Data for Endpoint Selection

Selecting appropriate endpoints is a central challenge in rare disease research, particularly when clinical outcomes are heterogeneous or poorly characterized. This session will explore how different data sources can inform endpoint selection and support endpoints that are clinically meaningful, feasible to measure, and interpretable for regulators and other decision-makers.

Moderator: Craig Lipset, Co-Chair, Decentralized Trials & Research Alliance; Clinical Innovation, Buffalo Initiative

Panelists:

• Gabrielle Conecker, MPH, Executive Director & Co-Founder, Decoding Developmental Epilepsies
• Gerald F. Cox, MD, PhD, FACMG, Consultant, Gerald Cox Rare Consulting, LLC.; Staff Physician in Genetics, Boston Children’s Hospital, a NORD Rare Disease Center of Excellence
• Amena Fine, MD, PhD, Assistant Professor of Neurology and Developmental Medicine, Kennedy Krieger Institute, a NORD Rare Disease Center of Excellence
• Amy Raymond, PhD, PMP, Executive Director, Therapeutic Strategy Lead, Rare Disease & Cellular and Genetic Medicines, Worldwide Clinical Trials

View PDF Presentation from this Session
View Slideshow Presentation from this Session