An update on our work regarding Tofersen

We’re using this opportunity to share an update on the action we’re taking following the National Institute for Health and Care Excellence (NICE) decision about its appraisal of the drug tofersen.

Tofersen, developed by Biogen, has been shown to slow the progression of symptoms in people living with MND who have alterations in the SOD1 gene. This is approximately 2% of the MND population, estimated to be between 60-100 people in the UK.

Ordinarily, products tailored towards rare diseases that affect less than 1 in 50,000 people in England are appraised through the Highly Specialised Technology (HST) route.

Back in March, NICE confirmed it would assess the drug through the Single Technology Appraisal (STA) route instead. We believe this decision is wrong and will prevent tofersen from being made available to people with SOD1 MND.

NICE agreed to speak with members of our team but, during that meeting, officials reiterated to us they would not change their decision despite extensive representations in recent weeks from people with MND, the MND Association, MND Scotland, My Name’5 Doddie Foundation and leading clinical experts.

Following that discussion, we have sought clarity on issues raised, and are waiting for a further response from NICE.  

In collaboration with other UK MND charities, we continue to engage with Biogen, the company that manufactures tofersen, about its next steps. We have a meeting with them in the coming weeks.

We have also written to the Secretary of State for Health and Social Care, Victoria Atkins MP, expressing our dismay at this decision.  

And we are continuing to work with the media to highlight the stories of those people this decision affects, who have generously agreed to share their experiences.

While we recognise NICE is an independent regulator, we are seeking meetings with Government representatives to discuss this situation.  

Although our immediate focus is on this incredibly frustrating position regarding tofersen, we also believe the decision by NICE goes against the Government’s ambition to lead the world in precision medicine and its vision for the UK being one of the ‘best locations to develop and commercialise genomics technologies’.  

It sets a worrying example for future precision medicines entering the UK market – both in treatment for MND and other rare diseases.

As always, we remain committed to ensuring that people with MND gain access to proven, effective treatments.