12:00 AM
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Nov 10, 2014
 |  BioCentury  |  Product Development

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 registry are twofold: helping patients share data and connect with researchers, and helping patients and researchers better understand the disease's natural history and variables involved in disease progression.

Pamela Gavin, COO at NORD, said the platform's focus on natural history helps patient organizations increase their role in researching and developing treatments.

"From a functional perspective, it takes a simple contact registry and gives it more dimensions. It enables patients and patient organizations to contribute and participate in the drug approval process," she said.

At the summit, both FDA Commissioner Margaret Hamburg and Director of the Center for Drug Evaluation and Research Janet Woodcock hailed the CGIP Databank as a tool that can improve the agency's ability to review treatments for rare diseases.

"Having such a resource can yield vital information about biomarkers, demographic, genetic and environmental factors...

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