BioWednesday London: Rare Diseases: Solutions for Increasing Access to Innovative Medicines

18:00 - 21:00 Wednesday 8 May 2019

We have reached full capacity for this event and registration is now closed. Please email if you'd like to be added to the waiting list.

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

20:05     Networking

21:00     Close



One Nucleus Members: Free

Non-Members: £50 +VAT

Academics: please contact Alicia


May 8th, 2019 6:00 PM   to   9:00 PM
Taylor Vinters - Tower 42
33rd Floor
25 Old Broad Street
London, London EC2N 1HQ
United Kingdom
Phone: 01223 896450
Phone: 01223 896456
Email: Email:

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