Description

One of the greatest challenges in the conduct of CF clinical trials is the designation of clinically relevant endpoints, which are sufficiently rigorous to reflect biological changes and to meet regulatory requirements. This session will include a broad variety of approaches to reporting outcomes in CF, from CFTR function to imaging changes to patient-reported outcomes. The final discussion will highlight current regulatory requirements in the context of outcomes, allowing attendees an understanding of ongoing challenges in the field.

Learning Objectives:

• Summarize state-of-the-art measurements of CF outcomes.
• Assess a broad panoply of potential measurements, including measures of CFTR, gene signatures, microscopy, radiology & patient-reported outcomes.
• Explain ongoing challenges in the field of biomarkers.

Speaker(s):

• Jane Davies, M.D., MBChB, MRCP, Prof - Paed Respirology & Experimental Medicine
• Mark O Wielpuetz, M.D., University of Heidelberg
• Ellison Suthoff, Vertex Pharmaceuticals
• Nico Derichs, M.D.
• Kengyeh K Chu, Ph.D., Research Fellow, Massachusetts General Hospital
• Milene T. Saavedra, M.D., Assistant Professor of Medicine, University of Colorado School of Medicine
• Eugene Sullivan, M.D., FCCP, EJS Consulting, LLC