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

Abstract

BackgroundThe 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.MethodsA 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.ResultsAccording 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).ConclusionsThe 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.

Monthly blood transfusions decrease after four months of azacitidine

“…Similarly, the population‐weighted mean cost of transfusing two units of red cells in Western Europe has been estimated to be €877·69 . Coupled with other studies suggesting that AZA is a cost‐effective treatment for higher risk MDS, this data support the notion that AZA has a beneficial effect on transfusion resource utilization . Our data also demonstrate that TI rates reported in clinical trials may exaggerate actual transfusion responses, as they do not report on patients who commence TI and become TD on study, while achieving other responses (e.g.…”

Cost-Effectiveness of Azacitidine Compared with Low-Doses of Chemotherapy (LDC) in Myelodysplastic Syndrome (MDS)

“…The partitioned survival analysis model used in this study is similar to the q-twist approach, a well-established analytic framework for evaluating oncology therapies (12), and also to the models used in numerous earlier economic assessments of treatments for MDS, including recent evaluations of the cost-effectiveness of azacitidine for the same disease (13,14).…”

Guía Metodológica de Evaluación Económica aplicada a Medicamentos Huérfanos.

“…Respecto a los datos de los costes, es necesario analizar varios aspectos, como son su identifi cación, la cuantifi cación de su uso, el precio asociado a cada uno de los recursos y su actualización a un valor actual neto. Se verá más claramente en el ejemplo de un análisis económico que estima la efi ciencia de un medicamento huérfano en el tratamiento del síndrome mielodisplásico 60 . Esta enfermedad agrupa un conjunto de condiciones médicas derivadas de la insufi ciencia progresiva de la médula ósea, originando una producción no adecuada de células sanguíneas.…”

“…En una evaluación económica de este síndrome mielodisplásico, una vez definida la perspectiva -la del fi nanciador del sistema de salud-, se estableció que se incorporarían los costes directos sanitarios 60 . Para ello, se identifi caron los recursos utilizados (estancias hospitalarias, consultas médicas del hematólogo, personal de enfermería, diversas pruebas de laboratorio, transfusiones de sangre y plaquetas y tratamiento de los eventos adversos, como neutropenia, leucopenia, neutropenia febril, pirexia, neumonía y sepsis).…”