The Duchenne Regulatory Science Consortium (D-RSC) was formed to develop tools to accelerate therapy development for Duchenne Muscular Dystrophy. Many clinical trials are currently underway for potential therapies for the disease, but further work is needed to optimize trial protocols to ensure that such trials are as effective and informative as possible. D-RSC aims to develop new tools to accelerate and improve trial protocol development and to reduce the numbers of patients needed to demonstrate the effect of new therapies.
D-RSC’s primary goal is to develop a clinical trial simulation tool for Duchenne muscular dystrophy. This will allow informed development of future clinical trial protocols across the spectrum of diseases. It will help clinical trial sponsors to make informed decisions on groups of patients most appropriate to take part in specific clinical trials endpoints to select, and how to analyze data from those trials. C-Path will seek regulatory endorsement for tools developed by the consortium from both the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA).
To do this, we have aggregated clinical data provided by partner organizations into a common database. The D-RSC database currently includes twelve integrated databases. Data were integrated using CDISC standards, and D-RSC has developed a CDISC Therapeutic Area User Guide. The D-RSC data platform will allow C-Path, members and possibly outside groups to analyze some or all of the integrated data for multiple purposes.
D-RSC was formed through collaboration between the Critical Path Institute and Parent Project Muscular Dystrophy. Founding members from industry and academia agreed to join the consortium, and representatives of FDA, EMA and NIH have joined the consortium coordinating committee as observers.