Agenda at a Glance
Orphan Drugs & Rare Diseases - Conference Day 2
Day 2: Wednesday - 15th September 2021
- Major challenges that each stakeholder encountered during the pandemic
- Lessons learned from Covid-19 and how we can benefit from it?
- How to build specific needs, support, and recommendations for sustainable healthcare systems
that provide directions for better preparedness in the future?
- The aim is to provide an overview of the recent discussions and updates from the European
- Commission concerning the revision of the orphan drug legislation in Europe.
Pedro Franco, Director for Global Regulatory & Scientific Policy (GRASP), Merck
- What mobile research nursing is all about.
- Utilising mobile research nursing in paediatric trials.
- Giving the child control over their participation in the trial.
Senior Representative, Illingworth Research
- How to improve patient engagement during studies while collecting standardized, regulatory-grade
- Clinical and hybrid studies to improve real-world data collection for drug development
Dr Femida Gwadry-Sridhar, Founder & CEO, Pulse Infoframe
- What is the state of play in rare and ultra-rare diseases and what needs to happen to have a
- Input gathered from 18 advocacy organisations/healthcare professionals
- Awareness / diagnosis / access to treatment / driving research and innovation / impact of covid /
patient perspectives / health economist perspectives of EU drug policy / industry perspective
- Public perception survey data around awareness of rare diseases and attitudes towards their prioritisation
Paul Greenland, President EMEA, Amryt Pharma
- How to close the gap between regulatory approval and funding agreement?
- How do the key HTA and payers operate?
- Samples of high profile reimbursement battles
- How to overcome struggles to meet the strict cost-effectiveness threshold for new therapeutics?
2:00 - 2:30, 2:35 - 3:05 pm - ROUND TABLE DISCUSSIONS - divided between 2 sessions of 30 mins. (5 mins. to change table) - Attendees have 2 round table options to attend
- The foundational value of building collaboration through trust, respect, transparency, and empathy
- Not all advocacy organizations are alike: assessing the landscape
- Real-world examples of innovative digital solutions driving collaboration among multiple
Tom Pulles, MD, VP, Head of Medical Affairs & Patient Advocacy, Ultragenyx Europe GmbH
- Mechanism: how it works
- The increasing number of RPDDs
- Past, present, and future value of Priority Review Vouchers
Christian Girard, Strategic Consultant, Orphan Drugs Industry Advisory & Intelligence
- Breaking down barriers for access
- Strengthening health systems around the world and accelerate pathways to diagnosis
- Stakeholders to address the rare diseases’ ecosystem gaps to collaboratively build a sustainable
roadmap for better health and a brighter future for the patients suffering from rare diseases.
- Challenges and opportunities in creating sustainable healthcare for all.