Following the recent successful ‘Prescribe Life’ campaign driven by the MND Association, which was supported by MND Scotland and My Name’5 Doddie Foundation, we welcome the announcement this morning from NICE that they will now appraise the drug tofersen via the Highly Specialised Technologies (HST) route.

NICE is the National Institute for Health and Care Excellence. NICE evaluates new health technologies for NHS use in England and Wales, considering clinical effectiveness and value for money.   

Tofersen, developed by Biogen, is targeted to treat people living with motor neuron disease (MND) who have alterations in the SOD1 gene. This is approximately 2% of the MND population, estimated at between 60 and 100 people in the UK.  

Ordinarily products targeted to rare diseases that affect less than 1 in 50,000 people in England are appraised through the HST route, but NICE had indicated that it would only conduct a standard appraisal on the basis that it does not view SOD1 MND as a clinically distinct disease. This would have meant that tofersen was unlikely to receive a positive outcome. NICE have now changed this decision. 

Whilst this change to NICE’s decision is very welcome, there is still a long way to go to make tofersen available to people with SOD1 MND in the UK.  

All drugs that are prescribed in the UK must have marketing authorisation by the Medicines and Healthcare products Regulatory Agency (MHRA). If this is achieved, then decisions need to be made about whether the drug will be made available on the NHS. In Scotland, this decision is taken by the Scottish Medicines Consortium which has yet to release details on how it plans to conduct its appraisal of tofersen. 

The MND charities recently produced a series of infographics outlining how the clinical trial and drug regulatory approval process works, and you can view those here. 

The three MND charities will be meeting with Biogen in the coming weeks for an update on their position and timelines. We hope that the removal of the blockage at NICE will encourage them to present an application to MHRA soon.  

In the meantime, the tofersen Early Access Programme in the UK, which is made available by Biogen, remains open to eligible patients. 

Dr Jane Haley, Director of Research at MND Scotland, said: “This is a very encouraging outcome and we congratulate the MND Association on their very successful ‘Prescribe Life’ campaign which MND Scotland was happy to support. We’d also like to thank NICE for re-considering their decision. While there is still some way to go, we hope that this is an important step in making tofersen available to people with SOD1 MND in the UK.” 

The three main UK MND charities are committed to working collaboratively to pursue every avenue available to us to make proven treatments available to people with MND as quickly as possible and on an equitable basis.