希少疾患の治験を成功させるためには

抄録

Therapeutic development in rare diseases shares many challenges, such as incomplete understanding of natural history to design clinical trials, and difficulties of recruiting patients to participation. To secure clinical studies and clinical research at national university hospitals in Japan, National University Hospital Clinical Research Promotion Initiative (NUH–CRPI) founded in October 2012. The topic group (TG2) promotes information sharing, standardization and coordination, and discusses the network of national university hospitals. They set up the web system of feasibility study assessing capacity in university hospitals for clinical trials in rare diseases. The support and training program for translational research that was initiated by the Ministry of Education, Culture, Sports, Science and Technology was launched in 2007. The second term of Translational Research Network Program started in 2012. In this program, they are setting up the patient registry system for screening for eligibility and enrollment in clinical trials.