21 March 2024 News

Tofersen is a proven, effective treatment. Clinical trials have shown it slows progression of MND in people who have the SOD1 gene alteration.  

That’s why there is so much confusion, upset and anger about the decision of the National Institute for Health and Care Excellence (NICE) to appraise the drug through the Single Technology Appraisal (STA) route rather than the Highly Specialised Technologies (HST) route. 

The decision has been based on the fact it doesn’t view SOD1 MND as a clinically distinct disease – against advice from leading MND clinical experts and organisations including the MND Association representing people with MND.

We’re already taking action.

  • Our Director of Research Development Dr Brian Dickie, is a member of the UK MND Clinical Studies Group and added his signature to the group’s strongly worded open letter to NICE urgently requesting it ‘reconsider the appraisal routing decision’. The group includes leading neurologists, healthcare professionals, charity representatives and patient representatives.
  • We attended NICE’s public board meeting earlier this week and were very disappointed to hear NICE say its decision was final. We were also perplexed by the reasoning given for the decision.
  • Tofersen is already approved for use in the US and has been recommended for approval in Europe. We are talking to colleagues at the International Alliance of ALS/MND Associations and are set to discuss the situation with our peers in other MND organisations around the world to understand and learn from their experiences.
  • We continue to engage regularly with Biogen and have requested a further meeting next week.
  • We raised the issue at the All Party Parliamentary Group on MND last week and were pleased to receive a supportive response. We are now following this up with further discussions.  
  • We are pursuing a number of other routes and will provide updates when we have them.

We are very grateful to all those people and organisations who have shared their dismay, frustration and anger at this decision, bringing it to wider attention. We will work with all those people who can help us raise this issue at the highest level.