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The NHS proposals for rare disease care - what's next?

Coordination, an alert card and effective transition from paediatric to adult care for those with rare diseases, along with better access to research - promises given by Conservative MP Nicola Blackwood on Rare Diseases day at the end of February this year. What does this mean for people in the UK with superficial siderosis and how might these developments have a more global impact?

I was invited to join a webinar with Rare Disease UK on Thursday 14th, hosted by Evan Wroe from Rare Disease UK and Fiona Marley, Head of Specialist Technology Commissioning for NHS England. I was joined online by a number of rare disease organisations and Fiona Marley spoke about how she intends to implement the proposals.

The Silent Bleed - our position

Considering that the UK’s strategy’s aims are to:

  • Facilitate earlier diagnosis and intervention

  • Improve care coordination

  • Promote research

...many Silent Bleed members in the UK are faced with inconsistency in care, doctors who are unaware of research and developments in chelation therapy, lack of awareness when accessing primary and emergency care and contraindications across therapies and medication.

So, consistency of provision, awareness and access to research are our priorities.

For further Silent Bleed discussion of the UK Rare Disease Strategy, see here.

What are the next steps?

Alert Card

There is some uncertainty over the form it will take and how long it will take to roll out:

  • The aim is to prevent contraindications and keep patients safe, the focus being A&E care but it may have other applications such as when seeing a locum GP, for example.

  • A physical card but with an electronic link (to say an online database) to carry the sheer weight of information needed for effective care.

  • A consultation with the Royal College of Emergency Medicine raised the issue of how readily accessible information needs to be in order to treat someone in an emergency situation, so the card would also need to be substantial enough to carry physically printed details, available in an instant.

  • Authenticity is an issue – there have been instances where patients have made up a condition in order to access narcotics.

  • Awareness is also important – making sure that providers know to look for the card and know how to use it.

  • There would be an easily recognisable logo to prevent fraud and aid visibility.

Coordinated care

The appointment of someone with oversight and overall responsibility for linked-up care across an array of disciplines (hearing, sight, neurology, medicines and pain management, for example):

  • Will be a member of medical staff already involved with treating the condition (either a specialist doctor or nurse).

  • Where a condition has a centre for excellence, the centre will organise the appointment.

  • There was some uncertainty for those without a centre for excellence (for example, patients with superficial siderosis). Will their own neurology department be responsible for making an appointment? Who knows?


The subject of research came up in the webinar, particularly how to make it easier to access information about trials.

Devolved administrations

There was some discussion on how these proposals would roll out across NHS Wales, Scotland and Northern Ireland as well. They are considered self-contained services, in terms of NHS provision and there was no clarity on how the roll-out would happen, save that it would.

The Silent Bleed – our response

As previously mentioned, we're already concerned about inconsistencies in care across the UK from area to area and consultant to consultant.

It's also important to consider that there is, as far as we know, no formal medical research into superficial siderosis in the UK. We've been supporting Dr Michael Levy's drug research at Johns Hopkins in Baltimore for years but are there other areas that UK institutions could be looking at such as cause, prevention and better for support for those who are unable to withstand the physical demands of treatment.

The core concepts behind the proposals are consistency, safety and progress but how fair is it that some patients will still receive better provision than others, depending on where they live? Is that not also an area of consistency that needs addressing?

Would outcomes for superficial siderosis patients be better if there was a centre for excellence?

How will other rare disease organisations fare under the new proposals?

Global implications

In seeking to become a beacon for rare disease provision within a universal healthcare system, NHS England have made mighty promises, for which they should made accountable. For other nations with a universal healthcare system, what lessons can be learned in terms of cost and availability? For those countries with a less centralised system, how do providers ensure safe emergency care, proper access to research and coordination?

What’s next for the Silent Bleed?

For a tiny charity, we have big dreams. We have a number of projects underway this year, including research on navigating UK rare disease policy, keeping our members updated, supporting medical research and stronger links with other organisations such as Rare Disease UK.

We will keep fighting to make sure our voice is heard.

The Silent Bleed is a UK registered charity caring for the needs of people with the rare, neurological condition, superficial siderosis. Like other rare disease groups, we're a small organisation determined to improve outcomes despite being few and far between. Join us.

Further reading:

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