Skip to main content
Top
Published in: Health Economics Review 1/2013

Open Access 01-12-2013 | Research

Cost-effectiveness analysis of azacitidine in the treatment of high-risk myelodysplastic syndromes in Spain

Authors: Carlos Crespo, Estela Moreno, Jordi Sierra, Suzan Serip, Marta Rubio

Published in: Health Economics Review | Issue 1/2013

Login to get access

Abstract

Background

The objective of the study was to analyse whether azacitidine is a cost-effective option for the treatment of myelodysplastic syndrome in the Spanish setting compared with conventional care regimens, including best supportive care, low dose chemotherapy and standard dose chemotherapy.

Methods

A life-time Markov model was constructed to evaluate the cost-effectiveness of azacitidine compared with conventional care regimens. The health states modelled were: myelodysplastic syndrome, acute myeloid leukemia and death. Variables measured included survival rates, progression probabilities and quality of life indicators. Resource use and cost data reflect the Spanish context. The analysis was performed from the Spanish National Health System perspective, discounting both costs (in 2012 euros) and future effects at 3%. The time horizon considered was end-of-life. Results were expressed in cost per quality-adjusted life-year gained and cost per life-year gained and compared with cost-effectiveness thresholds.

Results

According to the current use of each conventional care regimens options in Spain, azacitidine resulted in €34,673 per quality-adjusted life-year gained (€28,891 per life-year gained) with an increase of 1.89 in quality-adjusted life-years (2.26 in life-years). Azacitidine was superior to best supportive care and low dose chemotherapy in terms of quality-adjusted life-years gained, 1.82 and 2.03, respectively (life-years 2.16 vs. best supportive care, 2.39 vs. low dose chemotherapy). Treatment with azacitidine resulted in longer survival time and thus longer treatment time and lifetime costs. The incremental cost-effectiveness ratio was €39,610 per quality-adjusted life-year gained vs. best supportive care and €30,531 per quality-adjusted life-year gained vs. low dose chemotherapy (€33,111 per life-year gained vs. best supportive care and €25,953 per life-year gained vs. low dose chemotherapy).

