BioWednesday London: Rare Diseases: Solutions for Increasing Access to Innovative Medicines
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The development of drugs for the treatment of rare diseases benefits from legislative incentives, regulatory orphan designation developments and changes in the approval process. However, rare diseases inherently mean small sample sizes and consequently challenges recruiting sufficient numbers into clinical trials, less well understood pathophysiology of diseases and reimbursement challenges.
Our BioWednesday will explore key factors to consider for programme development, including innovative study design options, navigating regulatory landscapes and reimbursement strategies. We will also showcase an example of a company using artificial intelligence to repurpose existing compounds to encourage and accelerate patient access to treatment. This session will demonstrate drug development solutions to help patients to achieve better outcomes.
18:00 Registration and networking
18:30 Welcome from One Nucleus and Taylor Vinters
18:35 Welcome and Introductions: Joanna Segieth, Biosynetix
18:40 Using artificial intelligence to identify new treatment options for rare diseases: Michale Bouskila- Chubb, Healx
19:00 Maximise the value of your drug development programme: Joanna Segieth, Biosynetix
19:20 Regulatory tools and pathways to facilitate early access: Richard Huckle, Huron Consulting Group
19:40 Reimbursement challenges in rare diseases: Andrew Hobbs, Huron Consulting Group
20:00 Closing Remarks: Joanna Segieth, Biosynetix
25 Old Broad Street
London, London EC2N 1HQ