Actioning Intelligence: Trying to Create Solutions To Orphan Drug And Rare Disease Problems

In Orphan Drug Congress by Hugh Blackstaffe


Bob Dylan once sang “The Times They Are A-Changin'”, and whilst Ron Burgundy may have never heard that song, the rare disease community certainly has…and so have I. I am now beginning to transition to a new phase in the development of the World Orphan Drugs Congress, whereby I take ALL the information I’ve gathered from reading, emailing and telephone calling, put it on a big piece of paper and begin to break the prevalent topics down.

I know the areas I need to focus on, now all I have to do is find the details. It’s like knowing the correct section of the library, but are unsure of which book is most relevant to you.

Herein lies the next challenge. Getting the right details to make my event

A: Informative

B: Relevant

C: Progressively provocative

and from a commercial/sheer personal competitiveness point of view

D: Outclassing every other rare disease/orphan drug event on the market.

(I put that last bit in bold as I wanted it to read like I was bellowing it like Leonidas of Sparta)


This is essentially the stage that will make or break the event. I’ve got to sift through everything I’ve learnt over the past two months, find the key details, consider how they are commercially viable (so as to sell tickets) and all of the previously mentioned points of A-D…no pressure then.

The most satisfying thing is seeing all the little bits of information, all the little pieces of the puzzle, begin to fit together. It’s coming together on paper now, rather than my brain.

So, I have the information, it’s taken shape, so how does this affect the world of orphan drugs and rare diseases?

Well, I shall tell you.

The one big issue that keeps coming up again and again and again is the disparity between the various parts of the orphan drug chain. You have all these interlinked component parts, from biotechs to regulators to patients to pharmas, as well as the CMOs, CROs, HTAs and all the other acronyms you could think of who rely on each other to work with them efficiently and effectively, but sadly, as with most things in life, it doesn’t quite work out.

Now, I should point out to you, dear reader, that that is not a criticism. It is an observation. An observation I have made after looking at all the angles, stepping back, looking at all the angles again, going away to make some tea, then returning to look at them for a third time.

In fact, it’s practically a conclusion…a personal, observational conclusion if you will.

Now, we have identified the big issue, based on all the information. We have built up a picture of what we need to do, now we need to action it. I would say “Lets jump in the Terrapinn World Orphan Drugs Congress Batmobile and get’er done!” but sadly we don’t have one (yet) and honestly, it’s only a two seater, so we’d have to bunch up…put someone on the floor maybe…someone else goes in the boot (or ‘trunk’ for you American readers)…but I digress.

While I may not have a Batmobile (yet), what I do have is an event and an event with an agenda. These are two very powerful things to use to get people to talk to  each other, especially from a cross-sector perspective. Everyone who I have spoken to has identified the need for all stakeholder interaction, unifying perspectives and aligning goals to create a cohesive, comprehensive, united front to tackle rare diseases…which frankly is a massive task…hang on…going to need some coffee to tackle the next bit…

Right, I’m back…blast…spilt some…two secs…need to mop it up…eww…right, let’s try that again…

I want to bring everyone together in this event, all the sides, the groups, the factions, the representatives, and the organisations, because honestly, that’s what needs to be done. And to do this, I’m going to try and make the agenda as fair, balanced, inclusive and progressive as possible so that everyone can get involved. This years event has one eye firmly on the hear and now, and another on the future. Being able to deal with the immediate challenges by putting solutions into action right away means that we are very well positioned to take on the unforeseen future challenges that are emerging…which means another congress…which means I get to keep my job…It’s a win-win situation!

However, I am just a man (6 foot 2 and 13.5 stone of pure British handsome of course, but in the grand scheme of things, I am just a man), so what needs to happen if for everyone else to start pushing all the other parts to interact better, talk more, meet more and work closer. In the paraphrased words of Lord Kitchner “I Need You” to do your very best for each other. I’m creating the forum, the space and the event…now I need you to attend and start making a difference.

We’re all one big rare disease/orphan drug family, we all want the same thing, we just have to start working for the greater good, for the betterment of each other, putting aside the more self serving aims and start making some changes because if we don’t…we’ll be in trouble.

Thanks for reading

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Thanks again for reading


p.s. Sorry about the slightly ominous ending, but I felt it was needed.