Announcing the Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP), an integrated database and analytics hub that will provide data-driven solutions to help accelerate and optimize drug development across rare diseases. Developed as a collaboration between the Critical Path Institute (C-Path) and the National Organization for Rare Disorders (NORD) and funded through a collaborative grant from the FDA [Critical Path Public-Private Partnerships Grant Number U18 FD005320 from the US Food and Drug Administration], the RDCA-DAP will provide a centralized and standardized infrastructure to support and accelerate rare disease characterization.
The robust integrated platform will include aggregated rare disease data from various sources —including NORD’s IAMRARE™ registry platform— and an analytics tool that will allow efficient and effective analysis of data to accelerate rare disease research and inform clinical trial design and regulatory review.
There are more than 25 million people in the United States affected by one or more of the over 7,000 rare diseases that have been identified. Rare disease drug development is frequently impeded due to small patient populations, as well as a limited understanding of both disease progression and how to measure drug effects that matter most to patients. The RDCA-DAP will be a framework for a sustainable, cooperative scientific approach to clinical trial readiness in rare diseases by addressing these knowledge gaps.