Collaborating for Cures Networking Evening 2016

Rare diseases are leading the charge in research when it comes to collaboration. The unique selling point of this group of conditions is the willingness of all stakeholders to work together to achieve better health outcomes for patients. This is something we champion at Findacure; we truly believe collaboration between patients, the pharmaceutical industry, clinicians, academics, medical professionals, and other interested groups, is necessary for the rare disease community to move forward and confront its most pressing challenges.

To encourage further collaborations, we organised our second networking evening on Thursday, 19th May. More than 40 delegates joined us at Baroosh in Cambridge, with conversation flowing faster than the prosecco. We brought back our highly successful lightning talk format for the evening, with five 5-minute presentations from delegates interspersed with networking.

Programme and summaries

Dr Rick Thompson - Scientific Officer, Findacure

The evening opened with a presentation from our very own Scientific Officer, Dr Rick Thompson, who shared the story of our collaborations around repurposing a treatment for the rare disease congenital hyperinsulinism (CHI). This includes partnering with Costello Medical Consulting to develop a cost of illness model for CHI, speaking with GOSH and NORCHI teams to understand the treatment pathway for CHI, organising a focus group with CHI families to understand the impact of the condition, running a joint crowdfunding campaign with the Children’s Hyperinsulinism Charity, and working with Elsevier to data mine their papers to better understand CHI.

Sarah Venugopal - Communications and Engagement, Raremark

Our first lightning talk then came from Sarah Venugopal from Raremark, who shared Raremark’s work to inform patients around their conditions. When most families receive a rare diagnosis, it can be difficult to find accessible information online – if they can find any information at all. To address this, Raremark collate research papers from PubMed and develop lay summaries, working with Patient Ambassadors and clinicians. They then support patients joining clinical trials, educating them around areas such as informed consent and randomisation, to improve the clinical trial experience.

John Baker - VP Diagnostics, Abcam

Our next speaker, Dr John Baker from Abcam, picked up on this topic of improving clinical trials, from the perspective of improving diagnostic tools. John outlined the challenge of rare disease trials, where patients often present with very different manifestations of the same condition. This can lead to failed trials, even where the drug successfully treated a proportion of patients. John argued that with improved diagnostic tools, the condition could effectively be subcategorised to identify which patients the drug could be effective in and receive approval for treatment of those patients.

Tamzin Byrne - Science in Public, and Cambridge Rare Disease Network

Tamzin Byrne from Cambridge Rare Disease Network spoke next. Tamzin focused her talk on the different networks that exist in Cambridge to encourage collaboration. She highlighted two key successful collaborations, which set up a new company to improve genetic testing by clinicians and a new charity to bring together the companies and researchers in the Cambridge cluster to promote research into rare diseases.

Prof Steve Jackson - Frederick James Quick Professor of Biology, The Gurdon Institute

Our following speaker, Prof Steve Jackson from the Gurdon Institute, continued on this theme of Cambridge collaborations. Steve outlined his work looking at cell biology to identify new treatments for genetic conditions – starting first with cancer and transferring the technology to rare diseases. He then introduced his plans to set up a new institute in Cambridge – the Darwin Initiative for Disease Biology – to promote collaboration to bridge the chasm between basic science and clinical application.

Annabel Griffiths - Costello Medical Consulting

Our final presentation turned into a spontaneous five minute talk from Annabel Griffiths from Costello Medical, after our planned speaker was unable to attend the evening. Annabel delved more into the work Costello Medical have been conducting to understand the cost of rare diseases to the NHS, and the budget impact repurposed drugs could have if they were developed to treat them.

We would like to thank all of our speakers for their interesting presentations, which inspired a lot of conversations throughout the evening. We would also like to thank Abcam for kindly sponsoring the evening, and all of our delegates for making the evening such a success.