Being A Fortunate Son: Doing What I Can To Help With Orphan Drugs

In Orphan Drug Congress by Hugh Blackstaffe



It really is tough trying to find the best place to start tackling orphan drugs. There are so many players, payers, stakeholders, shareholders, biotechs, buy sides, sell sides, patients, policy makers, legislation shapers and every single one of those makes decisions that impacts the rest with tremendous repercussions, which can be positive and negative.

My role with orphan drugs, and rare diseases and the World Orphan Drug Congress puts me in a very fortunate position. By creating an event dedicated to tackling orphan drugs, with the intention of facilitating solutions to the challenges and hopefully getting things going to deliver treatments to the patients, because that is the ultimate end game. Getting people treated, and hopefully cured, is the goal for everyone involved in the orphan drug/rare disease world.

So what can I do? What can I do to help? I don’t have a rare disease or a multi-billion pound pharmaceutical company, I don’t have political influence or a scientific research lab…but what I do have is a job with one of the world leading Business to Business companies who created the first Orphan Drug event. An event which has lead the way in building bridges between all the stakeholders, putting the actual decision makers in the same room to make deals and shake hands and to actually make things happen. All I can do is build on this legacy, bring in my own ideas and try to make a difference.

I’m trying to make this years event as forward thinking as possible. I’m working to find the solutions to the big issues, the challenges that affect all stakeholders across the orphan drug and rare disease, with an eye on the future, gene therapies and new approaches to orphan drugs. It’s become clear that going down the same route, with the same techniques, tactics and strategies as have been used for Orphan drugs for years, often same routes for common disease drugs, just simply aren’t working. From an event point of view, having big and forward thinking topics, finding solutions and offering opportunities to delegates, is the best way to get people to attend. From a rare disease community point of view, being able to try and make a difference is hugely important. If there was one thing that has come out of my research, it’s this:

The need to engage with all stakeholders from the earliest possible stage.

Working with everyone seems to be the best way to overcome the challenges faced by the orphan drugs, to make the process as efficient and effective as possible. It takes a very long time for a therapy to be delivered, years at least, and this is a huge challenge for everyone involved, because there are lives at stake. It sounds dramatic, but it’s true.

So, with this in mind, this year, I am putting the idea of engaging with all stakeholders at the earliest stage at the core of my event. It’s one of the big headline acts. If there’s only one thing I can do with this event, it’s getting everyone to talk to each other, no matter their position/role in the rare disease world. Putting them in the same place, at the same time, will hopefully result in making a difference. It may only be a small thing, but I want to help as best I can.

Thanks for reading, please do have a look at the website for more information and have a read of my previous blogs here.

If you’d like to get in touch, feel free to send me an email at or tweet me @HBlackstaffe.