April 2019

Expert perspectives on improving cognitive measures from clinical trials to clinical practice.

Review of Recent FDA Draft Guidance for Rare Disease Clinical Trials

April 22, 2019

In this video, Dr. Pam Ventola reviews two recent FDA draft guidances for rare disease drug development, where she highlights the need for indication-specific outcomes, as well as how to get the most from natural history studies to determine acceptability/feasibility of endpoints and to inform future trial designs.