A longstanding priority for both Congress and FDA, there has been a significant evolution in recent years in the ways in which patients’ experiences and preferences have been incorporated into the drug development and regulatory review processes. Particularly when it comes to rare diseases for which natural history is often lacking, operationalizing learnings from the lived experiences of patients has become a critical goal for numerous stakeholders. In this session, we will discuss case studies and best practices in centralizing the voice of the patient/caregiver into the design of meaningful clinical trials (endpoints and methodologies), as well as ways patients can interact with FDA itself. In addition to biotech and pharma leaders in managing rare disease programs, the panel will include an FDA regulatory lawyer, with agency and congressional experience relating to patient-focused and rare disease drug development.