12 June 2024
Regulatory Dashboard | Navigating Rare Diseases: Considerations for the Development of Drugs and Biological Products Guidance for Industry
Rare diseases, affecting fewer than 200,000 people in the US, present unique challenges in drug development, particularly due to limited medical knowledge, unclear natural history, small patient populations, and a lack of development experience. This guidance provides comprehensive regulatory considerations for successfully developing small molecule- and biologics-based drug products for rare diseases, offering recommendations on natural history studies, nonclinical, quality, and clinical development.
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