Conclusions

The analysis showed that the use of azacitidine in the treatment of high-risk myelodysplastic syndrome is a cost-effective option compared with conventional care regimen options used in the Spanish setting and had an incremental cost-effectiveness ratio within the range of the thresholds accepted by health authorities.
Appendix
Available only for authorised users
Literature
1.
go back to reference Steensma DP, Bennett JM: The myelodysplastic syndromes: diagnosis and treatment. Mayo Clin Proc 2006, 81: 104–130. 10.4065/81.1.104PubMedCrossRef Steensma DP, Bennett JM: The myelodysplastic syndromes: diagnosis and treatment. Mayo Clin Proc 2006, 81: 104–130. 10.4065/81.1.104PubMedCrossRef
2.
go back to reference Greenberg P, Cox C, LeBeau MM, Fenaux P, Morel P, Sanz G, Sanz M, Vallespi T, Hamblin T, Oscier D, Ohyashiki K, Toyama K, Aul C, Mufti G, Bennett J: International scoring system for evaluating prognosis in myelodysplastic syndromes. Blood 1997, 89: 2079–2088.PubMed Greenberg P, Cox C, LeBeau MM, Fenaux P, Morel P, Sanz G, Sanz M, Vallespi T, Hamblin T, Oscier D, Ohyashiki K, Toyama K, Aul C, Mufti G, Bennett J: International scoring system for evaluating prognosis in myelodysplastic syndromes. Blood 1997, 89: 2079–2088.PubMed
4.
go back to reference Miller KB, Kim K, Morrison FS, Winter JN, Bennett JM, Neiman RS, Head DR, Cassileth PA, O'Connell MJ: The evaluation of low-dose cytarabine in the treatment of myelodysplastic syndromes: a phase-III intergroup study. Ann Hematol 1992, 65: 162–168. 10.1007/BF01703109PubMedCrossRef Miller KB, Kim K, Morrison FS, Winter JN, Bennett JM, Neiman RS, Head DR, Cassileth PA, O'Connell MJ: The evaluation of low-dose cytarabine in the treatment of myelodysplastic syndromes: a phase-III intergroup study. Ann Hematol 1992, 65: 162–168. 10.1007/BF01703109PubMedCrossRef
5.
go back to reference Alessandrino EP, Amadori S, Barosi G, Cazzola M, Grossi A, Liberato LN, Locatelli F, Marchetti M, Morra E, Rebulla P, Visani G, Tura S: Italian Society of Hematology, Evidence- and consensus-based practice guidelines for the therapy of primary myelodysplastic syndromes. A statement from the Italian Society of Hematology. Haematologica 2002, 87: 1286–1306.PubMed Alessandrino EP, Amadori S, Barosi G, Cazzola M, Grossi A, Liberato LN, Locatelli F, Marchetti M, Morra E, Rebulla P, Visani G, Tura S: Italian Society of Hematology, Evidence- and consensus-based practice guidelines for the therapy of primary myelodysplastic syndromes. A statement from the Italian Society of Hematology. Haematologica 2002, 87: 1286–1306.PubMed
6.
go back to reference Ossenkoppele GJ, Graveland WJ, Sonneveld P, Daenen SM, Biesma DH, Verdonck LF, Schaafsma MR, Westveer PH, Peters GJ, Noordhuis P, Muus P, Selleslag D, van der Holt B, Delforge M, Löwenberg B, Verhoef GE: Dutch-Belgian Hemato-Oncology Cooperative Group (HOVON), The value of fludarabine in addition to ARA-C and G-CSF in the treatment of patients with high-risk myelodysplastic syndromes and AML in elderly patients. Blood 2004, 103: 2908–2913. 10.1182/blood-2003-07-2195PubMedCrossRef Ossenkoppele GJ, Graveland WJ, Sonneveld P, Daenen SM, Biesma DH, Verdonck LF, Schaafsma MR, Westveer PH, Peters GJ, Noordhuis P, Muus P, Selleslag D, van der Holt B, Delforge M, Löwenberg B, Verhoef GE: Dutch-Belgian Hemato-Oncology Cooperative Group (HOVON), The value of fludarabine in addition to ARA-C and G-CSF in the treatment of patients with high-risk myelodysplastic syndromes and AML in elderly patients. Blood 2004, 103: 2908–2913. 10.1182/blood-2003-07-2195PubMedCrossRef
7.
