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Boletín Médico del Hospital Infantil de México

2013, Number 4

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Bol Med Hosp Infant Mex 2013; 70 (4)

Cost-effectiveness analysis of prophylaxis vs. “on demand” approach in the management in children with hemophilia A in Mexico

Salinas-Escudero G, Galindo-Suárez RM, Rely K, Carrillo-Vega MF, Muciño-Ortega E

Language: Spanish
References: 50
Page: 290-298
PDF size: 76.81 Kb.


ABSTRACT

Background. Hemophilia A is due to a deficiency of factor VIII. Treatment consists primarily of increasing the concentration of FVIII in the blood using replacement products. The aim of this study was to estimate the clinical and economic benefits of prophylactic management with factor VIII in children with hemophilia A in Mexico. We undertook this study to estimate the clinical and economic benefits of prophylactic management (PROF) with factor VIII (FVIII) in children with severe hemophilia in Mexico.
Methods. We carried out an economic evaluation of PROF vs. treatment on demand (OD). The strategies compared were management with PROF consisting of recombinant FVIII (rFVIII) 25 IU kg every other day vs. OD management consisting of plasma-derived FVIII (pdFVIII) 40 IU kg. A Markov model was performed with a time horizon of 16 years in patients with severe hemophilia for 2 years, reporting the number of bleeding events averted. We used a discount rate of 5%. The results are expressed in Mexican pesos (2012).
Results. The incremental cost of PROF regarding SD was $ 7,727,554 pesos. PROF management provides a reduction of 112 BA vs. OD management (162.9 vs. 50.7). Cost per averted bleed was $68,876 pesos.
Conclusions. Management with PROF reduces the number of bleeding events facing children with hemophilia A compared to OD management. PROF is a cost-effective alternative to reduce bleeding ($68,876 pesos per bleed averted) according to the willingness to pay established by health authorities in Mexico.


REFERENCES

  1. World Federation of Hemophilia. Report on the Annual Global Survey 2011. Canada; 2013. Disponible en: http://www1.wfh.org/publications/files/pdf-1488.pdf.

  2. World Federation of Hemophilia. Guidelines for the management of hemophilia. Canada; 2012. Disponible en: http://www1.wfh.org/publications/files/pdf-1472.pdf

  3. Skinner MW. WFH: closing the global gap—achieving optimal care. Haemophilia 2012;18(suppl 4):1-12.

  4. Zaiden R, Jardine LF, Lorenzana A, Besa EC, Crouch GD, Sawaf H, et al. Hemophilia A. Medscape Reference. Disponible en: http://emedicine.medscape.com/article/779322-overview#showall

  5. Bolton-Maggs PH, Pasi KJ. Haemophilias A and B. Lancet 2003;361:1801-1809.

  6. White GC 2nd, Rosendaal F, Aledort LM, Lusher JM, Rothschild C, Ingerslev J, et al. Definitions in hemophilia. Recommendation of the scientific subcommittee on factor VIII and factor IX of the scientific and standardization committee of the International Society on Thrombosis and Haemostasis. Thromb Haemost 2001;85:560.

  7. Rodriguez-Merchan EC. Aspects of current management: orthopaedic surgery in haemophilia. Haemophilia 2012;18:8-16.

  8. Liras A. Recombinant proteins in therapeutics: haemophilia treatment as an example. Int Arch Med 2008;1:4.

  9. Secretaría de Salud. Diagnóstico y Tratamiento de Hemofilia Pediátrica. México; 2009.

  10. Instituto Mexicano del Seguro Social. Guías de Práctica Clínica. Diagnóstico y Tratamiento de Hemofilia Pediátrica. México; 2009.

  11. Keeling D, Tait C, Makris M. Guidelines on the selection and use of therapeutic products to treat haemophilia and other hereditary bleeding disorders. A United Kingdom Haemophilia Center Doctors’ Organization (UKHCDO) guideline approved by the British Committee for Standards in Haematology. Haemophilia 2008;14:671-684.

