Global Access Concerns

Page last updated: 14th November 2019

We are often contacted by families, and sometimes adults, understandably desperate for access to treatment. This may be a treatment that is available in many countries but not in theirs. It may be a treatment which is looking promising, but clinical trials are not yet completed.

Some families may have had access to one treatment but have now heard about another one and want their child to have the chance to try this other one as well. None of the treatments now licensed and available, nor any of those under development, are a cure for SMA. For all of them it is early days in terms of knowing the long run outcomes. There is still much uncertainty.

Q. We’re thinking about applying to access a treatment that is available in another country, have you any advice?

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

Talk to your 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. Would your own Centre still be able to offer you / your child the Standards of Care and support needed?

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 these would be met
  • How often you would need to attend and for how long
  • What follow up is required
  • Where you would stay
  • 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

Q. We’re thinking about applying to access a treatment and raising funds to do so, what suggestions do you have?

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 treat them and want to if they can do so safely. These include:

If a treatment is repeated or ongoing, how much money needs to be raised before treatment starts? Potentially treatment may be stopped abruptly if funding runs out. If a treatment is a ‘one off’, regular and close follow up may still be needed.

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. Using an untrialled / unapproved combination of treatments presents similar dilemmas.

For us as a registered UK charity, web-based fundraising mechanisms such as crowdfunding present 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, these options are potentially open to abuse.

When we’re asked to post a personal campaign, 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.

For these reasons we regret we do not promote these campaigns.

In our view, no family or adult should be in the position of having to find funds for treatment either through fundraising or their own private means. This is not how treatment should be allocated.

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. This may involve these bodies addressing issues of high price and uncertainty over outcome.  For example, this could be by the regulatory authority reimbursing the company over time, based on the results for an individual. Pharmaceutical companies need to consider their ethical position when it comes to provision of treatment in developing countries, ensuring there are schemes that will enable access.

Q. We’re thinking about applying to ‘swap’ treatments or access a second one ‘in combination’ with the current treatment, are there any issues with this?

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.

To date, there have been no clinical trials of combination treatments so the risks and possible outcomes are unknown.

Q. There’s a treatment that we’ve heard about but you haven’t been reporting on it. What do you think about us going for this?

A. It is important to be aware that there are unauthorised or unlicensed ‘clinics’ in various parts of the world offering unconventional treatments, usually for large sums of money. The internet contains sophisticated websites which look appealing but make unsubstantiated claims about alternative treatments. It is critical to get an objective view from your clinical team before contacting such clinics or considering travelling abroad.