Ramblings of a CEO and rare dad

Estimated reading time: 7 minutes

“I have served a 25-year apprenticeship as a “rare dad”, and I am the group CEO of one of the world most respected rare patient organisations. We provide face-to-face advocacy and support, as well as expertise in heath technology assessments especially in the highly specialised area and advise globally on how to optimise the pathway for patients to receive new and innovative therapies. I don’t just work in rare, I live it.”

What is front of mind in your business currently?

There are always many areas in rare constantly on my mind—not just within the organisation but within the global environment for rare diseases.

Living a rare life better:

Having spent 25 years on this journey I know both the loneliness and despair it can bring but also the moments of sheer joy and sense of community. I am always thinking about this and how our organisation can help to make this journey easier.

We are an organisation that does real patient advocacy directly with families. We have a team that will visit the families, give school and GP training, challenge benefit decisions, provide whole family psychological support and provide events and functions where our community can come together and just be who they are. It is nothing more difficult than that—but I do believe that you have to live this life to fully understand what is needed.

Addressing inequalities within healthcare systems:

This is a complex area that has both similarities and differences depending on whether I am addressing global or UK specific problems.

In the UK we have a broken system built upon a fundamentally flawed equation when applied to rare diseases, namely the Quality Adjusted Life Years model (QALY). This is one of the most discriminatory models towards a population that was every thought up. Why should a person suffer through their life just because of a fate of being born with a rare condition?

The QALY manages to do this on a spectacular scale and treatments that are life altering are being refused by NICE on rationale of cost alone. Now, whilst not exact, it is true enough to say that most new medicines cost roughly the same to develop. The cost is divided by the patient population therefore, with rare and ultra-rare disease where you have tiny patient populations the price is exponentially more expensive. However, unlike many other treatments that have been developed, these treatments are transformative and over a lifetime. This is not fully captured in the modelling and leads to discrimination based upon cost and a tiny population. Some countries now are recognising this and beginning to move away from this model.

I do not have enough space to talk about newborn screening other than to say in the UK it is appalling. In Europe more than 20 countries screen for more than 20 conditions where here in the UK we screen for just nine. Our approach is doing harm, and the system must change. Other countries have left us behind and the gatekeepers in the UK need to make way for a new approach.

There are other areas such as NHS capacity and biomarkers as accepted endpoints in clinical trials but that is for another day…

Another part of my role is being plugged into as many parts of the global system as I can be so that I can take both a high-level view and when needed a more in-depth scrutiny of the in-country issues. This is why I attend so many meetings—it gives me more pieces of the puzzle and a greater understanding of the challenges.

The third area is all around patient organisations and finance:

When I first came into this arena I was staggered by the amount of politics that exists in patient organisations. Some of this is because we are now forced to compete for financial resources but some of it is ego and intransigence. Now I am not good at politics and frankly there is no place for it within patient organisations and yet it persists. My organisation has suffered directly as a result of this, and so too have patients. I appeal to everyone to stop this and to think solely of the communities they serve.

Finally, there is the financial questions all charities are facing. So many charities are using reserves to survive and there needs to be consolidation.  Having come from a commercial background I recognise this and am actively seeking collaboration between organisations with similar principals.

One of the most fun things that I like to do and that is important is giving lectures and talks globally. It gives me an opportunity to interact with other CEOs and the shop floor, in a way that would otherwise be impossible. Only by being as involved as I am, can I make more educated decisions that keep the organisation at the forefront of everything and ensure our sustainability.

I don’t have a job it’s a way of life—a rare life!

What are the greatest challenges you face as a CEO, what are your pain points and barriers to overcome?

Finance is always a big issue.

All charities are facing the most challenging set of circumstances in modern times.  We have a cost-of-living crisis, an increased tax burden, increasing demand on our services and more-on-more compliance which requires investment in infrastructure. I am increasingly horizon scanning for the next issue heading towards us and preparing the organisation.

This all leads to the even bigger issue and that is time!

There are simply not enough hours in the day to address the everyday problems and so time is our most finite resource. My family sometimes only see me for a couple of hours a week or even less when I am travelling. All I can say is thank goodness that we now have online conferencing so that I can at least see my family virtually through the screen.

What are the greatest opportunities yet to be realised or on the horizon for your business?

I will say that there are two from my perspective:

AI:

AI will revolutionise things in ways we cannot even comprehend. For instance, it could completely change the diagnostic odyssey of patients, by, for the first time enabling HCPs to diagnose via software that identifies likely diagnoses via complex algorithms. This is not science fiction but almost science fact and this is only made possible by AI.

Digital and social communities:

Now, some may say that this is already happening, but in rare not so much. We are only scratching the surface of what is possible. I have just had a podcast studio built at our offices and it is our intention to give our young adults a greater voice. They know how to reach out and break down traditional barriers, get to a larger and more influential audience and tap into new revenue streams. It comes with dangers, but my attitude is, so does a rare life so let’s embrace them.

If you could create overnight change, what would it be?

I would have a UK Rare Disease Task Force that took a small group of diseases and was allowed to show what “good” actually looks like. We could treat more people, more quickly, saving money and other resources and patients would be happier.

For this to work we need more individuals involved that have walked through and experienced the system and not purely the policy people.

Just look at the Rare Disease Framework and Action Plans. What have they actually achieved for patients? Not a lot, and partly because it’s the same old people involved that do not challenge the system.

What is the next ‘go-to’ event in your diary and why?

It’s actually a lecture to a CRO about living a rare life both as a dad and as a group CEO.

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