Navigating the tides of change

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“Charity CEO working in the rare disease space for just under a decade after many years in the housing and children’s policy sectors. Experience of rare disease in family. Very much a generalist rather than having one specific area of expertise as needed in the broad role of CEO.“

What is front of mind in your industry currently?

‘Money, money, money’…. I speak with a lot of leaders of patient support and advocacy organisations and a lot of industry, and everyone is talking about money. As the famous song from Cabaret states, money truly makes the world go round.

It sound both simplistic and a little needy to say that patient organisations need money to do our work, but it is true. Whatever we do, from family fun days to attending meetings to having websites, it is very unusual for anything connected to a group not to have some cost attached. At the very least, we need insurance and to pay the Information Commission’s Office our subs.

But it’s not only money for our organisations that is on my mind and being discussed a lot. It is money within the medicines industry—and not being too political here—the UK and other policies that are impacting investments on research and headcount in the UK and how that is affecting product launches that is being talked about. It is concerning to anyone in this space.

Lastly, the money for rare within the healthcare system is hugely topical. Will any new iteration of the Rare Diseases Framework come with budget? What about the NICE HST thresholds? How can cost savings created by innovative treatments or faster diagnosis have a full system benefit?

It’s all to do with money at the moment.

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

‘Ch-ch-changes’ are constant and a massive challenge to patient organisations, as the late, great David Bowie might have said. We know that the system needs to change. We hope that it will change to the benefit of our community. We worry that it won’t. But the number of changes all at the same time with the corresponding number of consultations and the need for us to make the case for our community and our loved ones, frankly, it’s a little overwhelming at the moment.

In a way, once the new system is in place, all the consultations are closed and responses submitted, then things will be less frenetic. Although, the chance to shape things is so welcome because the time is now to get it right. What happens now will set the pattern for what happens for our community for not only 10 years but potentially decades to come.

Thinking like that is quite a responsibility, quite a pressure, particularly for the very small volunteer-run organisations I speak with. Lack of capacity is a pain point for so many of us.

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

There are so, so many opportunities available to patient organisations as long as we ‘Don’t stop believing’.

I do believe that, if we have an inch of bandwidth to participate, influencing the multitude of current policy changes could have a positive impact on the people living with rare disease and their families.

I do believe that there are technological advances that could help our community and, honestly, could help our organisations. Maybe this will be seen in the form of speedier diagnostics or more diseases being identified so they can be researched. Maybe this will be new treatments that our community is given the choice to receive, or not, as they want. Maybe this will be helping patient organisations achieve more with the less we have currently.

I do believe there is more patient organisations can achieve together. We are rightly experts in our own disease areas and communities, but there are commonalities and we are not using those to the best advantage of our communities yet.

We just have to remember that we deal in hope. I believe that tomorrow will be a better day.

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

There is one change I think would alter the way that people living with rare disease and their families experience life, not only in the health service but across all their lives, and that is for the lives of our community to be understood in the same way as anyone else’s lives.

At the moment, we aren’t really understood by people without direct experience of a rare disease themselves. We get caught up in having to re-explain our lives repeatedly. We have to battle for a diagnosis. We have to search for information. We have to give evidence to social care teams, teachers, employers and healthcare professionals. We have to explain why things are significant to us that might not be significant to others including the healthcare system.

Could you imagine the impact on lives if we were just understood?

‘Is this just fantasy?’ Maybe at the moment, but I hope not forever.

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

There are many events to be ‘Steppin’ out’ to! I could be out every week, sometimes every day. There are some fixtures that go straight into the calendar for the next year, as the current year’s event ends, and those are PPP Genomics conference, the NHSE Genomics summit, the Festival of Genomics and Biodata, and Advanced Therapies.

Why these events? I have limited time and it’s useful to have all the people I want to see, hear or speak with in the same places at the same time. Plus, this combination tends to give me different perspectives on similar topics so the thinking I take back to the office is well-rounded.

incognito provides a candid look behind corporate closed doors in the RARE disease world.
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