children be given the same value as health gains by adults in an economic evaluation framework? In: Ungar WJ, ed. Economic Evaluation in Child Health. Oxford: Oxford University Press, 2010: 271-87.
48. Williams A. Intergenerational equity: an exploration of the 'fair innings' argument. Health Econ 1997; 6: 117-32.
49. Tsuchiya A, Dolan P, Shaw R. Measuring people's preferences regarding ageism in health: some methodological issues and some fresh evidence. Soc Sci Med 2003; 57: 687-96.
50. Walker I, Pai M, Akabutu J et al. The Canadian Hemophilia Registry as the basis for a national system for monitoring the use of factor concentrates. Transfusion 1995; 35: 548-51.
51. Hughes DA, Tunnage B, Yeo ST. Drugs for exceptionally rare diseases: do they deserve special status for funding? QJM 2005; 98: 829-36.
52. McKie J, Richardson J. The rule of rescue. Soc Sci Med 2003; 56: 2407-19.
53. Niezen MG, Stolk EA, Steenhoek A, Uyl-De Groot CA. Inequalities in oncology care: Economic consequences of high cost drugs. Eur J Cancer 2006; 42: 2887-92.
54. McCabe C, Tsuchiya A, Claxton K, Raftery J Orphan drugs revisited. QJM 2006; 99: 341-5; discussion 50-1.
55. Hey JD, Patel MS. Prevention and cure? or: is an ounce of prevention worth a pound of cure? J Health Econ 1983; 2: 119-38.
56. Resnik S. Blood Saga: Hemophilia, AIDS and the Survival of a Community. Berkeley and Los Angeles, CA: University of California Press, 1999.
57. Wilson K, Leonard B, Wright R et al. Application of the precautionary principle by senior policy officials: results of a Canadian survey. Risk Anal 2006; 26: 981-8.
58. Wilson K, Graham I, Ricketts M, Dornan C, Laupacis A, Hebert P. Variant Creutzfeldt-Jakob disease and the Canadian blood system after the tainted blood tragedy. Soc Sci Med 2007; 64: 174-85.
59. Tubiana M Conclusions. The precautionary principle: its advantages and risks. Bull Acad Natl Med 2000; 184: 969-93.
60. Eastaugh SR. Valuation of the benefits of risk-free blood. Willingness to pay for hemoglobin solutions. Int J Technol Assess Health Care 1991; 7: 51-7.
61. Camerer CF, Fehr E. When does 'economic man' dominate social behavior? Science 2006; 311: 47-52.
62. Evatt BL, Black C, Batorova A, Street A, Srivastava A. Comprehensive care for haemophilia around the world. Haemophilia 2004; 10(Suppl 4): 9-13.
63. Srivastava A. Factor replacement therapy in haemophilia - are there models for developing countries? Haemophilia 2003; 9: 391-6.
64. Poon MC, Luke KH. Haemophilia care in China: achievements of a decade of World Federation of Hemophilia treatment centre twinning activities. Haemophilia 2008; 14: 879-88.