Launching Registries & Natural History Studies

In the last decade, this has changed dramatically. The FDA Safety and Innovation Act incorporates the patient voice into drug development through listening sessions that allow patients and caregivers to share their experiences, needs, and priorities directly with FDA staff.

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 accelerates clinical development, makes it less expensive, and encourages new companies to develop rare disease drugs.

Getting the Most from the Data

Critical Path Institute (C-Path) and NORD, through a collaborative grant from the FDA, joined together to create the Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP®), an integrated database and analytics hub designed to be used in building novel tools to accelerate drug development across rare diseases. The platform promotes sharing patient-level data and encourage the standardization of new data collection.

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]