Patient-Focused Drug Development Forum (PFDD)

On October 23, 2020, PSC Partners Seeking a Cure hosted an interactive virtual public meeting giving patients and caregivers a unique opportunity to share how PSC has impacted their lives. They shared their first-hand experiences with existing treatments and their unique perspectives on the unmet treatment needs of PSC patients. The meeting was developed in conjunction with the U.S. Food and Drug Administration (FDA) as an externally-led Patient-Focused Drug Development (PFDD) meeting, to complement FDA’s PFDD initiative. The patient input collected at the meeting and reported here will have a lasting impact on the lives of PSC patients as it will inform the development and regulatory review of new drugs for this rare disease.

Making Our Voices Heard: Patient-Focused Drug Development Forum 

View Voice of Patient Executive Summary

View Voice of Patient Full Report

View full recording.

View this time-stamped agenda to help you navigate the recording. 

This video has been spliced into each agenda section. To only view a specific section, click links below. 

• Welcome, Introductions & Overview 
• PSC Clinical Symptoms & Quality of Life 
• Consequences of the Lack of Effective PSC Treatments 
• Pediatric & Adolescent Patient Perspectives 
• Improving Current PSC Treatments 
• Incorporating the Patient Perspective into PSC Clinical Trials 
• Summary, Next Steps & Closing Remarks