Registries and Real World Data as Development Platforms - NORD Scientific Symposium

Registries and real-world data sources are increasingly used to support multiple stages of rare disease research. This session will frame how these data assets can function as development platforms, informing natural history studies, clinical trial design, and evidence generation beyond traditional clinical trials. Moderator: Angela Waanders, MD, MPH, MS, Section Head, Neuro-Oncology, Ann and Robert H. Lurie Children’s Hospital of Chicago, a NORD Rare Disease Center of Excellence Panelists: Amy Palmer Laster, PhD, Chief Scientific Officer, Foundation Fighting Blindness Mayowa Azeez Osundiji, MD, PhD, Medical Geneticist, Mayo Clinic, a NORD Rare Disease Center of Excellence Srilakshmi (Sri) Raj, PhD, Assistant Professor of Genetics, Albert Einstein College of Medicine, a NORD Rare Disease Center of Excellence Theresa Strong, PhD, Director of Research Programs, Foundation for Prader-Willi Research