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.
The FDA’s guidance offers a flexible and comprehensive approach to developing drugs for rare diseases, acknowledging the unique challenges and encouraging early and continuous dialogue with the FDA. Ergomed, with its extensive expertise in regulatory affairs and rare disease clinical development, can provide invaluable support in implementing this guidance.
