New U.S. Initiative Launches to Accelerate Rare Disease Treatments
A new U.S. initiative, Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP), was launched to accelerate treatment innovation across rare diseases. The platform, a collaboration between the National Organization for Rare Disorders (NORD), the Critical Path Institute (C-Path), and the U.S. Food and Drug Administration (FDA), aims to share existing patient data and promote standardized data collection.
RDCA-DAP made its debut at the RDCA-DAP 2021 Virtual Workshop, attracting over 400 rare disease stakeholders. The platform seeks to advance understanding of disease progression, develop mathematical models, and improve clinical trial design. It addresses the pressing need for treatments, as over 90% of the estimated 7,000 rare diseases lack FDA-approved treatments.
The platform curates data from various sources, including clinical trials, observational studies, patient registries, and real-world data. It provides workspaces for advanced analytics, encouraging collaboration and candid dialogue among academic, clinical, regulatory, and patient groups. Interested parties can access rare disease data by finding datasets of interest and requesting access. RDCA-DAP also features patient profiles, highlighting its importance to patient groups.
RDCA-DAP is a significant step towards accelerating drug development for rare diseases. By promoting collaboration and data sharing, it aims to bring attention to relatively neglected rare diseases and ultimately, improve patient outcomes. The platform's success relies on the engagement of stakeholders, from academic institutions to regulatory bodies and patient groups.
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