go back to reference Sekeres MA, Stone RM, Zahrieh D, Neuberg D, Morrison V, De Angelo DJ, Galinsky I, Lee SJ: Decision-making and quality of life in older adults with acute myeloid leukemia or advanced myelodysplastic syndrome. Leukemia 2004, 18: 809–816. 10.1038/sj.leu.2403289PubMedCrossRef Sekeres MA, Stone RM, Zahrieh D, Neuberg D, Morrison V, De Angelo DJ, Galinsky I, Lee SJ: Decision-making and quality of life in older adults with acute myeloid leukemia or advanced myelodysplastic syndrome. Leukemia 2004, 18: 809–816. 10.1038/sj.leu.2403289PubMedCrossRef
8.
go back to reference Silverman LR, Demakos EP, Peterson BL, Kornblith AB, Holland JC, Odchimar-Reissig R, Stone RM, Nelson D, Powell BL, DeCastro CM, Ellerton J, Larson RA, Schiffer CA, Holland JF: Randomized controlled trial of azacitidine in patients with the myelodysplastic syndrome: a study of the cancer and leukemia group B. J Clin Oncol 2002, 20: 2429–2440. 10.1200/JCO.2002.04.117PubMedCrossRef Silverman LR, Demakos EP, Peterson BL, Kornblith AB, Holland JC, Odchimar-Reissig R, Stone RM, Nelson D, Powell BL, DeCastro CM, Ellerton J, Larson RA, Schiffer CA, Holland JF: Randomized controlled trial of azacitidine in patients with the myelodysplastic syndrome: a study of the cancer and leukemia group B. J Clin Oncol 2002, 20: 2429–2440. 10.1200/JCO.2002.04.117PubMedCrossRef
9.
go back to reference Fenaux P, Mufti GJ, Hellstrom-Lindberg E, Santini V, Finelli C, Giagounidis A, Schoch R, Gattermann N, Sanz G, List A, Gore SD, Seymour JF, Bennett JM, Byrd J, Backstrom J, Zimmerman L, McKenzie D, Beach C, Silverman LR: International Vidaza High-Risk MDS Survival Study Group. Efficacy of azacitidine compared with that of conventional care regimens in the treatment of higher-risk myelodysplastic syndromes: a randomised, open-label, phase III study. Lancet Oncol 2009,10(3):223–232. 10.1016/S1470-2045(09)70003-8PubMedCentralPubMedCrossRef Fenaux P, Mufti GJ, Hellstrom-Lindberg E, Santini V, Finelli C, Giagounidis A, Schoch R, Gattermann N, Sanz G, List A, Gore SD, Seymour JF, Bennett JM, Byrd J, Backstrom J, Zimmerman L, McKenzie D, Beach C, Silverman LR: International Vidaza High-Risk MDS Survival Study Group. Efficacy of azacitidine compared with that of conventional care regimens in the treatment of higher-risk myelodysplastic syndromes: a randomised, open-label, phase III study. Lancet Oncol 2009,10(3):223–232. 10.1016/S1470-2045(09)70003-8PubMedCentralPubMedCrossRef
10.
go back to reference Celgene Corp: Data on file: Clinical study report: A Randomized Phase III Controlled Trial of Subcutaneous 5-Azacitidine (NSC # 102816) vs. Observation in Myelodysplastic Syndromes (CALGB 9221). 2003. Celgene Corp: Data on file: Clinical study report: A Randomized Phase III Controlled Trial of Subcutaneous 5-Azacitidine (NSC # 102816) vs. Observation in Myelodysplastic Syndromes (CALGB 9221). 2003.
11.
go back to reference Spanish Group for Myelodysplastic Syndromes (GESMD) and Spanish Society of Haematology and Haemotherapy (SEHH): Spanish guidelines for the diagnosis and treatment of myelodysplastic syndromes and chronic myelomonocytic leukaemia. Grupo Acción Médica. Madrid 2012.,97(5): ISSN 1138–0381 Spanish Group for Myelodysplastic Syndromes (GESMD) and Spanish Society of Haematology and Haemotherapy (SEHH): Spanish guidelines for the diagnosis and treatment of myelodysplastic syndromes and chronic myelomonocytic leukaemia. Grupo Acción Médica. Madrid 2012.,97(5): ISSN 1138–0381
12.
