The Duchenne Regulatory Science Consortium (D-RSC) was formed in August of 2015 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 first goal was to aggregate clinical data provided by partner organizations into a common database. The D-RSC database currently includes 8 integrated databases. Data was integrated using CDISC standards, and D-RSC has developed a CDISC Therapeutic Area User Guide, which is expected to be completed by the end of 2017. 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. The initial goal of the consortium is to develop a disease progression model, based on analysis of these data. This model is envisioned to have three main purposes: 1) to serve as the backbone for the future development of a clinical trial simulation platform; 2) to serve as a quantitative clinical trial enrichment platform, allowing clinical trial sponsors to make informed decisions on groups of patients most appropriate to take part in specific clinical trials and how to analyze data from those trials; 3) to inform further biomarker efforts. 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).
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 NIH and FDA have joined the consortium coordinating committee as observers.