Health economic models in hemophilia A and utility assumptions from a clinician's perspective

Pediatric Blood and Cancer

Volumn 62, Issue 10, 2015, Pages 1826-1831

  Valente, Michele ^a,b   Cortesi, Paolo A ^c   Lassandro, Giuseppe ^d   Mathew, Prasad ^e,g   Pocoski, Jennifer ^e   Molinari, Angelo C ^f   Mantovani, Lorenzo G ^c   Giordano, Paola ^d  

^a BOCCONI UNIVERSITY   (Italy)

^b Bayer HEOR Intern   (United States)

^c UNIVERSITY OF MILANO BICOCCA   (Italy)

^d UNIVERSITY OF BARI   (Italy)

^e BAYER CORPORATION   (United States)

^f G GASLINI INSTITUTE   (Italy)

^g UNIVERSITY OF NEW MEXICO   (United States)

Author keywords

Cost Utility Analysis (CUA); Health Technology Assessment (HTA); Health related quality of life (HRQoL); Hemophilia; Prophylaxis; Quality adjusted life years (QALY)

Indexed keywords

RECOMBINANT BLOOD CLOTTING FACTOR 8;

ARTICLE; COST EFFECTIVENESS ANALYSIS; COST UTILITY ANALYSIS; HEALTH STATUS; HEMOPHILIA A; HUMAN; PRIORITY JOURNAL; PROPHYLAXIS; QUALITY ADJUSTED LIFE YEAR; COST BENEFIT ANALYSIS; ECONOMICS; HEALTH CARE QUALITY; HEMORRHAGE; STANDARDS; STATISTICAL MODEL;

COST-BENEFIT ANALYSIS; HEMOPHILIA A; HEMORRHAGE; HUMANS; MODELS, ECONOMIC; QUALITY ASSURANCE, HEALTH CARE;

EID: 84939564818     PISSN: 15455009     EISSN: 15455017     Source Type: Journal    
DOI: 10.1002/pbc.25543     Document Type: Article

References (31)