go back to reference McKenzie L, van der Pol M: Mapping the EORTC QLQ C-30 onto the EQ-5D Instrument: The Potential to Estimate QALYs without Generic Preference Data. Value Health 2009, 12: 167–171. 10.1111/j.1524-4733.2008.00405.xPubMedCrossRef McKenzie L, van der Pol M: Mapping the EORTC QLQ C-30 onto the EQ-5D Instrument: The Potential to Estimate QALYs without Generic Preference Data. Value Health 2009, 12: 167–171. 10.1111/j.1524-4733.2008.00405.xPubMedCrossRef
13.
go back to reference Kornblith AB, Herndon JE 2nd, Silverman LR, et al.: Impact of azacytidine on the quality of life of patients with myelodysplastic syndrome treated in a randomized phase III trial: a Cancer and Leukemia Group B study. J Clin Oncol 2002, 20: 2441–2452. 10.1200/JCO.2002.04.044PubMedCrossRef Kornblith AB, Herndon JE 2nd, Silverman LR, et al.: Impact of azacytidine on the quality of life of patients with myelodysplastic syndrome treated in a randomized phase III trial: a Cancer and Leukemia Group B study. J Clin Oncol 2002, 20: 2441–2452. 10.1200/JCO.2002.04.044PubMedCrossRef
14.
go back to reference Gray AM, Rivero-Arias O, Clarke PM: Estimating the association between SF-12 responses and EQ-5D utility values by response mapping. Med Decis Making 2006, 26: 18–29. 10.1177/0272989X05284108PubMedCrossRef Gray AM, Rivero-Arias O, Clarke PM: Estimating the association between SF-12 responses and EQ-5D utility values by response mapping. Med Decis Making 2006, 26: 18–29. 10.1177/0272989X05284108PubMedCrossRef
15.
go back to reference Dalziel K, Round A, Garside R, Stein K: Cost effectiveness of imatinib compared with interferon-alpha or hydroxycarbamide for first-line treatment of chronic myeloid leukaemia. Pharmacoeconomics 2005, 23: 515–526. 10.2165/00019053-200523050-00010PubMedCrossRef Dalziel K, Round A, Garside R, Stein K: Cost effectiveness of imatinib compared with interferon-alpha or hydroxycarbamide for first-line treatment of chronic myeloid leukaemia. Pharmacoeconomics 2005, 23: 515–526. 10.2165/00019053-200523050-00010PubMedCrossRef
18.
go back to reference Chalkidou K: Evidence and values: paying for end-of-life drugs in the British NHS. Health Econ Policy Law 2012,7(4):393–409. 10.1017/S1744133112000205PubMedCrossRef Chalkidou K: Evidence and values: paying for end-of-life drugs in the British NHS. Health Econ Policy Law 2012,7(4):393–409. 10.1017/S1744133112000205PubMedCrossRef
20.
go back to reference Naveršnik K, Rojnik K: Handling input correlations in pharmacoeconomic models. Value Health 2012,15(3):540–549. 10.1016/j.jval.2011.12.008PubMedCrossRef Naveršnik K, Rojnik K: Handling input correlations in pharmacoeconomic models. Value Health 2012,15(3):540–549. 10.1016/j.jval.2011.12.008PubMedCrossRef
21.
go back to reference Briggs AH, Weinstein MC, Fenwick EA, Karnon J, Sculpher MJ, Paltiel AD: ISPOR-SMDM Modeling Good Research Practices Task Force. Model parameter estimation and uncertainty: a report of the ISPOR-SMDM Modeling Good Research Practices Task Force--6. Value Health 2012,15(6):835–842. 10.1016/j.jval.2012.04.014PubMedCrossRef Briggs AH, Weinstein MC, Fenwick EA, Karnon J, Sculpher MJ, Paltiel AD: ISPOR-SMDM Modeling Good Research Practices Task Force. Model parameter estimation and uncertainty: a report of the ISPOR-SMDM Modeling Good Research Practices Task Force--6. Value Health 2012,15(6):835–842. 10.1016/j.jval.2012.04.014PubMedCrossRef
22.
go back to reference Iglesias Gallego M, Sastre Moral JL, Gayoso Diz P, García Costa A, Ros Forteza S, Mayán Santos JM: Incidence and characteristics of myelodysplastic syndromes in Ourense (Spain) between 1994–1998. Haematologica 2003, 88: 1197–1199.PubMed Iglesias Gallego M, Sastre Moral JL, Gayoso Diz P, García Costa A, Ros Forteza S, Mayán Santos JM: Incidence and characteristics of myelodysplastic syndromes in Ourense (Spain) between 1994–1998. Haematologica 2003, 88: 1197–1199.PubMed
