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Creating new drugs for patients with rare diseases poses special challenges for drug developers. Learn about the clinical trial process with a special focus on rare disease drug development.
Learn about the incentives for rare disease drug development created by the Orphan Drug Act.
The Orphan Drug Act is critical to ensuring that rare disease drug development continues as new rare diseases are identified.
Incentives provided by the Orphan Drug Act have encouraged a greater number of companies to develop therapies for rare diseases.
Nearly 30 million Americans suffer from a rare disease or condition, yet only four percent of rare diseases have an approved treatment.