CDER Continues to Make Rare Diseases a Priority with Drug Approvals and Programming to Speed Therapeutic Development
A rare disease is any disease that affects less than 200,000 people in the U.S. Drug development for the approximately 7,000 rare diseases can be complex for many reasons. Challenges exist with using well-established trial designs. Selecting endpoints (outcome measures) can be difficult if there is a limited understanding of the natural history of the disease. Because of the small patient populations, there may not be enough people available to participate in rare disease clinical trials. For these and other reasons, many of these diseases have few or no available treatments.
The FDA’s Center for Drug Evaluation and Research (CDER) has teams and programs that are dedicated to overcoming these hurdles and facilitating the development of therapies for the more than 30 million people living with a rare disease in the U.S. The combination of government incentives and scientific advancements has fueled extraordinary development of drugs for rare diseases.
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