One Small Step and One Giant Leap: Getting to Grips with Orphan Drugs

In Orphan Drug Congress by Hugh Blackstaffe

For someone who doesn’t come from a science background, plunging into the immense world of orphan drugs, rare diseases and the combination of chemistry and biology that totally stumped me at school was always going to be a real challenge. However, there is one key thing that helped me take that leap and dive head first into the vast sphere of Biotechs, CRO’s, Pharma, Patient Alliances. The work the orphan drug sector does saves lives and gives people hope.

I’ve only recently been inducted into the world of orphan drugs as my new role as Conference Manager for the World Orphan Drugs Congress 2014 and I have to be honest, it’s utterly fascinating and inspiring. The work that is done across the sector by impassioned people all working for the same aim, to cure the diseases, is remarkable and should be championed even more. It’s fired up my curiosity more than I could have imagined, and what’s really helped that is interacting with people who highly passionate about the work that they do.

My current task is constructing the agenda for the World Orphan Drugs Congress 2014, a task which I’m sure you understand is vital to not only the event, but the industry as a whole. As the pioneering international orphan drug event, the weight of responsibility to deliver and excel is great, but it is a weight that I take on eagerly. My aim is to build on the previous events and make it as progressive, beneficial, advisory, informative and forward thinking as possible.

I had a great chat with Nick Sireau, Chairman of the A.K.U. Society ( and founder of Findacure ( yesterday, who spoke at last year’s conference and gave me a wealth of great information and spoke more about the work his new organisation is working to understand both rare and common diseases, and as it’s a non-profit organisation, all the money donated goes straight to the research and development. Here is a fantastic example of work that benefits everybody, creating a united front against diseases that affect not only tens of people, but diseases that affect tens of thousands as well. It’s about improving everybody’s life, from the rarest of the rare to the commonest of the common.

I’m taking this concept as I develop my event. I want it to help not only the attending delegates and speakers though my agenda and networking opportunities, but the global community of rare disease patients and advocates a whole. It’s about being relevant without being inaccessibly niche and overly specific to a single group. In this every connected world of ours, fueled by social media and digital networking, accessibility and information sharing is paramount to ensuring progress. Keeping abreast of day to day news and staying on top of the ebb and flow of the orphan drug sector is vital to ensuring success. It’s what keeps the sector going and keeps it fresh in the minds of the people who can make a difference, from those at the top of governments to the generous donators to charity. We are increasingly becoming a global community, and while progress in some areas is rapid and advanced, others are lacking behind and as a community, we need to speed them up. Whilst it can take years for drugs to be developed and marketed, we must look at the present and plan for the future. Accurately predicting what’s round the corner and being prepared for it by building the tools and means now so as to tackle it is the best approach, and my aim with the World Orphan Drug Congress is to look at the present with a simultaneous firm grasp of the future.

If you’d like, please do have a look at the event website. I’m aiming to blog every week, so if there are any current issues, challenges or ideas you’d like to talk about, feel free to get in touch. If you are so inclined, you can follow me on Twitter @HBlackstaffe or email me at

Thanks for reading,