EP.88Development of clinical trial simulation tool for Duchenne muscular dystrophy through the Duchenne Regulatory Science Consortium

Developing clinical trial protocols that give definitive answers as to whether potential new therapies are effective for rare diseases is challenging due to the small population sizes, limitations on availability of natural history data and limited understanding of disease progression. In Duchenne muscular dystrophy (DMD) this has contributed to few trials meeting primary endpoints, and led to questions as to how best to evaluate efficacy of therapeutic candidates. The Duchenne Regulatory Science Consortium (D-RSC) is a public-private-partnership that aims to develop quantitative tools to accelerate drug development and seeks approval of such tools through regulatory pathways at FDA (Food and Drug Administration) and EMA (European Medicines Authority) to confirm utility and value.
Source: Neuromuscular Disorders - Category: Neurology Authors: Source Type: research