24 July 2024 News

The introduction of a new Government means we are now working hard to engage with new Members of Parliament, with our work around access to tofersen a priority for discussion. 

In addition, plans to launch a public campaign to raise awareness of what we see as an unjust decision by NICE are now underway. This is in addition to the conversations and meetings we are continuing to have with representatives of the National Institute for Health and Care Excellence (NICE) and Biogen, the pharmaceutical company responsible for developing tofersen. 

As an Association, we will do everything we can to make the case for a different approach to appraising tofersen. 

As a reminder, tofersen has been shown in clinical trials 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.  

Leading MND clinical and research experts have called for tofersen to be appraised by (NICE) through its Highly Specialised Technology (HST) route, which is designed for treatments aimed at very small target populations. In March 2024, 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. We have worked with leading clinicians and research experts to make this argument to NICE directly but, frustratingly, NICE has not changed its position.  

We are pleased Biogen’s early access programme for tofersen currently remains open to people with SOD1 MND. If you have SOD1 MND, are not currently taking tofersen but would like to, please speak to your consultant in the first instance.