Rare Disease Innovation and Collaboration – CamRARE at the Health Horizons Forum

CamRARE host rare disease session at Health Horizons

Cambridge Biotech week, which ran from the 25th to the 28th of June was a brand new festival of events launched by the Global Innovation Forum designed to accelerate scientific ideas and support investment and growth for companies in the field of life sciences. Events took place in key venues across Cambridge and included the Health Horizons Future
Healthcare Forum, the Milner Therapeutics Symposium, Digital Disruptors, Scaling up Success in Biotech hosted by One Nucleus and the Hong Kong Biotech Roadshow. As part of
the two day Health Horizons Forum, Cambridge Rare Disease Network hosted ‘Rare Disease Innovation and Collaboration’ at Corpus Christi College in Cambridge.

Health Horizons is a high calibre, two-day conference focusing on the future of the healthcare industry. Over 100 hard-hitting speakers gathered to address this challenge and share their thoughts with a global audience. The Cambridge Independent shared their “Five things we learned at Health Horizons” summing up that “it’s all about being interdisciplinary”, “research needs to be translated”, scientific co-creation can follow from serendipity” and “open innovation can accelerate progress”. Needless to say, we were delighted to bring the conversation around to the development of treatments and cures for rare diseases and promote the patient voice as essential to these interdisciplinary and collaborative approaches.

As with all events, it’s great to have a plethora of stakeholders in attendance. A global audience of patient groups and rare disease advocates, industry, healthcare, research and technology professionals were in attendance to benefit from the thought-provoking presentations of our four speakers who are at the heart of innovative breakthroughs in therapies and technology. Delegates were able to share in their expertise in co-creating innovative solutions to some of rare disease’s most challenging healthcare issues. Presentations and Q+A were followed by a lively panel discussion moderated by CamRARE Trustee Prof. Tim Cox.

Presentations in a Nut-Shell

Professor Tim Cox: Professor of medicine and trustee of Cambridge Rare Disease Network.

What is it to be rare?

From the diagnostic odyssey still faced by patients with rare diseases to the rise of drug-buyers clubs, Professor Cox discussed the current climate and how his belief in human connections and collaborative and combined thoughts and effort will have the greatest potential for solving the biggest challenges in rare disease drug development. Tim spoke with a passion and empathy which comes from many years of working closely with affected patients, their families and with patient groups.

“The race to introduce new medicines, provide healthcare and stimulate investment often misses the
point for patients at the centre of our network…”

Dr Tim Gulliams: CEO and Co-Founder of Healx.

Drug repurposing for rare diseases: patient group partnerships at the heart of AI

Dr Tim Guilliams spoke passionately about the importance of collaborating with patient groups and how invaluable this has been in their work in drug repurposing. Tim described some of the huge leaps forward that the Cambridge-based start-up company has made
using AI and big data to find drug-repurposing options for 100 rare diseases by 2025. But his message was clear, that their partnerships with patient groups who can share their lived experience is vital to their success.

Patricia Durao-Lewi: Co-Founder of CATS Foundation.

Patient organisations driving research: collaboration is the key

An inspirational presentation showing the sheer power of a united patient community. From being told they would never meet another Tay-Sachs patient to hosting their sixth European family conference and the creation of the European Tay-Sachs and Sandhoff Charity Consortium Patricia demonstrated how collaboration with other Tay-Sachs patient groups globally and a firm partnership with Prof Tim Cox and his team has allowed them to create a powerful and united narrative for Tay-Sachs and Sandhoff disease. Their purposeful and focussed collaborative approach has resulted in a comprehensive patient registry, successful funding bids and a promising research pipeline with clinical trial dates set for 2019 and 2020.

“I would hope that my talk helped you (the audience) understand that rare diseases have to be tackled from all angles. This means including patient organisations as a powerful resource and for these to work with researchers and pharma as a team.”
Patricia Durao-Lewi

Dr Rick Thompson CEO of Findacure and Professor Tim Cox

Dr Thompson took part in a fireside chat with Professor Tim Cox.

Rick and Tim explored Findacure’s role in upskilling and training small and new patient advocacy groups to develop the knowledge and confidence to be able to move their organisations forward and engage better with companies and pharma to benefit their patients. Findacure is a Cambridge-based charity with a UK-wide reach.

“Strong, forward-looking talks from all the speakers showed what can be achieved in true partnership – and how. Put simply: different means for each party needs to be understood if the common goal is to be achieved.” Professor Tim Cox.