Clinical trial network for the promotion of clinical research for rare diseases in Japan: muscular dystrophy clinical trial networkReport as inadecuate

Clinical trial network for the promotion of clinical research for rare diseases in Japan: muscular dystrophy clinical trial network - Download this document for free, or read online. Document in PDF available to download.

BMC Health Services Research

, 16:241

Organization, structure and delivery of healthcare


BackgroundDuchenne muscular dystrophy DMD is the most commonly inherited neuromuscular disease. Therapeutic agents for the treatment of rare disease, namely -orphan drugs-, have recently drawn the attention of researchers and pharmaceutical companies. To ensure the successful conduction of clinical trials to evaluate novel treatments for patients with rare diseases, an appropriate infrastructure is needed. One of the effective solutions for the lack of infrastructure is to establish a network of rare diseases.

MethodsTo accomplish the conduction of clinical trials in Japan, the Muscular dystrophy clinical trial network MDCTN was established by the clinical research group for muscular dystrophy, including the National Center of Neurology and Psychiatry, as well as national and university hospitals, all which have a long-standing history of research cooperation.

ResultsThirty-one medical institutions 17 national hospital organizations, 10 university hospitals, 1 national center, 2 public hospitals, and 1 private hospital belong to this network and collaborate to facilitate clinical trials. The Care and Treatment Site Registry CTSR calculates and reports the proportion of patients with neuromuscular diseases in the cooperating sites. In total, there are 5,589 patients with neuromuscular diseases in Japan and the proportion of patients with each disease is as follows: DMD, 29 %; myotonic dystrophy type 1, 23 %; limb girdle muscular dystrophy, 11 %; Becker muscular dystrophy, 10 %. We work jointly to share updated health care information and standardized evaluations of clinical outcomes as well. The collaboration with the patient registry CTSR, allows the MDCTN to recruit DMD participants with specific mutations and conditions, in a remarkably short period of time.

ConclusionCounting with a network that operates at a national level is important to address the corresponding national issues. Thus, our network will be able to contribute with international research activity, which can lead to an improvement of neuromuscular disease treatment in Japan.

KeywordsMuscular dystrophy clinical trial network MDCTN Orphan drugs Neuromuscular diseases Rare diseases Clinical trial network Muscular dystrophy Registry of Muscular Dystrophy Remudy  Download fulltext PDF



Related documents