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In this week’s blog, Mary Rose – our Head of Operations – tells us about Findacure’s recent trip to World Orphan Drug Congress, Europe.

This week, Rick – Findacure’s CEO – and I attended World Orphan Drug Congress (WODC) in Barcelona, Spain. Now in its 10th year, WODC has become one of the largest and well-established events on rare diseases and orphan drugs within Europe, catering for patient advocacy groups, regulators, and industry from across the continent. Rick has been on WODC’s advisory board for the last few years, but this year Findacure was a Supporting Partner of the conference, which meant we were also able to host an exhibition stand at the event.

Day one of WODC began with the pre-congress workshops – half day sessions focused on specific topics, including market access in Europe and US, creating sustainable business models and pathways for patient access, overcoming clinical and regulatory challenges, and drug repurposing.  

The latter of these workshops was hosted by our own Rick Thompson, and looked at two key areas; how to enable patient group involvement in drug repurposing, and how to encourage industry involvement and support in drug repurposing. The session also featured a drug repurposing success story, delivered by Prof. Ranganath, Director of the National Alkaptonuria Centre and Trustee of AKU Society, who shared their experience in repurposing nitisinone for an ultra-rare condition. (If you’d like to learn more about repurposing, why not join us for our annual Drug Repurposing for Rare Diseases Conference on 24th February 2020!)

For days two and three, Rick and I were delighted to be joined by Laura Harper- our new incoming Fundraising Manager, who will officially be joining our team in late November! With the conference now in full swing, we divided our time between talks on cell and gene therapies, patient advocacy, access to medicine, pricing and reimbursement, and more.

A highlight of mine were the conference roundtables. Of particular enjoyment was the session titled “The patient voice: an important consideration not only for late stage reimbursement, but also for early stage regulatory affairs”, led by Tamsyn Frost, CEO of IDEA Regulatory and Pushpa Hossain, Researcher at Metabolic Support UK. Alongside the other participants, we discussed why meaningful patient engagement is so critical to clinical development, and how to guarantee that this becomes more than just a ‘tick box exercise’ for industry partners. We also touched on the practical and financial benefits of listening to patient voices – ensuring that treatment development is based on patient priorities, and delivery of medicines is suitable for the cohort of patients you are serving.

Not quite over his time in the spotlight, the final day saw Rick delivering another presentation – this time on involving patients in research and drug development. Using the analogy of ‘you wouldn’t design a coffee machine without speaking to coffee drinkers’, Rick explained why patients should be consulted along every step of the drug development process and shared some great examples of patient group-led research initiatives, such as CATS Foundation’s involvement in research for Tay-Sachs and Sandhoff disease. Rick implored industry not to be scared of approaching patients, but to also ensure this is a fair process for those involved – not expecting to shift the burden of successful drug development onto patients, without providing them with the support and training they need.

Being able to exhibit at the conference also brought fantastic opportunities for us, and we were thrilled by just how many people stopped by to speak with our team. We were able to connect with rare advocates from all across the rare disease spectrum, spreading the word on our patient group training programme, drug repurposing conference, and our community building projects. Lots of people came to us having heard great things about The Patient Group Handbook: a Practical Guide for Research and Drug Development, written by our co-founders, Nick Sireau and Anthony Hall. If you’d like to get your hands on a copy, you can find it available here on Amazon.

All in all, we had a brilliant time at this year’s World Orphan Drug Congress, hearing lots of new updates, sharing ideas, and meeting up with our friends from across the rare space. We hope everyone who came to see us enjoyed hearing about our work and hope you will be in touch soon!


World Orphan Drug Congress 2019

by Mary Rose Roberts time to read: 4 min