Pharma
Progress
A virtual event from BresMed and Cello Health
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)
- 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
‘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.’
Event overview
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.
Attendees will hear about
- ‘Patient challenge’ new research, synergies and unmet need across the community of rare disease patients; implications for pharma
- How rare diseases are appraised internationally and the implications and opportunities for patients
- Developing breakthrough therapies in partnership with the patient and preparing for the transition to commercialization
Agenda
3-hour virtual event 0800 (EDT) / 1300 (BST) / 1400 (CEST)
5 mins
Welcome and Introduction
50 mins
Understanding patients with rare diseases
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."
New Social Media Listening (SML) insights: Patients' perspectives in rare diseases
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
Break
50 mins
Rare disease appraisal landscape and bringing novel technologies to market
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
Break
50 mins
Partnering with the Patient to Innovate and Prepare for the Transition to Commercialization
PhD, Executive Vice President & Chief Research Officer,
Muscular Dystrophy Association
VP, MPH, Chief Patient Officer and Head of Government Affairs,
Taysha Gene Therapies
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.
Additional patient and industry speakers to follow.
5 mins
Closing
