Workshop E: A Regulatory Perspective: Understanding the Approach Towards Selecting Efficacy Endpoints for Rare Disease Clinical Trials
Time: 11:30 am - 2:30 pm
day: Workshop Day Track B
Details:
Attend this workshop to better understand how to demonstrate clinically meaningful and statistically significant endpoints to identify the windows of opportunity for the future treatments of IgAN, FSGS and ADPKD.
- Harmonizing global regulations to standardize endpoints for rare and genetic kidney diseases
- Developing disease-specific endpoints for rare kidney diseases with different phenotypic presentations and disease pathology
- Navigating endpoints beyond total kidney volume and eGFR slope
- Understanding how to go about INDs and when to request a meeting
- Leveraging registries to aid in validating endpoints
- Selecting endpoints for rare kidney disease clinical trials for efficient assessment of clinical signal for decision making in drug development