1. O'Mahony B, Noone D, Tolley K. An introduction to key concepts in health economics for haemophilia organizations. Haemophilia Organization Development. http://haemophilia.ie/PDF/Health%20Economics.pdf. Accessed February 16, 2015.
2. Briggs A, Sculpher M, Claxton K. Decision Modelling for Health Economic Evaluation. Oxford: Oxford University Press; 2006.
3. Briggs AH, Goeree R, Blackhouse G, O'Brien BJ. Probabilistic analysis of cost-effectiveness models: Choosing between strategies for gastroesophageal reflux disease. Med Decis Making 2002;22: 290-308.
4. Szende A, Oppe M, Devlin N, editors. EQ-5D Value Sets: Inventory, Comparative Review C and User Guide Series: EuroQol Group Monographs Vol. 2. Dordrecht: Springer 2007, 102 p.
5. Turchetti G, Scalone L, Della Casa Alberighi O, Mosca M, Montella S, Cortesi PA, Mantovani LG. The rationale of pharmacoeconomic analysis in rheumatologic indications. Clin Exp Rheumatol 2012; 30:S64-S71.
6. Gravelle S, Smith D. Discounting for health effects in cost-benefit and cost-effectiveness analysis. Health Econ 2001; 10:587-600.
7. Scalone L, Cortesi PA, Ciampichini R, Belisari A, D'Angiolella LS, Cesana G, Mantovani LG. Italian population-based values of EQ-5D health states. Value Health 2013; 16:814-822.
8. Bolton-Maggs PH, Pasi KJ. Hemophilias A and B. Lancet 2003; 361:1801-1809.
9. Ljung R. Prophylactic therapy in hemophilia. Blood Rev 2009; 23:267-274.
10. Collins PW. Personalized prophylaxis. Haemophilia 2012; 18:131-135.
11. Xi M, Makris M, Marcucci M, Santagostino E, Mannucci PM, Iorio A. Inhibitor development in previously treated hemophilia A patients: A systematic review, meta-analysis, and meta-regression. J Thromb Haemost 2013; 11:1655-1662.
12. Aledort LM, Haschmeyer RH, Pettersson H. The Orthopaedic Outcome Study Group. A longitudinal study of orthopaedic outcomes for severe factor-VIII-deficient haemophiliacs. J Intern Med 1994; 236:391-399.
13. Gringeri A, Ewenstein B, Reininger A. The burden of bleeding in haemophilia: Is one bleed too many? Haemophilia 2014; 20:459-463.
14. Fischer K, Hermans C. European Hemophilia Therapy Standardisation Board. The European principles of hemophilia care: A pilot investigation of adherence to the principles in Europe. Hemophilia 2013; 19:35-43.
15. Colombo GL, Dimatteo S, Mancuso ME, Santagostino E. Cost utility analysis of prophylaxis versus treatment on demand in severe hemophilia A. Clinico Econ Outcomes Res 2011; 3:55-61.
16. 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 hemophilia A. Hemophilia 2008; 14:743-752.
17. Miners AH, Sabin CA, Tolley KH, Lee CA. Cost-utility analysis of primary prophylaxis versus treatment on-demand for individuals with severe hemophilia. Pharmacoeconomics 2002; 20:759-774.
18. Miners AH. Economic evaluations of prophylaxis with clotting factor for people with severe haemophilia: Why do the results vary so much? Haemophilia 2013; 19:174-180.
19. Lippert B, Berger K, Berntorp E, Giangrande P, van den Berg M, Schramm W, Siebert U. European Haemophilia Economic Study Group. Cost effectiveness of haemophilia treatment: A cross-national assessment. Blood Coagul Fibrinolysis 2005; 16:477-485.
20. Farrugia A, Cassar J, Kimber MC, Bansal M, Fischer K, Auserswald G, O'Mahony B, Tolley K, Noone D, Balboni S. Treatment for life for severe hemophilia A. A cost-utility model for prophylaxis vs. on-demand treatment. Hemophilia 2013; 19:228-238.
21. Miners A. Revisiting the cost-effectiveness of primary prophylaxis with clotting factor for the treatment of severe haemophilia A. Hemophilia 2009; 15:881-887.
22. Schramm W, Royal S, Kroner B, Berntorp E, Giangrande P, Ludlam C, Gringeri A, Berger K, Szucs T. Clinical outcomes and resource utilization associated with hemophilia care in Europe. Hemophilia 2002; 8:33-43.
23. Naraine VS, Risebrough NA, Oh P, Blanchette VS, Lee S, Stain AM, Hedden D, Teitel JM, Feldman BM. Health-related quality of life treatments for severe haemophilia: Utility measurements using the Standard Gamble technique. Haemophilia 2002; 8:112-120.
24. Miners AH, Sabin CA, Tolley KH, Jenkinson C, Kind P, Lee CA. Assessing health related quality of life in individuals with hemophilia. Hemophilia 1999; 5:378-385.
25. Noone D, O'Mahony B, Van Dijk JP Prihodova L. A survey of the outcome of prophylaxis, on-demand treatment or combined treatment in 18-35-year old men with severe hemophilia in six countries. Hemophilia 2013; 19:45-50.
26. Fischer K, Steen-Carlsson K, Petrini P, Holmstrom M, Ljung R, Berntop E. Swedish vs. Dutch prophylactic strategy for hemophilia: Difference in outcome is small but cost-difference is large. J Thromb Hemost 2011; 9:744.
27. 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 Hemost 2011; 9:700-710.
28. Manco-Johnson MJI, Abshire TC, Shapiro AD, Riske B, Hacker MR, Kilcoyne R, Ingram JD, Manco-Johnson ML, Funk S, Jacobson L, Valentino LA, Hoots WK, Buchanan GR, DiMichele D, Recht M, Brown D, Leissinger C, Bleak S, Cohen A, Mathew P, Matsunaga A, Medeiros D, Nugent D, Thomas GA, Thompson AA, McRedmond K, Soucie JM, Austin H, Evatt BL. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med 2007; 357:344-535.
29. Duncan N, Roberson C, Lail A, Donfield S, Shapiro A. A hemophilia disease management programme targeting cost and utilization of specialty pharmaceuticals. Hemophilia 2014; 20:519-526.
30. National Institute for Health and Care Excellence (NICE). Consultation paper - value based assessment of health technologies. http://www.nice.org.uk/Media/Default/About/what-we-do/NICE-guidance/NICE-technology-appraisals/VBA-TA-Methods-Guide-for-Consultation.pdf Published April 2013, Accessed May 14, 2014.
31. Gringeri A, Mantovani L, Mackensen SV. Quality of life assessment in clinical practice in hemophilia treatment. Hemophilia 2012; 12:22-29.