On May 9, the Duchenne Regulatory Science Consortium (DRSC) held its Annual Meeting in Crystal City, VA. In the audience of about 40 people, there were industry and academic Consortium members, potential industry members, five FDA representatives, two NIH representatives, C-PATH representatives, and Buddy Cassidy, the DRSC patient rep and member of PPMD’s Adult Advisory Committee.


The objectives of the meeting were to recap work DRSC has accomplished and discuss with the Consortium where to go next, focusing on gaps in data and gaining preliminary approval for a framework for the disease progression model.

Buddy Cassidy, patient rep, DRSC, opening remarks

DRSC currently has seven data sets in house, with one currently in the process of being transferred to C-PATH. These datasets will be aggregated so they can be used to develop a disease progression model. As mentioned in previous blogs, in order to facilitate a data sharing platform that houses multiple data sets, there needs to be a common language so these data sets can “inter-operate.” In the drug development world, these are CDISC standards and are overseen by an organization that maintains the common data standards framework for all therapeutic areas. The CDISC working group of DRSC has worked for over a year to put together a draft guidance and it is ready to go out for public review, which is a huge accomplishment.


I know this may not sound like sexy stuff. Think of it this way though. It is as important as the behind-the-scenes ATM inter-operability that is in place, so that when you insert your ATM card at one bank, it can talk with your bank and seamlessly carry out whatever task you need to accomplish.  So kudos to the CDISC standards working group for all their hard work!


Most of the meeting focused on what we know about the disease progression based on current endpoints and analysis, and emerging concepts. Duchenne is a complex heterogeneous disease and may require the use of multiple endpoints in clinical studies. Much of the analysis focused on predicting one outcome over a period of time (for ex loss of ambulation over two years) by a baseline value (such as the 4-stair climb assessment).   Additionally, we know that some endpoint measures are prognostic, meaning they can predict how a patient will progress (rise from floor > 6 seconds prognostic for functional decline, and if > 10 seconds prognostic for loss of ambulation, 6-minute walk test < 300 meters prognostic for rapid decline and lack of treatment response in  a 12 month study).  


All this analysis feeds in to how DRSC develops a disease progression model that can span all ages and across ambulatory stages.


The meeting closed with a discussion about a proposed disease progression model using FVC mapped to various endpoints across the disease progression. This will be developed as DRSC goes forward, with regulatory and consortium input along the way. There was much discussion with the regulators and Consortium members about the feasibility of the proposed model and important concepts to consider.


I had dinner with some Consortium members the night before and was struck with their level of engagement and commitment to this project. They see the necessity of getting the CDISC standards in place and the shared data platform up and running before jumping into the modelling. PPMD applauds them for sticking with it. Good things often take longer than we would like, but these partners bring incredible knowledge and focus. I am looking forward to the next phase of the DRSC project and working together to develop the disease progression model.


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