There are more than 7000 rare diseases affecting an estimated 3.5 million people in the UK1. To put that into context, this means 1 in 17 people are affected at some point in their lives. So next time you are on a bus, train or tube, look around you, it’s highly likely one or more of the people you see are affected by a rare disease. What’s more, 75% of rare diseases affect children1. Rare doesn’t seem to be quite so ‘rare’ now does it?
Rare diseases have their own individual challenges. Imagine it taking years to be diagnosed, your local doctor not knowing how to treat your condition, friends and family never having heard of it, and needing to travel hours or days to see a specialist (missing school or work and incurring expensive costs). Then finding out that treatment options are limited, unavailable or may come with unpleasant side effects. The European Medicines Agency (EMA) supports the development and authorisation of medicines for rare diseases (often referred to as ‘orphan’ diseases) through incentives such as protocol assistance, grants, and reduced fees2. However, this does not mean they are immediately available to all patients.
At Sciterion, we are passionate about improving the lives of people living with rare diseases and we’ve had the opportunity to work with a number of our clients on rare diseases in recent years.
Treatment limitations and the rare nature often severely limits HCPs’ knowledge around diagnosis, referral and management of patients, causing detrimental effects on patient outcomes3. To address these challenges, we’ve worked with our clients on a number of initiatives. Working Groups have helped us identify unmet needs and develop practical materials to support identification and referral of rare diseases. To reach a wider audience we’ve delivered award winning standalone meetings to 100+ attendees, both in person and virtually when COVID limited our activities.
We’ve seen these initiatives make a difference to patients’ lives across Europe by educating HCPs to recognise, refer and manage patients with rare diseases. Supporting these small networks of HCPs to engage, educate and grow in numbers has been not only been a fascinating, but also a rewarding experience for the teams. And over that time seeing the advances in management reflected in patient case studies, videos and photos has been an emotional experience.
So why work in rare diseases? Will your events and initiatives garner the interest of other more common diseases? Maybe not. Do you experience working with a close knit, dedicated and boundary pushing faculty who change patients’ lives? Yes, and that makes it all worth it.
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