Funded by a cooperative agreement with the US Food and Drug Administration (FDA), the Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP) will be created with Critical Path Institute (C-Path) and the National Organization for Rare Disorders (NORD).
Peter Saltonstall, president and CEO of NORD, stated that there are more than 25 million people in the US affected by one or more rare diseases, of which there are 7,000 known.
RDCA-DAP includes integrated rare disease data from sources such as clinical trials, observational studies, real-world data (RWD), and patient registries. NORD’s IAMRARE registry platform, focused on analytics, also will be included within the collaborative platform, which will help the organizations find and address knowledge gaps.
Joseph Scheeren, president and CEO of C-path stated, “By leveraging the rare disease community access and data of NORD and the data curation, aggregation, governance, and advanced analytics expertise of C-Path, we are poised to make a significant impact on rare disease drug development by providing quality data that will inform clinical trial design and accelerate the development of therapies.”
NORD and C-Path will use the knowledge of the natural course of disease established through the platform to determine an approach for clinical trial readiness, evaluate new treatments, and generate solutions to inform clinical trial design and regulatory review.
“Drug development for these diseases is often impeded due to the low affected patient numbers and a limited understanding of how rare diseases progress or how to measure clinical improvements. FDA-approved treatments exist for only 10% of rare diseases; with this collaboration we can change that statistic for the better for our rare community,” said Scheeren.