Hostname: page-component-77c89778f8-9q27g Total loading time: 0 Render date: 2024-07-18T10:51:40.491Z Has data issue: false hasContentIssue false

COST-UTILITY ANALYSIS OF PRIMARY PROPHYLAXIS, COMPARED WITH ON-DEMAND TREATMENT, FOR PATIENTS WITH SEVERE HEMOPHILIA TYPE A IN COLOMBIA

Published online by Cambridge University Press:  06 December 2016

Héctor Eduardo Castro Jaramillo
Affiliation:
Instituto de Evaluación Tecnológica en Salud – IETShcastro@hsph.harvard.edu; dochecj@hotmail.com
Mabel Moreno Viscaya
Affiliation:
Instituto de Evaluación Tecnológica en Salud – IETS
Aurelio E. Mejia
Affiliation:
Instituto de Evaluación Tecnológica en Salud – IETS

Abstract

Objectives: This article presents a cost-utility analysis from the Colombian health system perspective comparing primary prophylaxis to on-demand treatment using exogenous clotting factor VIII (FVIII) for patients with severe hemophilia type A.

Methods: We developed a Markov model to estimate expected costs and outcomes (measured as quality-adjusted life-years, QALYs) for each strategy. Transition probabilities were estimated using published studies; utility weights were obtained from a sample of Colombian patients with hemophilia and costs were gathered using local data. Both deterministic and probabilistic sensitivity analysis were performed to assess the robustness of results.

Results: The additional cost per QALY gained of primary prophylaxis compared with on-demand treatment was 105,081,022 Colombian pesos (COP) (55,204 USD), and thus not considered cost-effective according to a threshold of up to three times the current Colombian gross domestic product (GDP) per-capita. When primary prophylaxis was provided throughout life using recombinant FVIII (rFVIII), which is much costlier than FVIII, the additional cost per QALY gained reached 174,159,553 COP (91,494 USD).

Conclusions: using a decision rule of up to three times the Colombian GDP per capita, primary prophylaxis (with either FVIII or rFVIII) would not be considered as cost-effective in this country. However, a final decision on providing or preventing patients from primary prophylaxis as a gold standard of care for severe hemophilia type A should also consider broader criteria than the incremental cost-effectiveness ratio results itself. Only a price reduction of exogenous FVIII of 50 percent or more would make primary prophylaxis cost-effective in this context.

Type
Assessments
Copyright
Copyright © Cambridge University Press 2016 

Access options

Get access to the full version of this content by using one of the access options below. (Log in options will check for institutional or personal access. Content may require purchase if you do not have access.)

