Tofersen access update

We want to share an update on work we have been doing to improve access to the drug tofersen across the UK, for people living with SOD1 motor neuron disease (MND). We have been speaking to people living with the disease, patient advocates and stakeholders across the community to establish how we can help the situation as quickly as possible.

You may have seen recent coverage of this drug, which is the first truly effective life-extending treatment for people living with SOD1-MND.

The drug is licensed in the UK but still needs to be approved for NHS use by the National Institute of Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC). While this process goes on, the manufacturer, Biogen, is making the drug free of charge to eligible patients on a ‘named patient basis’. This means NHS Trusts and Health Boards can choose to make the drug available and currently, only some have done so. This postcode lottery is preventing some eligible patients accessing the treatment. This is a particular issue in Scotland, where no Health Boards are currently offering it at all.

We believe this is unacceptable and we’ve been looking urgently into how we can help resolve it. We have also been establishing exactly how many people this affects, and where in the country it is a problem. 

We are taking three steps to address this: 

1. Earmarking funding to provide practical interim support to NHS Trusts and Health Boards to enable delivery of tofersen until it has been approved by NICE/SMC
2. Working with senior management and doctors at NHS Trusts and Health Boards, to support the expansion of existing services and the establishment of new ones
3. Seeking to meet key politicians in Westminster and Holyrood to highlight the current situation and provide solutions for them to help us address the situation 

If you or a family member is living with SOD1-MND and cannot currently access tofersen, please get in touch at research@myname5doddie.co.uk

If you are an MND clinician and could establish or expand your tofersen service with additional support or funding, please also get in touch at research@myname5doddie.co.uk

We believe research has the capacity to change the reality of an MND diagnosis. But the whole system has to work together to make this possible.