3-hour virtual event 0800 (EST) / 1300 (GMT) / 1400 (CET)
“It’s all about collaboration. By itself, a rare disease patient group can’t really accomplish that much, but when you work with scientists, industry, other patient groups, clinicians and regulators, that’s when we can make a really significant difference.”
CEO and Chair of Trustees, AKU Society
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.
3-hour virtual event 0800 (EST) / 1300 (GMT) / 1400 (CET)
5 mins
50 mins
0805 (EST) / 1305 (GMT) / 1405 (CET)
CEO and Chair of Trustees, AKU Society
"I am very pleased and excited to be part of this event and to showcase the struggles of rare disease patients and their supporters in the quest to bring treatments to their communities. I look forward to exploring solutions together to bring innovation and urgency to the need for better market access, policy, and stakeholder engagement. By definition, rare disease means fewer patients, but our unmet needs are significant and our vision is to help to bring treatments to rare disease patients that are also sustainable for health services."
Managing Director, Cello Health Logic
Chairman, Cello Health Communications
We explore the challenges of living with a rare disease and highlight synergies across rare disease communities, as well as the implications for pharma.
10 mins
50 mins
0905 (EST) / 1405 (GMT) / 1505 (CET)
Senior Director, Global Health Economics and Outcomes Research, Novartis Gene Therapies
PhD FRCP, Chair, NICE HST Committee
Principal Insight Analyst, BresMed
Principal Insight Analyst, BresMed
Examining the appraisal landscape for rare diseases looking at best practices for value assessment and patient engagement.
10 mins
50 mins
1005 (EST) / 1505 (GMT) / 1605 (CET)
PhD, Executive Vice President & Chief Research Officer,
Muscular Dystrophy Association
MPH, Senior Director, Community Engagement,
Taysha Gene Therapies
CEO and Chair of Trustees,
AKU Society
VP, Head of Advanced Therapeutics
Cello Health BioConsulting
Exploring patient advocacy’s success in raising awareness of unmet needs and unique ability to source potential early-stage therapeutic solutions. Plus, a view of the unique challenges that rare disease drug developers face when transitioning from a R&D focus to preparing for commercialization.
5 mins
‘BresMed and Cello Health are excited to host the second Pharma Progress conference, one of a series of virtually enabled, short and focused events. The conference will focus on the implications of rare diseases for patients and pharma and how we as an industry can work with those affected to support the development of treatments and care pathways which meet the needs of those living with rare diseases.’
