Emmes Endpoints Solutions Advances Duchenne Muscular Dystrophy Research with Innovative FDA Submission

28 February 2024 | Wednesday | News

Ahead of Rare Disease Day, Emmes Endpoints Solutions has made a significant stride in Duchenne muscular dystrophy (DMD) research by submitting a Duchenne Video Assessment (DVA) qualification plan to the FDA's Center for Drug Evaluation and Research (CDER). This pioneering submission aims to introduce a novel clinical outcome assessment for DMD, potentially revolutionizing patient participation in clinical trials.
Image Source : Public Domain

Image Source : Public Domain



The DVA is designed to assess the progression and severity of DMD through videos of patients performing functional tasks at home, eliminating the need for travel to clinical sites. This method allows for the inclusion of participants at any stage of their disease, offering a broader and more inclusive approach to clinical trials. By utilizing certified central raters and pre-specified scorecards, the DVA ensures objective and blinded evaluations of patient function, setting it apart from other anecdotal video capture methods.

Emmes' innovative approach could significantly enhance clinical trial accessibility and effectiveness for DMD patients, a group currently underserved by approved therapies. The company expects initial feedback from the FDA by July 2024, with further data collection and analysis planned over the next two years.

If successful, the DVA could pave the way for the use of home-based video assessments as primary endpoints in clinical trials across a range of rare diseases, including neuromuscular and neurodevelopmental disorders. This approach not only addresses methodological challenges but also supports the decentralization of clinical trials, reducing logistical hurdles for patients and potentially improving trial outcomes.

Marielle Contesse, PhD, at Emmes Endpoint Solutions, emphasized the DVA's development by caregivers of people with Duchenne, highlighting its potential to provide a more accurate reflection of drug efficacy by capturing changes in patient function that are noticeable in the home environment. This could significantly impact how clinical trials are conducted and evaluated in the future, especially for rare diseases.

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