Sep. 11, 2018
Posted by Valaree DonFrancesco
Washington, D.C., September 11, 2018—The National Organization for Rare Disorders (NORD), the leading independent nonprofit organization representing the 30 million Americans with rare diseases, has announced the addition of ten new rare disease registries, made possible through a multi-year grant award from Shire.
NORD’s IAMRARETM Registry Program provides a web-based tool to collect patient experience and patient-reported outcomes data across rare conditions. NORD works in direct partnership with patient advocacy groups to establish registries and provide programmatic services to support and address the needs of patients across the research continuum; from study implementation to maintenance and support, including training, research guidance, and IRB services. A longitudinal, natural history study systematically collects comprehensive health information with the intent of advancing the understanding of a disease and how it is expressed over time. Data from the IAMRARETM Registry Program establishes a foundation of knowledge which can be used to inform diagnostics, treatment protocols, standards of care, and the development of patient-centric clinical trials by providing contextual and complementary evidence.
In an effort to further support and expand access, NORD has dedicated a portion of the funding from Shire to develop ten registries to capture high-quality, longitudinal, natural history data for rare disease communities that do not currently have a formalized patient organization to advocate on their behalf.
“Patient-powered registries are changing the landscape of rare disease research,” said Pamela Gavin, NORD’s Chief Strategy Officer. “We are so thankful to Shire for their generous support and for their belief in our vision of the power of patients.”
NORD is proud to provide this important new registry model to the rare disease community and to work with patients as partners to improve the quality of life of individuals and families affected by rare diseases.
Approximately 7,000 rare diseases have been identified, affecting 30 million Americans. Of the 7,000 that exist, less than 500 have FDA-approved treatments. Let’s work together to change this!
About the National Organization for Rare Disorders (NORD)®
The National Organization for Rare Disorders (NORD) is the leading independent advocacy organization representing all patients and families affected by rare diseases. NORD is committed to the identification, treatment and cure of the 7,000 rare diseases that affect 30 million Americans, or 1 in every 10 people. NORD began as a small group of patient advocates that formed a coalition to unify and mobilize support to pass the Orphan Drug Act of 1983. For more than 35 years, NORD has led the way in voicing the needs of the rare disease community, driving supportive policies and education, advancing medical research, and providing patient and family services for those who need them most. NORD represents more than 280 disease-specific member organizations and their communities and collaborates with many other organizations in specific causes of importance to the rare disease patient community.
Shire is the global biotechnology leader serving patients with rare diseases and specialized conditions. We seek to push boundaries through discovering and delivering new possibilities for patient communities who often have few or no other champions. Relentlessly on the edge of what’s next, we are serial innovators with a diverse pipeline offering fresh thinking and new hope. Serving patients and partnering with healthcare communities in over 100 countries, we strive to be part of the entire patient journey to enable earlier diagnosis, raise standards of care, accelerate access to treatment, and support patients. Our diverse portfolio of therapeutic areas includes Immunology, Hematology, Genetic Diseases, Neuroscience, Internal Medicine, and Ophthalmics.