The FDA awarded a $250,000 grant to the National Organization for Rare Disorders (NORD).
NORD will use the grant to develop 20 natural history studies for 20 rare diseases based on a lottery system. The studies fill the unmet need of a generalized, standard approach to natural history study development across rare diseases. NORD will work with disease patient groups to develop a registry toolkit containing best-practice tools and templates that will aid future organizations to initiate and conduct natural history studies that are designed to support drug development programs. The grant will fund a 1-year cooperative agreement between NORD, patient groups and the FDA.
There are 7,000 rare diseases that affect 30 million Americans, 20 million of whom are children. NORD hopes that more understanding and documentation of rare diseases will aid researchers with clinical trials.
Patient groups can enter the lottery for the Natural History Study Project by applying on the NORD website: www.rarediseases.org/patient-registries/nord-fda
To qualify for the natural history study project, patient groups must be 501©(3) Organizational Member of NORD with an unmet medical need and a limited natural history and research gaps that could potentially be addressed by a natural history study.
“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,” said Peter L. Saltonstall, president and CEO of NORD. “The FDA’s support enables us to expand this important program with our shared goal of supporting patient organizations and helping patients. This program will fill the unmet need of a generalized, standard approach to natural history study development across rare diseases.”
“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,” said Jacqueline Kraska. research program manager at NORD. “Studying one rare disease can lead to findings about another.”
FDA Awards National Organization for Rare Disorders (NORD) $250,000 Grant to Support the Development of 20 Natural History Studies for Rare Disease Research [news release]. Washington DC; NORD: December 10, 2015.