Despite high unmet medical needs, investment in rare cancer drug development has stagnated, likely because the potential market for such drugs is small. In this context, we hypothesized that rare cancer drugs could achieve a higher sales margin. A dataset was created from publicly available information obtained from the IQVIA Solutions Japan K.K. Pharmaceutical Market database on the website of the Pharmaceuticals and Medical Devices Agency/Ministry of Health, Labour and Welfare of Japan. The total amount of sales and prescription volumes between 2010 and 2016 for drugs whose indications include chronic myelogenous leukemia (CML) and neuroendocrine tumor (NET) were investigated. Regarding drugs for CML, the sales and prescription volumes of imatinib have been decreasing every year, whereas those of dasatinib and nilotinib have been increasing. Regarding drugs for NET, the sales and prescription volumes of sunitinib, everolimus, and streptozocin have been increasing every year. The present study revealed two sales models for the development of rare cancer drugs. First, sales amounts can be assured if clinical positioning with other existing drugs is sufficiently clear. Second, obtaining a label for rare cancers can stimulate drug development for more common cancers. These findings suggest that rare cancer drugs can offer high market value and profit potential; thus, to meet high unmet medical needs, clinical development programs for the development of rare cancer drugs should be promoted. https://doi.org/10.21423/jrs-v07shibat
