Constructive, Critical, Creative: Revealing The Content of the World Orphan Drug Congress 2014

In Orphan Drug Congress by Hugh Blackstaffe

Oh Ladies and Gentlemen, I cannot express just how excited I am to be able to write this blog post. It’s been over a week now since my last one, and that has mainly been due to everything with the event building up at a steady rate of about 65 miles an hour, then discovering it has to be taken up to 100 miles an hour for the next few weeks, balanced with the arrival of Easter (the only Christian celebration still subject to the state of the moon…just a little fun fact for you there) and my first major international business trip (more than just a soggy day in Brussels) over to Washington DC to attend our sister event. So it’s going to be 100mph for a good 12 days, hence the lateness of my blog…and is likely to be my last one till May (sorry).

I am proud to say that the agenda for the World Orphan Drug Congress is rapidly approaching the stage of finalisation. And my-oh-my is it a good’un. I’m very proud to say that we have representatives from the European Commission, multiple European governments (British, Belgian, German, Italian, French currently), highly respected academics, industry leaders such as EuropaBio, and exceptional patient alliances including the AKU Society and Findacure, SCAD Alliance, Ben’s FriendsRare Disease UK, CORD, and the Croatian Alliance for Rare Diseases. We also have health care providers, established journalists and research organisations. And I am very proud to say we have the might and power of the biggest pharmaceutical, biotech and research companies in the sector, including (in slightly particular order): Shire, Alexion, Pfizer, NPS, Clinigen, ProMetic, Quintiles, Oxford PharmaGenesis, Ergomed and PSR Agility, Moorfields, Durbin, and Cote Orphan.

So, it’s quite clear that we are certainly working with the biggest and best…I’d expect nothing less for the event. And if you’d like to come along and engage with all of these organisations, and many many more, you can sign up here.

But enough of this shameless sales pitching. Let’s get stuck into what these people will be talking about (though only to a certain extent, as the agenda is not yet fully confirmed) and what is on offer for our potential delegates.

We’ve got presentations from the European Commission on the future of R&D with the rise of Horizon 2020, we have a panel discussion featuring the heads of several of the biggest Health Technology Assessment organisations in Europe, and we have keynote speeches from the world leading pharmaceutical companies. There is a truly epic line up, with more speakers being added each day. The aim of this year’s Congress is to present a balanced, indepth agenda, that appeals to the entire orphan drug sector. It is designed to be interactive and thought inducing. The content is focused on tackling the immediate and future issues in the sector. It’s dealing with the big problems of working in such a complex and diverse market as the Euro zone is, as well as further afield, across Eurasia and around the world.

The agenda is almost ready to be fully released in May, but as it stands, we have topics such as genetic medicine in rare diseases, integrating all stakeholders, developing patient networks digitally,patient driven research, funding and clinical studies, dealing with the challenges facing the European biotech, examining the state of Orphan Drugs in Eastern and South Eastern Europe, and developing new and effective appraisal process for expensive orphan medicines. And that’s less than half of the total agenda. There’s still so much more to come.

Following some criticism from last year’s Congress, I took it upon myself to build an agenda that moved away from the ‘pay to play’ and ‘corporate showcase’ accusations, into a fairer and better balanced event. Orphan drugs has so many players, that giving preference to one over the rest doesn’t achieve anything…but of course, one must remember that having the big names enables us to deliver the Congress every year. Without their partnership, we would not be able to assist patient groups attending the event, nor would we be be able to put on the Congress at all!

I’ve got a tough few weeks ahead of me…actually, less than a few…more like one and a bit. I will try and blog more as and when I can, but being as up against it as I am, it may be a while.


Thanks for reading. For more information, please do visit the website, or feel free to email me at or tweet me @HBlackstaffe.


This week, I wrote the blog listening to the full soundtrack of ‘Lord of the Rings’ (Peter Jackson, 2010, 2011, 2013)