Externally-Led Patient-Focused Drug Development Meeting for ARPKD

Agenda

*all times in CST

1:00-1:05 p.m.	Opening Remarks	Matt Becka – PKDF
1:05-1:20 p.m.	Recap of EL-PFDD: What’s Important to ARPKD Patients/Caregivers	Larry Bauer – Hyman, Phelps & McNamara
1:20-1:30 p.m.	Framework for Integrating the Patient Voice into Drug Development	James Valentine – Hyman, Phelps & McNamara
1:30-1:40 p.m.	Opportunities to Conduct Clinical Trials in ARPKD	Lisa Guay-Woodford – CHOP
1:40-2:15 p.m.	Panel Discussion: Perspectives on Facilitating Clinical Trials in ARPKD
2:15-2:25 p.m.	Landscape Analysis of Tools to Evaluate What’s Important to ARPKD Patients	Katherine Dell – Cleveland Children’s
2:25-2:55 p.m.	Panel Discussion: Setting a Patient-Focused Research Agenda for Drug Development Tools
2:55-3:00 p.m.	Closing Remarks	Matt Becka – PKDF

Panelists:
– Lisa Guay-Woodford, CHOP
– Katherine Dell, Cleveland Children’s
– Erum Hartung, CHOP
– Max Liebau, University of Cologne
– Djalila Mekahli, KU Leuven