Hopefully many more rare diseases will soon have disease modifying treatments that have the potential to transform patient lives. However if diagnostic, treatment and management pathways are not ready or fit for purpose then patient access will be delayed. This panel will discuss recent experiences in Cystic Fibrosis during a time when disease modifying agents were introduced. The panel will discuss this revolution in the treatment of this disease through the eyes of patients, parents and physicians to explore what can be applied to accelerate pathway development in other rare diseases.
Panelists
Tracey Daniels – Clinical Lead, York Teaching Hospital Foundation Trust
Ella Balasa – Patient Advocate and Health Engagement Consultant
Eleanor Thistleton – Parent and Medical Communications Expert, OPEN Health
Tom Smith – Patient Advocate and Life Science Public Engagement Expert
Panel Chair – Gavin Jones, Global Advisor, Rare Diseases, OPEN Health
