Archives: Agenda

Interactive roundtable sessions offer a unique opportunity to come together with your peers to share best practice and develop solutions to critical challenges facing the industry as a whole. Each discussion will be led by a table moderator and will focus on a different challenge within rare disease clinical trials. Roundtables are an exciting, interactive way to build your personal network and learn from the experience and expertise of others.

After 30 minutes, delegates will have the opportunity to swap and choose a different table, and each roundtable will run twice.

• How to approach the design and conduct of a clinical development plan in the rare disease space from a biotech perspective
• Maximizing organizational and operational efficiency, and key considerations for regulatory adherence
• How should pharma work with patient organisations and what are the best ways to engage with them?

• Overview of rare diseases in neurology
• Pharma focus on rare diseases in CNS for drug development – Where do industry interests lie?
• Challenges in rare disease trials in neurology – biomarkers, endpoints, patient populations
• Case study – R&D strategies in amyotrophic lateral sclerosis (ALS)

• Implementing alternative methodology like RWD or AI to obtain and model data when clearly defined clinical trial roadmaps and regulatory pathways seldom exist
• Discussing the necessity and ethics of placebos for orphan drug trials: Can we do something different?
• How can you run studies with limited populations, does the clinical trial model work in this area of research?

• Turning to patients to help reduce waste, shorten development times and ensure the right outcomes are selected and measured effectively
• Shedding light on the ACT-EU Multistakeholder partnership advisory board: an overview from a patient and policy perspective