What is the petition about?
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 time, it looked like tofersen would not be available on the NHS. The National Institute for Health and Care Excellence (NICE) is the body that decided which treatments the NHS would fund. Earlier this year NICE had decided not to evaluate tofersen using the approach that gave it the best chance of being approved, saying MND wasn’t rare enough to qualify.
Yet NICE’s own guidelines said it could make exceptions to the rule, and we believed tofersen was worth making an exception for.
Based on this, in October we launched a petition calling on NICE to reconsider and adopt the right approach (called the Highly Specialised Technology or HST route) to appraising tofersen, so that it had the best chance of being made available to patients on the NHS.
Our Director of Engagement, Richard Evans, explained the situation at the start of the campaign in more detail.
What is motor neurone disease (MND)?
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.
How will signing the petition make a difference?
Your signature on this petition joined more than 15,000 other names to show NICE that this issue mattered hugely – to the MND community and beyond.
The campaign was also covered in the media and championed on social media, and throughout we continued to talk with NICE and other decision-makers to make our case.
We hoped that NICE would see both the strength of public opinion and the justice of our position. We are delighted that it has now accepted that tofersen is worth making an exception to its normal criteria for, giving it a better chance of becoming available on the NHS.
What is NICE and what does it do?
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 disagreed with their position on tofersen.
Can my MP do anything?
NICE is a public body, but it is not a government department and acts independently of government.
The role of MPs in this situation was therefore limited. But we have asked them to share their support for the issue and the petition on social media, and we briefed them on tofersen at the meeting of the All-Party Parliamentary Group on MND in November 2024.
We will continue to keep MPs updated on progress. If you have a good relationship with your MP, contact us at [email protected] or 02038758910, and we can provide you with a briefing to share with them.
I have MND. Can I take tofersen?
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.
I have SOD1 MND. Can I take tofersen?
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.
I have SOD1-type MND. If tofersen won’t be available on the NHS, can I access it privately?
No. Biogen has not applied for marketing authorisation in the UK, meaning tofersen can’t be prescribed privately.
If I can’t access tofersen privately in the UK, could I go abroad to get it?
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.
What about the MIROCALS trial?
MIROCALS is completely separate from tofersen. Read more about the MIROCALS trial here along with our September 2024 update.
I have another question
Please get in touch with the Campaigns Team on [email protected] or 02038758910 and we will do our best to help.
Click here for a 1-minute summary on tofersen
As the campaign goes forward, we'll try and answer as many of your questions as we can here.