The US Food and Drug Administration (FDA) just updated its 2015 draft guidelines for drug discovery in rare diseases. The January update, Rare Diseases: Common Issues in Drug Development, seeks to help pharmaceutical companies and other sponsors perform more efficient development programs for drugs and biological products and provides new insights on complex elements of the development process.
In this webinar, participants will learn about the core elements of the new guidance plus the latest recommendations and instructions from the agency. Participants will hear about practical examples and applications.
Subjects will include:
– Natural history studies
– Issues for evaluation and validation of surrogate biomarkers
– Nonclinical flexibility
– External controls and early randomization
– Additional agency considerations and interactions