2021 Volume 11 Issue 2 Pages 111-123
Rare cancers include a wide variety of malignancies and despite new target treatment focusing on their characteristics are expected, but the small number of patients often cause high price for those drugs. Number of countries introduced Health Technology Assessment (HTA), which includes cost-effectiveness, and utilized it to assess the reasonable price and reimbursement. In Japan, a cost-effectiveness evaluation regulation was also introduced to adjust drug prices. Our study investigated the cases of HTA in other countries to get the insight for future applicability of HTA to evaluate the value of rare cancer drugs. Extracted 42 rare cancer drugs approved during 2009-2019 in Japan, 2.9-12.5% were not recommended and 0-22.2% of 44 other orphan drugs in the UK, Canada and Australia. The major reason for negative recommendation was “uncertainty regarding clinical effectiveness”. Many of the “not-recommended” drugs were priced by the cost-calculation method, which means that there are no comparable drug available. Another tendency was observed that no active control was set and only small number of subjects were included in the clinical trials. In order to reduce the uncertainty in clinical effectiveness, more data collection should be attempted, such as utilizing global studies, industry-academic consortia and web recruitment. On the other hand, there is also limitation of difficulty to set control due to lack of standard treatments and/or ethical reason from insufficient efficacy of available treatments. Therefore, we should attempt to utilize real-world data to obtain control group data in the development stage and to accumulate more clinical data after marketed.
