Shining a Light on Rare Diseases

The 29^th of February has a special feel to it simply because of its relative rarity, whether or not it’s your ‘true’ birthday, or it’s the day that girlfriends propose to their boyfriends. For those of us working in the pharmaceutical sector, it has a significance that transcends all of these considerations, and that is its association with Rare Diseases. 

In most branches of medicine, there are statistics galore that help to define the prevalence, occurrence and severities of a particular condition. For example, we are all probably aware of the statistic that almost half of us will develop some form of cancer in our lifetime (ref. www.nhs.uk), but for rare diseases, such simple, tangible stats are harder to define. Of course this is largely because of their very nature – being rare – but thanks to the existence and outreach of Rare Disease Day, we are starting to shed more light on the situation. 

Importantly, what we are learning is that ‘Rare’ maybe something of a misnomer. For example, as stated on the Orphanet website, ‘Rare diseases are rare, but rare disease patients are numerous.’

So let’s highlight two important statistics, taken from the UK government’s website (the Rare Disease Framework):

• Globally, approximately 1 in 17 people are affected by a rare disease, which in the UK equates to around 3.5 million individuals. 
• Children are disproportionately affected and of those who are, over 30% of them will die before their fifth birthday. 

The good news is that, although these numbers seem daunting, real progress is being made in the research and developments of new therapies. In the four years since we last experienced the extra February day, the EMA have approved 141 drugs for use in a rare disease or a complication of a rare disease. For all those living with a condition that one of these new drugs can treat, it is obviously a momentous time, but there remains a very large number of rare disease patients for whom the wait is long and frustrating. 

Here at Dice, we are proud to have been entrusted by many of our clients to help them bring some of these new drugs to market. Rare disease campaigns now represent almost half of our workload and it’s a proportion that we feel sure will increase in the years to come. 

Working in this sector not only requires in-depth scientific understanding and skilful medical writing, but also sensitivity and empathy especially when dealing with patient support groups. With 12 rare disease brands now being supported by the Dice team, it’s clear that we manage to get the balance right. 

The Rare Disease Day campaign continues to do a great job in raising awareness about these important issues and Dice are more than happy to donate to their cause. We encourage others in the pharma industry and beyond to support them in whatever way they can. 

For more information on Rare Disease Day, visit their dedicated website here: https://www.rarediseaseday.org/.