References

REFERENCES

1. Haemophilia, Stachnik J.: Etiology, complications, and current options in management. Formulary. 2010;45:218227.Google Scholar
2. White, GC, Rosendaal, F, Aledort, LM, et al. Factor VIII and Factor IX Subcommittee. Definitions in haemophilia. 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.Google Scholar
3. Robledo, S. Colombian League for Haemophilia/Liga Colombiana de hemofílicos y otras deficiencias sanguíneas. Secondary data provided to the researcher by its Chair. 2011 Oct 15.Google Scholar
4. Castro, HE, Briceño, MF, Casas, CP, Rueda, JD. The history and evolution of the clinical effectiveness of haemophilia type a treatment: A systematic review. Indian J Hematol Blood Transfus. 2014;30:111.Google Scholar
5. Giangrande, PL. Blood products for haemophilia: Past, present and future. BioDrugs. 2004;18:225234.Google Scholar
6. Nilsson, IM, Berntorp, E, Löfqvist, T, Pettersson, H. Twenty-five years' experience of prophylactic treatment in severe haemophilia A and B. J Intern Med. 1992;232:2532.Google Scholar
7. Aznar, JA, Magallón, M, Querol, F, Gorina, E, Tusell, JM. The orthopaedic status of severe haemophiliacs in Spain. Haemophilia. 2000;6:170176.Google Scholar
8. Coppola, A, Di Capua, M, De Simone, C. Primary prophylaxis in children with haemophilia. Blood Transfus. 2008;6 (Suppl 2):s4s11.Google Scholar
9. Berntorp, E. Joint outcomes in patients with haemophilia: The importance of adherence to preventive regimens. Haemophilia. 2009;15:12191227.Google Scholar
10. Miners, AH. Economic evaluations of prophylaxis with clotting factor for people with severe haemophilia: Why do the results vary so much? Haemophilia. 2013;19:174180.Google Scholar
11. Geraghty, S, Dunkley, T, Harrington, C, et al. Practice patterns in haemophilia A therapy – Global progress towards optimal care. Haemophilia. 2006;12:7581.Google Scholar
12. De Moerloose, P, Urbancik, W, Van Den Berg, HM, Richards, M. 2008, A survey of adherence to haemophilia therapy in six European countries: Results and recommendations. Haemophilia. 2008;1:931938.Google Scholar
13. Daliri, AA, Haghparast, H, Mamikhani, J. Cost-effectiveness of prophylaxis against on-demand treatment in boys with severe haemophilia A in Iran. Int J Technol Assess Health Care. 2990;25:584587.Google Scholar
14. Miners, A. Revisiting the cost-effectiveness of primary prophylaxis with clotting factor for the treatment of severe haemophilia A. Haemophilia. 2009;15:881887.Google Scholar
15. Lalezari, S, Coppola, A, Lin, J, et al. Patient characteristics that influence efficacy of prophylaxis with rFVIII-FS three times per week: A subgroup analysis of the LIPLONG study. Haemophilia. 2014;20:354361.Google Scholar
16. Kempton, C, Valluri, S, Reding, M, et al. Regional differences in baseline patient-reported outcomes in a randomized, controlled, prospective trial of secondary prophylaxis VS on-demand treatment in patients with severe haemophilia A. Haemophilia. 2012;18:25.Google Scholar
17. Olivieri, M, Kurnik, K, Pfluger, T, Bidlingmaier, C. Identification and long-term observation of early joint damage by magnetic resonance imaging in clinically asymptomatic joints in patients with haemophilia A or B despite prophylaxis. Haemophilia. 2012;18:369374.Google Scholar
18. Hilberg, T, Czepa, D. Cross sectional study to investigate the influence of treatment regimes on the development of haemophilic arthropathy. Hamostaseologie. 2009;29 (Suppl 1):S77–S79.Google Scholar
19. Noticiero del llano/Southwest regional news Colombia. Familias que tienen enfermos de hemofilia, venden los medicamentos a las EPS, por ser de alto costo. Noticiero del llano [Internet]. 2013 May [cited 2014 Apr 29]; [about 1 p.]. http://www.llanera.com/?id=16281 (accessed April 29, 2014).Google Scholar
20. Darby, SC, Kan, SW, Spooner, RJ, et al. Mortality rates, life expectancy, and causes of death in people with haemophilia A or B in the United Kingdom who were not infected with HIV. Blood. 2007;110:815825.Google Scholar
21. Manco-Johnson, MJ, Abshire, TC, Shapiro, AD, et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe haemophilia. N Engl J Med. 2007;357:535544.CrossRefGoogle Scholar
