Rare Disease
A condition affecting a small share of the population, defined in the United States as fewer than 200,000 people. Rare diseases often face diagnostic delays and limited therapies, and qualify drugs for orphan-drug development incentives.
What is a Rare Disease?
A Rare Disease is a medical condition that affects only a small portion of the population. In the United States, the legal threshold is a condition affecting fewer than 200,000 people nationwide at any given time.
Because each rare disease touches relatively few patients, knowledge tends to be concentrated among a handful of specialists, and many conditions have no approved treatment at all. Patients frequently spend years navigating misdiagnoses before the correct condition is identified.
Why do Rare Diseases matter?
Although any single rare disease is uncommon, there are thousands of them, and collectively they affect a large number of people. The diagnostic delays, limited therapeutic options, and high per-patient costs make them a significant clinical and economic challenge.
The rare-disease designation also unlocks orphan-drug development incentives, such as extended market exclusivity and research support, intended to make it commercially viable for manufacturers to pursue treatments that would otherwise serve too few patients to justify the investment.
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