CamRARE Companies Forum 2020: Connecting the dots

CamRARE Companies Forum 2020: Connecting the dots

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Can CamRARE’s Companies Forum 2020 connect the dots?

Active stakeholders in the rare disease forum are all too aware of the complex landscape that surrounds the development of treatments for such conditions. From small patient populations and lack of real-world evidence to the NICE Impact model (QALY) and big price tags, taking a drug from compound to the patient still remains an enormous challenge, where 95% of rare diseases currently have no viable treatment, a challenge that significantly burdens patients, families and indeed healthcare budgets.

COMPANIES FORUM: Recognising the Challenges

CamRARE, at the heart of Cambridge and its world class life science heritage, recognised this challenge and the opportunities it presents for the charity to create a climate of positive and meaningful collaboration that could become a driving force towards improved outcomes for rare disease drug development.

Our first step in recognising the challenges in patient access to drugs was to identify the drivers for stakeholders.  Science, economic viability and, running through both, measuring improved health outcomes (real-world evidence).

Step two was to bring together a broad range of stakeholders to develop a joined-up approach within the forum for supporting and influencing drug development strategy. To find out more about our Companies Forum we asked CamRARE Trustee Dr. Ron Jortner the charity’s Companies Forum lead, to tell us why he feels this initiative is an important part of the Cambridge rare disease eco-system.

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Dr. Ron Jortner, CamRARE Trustee and Founder and CEO of Masthead Bioscience

In conversation with Dr Ron jortner, Companies forum lead

What is the main aim of the Companies’ Forum?
“It is only through collaborative effort and stakeholder interaction that treatments and care can be developed for the benefit of those affected by rare diseases. Our mission at CamRARE is to create those networks, by bringing together the relevant stakeholders and facilitating these interactions. Companies’ Forum is a special case of that, focused on industry. Its aim is to encourage collaboration between industry leaders and in turn, other stakeholders, with the goal of advancing the development of treatments and care options for rare conditions.”


What sort of companies get involved?

“We welcome industry leaders with an interest in making a mark in the rare disease area. Many of our members are companies doing their own R&D; this includes some pharma and biotech companies who specialise in rare disease, as well as other, larger pharma companies for whom rare disease is just one of their focus areas. There are also members involved in drug re-purposing, in home care, and we have recently seen interest from some rare-disease-focussed CROs.  As with all CamRARE activities, it is this variety that creates synergies and opportunities for collaboration, which is so essential in the rare disease field. I hope we continue attracting such a variety of companies.”


How are COMPANIES FORUM meetings structured?

“Meetings are small, limited to 20 attendees. They typically last half a day, including a networking lunch or dinner. The atmosphere is very relaxed and informal, characterised by open discussion and free exchange of ideas and insights; we follow the Chatham House Rules to encourage this. Each meeting has a high-profile keynote speaker or panel, and attendees get a chance to engage in open discussion with them, workshop-style. As each meeting is small, informal and the atmosphere is intimate, discussions usually go deep.   Attendees have often complimented us on the level of content they get from our meetings.”

 and Tell us more about the next meeting…

“Our next Companies Forum meeting will be held at the Royal Society of Medicine on November 29th. It will focus on collaborative strategies to expedite drug development in rare diseases. The meeting will feature Prof. Chas Bountra, Pro-Vice Chancellor of Innovation at Oxford, and a thought leader on translational medicine, and several patient advocacy group CEOs – Daniel Lewi from CATS Foundation, Tess Harris from PDUK and Allison Watson from Ring20.  We will discuss ideas for expediting and streamlining the development of treatments for rare conditions and how patient groups can help in this – a topic of high interest to everyone involved. We’ll be convening in one of the RSM’s beautiful meeting rooms, and also have a nice lunch during the four-hour meeting.”

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Prof. Chas Bountra, Pro-Vice-Chancellor of Innovation at Oxford University Innovation

Do you see this forum affecting change and if so, at what level?

“Absolutely.  Firstly, as I already said, stakeholder interaction is itself a catalyst for progress in developing treatments. For example, a pharma company may already have a molecule in its portfolio which can benefit patients with some rare condition, but the company may not have ever heard of that rare disease. Such case exactly, where a company suddenly became aware of a condition, led to such realisation and now there’s a Phase 2 clinical trial for a drug that could potentially treat the fibrosis element of Alström Syndrome. We can’t even imagine how many such opportunities exist that have not been realised yet. Secondly, our vision for Companies Forum is that it becomes a taskforce, taking on one or more missions in the rare-disease field with members working on them together. Our members have already discussed a collaborative project on identifying unmet needs in patient homecare. We are brainstorming around these ideas just now.”


HOw do you like to describe the forum?

“I would describe it as a progressive group of industry leaders from pharma, biotech and healthcare who meet regularly to exchange ideas and do collaborative work on rare-disease projects with patients firmly in mind.”


 If you would like to participate in our next Companies Forum visit our website  for more information, and contact Jo Balfour for membership details.