Rare Diseases at FDA | FDA

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The .gov means it’s official.Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you're on a federal government site. The site is secure. The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely. Over 7,000 rare diseases affect more than 30 million people in the United States. Many rare conditions are life-threatening and most do not have treatments. Drug, biologic, and device development in rare diseases is challenging for many reasons, including the complex biology and the lack of understanding of the natural history of many rare diseases. The inherently small population of patients with a rare disease can also make conducting clinical trials difficult. Since the Orphan Drug Act was signed into law in 1983, the FDA has approved hundreds of drugs for rare diseases, but most rare diseases do not have FDA-approved treatments. The FDA works with many

People with colored puzzle pieces On this page: What is a rare disease? | What is an orphan drug? | How does the FDA encourage product development for rare diseases? | What is the Orphan Drug Act? | How do the FDA medical product centers support rare disease product development? | What is the FDA Rare Disease Innovation Hub? | What does the FDA Office of Orphan Products Development do? | How can patients become involved in FDA’s work on rare diseases? What is a rare disease? The Orphan Drug Act defines a rare disease as a disease or condition that affects less than 200,000 people in the United

Rare Diseases at FDA | FDA

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