  12. Colvin BT, Astermark J, Fischer K, Gringeri A, Lassila R, Schramm W, et al. European principles of haemophilia care. Haemophilia 2008;14:361-374.

  13. Mannucci PM, Tuddenham EG. The hemophilias–from royal genes to gene therapy. N Engl J Med 2001;344:1773-1779.

  14. Fischer K, van der Bom JG, Molho P, Negrier C, Mauser-Bunschoten EP, Roosendaal G, et al. Prophylactic versus on-demand treatment strategies for severe haemophilia: a comparison of costs and long-term outcome. Haemophilia 2002;8:745-752.

  15. Berntorp E, Astermark J, Björkman S, Blanchette VS, Fisher K, Giangrande PL, et al. Consensus perspectives on prophylactic therapy for haemophilia: summary statement. Haemophilia 2003;9(suppl 1):1-4.

  16. Miners A. Revisiting the cost-effectiveness of primary prophylaxis with clotting factor for the treatment of severe haemophilia A. Haemophilia 2009;15:881-887.

  17. Roosendaal G, Lafeber F. Prophylactic treatment for prevention of joint disease in hemophilia—cost versus benefit. N Engl J Med 2007;357:603-605.

  18. Lippert B, Berger K, Berntorp E, Giangrande P, van den Berg M, Schramm W. et al. Cost effectiveness of haemophilia treatment: a cross-national assessment. Blood Coagul Fibrinolysis 2005;16:477-485.

  19. Global Market Access Solutions. ADVATE European Value Dossier. Baxter Healthcare.

  20. Risenbourgh 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;14:743-752.

  21. Ljung R. Prophylactic therapy in haemophilia. Blood Rev 2009;23:267-274.

  22. Heemstra H, Zwaan T, Kern M, Feldman B, Blanchette V, Hemels MEH, et al. Long term cost of illness study for severe hemophilic children in Toronto. Value in Health 2003;6:339-340 [Abstract PPT5].

  23. Notas Fiscales. Indicadores Financieros y Fiscales. Tipo de cambio peso-dólar. 2 de agosto 2012. Disponible en: http://www.notasfiscales.com.mx/indicadores.html

  24. Bureau of Labor Statistics. Consumer Price Index 1913-2012. Disponible en: ftp://ftp.bls.gov/pub/special.requests/cpi/cpiai.txt

  25. Martínez-Murillo C, Quintana S, Ambriz R, Benitez H, Berges A, Collazo J, et al. An economic model of haemophilia in Mexico. Haemophilia 2004;10:9-17.

  26. Wyeth Research. Protocol No. 3082B2-310-WW. A randomized two-way blinded crossover-design study to establish the bioequivalence of B-domain deleted recombinant factor VIII (BDDrFVIII, moroctocog alfa [AF-CC]) with a full-length recombinant factor VIII preparation (FLrFVIII, Advate), followed by an open-label trial of the safety and efficacy of moroctocog alfa (AF-CC) in previously treated patients with hemophilia A. US Food & Drug Administration 2007. Disponible en: http://www.fda.gov/BiologicsBloodVaccines/BloodBloodProducts/ApprovedProducts/LicensedProductsBLAs/FractionatedPlasmaProducts/ucm056034.htm

  27. Fischer K, Pouw ME, Lewandowski D, Janssen MP, van den Berg HM, van Hout BA. A modeling approach to evaluate long-term outcome of prophylactic and on demand treatment strategies for severe hemophilia A. Haematologica 2011;96:738-743. doi:10.3324/haematol.2010.029868

  28. Molho P, Rolland N, Lebrun T, Dirat G, Courpied JP, Croughs T, et al. Epidemiological survey of the orthopaedic status of severe haemophilia A and B patients in France. The French Study Group. Haemophilia 2000;6:23-32.

  29. Fischer K, van der Bom JG, Mauser-Bunschoten EP, Roosendaal G, Prejs R, Grobbee DE, et al. Changes in treatment strategies for severe haemophilia over the last 3 decades: effects on clotting factor consumption and arthropathy. Haemophilia 2001;7:446-452.

