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Archives: Agenda
Afternoon Break and Networking
Setting the Scene & Welcome to the Forum
END OF CONFERENCE
HEAR FROM ACORSS THE POND, VIRTUAL SESSION Innovation and digital health for rare diseases
- Challenges and limitations of rare disease research: how new solutions can help to overcome this
- Embracing a patient centric approach in rare disease trials through digital health
- Opportunities for innovation for orphan drug trials in the US
INTERACTIVE PANEL DISCUSSION: Navigating the complexities of insufficient resources to allow your rare disease trial to succeed in an increasingly competitive landscape
- Battling site competitiveness to reduce delays on study start-up: what can you do differently to tackle these operational challenges?
- When there are immense pressures on the trial ecosystem, how can we build and ensure quality in an innovative way
- Developing an optimized strategy as a small to medium-sized biotech with limited resources to stay afloat
- Discussing the importance of protocol optimization and specialized statistical support to reduce the pressure on sites and relieve bottlenecks
- Global health inequalities: Are rare diseases rare everywhere?
END OF DAY 1 AND NETWORKING DRINKS
Real World Data/ Evidence in translational medicine and rare disease clinical trials
- AMR, Evolution and history of the natural world
- The innate immune system – a billion years Proof of concept
- RWD RWE – surgical sites and improved outcomes in oral surgery
- RWD RWE – Serious burns and a survival benefit
- COVID pandemic RWD RWE pulmonary administration
- Safety studies and tolerability
- The evolutionary successor to antibiotics is a multi-modal anti-inflammatory via TNF-a, Il6 and Il-10
- Key milestones and next steps: CHMP and Orphan designation
Miransertib—an interesting path to an interesting disease
- Developmental history of miransertib
- Past clinical trial history in rare diseases
- Current research
- Future directions
Placing fear reduction at the core of the patient experience strategy: assessing the impact of diminished anxiety and increased sense of control for rare disease clinical trials
- Improving the sense of security to reduce unnecessary suffering and distress for patients
- Understanding the different types of patients and their individual response behaviours
- Being hyper aware of cultural differences and biases to ensure sensitivity and effective outcomes for all
- Sharing best practice on effective strategies with case studies and examples to help reduce patient drop out rate