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Successful collaborations: engaging the people that matter

22nd September, 2020 – 24th September, 2020

Back in September, we hosted a three-day online workshop that focused on how patient groups can successfully collaborate with stakeholders from across the rare disease spectrum. The goal of the workshop was to help patient groups collaborate with industry, pharma and other rare disease patient groups so that the rare community can advocate for change as one, united force.

Knowing how many patient group organisations exist with similar ethos and goals across the globe, we wanted to help our patient group representatives understand how they can utilise their global community’s shared passion to connect with people who want to help them and advance their cause.


Our three-day workshop was kicked off by our very own Rick Thompson, who covered some of the basics of engaging with the pharmaceutical industry. It was a great introduction to the next few days, as we learned why working with industry can be so beneficial. We also discussed the different kinds of interactions that can be fostered with pharmaceutical professionals.

Day one of our workshop saw a fantastic talk from Peter Wasson (BioIndustry Association) and Daphnee Pushparajah (Alexion). They described what industry partners are looking for from patient groups and taught us how best to reach out to them. We also learnt about the need for integrity and transparency in all interactions with industry. The day’s talks were rounded off by Taylor Fields (Intrabio) and Will Evans (NPUK), as they discussed the successful relationship between the two and gave us their top tips on successful collaborations.

On day two, we focused on collaborations between patient organisations. We heard great talks throughout the day, with the first being hosted by Becky Jenner and John Sharpe of Rett UK and Reverse Rett. They discussed their successful patient group collaboration and demonstrated how two patient groups can form a successful alliance, which benefits both rare disease communties. Their presentation was followed by Toni Mathieson from NPUK,who spoke of the success of LSD Collaborative, which was set up to advocate on behalf of patients and families with Lysosomal storage disorders in the UK. Rick rounded off the day by talking about the importance of collaboration agreements when looking to establish a professional relationship with another group.

On the third and final day of the workshop, it was time to discuss collaborating with medical professionals. We kicked off the day with an interactive session lead by clinical geneticist. Dr Melita Irving, where we tackled questions, such as‘How do you begin to capture the needs of your community and drive your own agenda?’ Melita’s session was followed by three lightning talks where we had the privilege of learning more about the different strategies patient organisations deploy when seeking to engage with clinicians. The workshop concluded with a talk from Emily Cutting at Ataxia UK, who presented us with advice on building medical and scientific advisory boards.

Read our day-by-day breakout of topics and events below and attend our next online Diversity Workshop on 12-14th January 2021 – Register now!

Rick Thompson, CEO, Findacure. Peter Wasson, Policy and Public affairs, BIA. Daphnee Pushparajah, Director of patient advocacy and engagement, Alexion. Will Evans, Chairman, Niemann Pick UK. Taylor Fields, Senior VP, IntraBio.

Day one was a jampacked day, as we heard from a range of experts who have extensive experience in how to best engage with industry partners!

Rick kicked off the workshop with an introduction to engaging with industry. He covered the reasons why patient organisations should work with industry professionals and highlighted the key role industry plays in drug development. Rick introduced us to the importance of understanding the role of the ABPI code and the fact that it exists to encourage interaction between pharma and patient organisations rather than to deter it. He then covered the importance of making sure that an interaction with industry is the perfect fit for your patient group before proceeding with establishing one with them. He ended his speech by giving some top tips for engagements, such as making sure to retain your independence and recognise your own value and expertise in the interaction. Amen to that!

Next in our line-up was Peter Wasson, who discussed how to engage and collaborate with biotech companies. Peter stressed why engagement is so key for patient organisations and emphasised the importance of bringing together different but complementary strengths to drive research collaborations forward. He demonstrated the importance of the values of organisational independence, integrity and transparency through the case study of a collaborative project with Parkinson’s UK. Peter also gave us some pointers on what patient organisations can do to provide funding streams for research, support clinical trial access and research infrastructure and support access to medicines after regulatory approval.

Daphnee from Alexion then built upon what Peter addressed and focused on the role of patient advocacy at Alexion and why they want to engage with patient communities. Daphnee focused particularly on the building blocks of good patient and industry exchanges. She emphasised the need for industry to listen to and learn from patient communities, as well as understand and respect their perspective on matters. Daphnee also said inudstry must practice transparency, identify a patient community’s needs properly and protect a patient’s privacy.

Day one ended with Taylor Fields and Will Evans discussing the relationship between Niemann Pick UK and IntraBio. They shared their journey of establishing a successful collaboration and working together to develop a treatment for Niemann Pick. They also shared with us their top tips for successful collaborations, including making sure to understand and acknowledge each other’s expertise as well as your own. Be sure to speak up and voice opinions and reach out to those working in your area. They assured us that once you have collaborate on a project once, that it is easier to collaborate with someone else again in the future.

To keep our workshop interactive, we concluded the session with a discussion between attendees. They identified where they may be able to provide value to an industry partner, what types of corporate interactions they had previously engaged in and what concerns they had about beginning to interact with industry. Overall, day one was a very informative session that set us up well for the rest of the workshop ahead!

Becky Jenner, CEO, Rett UK. John Sharpe, Chair, Reverse Rett. Toni Mathieson, Chair, LSD Collaborative. Rick Thompson, CEO, Findacure.

