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 initial goal was to develop a clinical trial simulation platform for Duchenne muscular dystrophy based on progression models of five endpoints. This will allow us to optimize the development of future clinical trial protocols across the spectrum of disease. It will help clinical trial sponsors to make informed decisions on groups of patients most appropriate to take part in specific clinical trials, which endpoints to select, and how to analyze data from those trials. C-Path is seeking regulatory endorsement for these models and the platform from both the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA), and they will be made publicly available on completion. D-RSC has now initiated new projects to develop patient reported outcome measures for Duchenne and is building new models to further optimize the use of outcome measures and to support the use of biomarkers.

To support this work, we have aggregated clinical data provided by partner organizations into a common database. The D-RSC database currently includes twenty integrated databases. Data were integrated using CDISC standards, and D-RSC has developed a CDISC Therapeutic Area User Guide to inform the community on how best to structure Duchenne data. The D-RSC data platform will allow C-Path, members and 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.