Economic evaluations of prophylaxis with clotting factor for people with severe haemophilia: Why do the results vary so much?

Haemophilia

Volumn 19, Issue 2, 2013, Pages 174-180

  Miners, A H ^a  

^a LONDON SCHOOL OF HYGIENE AND TROPICAL MEDICINE   (United Kingdom)

Author keywords

Cost; Economics; Haemophilia; Prophylaxis and economic evaluation

Indexed keywords

BLOOD CLOTTING FACTOR;

COST BENEFIT ANALYSIS; COST EFFECTIVENESS ANALYSIS; DECISION MAKING; DRUG COST; ECONOMIC EVALUATION; EMPLOYMENT; EVIDENCE BASED MEDICINE; HEMOPHILIA; PRIORITY JOURNAL; REVIEW; TREATMENT DURATION;

COAGULANTS; COST-BENEFIT ANALYSIS; DRUG COSTS; FACTOR VIII; HEMOPHILIA A; HUMANS; QUALITY-ADJUSTED LIFE YEARS;

EID: 84874075767     PISSN: 13518216     EISSN: 13652516     Source Type: Journal    
DOI: 10.1111/hae.12009     Document Type: Review

References (38)

1. Berntorp E, Astermark J, Baghaei F et al. Treatment of haemophilia A and B and von Willebrand's disease: summary and conclusions of a systematic review as part of a Swedish health-technology assessment. Haemophilia 2012; 18: 158-65.
2. Swedish Council on Health Technology Assessment. Treatment of Hemophilia A and B and von Willebrand Disease. Mmlnlycke, Sweden: Elanders, 2011. No 208E.
3. Drummond MF, Sculpher MJ, Torrance GW, O'Brien B, Stoddart GL. Methods for the Economic Evaluation of Health Care Programmes. 3rd edn. Oxford: Oxford University Press, England, 2005.
4. Griffiths UK, Miners A. Economic evaluations of Haemophilus influenzae type b vaccine: systematic review of the literature. Exp Rev Pharmacoecon Outcomes Res 2009; 9: 333-46.
5. 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: 745-52.
6. Gringeri A, Fusco F, Riva S et al. Direct costs of children with haemophilia A undergoing prophylaxis or episodic treatment: results from the ESPRIT study. J Thromb Haemost 2011; 9: 927.
7. Fischer K, Steen-Carlsson K, Petrini P, Holmstrom M, Ljung R, Berntorp E. Swedish vs. Dutch prophylactic strategy for hemophilia: difference in outcome is small but cost-difference is large. J Thromb Haemost 2011; 9: 744.
8. Fischer K, Pouw ME, Lewandowski D et al. A modeling approach to evaluate long-term outcome of prophylactic and on demand treatment strategies for severe hemophilia A. Haematologica 2011; 96: 738-43.
9. Matthew P, Boer R, Lalla A et al. Cost-effectiveness analysis of primary prophylaxis versus on-demand treatment for individuals with severe hemophilia based on the Joint Outcome Study. Haemophilia 2010; 16: 58.
10. Tagliaferri A, Rivolta G, Coppola A et al. Prophylaxis versus on-demand therapy through economic report (P.O.T.T.E.R.) study. J Thromb Haemost 2009; 7: 819.
11. Liou WS, Tu TC, Cheng SN et al. Secondary prophylaxis treatment versus on-demand treatment for patients with severe haemophilia A: comparisons of cost and outcomes in Taiwan. Haemophilia, 2011; 17: 45-54.
12. Boer R, Lalla A, Mathew P et al. US cost effectiveness analysis of primary prophylaxis versus on-demand treatment in hemophilia: design and rationale of a comprehensive model. Value Health 2010; 3: A216.
13. Mucino-Ortega E, Leyva-Bravo V, Gutierrez C et al. Cost utility analysis of the profilaxis versus on-demand treatment with recombinant factor IX for the treatment of hemophilia B in Mexico. Value Health 2012; 15: A103-4.
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. Bohn RL, Avorn J, Glynn RJ et al. Prophylactic use of factor VIII: an economic evaluation. Thromb Haemost 1998; 79: 932-7.
16. Briggs AH, O'Brien B. The death of cost-minimisation analysis?. Health Econ 2001; 10: 179-84.
17. Nilsson IM, Berntorp E, Lofqvist T et al. Twenty-five years' experience of prophylactic treatment in severe haemophilia A and B. J Intern Med 1992; 232: 25-32.
18. Lippert B, Berger K, Berntorp E et al. Cost effectiveness of haemophilia treatment: a cross-national assessment. Blood Coagul Fibrinolysis 2005; 16: 477-85.
19. Miners A. Revisiting the cost-effectiveness of primary prophylaxis with clotting factor for the treatment of severe haemophilia A. Haemophilia 2009; 15: 881-7.
20. Miners AH, Sabin CA, Tolley KH et al. A cost-utility analysis of primary prophylaxis versus treatment on-demand for individuals with severe haemophilia. PharmacoEconomics 2002; 20: 759-74.
21. Risebrough NA, 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: 743-52.
22. Institute for Quality and Efficiency in Health Care. General Methods for the Assessment of the Relation of Benefits to Costsv 1.0. 2009 02/09/10]; Available from: http://www.iqwig.de/download/General_Methods_for_the_Assessment_of_the_Relation_of_Benefits_to_Costs.pdf.
23. National Institute for Health and Clinical Excellence. Guide to the Methods of Technology Appraisal. London, UK: ???????, 2008
24. Cairns J. Discounting and health benefits: another perspective. Health Econ 1992; 1: 76-9.
25. 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; 357: 535-44.
26. Steen Carlsson K, Hojgard S, Lethagen S. et al. Willingness to pay for on-demand and prophylactic treatment for severe haemophilia in Sweden. Haemophilia, 2004; 10: 527-41.
27. Steen Carlsson K, Höjgård S, Lindgren A et al. Costs of on-demand and prophylactic treatment for severe haemophilia in Norway and Sweden. Haemophilia 2004; 10: 515-26.
28. Drummond MF, Jefferson TO. Guidelines for authors and peer reviewers of economic submissions to the BMJ. The BMJ economic evaluation working party. BMJ 1996; 313: 275-83.
29. Weinstein MC, O'Brien B, Hornberger J et al. Principles of good practice for decision analytic modeling in health-care evaluation: report of the ISPOR task force on good research practices - Modeling studies. Value Health 2003; 6: 9-17.
30. The EuroQol G. EuroQol - a new facility for the measurement of health related quality of life. Health Policy 1994; 16: 655-62.
31. 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.
32. Ryan M. Discrete choice experiments in health care. BMJ 2004; 328: 360-1.
33. Culyer AJ, Bombard Y. An equity framework for health technology assessments. Med Decis Making 2011;. doi:10.1177/0272989X11426484.
34. Smith PS, Teutsch SM, Shaffer PA et al. Episodic versus prophylactic infusions for hemophilia A: a cost-effectiveness analysis. J Pediatr 1996; 129: 424-31.
35. Szucs TD, Offner A, Schramm W. Socioeconomic impact of haemophilia care: results of a pilot study. Haemophilia 1996; 2: 211-7.
36. Miners AH, Sabin CA, Tolley KH et al. Assessing the effectiveness and cost-effectiveness of prophylaxis against bleeding in patients with severe haemophilia and severe von Willebrand's disease. J Intern Med 1998; 244: 515-22.
37. Daliri AAK, 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: 584-7.
38. 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.