| The rare disease community includes diverse perspectives from patients, families, caregivers, and advocates. Each of these perspectives is important and can contribute to the work of advancing rare disease treatments. Input from the rare disease community is crucial to the development of medical products to diagnose and treat conditions, as well to as maintain or increase quality of life. This year, the U.S. Food and Drug Administration is part of the worldwide community observing Rare Disease Week—February 28th through March 4th. Our participation includes hosting FDA's Rare Disease Day on March 4th. Our theme is: "Sharing Experiences in Rare Diseases Together." What do we mean by "Sharing Experiences in Rare Diseases Together?" We know how important it is to hear and understand the patient voice in all drug development activities. Ultimately, in the review of new medical products, what really matters is the patient perspective—does a drug or device improve how the patient feels, functions, or survives? Notice the word "review." That's one small word for everything the FDA does to approve new medical products. "Review" at the FDA encompasses widely disparate activities across many scientific disciplines. | | | |
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