[Progress of MM/CML treatment and medical economics]

Cost-effectiveness of the sequential application of tyrosine kinase inhibitors (TKIs) is an important issue in chronic myelogenous leukemia (CML). Similarly, although overall survival (OS) has tremendously improved, the treatment of multiple myeloma (MM), including 12 new drugs, typically costs more than $100,000 per year. This price should not be judged in isolation but rather evaluated in the context of clinical benefit, which can be assessed by cost-effectiveness analyses such as the total cost, quality-adjusted life years (QALYs), and incremental cost-effectiveness ratios. Physicians change drugs several times to prolong individual patient survival over the course of their therapy, which means there are variations in treatment choices and treatment sequence. To achieve a clinical response by each treatment is very important, and the overall response rate and OS are key pieces of information to assess this. The additional treatment goal is sustained minimal residual disease (MRD) negativity. In Japan, medical costs for MM are mostly covered by public health insurance. Modifications in treatment patterns for CML and MM drugs and medical costs for patients are important issues; how to cure the disease or prolong the therapy-free interval with lower costs is an urgent requirement. Total medical costs have remained stable since 2010, but there are important issues to consider in the use of generic drugs, Phase 2 approval of new drugs, and decreasing costs of other care, particularly for hospitalization that includes treatment costs other than that of CML and MM drugs. We must aim for treatment-free remission and a clinical cure in the future. We can stop treatment with a 2-year sustained molecular response (MR)^4.5 in CML and MRD negative (<10^-5) in MM with full informed consent and careful watch by the BCR-ABL international scale and next-generation flow.