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Information for People Living Outside the UK

Information for People Living Outside the UK

Page last updated: 31st March 2022

As a small UK charity, we regret we do not have the capacity to respond personally to individual enquiries when the person who has SMA is not resident in the UK. We hope the information on this page may answer some of your questions and be helpful to you.

A. These sections are probably most useful but please bear in mind they are written for people living in the UK with access to UK health services:

About SMA >

Recently Diagnosed with SMA >

A. These sections are probably most useful but please bear in mind they are written for people living in the UK with access to UK health services:

Nusinersen (SPINRAZA™) >

Risdiplam (EVRYSDI™) >

Onasemnogene Abeparvovec (ZOLGENSMA™) >

A. To get free NHS healthcare in England your child will need to be "ordinarily resident" in the UK. This means you must be living in the UK on a lawful and properly settled basis for the time being. You may be asked for evidence of this.

For a detailed definition of what being ordinarily resident means, see the GOV.UK guidance.

You / your child will also have to meet the eligibility criteria for the treatment. These criteria vary between treatments and between the UK countries – England, Scotland, Wales and Northern Ireland. You can find out more in this pages:

Nusinersen (SPINRAZA™) >

Risdiplam (EVRYSDI™) >

Onasemnogene Abeparvovec (ZOLGENSMA™) >

If you are considering a move to the UK, you can find further advice in these pages:

Moving to England from EU countries or Norway, Iceland, Liechtenstein or Switzerland >

Moving to England from outside the European Economic Area (EEA) >

A. There are lots of considerations. We suggest you would need to:

Talk to your / your child’s medical team about:

  • The clinical trial results and what they suggest might be the outcome for you / your child
  • Your health / the health of your child and any risks of travel for treatment
  • Their view of how close you should be to the treating Centre and for how long this should be, in case of complications arising from the treatment
  • Any difficulties there might be with having a treatment provided in one place and ongoing care in another. For example, would your own Centre still be able to offer you / your child the Standards of Care (scroll down on this page) and support needed?
  • If they are aware of where in the world you / your child may be able to access treatment

Talk to the overseas centre you are considering about all of the above plus:

  • Their eligibility criteria and referral process – how they would assess you / your child to see if you / your child are eligible for treament
  • How often you would need to attend and for how long treatment would continue
  • What follow up is required at the overseas centre
  • Where you would stay during and after treatment
  • How much it would all cost – the treatment, the travel, accommodation and whether any of this would be funded by the overseas centre

We suggest you consider and talk to your family / support network about:

  • Travel insurance to cover your child’s / your and any travelling companions’ health and other needs
  • Managing this commitment practically, emotionally and financially – perhaps on an ongoing basis

​A. The International Standards of Care (SoC) for SMA describe the intervention and care recommended for best quality of life. Many families find it helpful to discuss these with their medical team.

You can download a copy through the link here. If you scroll down the page you’ll come to a section of flags which link to the Family Care Guides in different languages.

A. Patient groups for countries in Europe are listed on the SMA Europe website. You will find other groups listed on the TREAT-NMD website – scroll right down to the bottom to the section ‘Patient Organisations’.

A. We understand people’s desperation, but we are concerned by the uncertainties and pressures this potentially creates for families and adults and for the clinicians who are asked to and want to treat them. Things to consider include:

  • If a treatment is repeated or ongoing:
    • How much is needed for it to start and then continue
    • What happens if money runs out and it then stops?
  • If a treatment is a ‘one off’:
    • regular and close follow up may still be needed.
  • In addition to the actual cost of treatment, you will need to be able to over other costs such as:
    • travel
    • food and accommodation
    • travel insurance to cover your child’s / your and any travelling companions’ health and other needs,
    • unexpected delays or extension of time away

A. Sorry, but we don’t know of any UK charities that are able to help families from overseas with fundraising for treatment

For the following reasons we regret we cannot help with or promote any personal fundraising campaigns:

  • In our view, no family or adult should have to find funds for treatment either through fundraising or their own private means. This is not how treatment should be allocated.
  • For us as a registered UK charity, web-based fundraising, such as crowdfunding, presents potential issues:
    • Though most people are genuine and will later post evidence of the outcome of their campaign, web-based platforms have no requirement for this and there’s no accountability to the people who’ve made donations as to how the money is actually used. Sadly, this means these options are potentially open to abuse.
    • We don’t have the resources to undertake these very important checks. Nor would we wish to judge which campaigns are ‘worthy’ and which less so.

 

  • In our view, regulatory authorities and pharmaceutical companies need to find ways for treatments to reach all those in need for whom there is clinical evidence that this is an appropriate treatment.

​A. These pharmaceutical companies can be contacted by email:

 

 

A. Talk to your medical team about whether this is a possibility and, if it is, how this should be managed and what risks this may pose for you / your child.

A. There are unauthorised or unlicensed ‘clinics’ in various parts of the world offering unconventional treatments, usually for large sums of money. They may have a website that looks good but the claims they make about a treatment have not been proved. Always talk to your clinical team before going any further.

If a clinician is asked to treat a patient ‘off label’, for example a patient with a clinical diagnosis of an SMA Type for which there is no licensing authority, they are potentially liable if something goes wrong.

Clinicians are also potentially liable if they use an untrialled / unapproved combination of treatments and something goes wrong.

​A. You can search for globally registered trials by drug treatment or condition name via this international website.

Patient registries that collect data from patients with SMA sometimes share information about clinical trials. If your country has a national SMA Patient Registry, you may wish to sign up here.