Rare Diseases: Access All Areas
Opportunities and Implications for Rare Disease Patients
Tuesday, 9th November 2021
3-hour virtual event 0800 (EDT) / 1300 (BST) / 1400 (CEST)
Register for our complimentary event!
- Attendance is open to professionals in the biotech and pharma industries
- Register below to ensure you also receive post-event content
- Attend as many sessions as you would like on the day
- Live Q&A – ask questions and comment via the live chat function
Starting with the patients’ perspective, we explore the challenges of living with a rare disease and highlight new research and synergies across rare disease communities, as well as the implications for pharma. We also examine the appraisal landscape for rare diseases, looking at best practices for value assessment and patient engagement. Finally, we delve into patient advocacy’s success in raising awareness of unmet needs and unique ability to source potential early-stage therapeutic solutions.
Key speakers include:
- Nick Sireau, CEO and Chair of Trustees, AKU Society
- Sharon Hesterlee, PhD, Executive Vice President & Chief Research Officer, Muscular Dystrophy Association
- Matthias Bischof, Senior Director, Global Health Economics and Outcomes Research, Novartis Gene Therapies
- Peter Jackson, PhD FRCP, Chair, NICE HST Committee