On day two, we honed our focus on collaborating with patient organisations and other charities. The day began with a talk from Becky Jenner and John Sharpe, who discussed the successful collaboration between Rett UK and Reverse Rett through the Rett Disorders Alliance. Becky and John covered the history of the two organisations and the small steps which led to the establishment of the alliance in 2015. They emphasised the fact that each charity has a distinct focus but common goals, which are conducive to a successful collaboration. Becky and John demonstrated how a great collaboration can benefit patient communities by showing us the Rett Syndrome Health Checklist, which was produced with consultation from all stakeholders in the alliance. They finished off their brilliant presentation by sharing their top tips with us. These included: making sure you have a neutral facilitator present for all the meetings, having mutual respect for the value each other’s work brings, being open and honest and most importantly, ensuring that all decisions are made with the best interest of patients in mind.

Their talk was followed by a recorded presentation by Toni Mathieso, who introduced us to the LSD Alliance and the role it plays in helping patients with Lysosomal Storage Disorders. Toni took us through the history of the LSD Alliance and stressed the need for establishing a terms of reference prior to establishing a collaboration. Toni then highlighted the areas that the LSD Alliance works in, including the Patient Professional Partnership Network, lobbying and newborn screening for those with LSD’s. Toni also gave great insight into some of the challenges that they faced as an alliance, such as the impact of changing personnel, struggling to get financial stability and trying to find time and capacity to fulfil objectives. Toni showed us the real benefits of collaboration and highlighted why shared learning helps everyone involved. She even showed us why coming together gives patients a stronger voice to influence policy and lobby for change! Before Toni concluded her talk, she briefly covered the international work that is being done by The International Niemann Pick Disease Alliance (INPDA) to help further highlight how collaboration can be successfully scaled up to help even more patients. Toni finished off her presentation by emphasising that good communication is key and that time should be taken to understand each other’s aims and objectives fully.

Day two concluded with Rick Thompson covering the value of collaboration agreements when starting and maintaining professional relationships. He showed us an example of a collaboration agreement to provide a basis for creating our own. He also highlighted the key aspects that the agreement should include, such as providing the background for the collaboration, stating the commitments of stakeholders, stating the intent of the relationship and having a declaration and signatures from all involved. It was a great activity, as it really broke down the steps involved in creating a collaboration agreement into easy, bite-sized chunks.
Day two gave us some key insights into the challenges and benefits associated with collaboration and taught us that above all else, great communication=successful collaborations.

Dr. Melita Irving, Guys and St. Thomas', Julia Boonak, CDG UK, Leslie Harrison, AKU Society, Tracy Lynch, Wolfram Syndrome, Emily Cutting, Ataxia UK

Our third and final day was kicked off by the wonderful Melita Irving. She got us all interacting and discussing key prompts associated with the topic of engaging with clinicians right from the start! In Breakout rooms, we used whiteboards to jot down our answers to key questions, such as ‘How do you capture the needs of your patient community to drive your own agenda?’, ‘How do you find clinicians and researchers?’ and ‘What has been your experiences in engaging with clinicians so far?’

After we brainstormed in our groups, we took the time to feed back to the room what we learned from each other and how we had answered the questions. It was really interesting to hear the different approaches that people favoured and it was extremely insightful to see which techniques have already been tried when approaching clinicians – even if it did take a little while to get used to the white boards!

After Melita’s presentation and activity, we were lucky enough to not have one, but three lightning talk speakers from across the rare disease spectrum take the time to speak with us about their experience engaging with healthcare professionals. First to take the stage was Julia Boonak, who discussed the CDG UK project of delivering information packs about CDG to clinicians. It was great to see the work CDG put into identifying the need for the packs and delivering a successful outcome. They have managed to deliver 200 packs to hospitals since Rare Disease Day 2019 and have made contacts with two children’s hospitals as a result.

Next up, we had Leslie Harrison from the AKU society, who talked about what they have done to raise awareness of AKU amongst healthcare professionals. Their project centred around providing e-learning modules on AKU, which could be accessed by clinicians. They were able to launch modules with both the Royal College of General Practitioners (RCGP) and the Royal College of Nursing Institute (RCNI), making the project an overall success despite facing a few hurdles along the way.

Our final lightning talk was brought to us by Tracy Lynch from Wolfram Syndrome UK. Tracy shared the Charity Road Trip that Wolfram Syndrome UK volunteers completed back in 2014. The challenge was to complete the largest ever awareness campaign for Wolfram Syndrome and the Charity Road trippers were tasked with delivering a comprehensive medical information pack to the 24 main Children’s Hospitals within the UK. The aim was to help doctors and medical professionals make the prompt and correct diagnosis of Wolfram Syndrome. Overall, the project was a great success and the volunteers travelled over 1,600 miles across the UK to deliver the valuable information to hospitals and clinicians!

After the brilliant lightning talks, our collaborations workshop was rounded off with a talk from Emily Cutting on the subject of building medical and scientific advisory boards. Emily started off by giving us an overview of the roles the two boards play at Ataxia UK. The scientific board is made up of researchers who are not active in the field of Ataxia and the medical panel is comprised of healthcare professionals who have a specific interest in Ataxia. She emphasised that having this distinction is important, because it keeps the scientific advisors unbiased, whilst making sure that the medical panel is adequately informed to provide expertise. Emily went on to demonstrate the process of setting up the two structures and highlighted some of the challenges associated with each, specifically the difficulty of finding new members. She shared a success story of the medical advisory panel, which was the production of Ataxia UK’s medical guidelines, and gave a great insight into the value of having these types of panels in place. She also showed us the work and effort needed to make sure that they can be maintained successfully.

Day three was a great round off to our workshop and it was inspiring to hear examples of engagement that has paid off and raised awareness for rare patient communities. Another successful workshop for Findacure!



Successful collaborations: engaging the people that matter

by Blayne Baker time to read: 11 min