Infrastructure for new drug development to treat muscular dystrophy: current status of patient registration (remudy)

Clinical trials for new therapeutic strategies are now being planned for Duchenne and Becker muscular dystrophies (DMD/BMD); however, many challenges exist in the planning and conduction of a clinical trial for rare diseases. The epidemiological data, total number of patients, natural history, and clinical outcome measures are unclear. Adequate numbers of patients are needed to achieve significant results in clinical trials. As solutions to these problems, patient registries are an important infrastructure worldwide, especially in the case of rare diseases such as DMD/BMD. In Europe, TREAT-NMD, a clinical research network for neuromuscular disorders, developeda global database for dystrophinopathy patients. We developed a national registry of Japanese DMD/BMD patients in collaboration with TREAT-NMD. The database includes clinical and molecular genetic data as well as all required items for the TREAT-NMD global patient registry. As of July 2011, 750 patients were registered in the database. The purpose of this registry is the effective recruitment of eligible patients for clinical trials, and it may also provide timely information to individual patients about upcoming trials. This registry data also provides more detailed knowledge about natural history, epidemiology, and clinical care. In recent years, drug development has become dramatically globalized, and global clinical trials (GCTs) are being conducted in Japan. It is appropriate, particularly with regard to orphan diseases, to include Japanese patients in GCTs to increase evidence for evaluation, because such large-scale trials would be difficult to conduct solely within Japan. GCTs enable the synchronization of clinical drug development in Japan with that in Western countries, minimizing drug approval delays.