Azacitidine and decitabine are used to treat patients with myelodysplastic syndromes (MDS) in the United States (US). This study sought to assess their relative cost-effectiveness.
The authors developed a cost-effectiveness Markov model (1-month cycles) tracking hypothetical cohorts of MDS patients treated with azacitidine or decitabine over 2 years. The model used a US payer perspective and 2009 costs. Health states modeled included MDS with Transfusion Dependence, MDS with Transfusion Independence, Progression to Acute Myelogenous Leukemia (AML), and Death. Incremental cost-effectiveness outcomes included cost per quality-adjusted life year (QALY), cost per life year (LY), cost per patient-month of transfusion independence, and cost per case of AML progression avoided. One-way sensitivity analyses were performed on key model parameters.
Compared to decitabine, azacitidine was associated with better survival (1.512 LYs vs 1.292), more QALYs gained (1.041 vs 0.870), more patient-months with transfusion independence (8.328 vs 6.224), and a greater proportion of patients avoiding progression to AML (50.9% vs 28.5%). Total per-patient costs over 2 years for azacitidine were lower than for decitabine ($150,322 vs $166, 212).
To inform and update the model over time, it will be important that randomized or observational clinical studies be conducted to directly compare azacitidine and decitabine, provide new information on how these medicines are used, and on their relative clinical effectiveness.
Results demonstrate that azacitidine provides greater clinical benefit and costs less than decitabine across all key outcomes. These results accentuate the positive role of azacitidine in providing cost-effective care for MDS.
"A recent study compared azacitidine with decitabine and found that azacitidine was a cost-effective treatment for MDS according to U.S. National Healthcare Input data , with a comparative gain of 0.171 more QALYs and savings of €15,890 over a 2-year period. However, the relevance of the study is limited, mainly because survival data was retrieved from two different phase III trials and no direct comparison was made. "
[Show abstract][Hide abstract] ABSTRACT: 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.
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.
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).
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.
[Show abstract][Hide abstract] ABSTRACT: Our goal was to determine the economic value of azacitidine in Canada compared with conventional care regimens (ccrs), including best supportive care (bsc) and low- or standard-dose chemotherapy plus bsc in the treatment of higher-risk myelodysplastic syndromes (mdss) and acute myeloid leukemia (aml) with 20%-30% blasts.
The cost-utility model is a lifetime probabilistic Markov model with a 35-day cycle length consisting of 3 health states: mds; transformation to aml with more than 30% blasts; and death. A third-party public payer perspective was adopted. Overall survival was extrapolated beyond the time horizon of the aza-001 trial comparing azacitidine with ccr. Resource use was determined through a questionnaire completed by Canadian hematologists. Utility values were obtained from two studies in which EQ-5D health questionnaire values were mapped from the European Organization for Research and Treatment of Cancer qlq-C30 survey, and SF-6D scores were mapped from the Short Form 12, elicited from 191 and 43 patients in two different trials.
In the base case, azacitidine had an incremental cost-effectiveness ratio (icer) of $86,182 (95% confidence limits: $69,920, $107,157) per quality-adjusted life year (qaly) gained relative to ccr. Comparing azacitidine with bsc, low-dose chemotherapy plus bsc, and standard-dose chemotherapy plus bsc, the icers were, respectively, $86,973, $84,829, and $2,152 per qaly gained. Results were most sensitive to the utility for azacitidine after 6 months of treatment and to overall survival.
The prolonged 9-month median overall survival with azacitidine relative to ccr fills a gap w hen treating patients with higher-risk mds and aml with 20%-30% blasts. The economic value of azacitidine is within the threshold of willingness-to-pay for third-party public payers for oncology treatments in Canada.
Current Oncology 02/2014; 21(1):e29-40. DOI:10.3747/co.21.1311 · 1.79 Impact Factor
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