Date: 24 April 2019
Time: 14:00–15:00 BST

There are currently around 500 drugs in development for the treatment of rare diseases, encompassing many different therapeutic areas. Despite the regulatory incentives and financial support introduced by a number of legislations, drug development programmes for the treatment of rare diseases still face many challenges.

An accelerated or optimised access pathway is a regulatory concept designed to improve patient access to breakthrough technologies and treatments in a cost-effective model. It relies on an iterative development plan which must be linked to unmet medical needs, prospective design, utilisation of real-world evidence data (safety & efficacy) and involve healthcare stakeholders (such as Health Technology Assessment (HTA) bodies) throughout development. Patients with rare diseases inherently mean a relatively small sample size and consequently challenges recruiting sufficient numbers into randomised clinical trials. These small patient populations are particularly well suited to the concept of accelerated access pathways and innovative study designs such as umbrella/basket/platform or adaptive design approach. 

This webinar will explore drug development solutions to help patients to achieve better outcomes. 

Speakers and topics

• Richard Huckle, Senior Consultant Regulatory Affairs with Huron Consultancy, will speak on the regulatory tools for pathways to facilitate early access. (20 minutes)
• Joanna Segieth, Founder and Director of Biosynetix Ltd., will talk about 'Utilising multiple compound study designs to successfully achieve a higher return on existing tried and tested methods' (20 minutes)

This will be followed by a Q&A session. (20 minutes)

The event is fully booked and the registration is now closed. 
Please contact membership@topra.org to be added to the waiting list.