Abstract Background For rare cancers, it has been difficult to develop drugs due to the small patient population. Methods Rare Cancers Japan (RCJ) members studied cancer patient support groups in the EU and USA to learn from their experience in cancer drug development. Also, RCJ members participated in clinical trial workshops organized by the European Organization of Research and Treatment for Cancer (EORTC) for patients and caregivers interested in clinical trials to learn what is expected of patient support group leaders. Results In Europe and the United States, the patient support groups actively participate and support cancer research and clinical trials from their early stages, and there is a mechanism to review research results. In addition, multi-stakeholder conferences are held regularly to discuss various issues related to the latest discovery in research as well as clinical trials. Patient groups are engaged through a program such as Patient Public Involvement (PPI) in UK, from early stages of clinical trials. Conclusions In Japan, in the area of new drug development for rare cancers, further cooperation is needed among key stakeholders, such as patient groups, academic societies, pharmaceutical companies, regulatory agencies across national borders in Asia. On this First Rare Cancers in Asia Symposium, Rare Cancer Japan, a network of Japanese rare cancer patient group, would like to discuss the unmet needs of Japanese rare cancer patients, and together discuss what new initiatives are needed in Japan and Asia to quicken the delivery of new drugs to rare cancer patients.