Who we are
Duchenne Regulatory Science Consortium (D-RSC) is a global collaboration formed by the Critical Path Institute (C-Path) and Parent Project Muscular Dystrophy (PPMD), a nonprofit patient advocacy organization. The consortium includes representatives of the pharmaceutical industry, academic and clinical researchers, patient advocates and non-profit groups working on Duchenne muscular dystrophy (DMD), and is advised by the U.S. Food and Drug Administration (FDA), European Medicines Agency (EMA), and the National Institutes of Health (NIH). Drug development in DMD has been hampered by the small patient population, the lack of shared natural history data, and paucity of well characterized endpoints and biomarkers. Using C-Path’s successful consortia model in other disease areas, D-RSC was established to develop novel drug development tools and to strengthen regulatory engagement to help inform development of clinical trial protocols that can use fewer patients and/or can be carried out over shorter periods while remaining informative.
To accelerate development of patient-centric therapies for Duchenne muscular dystrophy by serving as an international collaborative hub to develop new, regulatory endorsed drug development tools based on the most current scientific insights supported by an integrated database of detailed Duchenne patient data, and to augment expertise in clinical trial design and disease progression.
D-RSC is currently working on:
- Development of disease progression models of five endpoints to integrate into a clinical trial simulation tool
- Qualification of glutamate dehydrogenase as a liver safety biomarker in trials involving patients affected by muscle disorders
- Regulatory acceptance of a patient reported outcome for DMD
- Regulatory acceptance of Magnetic Resonance biomarkers for DMD and their relationship to disease progression, in collaboration with the ImagingDMD Consortium
- Models of additional endpoints as data becomes available
How we do it
D-RSC has created an integrated database of patient-level clinical data for DMD, which is available for analysis by the Duchenne community as permitted by the owners of each dataset. D-RSC uses Clinical Data Interchange Standards Consortium (CDISC) standards to integrate data and has written the CDISC Duchenne Muscular Dystrophy Therapeutic Area User Guide, which is available to the community. D-RSC is using its rich datasets to develop a clinical trial simulation platform for DMD based on models of five endpoints and is seeking regulatory endorsement of these models. In collaboration with C-Path’s Predictive Safety Testing Consortium, we are developing glutamate dehydrogenase (GLDH) as a biomarker for liver toxicity in patients with underlying muscle damage and have received a formal “letter of support” from EMA and positive response to the Qualification Plan for GLDH from the FDA. Future projects may include supporting the regulatory acceptance of other DMD-relevant biomarkers and patient reported outcomes or development of additional models to be leveraged for drug development.