Action against NICE’s decision on tofersen

Last week, we heard that despite an appeal and evidence being submitted by leading MND clinicians, National Institute for Health and Care Excellence (NICE) confirmed its decision to appraise the drug tofersen through the Single Technology Appraisal (STA) route. This means that it’s highly unlikely that tofersen will be approved for use in England and Wales.

Tofersen is the first treatment for MND that has effectively slowed down and even stopped disease progression in some people living with MND. NICE’s decision is therefore extremely perplexing and sets a worrying example for future precision medicines entering the UK market.

What has happened since our announcement last week?

• The UK MND Clinical Studies Group have issued an open letter to NICE asking it to reconsider its decision. As a member of the CSG, we fully support this letter.
• My Name’5 Doddie Foundation has continued to work closely with MND Association and MND Scotland to take action against the decision, we met earlier this week to coordinate our activities.
• We are making our international colleagues aware of the situation, through our membership of the International Alliance of ALS/MND Associations.
• We attended the All-Party Parliamentary Group on MND last week, organised by the MND Association, where this issue was raised and were pleased to see support from its members.
• Along with the other UK MND charities, we continue to engage with Biogen, the company that has made tofersen.

Our next steps

We will continue to work with all those who share our frustration and anger with this decision and will ensure that we combine our influence and voice to take action on behalf of the MND community.