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Orphan Drug Act

Meaning

The Orphan Drug Act is a significant piece of United States federal legislation enacted in 1983 to incentivize the pharmaceutical industry to develop therapeutic agents for rare diseases and conditions, defined as those affecting fewer than 200,000 people in the U.S. This act provides specific financial and regulatory benefits, including tax credits for clinical research costs, waived user fees, and a crucial seven years of market exclusivity upon approval. In the hormonal health space, this law is vital for encouraging research into treatments for uncommon endocrine disorders, such as certain congenital adrenal hyperplasias or rare pituitary adenomas, which would otherwise be commercially unviable to pursue.