Researchers from the National Organization for Rare Disorders (NORD) will have the opportunity to delve further into studies of rare conditions thanks to a $250,000 grant from the U.S. Food and Drug Administration (FDA).
The funds will be used for studies that take a deeper look at the natural history linked to certain diseases.
“NORD’s Natural History Study Project tackles one of the greatest needs and an inherent challenge of the rare disease community: having enough longitudinal data to help medical researchers better understand how these diseases develop and progress over time,” NORD President and CEO Peter Saltonstall said. “The FDA’s support enables us to expand this important program with our shared goal of supporting patient organizations and helping patients.”
NORD's Natural Study Program began last year. To date, 7,000 diseases have been designated as rare -- and there are therapies on the market for just five percent of them.
“Natural history studies can yield vital information that is essential to clinical trial design such as biomarkers, demographics, genetic and environmental variables, disease progression and patient perspectives on the challenges of living with the disease,” Jacqueline Kraska, NORD's research program manager, said.
Under the terms of the grant, the FDA and patient groups will have access to information from the study, ensuring that future natural history studies will not duplicate research.
“This program will fill the unmet need of a generalized, standard approach to natural history study development across rare diseases,” Saltonstall said.
The study will include 20 rare diseases to be chosen by lottery. Patient groups that wish to be considered for inclusion can apply online at www.rarediseases.org.
