Key result
The Duchenne Regulatory Science Consortium met to formulate plans for developing a disease progression model to forecast changes in clinically meaningful endpoints for Duchenne Muscular Dystrophy.
The D-RSC meeting established plans to develop a disease progression model for Duchenne Muscular Dystrophy to inform clinical trial design and data analysis.
May inform future DMD trial designs; leaves open prospective validation before clinical or regulatory adoption.
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.
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Larkindale et al. (2017) studied Duchenne Muscular Dystrophy. The Duchenne Regulatory Science Consortium met to formulate plans for developing a disease progression model to forecast changes in clinically meaningful endpoints for Duchenne Muscular Dystrophy.
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