Day 2

• The National Disease Registration Service (NDRS, NHS England) collects patient data on cancer, congenital anomalies and rare diseases, and provides expert, timely analysis to support clinical teams, academics, charities and policy makers to help plan and improve treatments and healthcare in England.
• The talk will illustrate how collaboration with NDRS can provide whole-population actionable insights into rare disease occurrence, treatment and outcomes.
• This can inform health policy and deliver pioneering rare disease research

Peter Lanyon, Rare Diseases Clinical Lead, National Disease Registration Service, NHS England

• International Collaboration and partnerships
• Access to diagnostics
• Access to treatments
• Diverse populations
• Future directions of RD research

David Pearce, President of Innovation, Research & World Clinic, Sanford Health

• The key challenges for Rare Disease drug development and access
  what is the EU HTA
• Description of EU HTA principles and timeline
• A SWOT analysis of the situation
• What has been done so far and what next

Sheela Upadhyaya, Chair Elect, Rare Disease Special Interest Group, ISPOR

• Challenges for commercialization in small markets
• Changes in the market access and regulatory environment
• How to priorotize markets – and how not to
• Success stories

Rüdiger Schulze, VP & GM, Germany & Central/Eastern Europe, Ultragenyx