Abstract
Introduction: The Duchenne Regulatory Science Consortium (D-RSC) was established to develop tools to accelerate drug development for DMD. The resulting tools are anticipated to meet validity requirements outlined by qualification/endorsement pathways at both the U.S. Food and Drug Administration (FDA) and European Medicines Administration (EMA), and will be made available to the drug development community. The initial goals of the consortium include the development of a disease progression model, with the goal of creating a model that would be used to forecast changes in clinically meaningful endpoints, which would inform clinical trial protocol development and data analysis. Methods: In April of 2016 the consortium and other experts met to formulate plans for the development of the model. Conclusions: Here we report the results of the meeting, and discussion as to the form of the model that we plan to move forward to develop, after input from the regulatory authorities.
Highlights
The Duchenne Regulatory Science Consortium (D-RSC) was established to develop tools to accelerate drug development for Duchenne Muscular Dystrophy (DMD)
Therapeutics that provide a transient improvement of function may be studied in for shorter durations
It is clear that in order to conclusively demonstrate that drugs are effective, we need a clearer understanding of sources of variability in disease progression of DMD patients, so that appropriate endpoints can be investigated in appropriately selected patient subgroups
Summary
Members of D-RSC (including PPMD) are authors of this paper
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