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The Food and Drug Administration has released a draft guidance document for navigating the unique challenges of developing treatments for rare diseases. It emphasizes the importance of understanding the natural history of a disease, creating study designs with clinically meaningful end points, establishing product safety and effectiveness, and ensuring product quality.
The Food and Drug Administration has released a draft guidance document for navigating the unique challenges of developing treatments for rare diseases. It emphasizes the importance of understanding the natural history of a disease, creating study designs with clinically meaningful end points, establishing product safety and effectiveness, and ensuring product quality.
The Food and Drug Administration has released a draft guidance document for navigating the unique challenges of developing treatments for rare diseases. It emphasizes the importance of understanding the natural history of a disease, creating study designs with clinically meaningful end points, establishing product safety and effectiveness, and ensuring product quality.