  30. Instituto Mexicano del Seguro Social (IMSS). Portal de Transparencia. IMSS va a comprar, IMSS compró. Periodo enero-julio 2012. Disponible en: http://transparencia.imss.gob.mx/trnsp/ncompro.aspx?c=1

  31. Diario Oficial de la Federación (DOF). Instituto Mexicano del Seguro Social (IMSS). Costos Unitarios de Atención Médica. Miércoles 9 de mayo, 2012.

  32. Consejo Nacional de Población (CONAPO). Proyecciones de la población de México 2005-2050. Disponible en: http://www.conapo.gob.mx/en/CONAPO/Proyecciones_de_la_poblacion_de_Mexico_2005-2050

  33. Academia Mexicana de Pediatría. Tabla de talla y pesos del niño mexicano. Disponible en: http://www.drscope.com/tablas/tabla_01.htm

  34. Cámara Nacional de la Industria del Vestido (CANAIVE). ¿Cuánto mide México? El tamaño sí importa. 2012. Disponible en: http://www.canaive.org.mx/detalleNoticias.php? id_nota=832

  35. Consejo de Salubridad General (CSG). Guía para la conducción de estudios de evaluación económica para la actualización del Cuadro Básico de Insumos del Sector Salud en México; 2008. Disponible en: http://www.csg.salud.gob.mx/descargas/pdfs/cuadro_basico/GUxA_EVAL_ECON25082008_2_ech.pdf

  36. Banco de México (BANXICO). Datos económicos y financieros de México. Disponible en: http://www.banxico.org.mx/SieInternet/consultarDirectorioInternetAction.do?accion=consultarCuadroAnalitico&idCuadro=CA126§or=12&locale=es

  37. Instituto Nacional de Estadística y Geografía (INEGI). Censo de Población y Vivienda 2010. Disponible en: http://www.inegi.org.mx/est/contenidos/proyectos/ccpv/cpv2010/principales2010.aspx

  38. Briggs A, Sculpher M, Buxton M. Uncertainty in the economic evaluation of health care technologies: the role of sensitivity analysis. Health Econ 1994;3:95-104.

  39. Comisión Europea. Asuntos Económicos y Financieros. Disponible en: http://ec.europa.eu/economy_finance/focuson/inflation/inflation_and_eu_es.htm

  40. Banco de México (BANXICO). Mercado cambiario (Tipos de cambio). Disponible en: http://www.banxico.org.mx/portal-mercado-cambiario/index.html

  41. Miners AH, Sabin CA, Tolley KH, Lee CA. 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;244:515-522.

  42. 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;25:584-587.

  43. Smith PS, Teutsch SM, Shaffer PA, Rolka H, Evatt B. Episodic versus prophylactic infusions for hemophilia A: a cost-effectiveness analysis. J Pediatr 1996;129:424-431.

  44. Lippert B, Berger K, Berntorp E, Giangrande P, van den Berg M, Schramm W, et al. Cost effectiveness of haemophilia treatment: a cross-national assessment. Blood Coagul Fibrinolysis 2005;16:477-485.

  45. Colombo GL, Di Matteo S, Mancuso ME, Santagostino E. Cost–utility analysis of prophylaxis versus treatment on demand in severe hemophilia A. Clinicoecon Outcomes Res 2011;3:55-61.

  46. 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;20:759-774.

  47. Manco-Johnson MJ, Abshire TC, Shapiro AD, Riske B, Hacker MR, Kilcoyne R, et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med 2007;357:535-544.

  48. Pipe SW. Recombinant clotting factors. Thromb Haemost 2008;99:840-850.

  49. World Federation of Hemophilia (WFH). The World Federation of Hemophilia’s Fifth Global Forum on the Safety and Supply of Treatments for Bleeding Disorders. Proceedings. Canada: World Federation of Hemophilia; 2007. Disponible en: http://www1.wfh.org/publications/files/pdf-1275.pdf

  50. Fischer K, van Hout BA, van der Bom JG, Grobbee DE, van den Berg HM. Association between joint bleeds and Pettersson scores in severe haemophilia. Acta Radiol 2002;43: 528-532.




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