Where Does The Power Truly Lie In Orphan Drugs And Rare Diseases?

In Orphan Drug Congress by Hugh Blackstaffe


With my event, it is my intention to attract the people who can make a difference. I have said before that my goal is to construct an event that can literally make a change the world of orphan drugs and rare diseases, putting the best people in the right place to start making a difference to peoples lives. I want to secure the best possible speakers to present to the best possible delegates to then go and turning ideas into actions and make a

It is with this thought, and a question that has been building up in my mind over the last few weeks of research calls, meetings and emails, that I write this blog. So, let us begin…


Where does the power lie in orphan drugs and rare diseases? Who has the ability to change everything?


There are so many links in the chain, each link intersecting and interacting with the other rest from a global level to a local level, and therefore it’s difficult to get a handle on where the true power lies.

Each link certainly has the capability to affect each of the other links to a certain extent. Though which, out of all of them, has the ability to affect them all significantly? Which one can cause great change and significant progress?

There are so many issues and factors to consider here. The diversity of roles within the sector shows that there is no clearly defined ‘executive power’, no one section has a majority of influence over others, instead the power lies in the collective. Each section brings its own pressures, objectives, focuses, goals, financial clout (or lack there of), agenda and history, and from there derives the power.

However, is that power being wielded correctly? Is what’s being done the right way of doing it? Is each link working in the way that is best for orphan drugs? And If not, how do we go about making it work as well as possible?

This is all quite high minded stuff, if I’m to be honest. I’m sitting here in my office in London typing away, briefly looking out the window to ponder a point, feeling a little under the weather and I’m trying to wrestle with the concept of power, geopolitics, the corporate world, whether man kind can truly change and evolve and how to change the world for the better…and my headache really isn’t helping matters.

We are dealing with a multibillion pound industry that has hundreds of thousands of lives at stake, spanning every country of the globe, working with tiny populations of people with rare diseases, navigating through the legislative, political, regulatory and financial complexities of the 196 countries in the world (I counted…via Google) in order to treat those tiny populations of people for their respective diseases…whilst still making enough money to perform that service over several years, as well as their others. And they have to operate from a global level down to the most local. So who has the power to make changes to make every single part of the process

There are a lot of stakeholders involved in every facet of the sector, and there are literally lives at stake. And whilst several elements of the system are working, there are sections lagging behind, parts that aren’t functioning as well as they should be, but who is going to step in and make them work? Which link. group, organisation or person is going to lead the charge to bring about significant sweeping changes, kick down some doors and bring about the innovation revolution?

There are many factors at play, many moving parts, many links in the orphan drug chain…so how is it going to start working better? Functioning with greater efficiency, speed and accuracy? This is a hugely important challenge for the sector to overcome and solve, and it involves everyone, because that is where the power lies.


Thanks for reading, please visit the website for more information and feel free to email hugh.blackstaffe@terrapinn.com or follow me on Twitter @HBlackstaffe.


This blog was written predominately to three computer game soundtracks: ‘Skyrim’, ‘Assassins Creed: Black Flag’, and ‘Fable 3’.