A 30-year retrospective: National Organization for Rare Disorders, the Orphan Drug Act, and the role of rare disease patient advocacy groups

Abstract: This 30-year retrospective looks at the history of the US Orphan Drug Act and how it originally came to be enacted, with particular emphasis on the role of patient advocacy organizations. The article explores how rare-disease patient advocacy groups learned to work with government partners in the National Institutes of Health and US Food and Drug Administration, members of Congress, and the media to promote their cause. It also discusses their awakening to the value of collaboration, which led to the formation of an umbrella organization to represent their shared interests. Milestones over the years are briefly discussed, and current areas of focus for rare disease patient organizations – most notably, globalization – are also examined. Keywords: rare diseases, orphan products, NORD, Rare Disease Day, global patient communities, EURORDIS, NZORD, TFORD, CORD, rare disease research