Prescribe Life - frequently asked questions

Tofersen is a ground-breaking new treatment for a small proportion of people with motor neurone disease (MND), a devastating and life-limiting neurological condition. In the UK there is currently only one treatment available (riluzole) which extends life by a matter of months.

But at the moment it looks like tofersen will not be available on the NHS. The National Institute for Health and Care Excellence (NICE) is the body which decides which treatments the NHS will fund. NICE has decided not to evaluate tofersen using the approach that gives it the best chance of being approved, saying MND isn’t rare enough to qualify.

Yet NICE’s own guidelines say it can make exceptions to the rule, and tofersen is worth making an exception for.

Our Director of Engagement Richard Evans explains the situation in more detail.

Sign the petition. 

MND is a fatal, rapidly progressing disease that affects the brain and spinal cord. It kills a third of people within a year of diagnosis and more than half within two years. MND leaves people locked in a failing body, unable to move, talk and eventually breathe. Six people every day die from MND in the UK. Another six are diagnosed.

There is no cure. 

More information on MND.

Your signature on this petition will join with thousands of other names to show NICE that this issue matters hugely – to the MND community and beyond.

We hope that NICE will see the strength of public opinion and will accept that tofersen is worth making an exception to its normal criteria for, giving it a better chance of becoming available on the NHS.

We will be presenting the petition to the decision-makers at NICE in the new year in the hope of influencing their thinking.

The National Institute for Health and Care Excellence (NICE) is an independent public body which helps to get patients the best care, while ensuring value for money for the taxpayer.

One of its roles is to assess new treatments for diseases and decide whether to make them available on the NHS for people who could benefit from them.

We have worked well with NICE on other projects – they are, for example, responsible for the clinical guidelines for health and social professionals on MND care.

But we disagree with their position on tofersen.

Please share the petition on your social media and in your networks to encourage others to sign. This is even easier if you sign the petition yourself – you are then taken to a page to help you share the petition via Facebook, Twitter, WhatsApp and LinkedIn.

A resource page will be available to help with this shortly too.

If you have a good relationship with your MP, we can support you to raise the issue with them (see next question).

NICE is a public body but it is not a government department and acts independently of government.

The role of MPs in this situation is therefore limited. But we will be asking them to share their support for the issue and the petition on social media, and we will be briefing them on tofersen at the meeting of the All-Party Parliamentary Group on MND in November 2024.

As a supporter, the most important thing you can do with your MP right now is to invite them to the APPG so they can hear about tofersen and our campaign.

If you also have a good relationship with your MP, contact us on [email protected] or 02038758910 and we can provide you with a briefing to share with them.

Tofersen is only effective for people who have a type of MND caused by a variation in a gene called SOD1, which is about 2% of people with MND. The only way to find out if you have a gene change, such as SOD1, associated with MND is to have genetic testing. 

If you are interested in this, please talk to your GP or neurologist. Deciding to have genetic testing can be difficult, as it can impact family members, as well as yourself.

Therefore, we always recommend having genetic counselling before you decide to have genetic testing. The counsellor will help explain the possible outcomes and impacts, to help you choose whether to go ahead. 

For more information, please read our booklet on inherited MND and genetic testing. 

If you have SOD1-type MND and are not receiving tofersen, ask your clinician to refer you to the Early Access Programme (EAP). Biogen, the pharmaceutical company that makes tofersen, is currently funding access to tofersen for SOD1 patients through the EAP.

No. Biogen has not applied for marketing authorisation in the UK, meaning tofersen can’t be prescribed privately.

Tofersen has been authorised in the EU and US. Please speak to your GP or neurologist in the first instance. We are unable to provide medical advice.

MIROCALS is completely separate from tofersen. Read more about the MIROCALS trial here along with our September 2024 update.

Please get in touch with the Campaigns Team on [email protected] or 02038758910 and we will do our best to help.