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Accelerating Cures and Treatments

Over 350 million people in the world have a rare disease, but only about 10% of rare diseases have an FDA-approved treatment. Drug development can be extremely challenging for rare diseases because of the often small patient populations and a limited understanding of how these diseases can manifest and progress. If we can bring together the experience of patients, along with clinical data, we can pave a clearer path for producing new treatments with better defined and more meaningful outcomes and improve our understanding of the progression of a disease. This would accelerate clinical development, make it less expensive, and encourage new companies to develop rare disease drugs.

The Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP®) is an integrated database and analytics hub that is designed to be used in building novel tools to accelerate drug development across rare diseases. It is being developed by the Critical Path Institute (C-Path) and NORD through a collaborative grant from the FDA [Critical Path Public-Private Partnerships Grant Number U18 FD005320 from the US Food and Drug Administration].

RDCA-DAP’s goal is to use data to accelerate clinical development, lowering costs and encouraging even more companies and researchers to get involved in rare disease research and innovation. The platform will promote sharing patient-level data and encourage the standardization of new data collection, resulting in a fuller understanding of a rare disease. This tool can do a lot to drive innovation and aid those looking for novel treatments. To learn more about the technical components and vision, please visit C-Path’s site.

If you would like to learn more or talk with the team at NORD about how to get involved please contact us at: [email protected]

If you would like to talk about contributing data or get involved in discussions around needed analytics please contact: [email protected]


*RDCA-DAP® is fully funded with a federal grant awarded to the Critical Path Institute.