With our annual conference, Drug Repurposing for Rare Diseases, just over a week away, things are getting busy in the office. We are preparing delegate packs, writing talks, and chatting to our venue and speakers to make sure the event is our best ever.  

Earlier this week we released our finalised conference programme, which you can view on our conference website or download here.  So, in today’s blog we thought we would preview a few more of the talks we have planned for the day, hopefully encouraging the few of you who still haven’t signed up to buy a ticket and join us to learn more about repurposing.

Reimbursement in repurposing; a NICE perspective

The National Institute for Health and Care Excellence (NICE) plays a central role in determining UK patients’ access to medicines. In recent times they have been increasingly aware of the unique challenges facing rare disease patients and orphan drug developers, and have developed the Highly Specialised Technologies (HST) programme in response to these challenges, and to give a fairer platform within which ultra-orphan medications can be assessed for cost effectiveness. As the field has advanced NICE has begun to think about the role of repurposed medicines in the rare disease space, the value they offer to the healthcare system, and the challenges of fair pricing in this sector. In this talk, Sheela Upadhyaya, HST and Topic Selection specialist at NICE, will review NICE’s role in enabling access to new technologies, and review some of the challenges and opportunities repurposed drugs for rare disease present for their processes.

Sheela Upadhyaya – HST and Topic Selection Specialist, NICE

Dual deprivation: understanding the psychological burden that coexists with the physical struggles in individuals with rare diseases

Sandy is one of the runners up in this year’s Student Voice Prize – a collaborative project run by Findacure and Medics4RareDiseases that aims to raise awareness among medical students about rare diseases. When writing her essay, Sandy spent some time working with the rare disease patient group FOP Friends, who gave her the opportunity to speak to an individual living with FOP (or stoneman syndrome). Sandy used this opportunity to gain an insight into the psychological struggles that many rare disease patients face and manage alongside their physical symptoms. Sandy’s talk will recount her experience writing her essay and talking to FOP patients, and her thoughts on the challenges of mental health facing rare disease patients.

Sandy Ayoub,
Student Voice Prize Runner up, St George’s University London

Lightning talk session

The conference will once again see the return of our ever-popular lightning talk session. This features five brave souls who have agreed to take the stage for a timed five-minute talk against the bell. Each will be speaking on a topic that they have proposed, giving us a great insight into the projects that matter to our audience. This year’s programme features the work of three patient perspectives: SRUK’s Fatima Sulaiman giving us an insight into their approach to driving research for Scleroderma; Tony Thornburn of Behcet’s UK highlighting the challenge facing their patient population; and Karen James of MEBO research highlighting the real cost of the rare condition trimethylaminuria. The rest of the programme will feature Paul Wicks, highlighting the potential of digital health to drive forward drug discovery, and Lisa Dilworth of Synteract discussing the role that CROs (Clinical Research Organisations) can play in the development of effective rare disease drugs.

As you can see, the conference is set to be an excellent day, with engaging talks that will highlight some real successes and innovations in the rare repurposing space. We’d love to see you there, ticket sales end at midnight on Wednesday 19th of February, so secure your ticket today.

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Speaker Spotlight – Drug Repurposing for Rare Diseases 2020

by Rick Thompson time to read: 5 min
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