25.
26.
go back to reference Gildea TR, Shermock KM, Singer ME, Stoller JK: Cost-effectiveness analysis of augmentation therapy for severe alpha1-antitrypsin deficiency. Am J Respir Crit Care Med 2003, 167: 1387–1392. 10.1164/rccm.200209-1035OCPubMedCrossRef Gildea TR, Shermock KM, Singer ME, Stoller JK: Cost-effectiveness analysis of augmentation therapy for severe alpha1-antitrypsin deficiency. Am J Respir Crit Care Med 2003, 167: 1387–1392. 10.1164/rccm.200209-1035OCPubMedCrossRef
28.
go back to reference Hoyle M, Green C, Thompson-Coon J, Liu Z, Welch K, Moxham T: Cost-effectiveness of temsirolimus for first line treatment of advanced renal cell carcinoma. Value Health 2010, 13: 61–68. 10.1111/j.1524-4733.2009.00617.xPubMedCrossRef Hoyle M, Green C, Thompson-Coon J, Liu Z, Welch K, Moxham T: Cost-effectiveness of temsirolimus for first line treatment of advanced renal cell carcinoma. Value Health 2010, 13: 61–68. 10.1111/j.1524-4733.2009.00617.xPubMedCrossRef
29.
go back to reference Paz-Ares L, García Del Muro X, Grande E, González P, Brosa M, Díaz S: Cost-effectiveness analysis of sunitinib in patients with metastatic and/or unresectable gastrointestinal stroma tumours (GIST) after progression or intolerance with imatinib. Clin Transl Oncol 2008, 10: 831–839. 10.1007/s12094-008-0297-3PubMedCrossRef Paz-Ares L, García Del Muro X, Grande E, González P, Brosa M, Díaz S: Cost-effectiveness analysis of sunitinib in patients with metastatic and/or unresectable gastrointestinal stroma tumours (GIST) after progression or intolerance with imatinib. Clin Transl Oncol 2008, 10: 831–839. 10.1007/s12094-008-0297-3PubMedCrossRef
30.
go back to reference Picavet E, Dooms M, Cassiman D, Simoens S: Drugs for rare diseases: Influence of orphan designation status on price. Appl Health Econ Health Policy 2011,9(4):275–279. 10.2165/11590170-000000000-00000PubMedCrossRef Picavet E, Dooms M, Cassiman D, Simoens S: Drugs for rare diseases: Influence of orphan designation status on price. Appl Health Econ Health Policy 2011,9(4):275–279. 10.2165/11590170-000000000-00000PubMedCrossRef
34.
go back to reference Gidwani R, Khan ZM, Fenaux P, Beach CL, Pashos CL: A cost-effectiveness analysis of using azacitidine versus decitabine in treating patients with myelodysplastic syndromes. J Med Econ 2012, 15: 145–154. 10.3111/13696998.2011.631067PubMedCrossRef Gidwani R, Khan ZM, Fenaux P, Beach CL, Pashos CL: A cost-effectiveness analysis of using azacitidine versus decitabine in treating patients with myelodysplastic syndromes. J Med Econ 2012, 15: 145–154. 10.3111/13696998.2011.631067PubMedCrossRef
35.
go back to reference Aul C, Giagounidis A, Germing U: Epidemiological features of myelodysplastic syndromes: results from regional cancer surveys and hospital-based statistics. Int J Hematol 2001, 73: 405–410. 10.1007/BF02994001PubMedCrossRef Aul C, Giagounidis A, Germing U: Epidemiological features of myelodysplastic syndromes: results from regional cancer surveys and hospital-based statistics. Int J Hematol 2001, 73: 405–410. 10.1007/BF02994001PubMedCrossRef
Metadata
Title
Cost-effectiveness analysis of azacitidine in the treatment of high-risk myelodysplastic syndromes in Spain
Authors
Carlos Crespo
Estela Moreno
Jordi Sierra
Suzan Serip
Marta Rubio
Publication date
01-12-2013
Publisher
Springer Berlin Heidelberg
Published in
Health Economics Review / Issue 1/2013
Electronic ISSN: 2191-1991
DOI
https://doi.org/10.1186/2191-1991-3-28

Other articles of this Issue 1/2013

Health Economics Review 1/2013 Go to the issue