22. Petrini, P. Identifying and overcoming barriers to prophylaxis in the management of haemophilia. Haemophilia. 2007;13 (Suppl 2):S16–S22.Google Scholar
23. Mingot, ME, Heiniger, AI, García, M, Fernández, JA. Impact of FVIII product change in severe haemophilia A patients previously treated with plasma-derived FVIII. J Thromb Haemost. 2009;7 (Suppl 2):1124.Google Scholar
24. Gouw, SC, van der Bom, JG, Ljung, R, et al. Factor VIII products and inhibitor development in severe haemophilia A. N Engl J Med. 2013;368:231239.Google Scholar
25. Farrugia, A, Cassar, J, Kimber, MC, et al. Treatment for life for severe haemophilia A- A cost-utility model for prophylaxis vs. on-demand treatment. Haemophilia. 2013;19:e228–e238.Google Scholar
26. Lusher, JM. Haemophilia treatment. Factor VIII inhibitors with recombinant products: Prospective clinical trials. Haematologica. 2000;85 (Suppl 10):S2S6.Google Scholar
27. Scharrer, I, Bray, GL, Neutzling, O. Incidence of inhibitors in haemophilia A patients–a review of recent studies of recombinant and plasma-derived factor VIII concentrates. Haemophilia. 1999;5:145154.Google Scholar
28. Kurnik, K, Bidlingmaier, C, Engl, W, et al. New early prophylaxis regimen that avoids immunological danger signals can reduce FVIII inhibitor development. Haemophilia. 2010;16:256262.Google Scholar
29. Smith, PS, Teutsch, SM, Shaffer, PA, Rolka, H, Evatt, B. Episodic versus prophylactic infusions for haemophilia A: A cost-effectiveness analysis. J Pediatr. 1996;129:424431.Google Scholar
30. Mannucci, PM. International clinical experience with plasma-derived factor VIII and IX concentrates. Haematologica. 2006;91 (Suppl 3):S26S31.Google Scholar
31. Wong, T, Recht, M. Current options and new developments in the treatment of haemophilia. Drugs. 2011;71:305320.Google Scholar
32. Vdovin, V, Andreeva, TA, Chernova, TA, et al. Prophylaxis with once, twice or three-times weekly dosing of rFVIII-FS prevents joint bleeds in a previously treated pediatric population with moderate/severe haemophilia A. J Coagul Disord 2011;3:1.Google Scholar
33. Fischer, K, Van Den Berg, M. Prophylaxis for severe haemophilia: Clinical and economical issues. Haemophilia. 2003;9:376381.CrossRefGoogle ScholarPubMed
34. Gringeri, A, Lundin, B, von Mackensen, S, et al. A randomized clinical trial of prophylaxis in children with haemophilia A (the ESPRIT Study). J Thromb Haemost. 2011;9:700710.Google Scholar
35. Consejo Directivo del Instituto de Seguros Sociales. Acuerdo 256 de 2001- Manual de tarifas de la Entidad Promotora de Salud del Seguro Social EPS-ISS. 2001. [cited 2012 Jan]. https://lexsaludcolombia.files.wordpress.com/2010/10/tarifas-iss-2001.pdf (accessed January 10, 2012).Google Scholar
36. Dolan, P, Gudex, C, Kind, P, Williams, A. A social tariff for EuroQoL: Results from a UK general population survey [Discussion No 138]. Centre for Health Economics: University of York; 1995 [cited 2014 Feb 16]. http://www.york.ac.uk/media/che/documents/papers/discussionpapers/CHE%20Discussion%20Paper%20138.pdf (accessed February 16, 2014).Google Scholar
37. Feldman, B, Funk, S, Bergstrom, BM, et al. Validation of the new pediatric joint scoring system from the international haemophilia prophylaxis study group: Validity of the heamophilia joint health score. Arthritis Care. 2011;63:223230.Google Scholar
38. Risebrough, N, 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;14:743752.Google Scholar
39. Gravelle, H, Smith, D. Discounting for health effects in cost benefit and cost effectiveness analysis. CHE Technical Paper Series 2000 Oct;(20). https://www.york.ac.uk/che/pdf/tp20.pdf (accessed June 30, 2015).Google Scholar
40. Van der Poel, CL, Reesink, HW, Mauser-Bunschoten, EP, et al. Prevalence of anti-HCV antibodies confirmed by recombinant immunoblot in different population subsets in the Netherlands. Vox Sang. 1991;61:3036.Google Scholar
41. Mannucci, PM, Tuddenham, EG. The haemophilias - From royal genes to gene therapy. N Engl J Med. 2001;344:17731779.Google Scholar
Supplementary material: File

Castro Jaramillo supplementary material

Table S1 and Figures S1-S3

Download Castro Jaramillo supplementary material(File)
File 307.6 KB