Even as the FDA and other government agencies have incentivized drug development for rare diseases, significant hurdles remain. Chief among them is the inherent difficulty in designing effective clinical trials for newly defined diseases, or those with extremely small patient populations, and oftentimes without regulatory precedent or established endpoints.
Attendees will hear from biotech companies who are meeting this challenge head on through innovative strategies for identifying potential enrollees, data gathering and even identifying new biomarkers to serve as clinical endpoints. The ultimate goal is to produce trials that yield rich and compelling data needed for drug approval.
Ability Level: All
Session ID: 503690