National Organization for Rare Disorders (NORD) evolved from a group of leaders of different rare disease patient organizations. All the groups in NORD were unified to mobilize support to pass the Orphan Drug Act. Later, NORD was instrumental in the Orphan Drug Act of 1983 to raise financial support for treating rare diseases. NORD has raised the voice for the need of rare disease community.
NORD is the country’s leading advocacy body meant to represent all rare disease patients and families. NORD and its 280 members of the patient organization are committed to identifying, evaluating, and treating rare disorders through education, research, and patient support programs. NORD collaborates with many government agencies, importantly the National Institutes of Health (NIH) and the Food and Drug Administration (FDA). Rare Disease Day is an observance day which is held on the last day of February each year worldwide with the aim of creating awareness for rare diseases and improving access to treatment for individuals with rare diseases and their families. NORD is the official sponsor for Rare Disease Day in the U.S.
The NORD-sponsored study states that the increased price of drugs shouldn’t be implied on the orphan drugs. The orphan indication is helping the drug market or medical sphere with better drugs to combat or fight diseases and give a better diagnosis for rare diseases. The prices of the medical therapy have not been drastically affected by the introduction of orphan indication. In the U.S., around 25 percent of orphan drug approvals target populations smaller than 5,000 patients. Of the 503 orphan-indicated therapies approved at the time of the research, 217 are no longer protected by orphan exclusivity or patent protection. Of these 217 treatments, 116 are generic or biosimilar. This report surely provides enough information that is needed to make informed decisions. “The data shows that the seven-year market exclusivity provision is working as intended as an incentive for developing rare disease therapies and is not being abused,” says NORD’s President and CEO, Peter L. Saltonstall. Approximately, 25-30 million American’s are affected by more than 7,000 rare diseases that are identified. About 80 percent of the rare diseases are known to be genetic which are chronic in nature and life-threatening.
In at least two instances of rare disease research grant program, NORD has resulted in FDA-approved orphan therapies. NORD has implemented Rare Action Network for the promotion of state advocacy. It has given a home to the people who had been orphaned by the medical community.