Four-plus decades after the bipartisan Orphan Drug Act was signed into law on January 4, 1983, by then-President Ronald Reagan,1 rare disease research has accelerated dramatically. At the time when the legislation was spearheaded by Sen Orrin Hatch (R, Utah) and Rep Henry Waxman (D, California), approximately 6000 rare diseases had been identified as affecting 25 million individuals in the US.2 Since then, major milestones have emerged, including the accelerated approval pathway, the Rare Diseases Act, the completion of the Human Genome Project, the first gene therapy approval, and the founding of the National Organization for Rare Disorders. The number of treatments on the market for rare diseases also has grown exponentially, from 34 to approximately 800,2-4 with more than 1300 approved indications and over 7500 designations, according to an FDA database search conducted on January 29, 2026.5
