Charting rare stars
How NORD is putting patients in control of rare disease natural history studies
A databank constructed by the National Organization for Rare Disorders and the VHL Alliance could serve as a model for how patient organizations can generate natural history data to help speed development and FDA review of new treatments.
The Cancer in Our Genes International Patient (CGIP) Databank was formally unveiled in May and discussed at NORD's Rare Diseases and Orphan Products Breakthrough Summit held Oct. 21-22. CGIP is the first public launch from a NORD platform designed to help patient organizations manage their own registries as a vehicle for natural history studies and other research.
NORD developed the platform because it saw a need for patient registries that are extensive and sophisticated enough for longitudinal studies of diseases that are not well understood, but also inexpensive and user-friendly so small patient communities can use them.
VHLA's goals for the