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The Next Generation of Rare Disease Drug Policy

April 7, 2022

The Next
The United States defines a rare disease as a condition affecting fewer than 200,000 people in the
country or one in which “there is no reasonable expectation” of recovering research and
development costs.1 Examples of rare diseases include genetically-linked cancers, cystic fibrosis,
and debilitating pediatric conditions like Gaucher disease and spinal muscular atrophy

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