Abstract
The aim of this study was to assess the cost-effectiveness of azacitidine therapy for patients with myelodysplastic syndromes. A Markov model was developed to estimate the total additional direct cost and quality adjusted life years (QALYs) gained with azacitidine therapy versus best-supportive care in patients with high-risk MDS. The cost-effectiveness of azacitidine was evaluated with incremental cost-effectiveness ratio, which represents the additional cost per QALY gained from the more effective treatment. Azacitidine therapy was 1.83 million yen more costly per patient but yielded an additional 0.353 QALYs. The ICER (Increment of Cost-effectiveness Ratio) was 5.18 million yen per QALY. In conclusion, because the ICER was less than the threshold for acceptable cost-effectiveness in Japan, azacitidine therapy for MDS patient was assumed to be cost-effective.