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Statement in response to NICE decision regarding tofersen

13 March 2024

Last week we heard the extremely disappointing news that the National Institute for Health and Care Excellence (NICE) has confirmed its decision to appraise the drug tofersen through the Single Technology Appraisal (STA) route rather than the Highly Specialised Technologies (HST) route.

Tofersen, developed by Biogen, is targeted to treat people living with MND who have alterations in the SOD1 gene. This is approximately 2% of the MND population, estimated at 60-100 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 has confirmed it will conduct a standard appraisal on the basis that it does not view SOD1 MND as a clinically distinct disease.

This decision goes against advice from patient representative organisations, including My Name'5 Doddie Foundation, and leading MND clinical experts, which have made extensive representations to NICE throughout the process. Unfortunately, we have been advised it is highly unlikely that tofersen will receive a positive decision if appraised through the standard route, meaning that it will not become accessible to patients through the NHS.

Although the chances of changing NICE’s decision are small, My Name'5 Doddie Foundation will raise this matter further with NICE and with the Government. We believe the decision calls into question the fitness for purpose of NICE’s processes, particularly for precision medicine treatments targeted at small sub-populations of patients.

We are, of course, acting as quickly as possible. Tofersen has proven effective in clinical trials at slowing the progression of MND in people with the SOD1 alteration. We want people with MND to be able to access effective treatments as quickly as possible, including those with SOD1 MND.

We believe the decision has further reaching consequences too. It goes against the Government’s desire to be a leader in genomics and personalised medicine. It has serious implications for precision medicine - not just in MND but other diseases where there are proven treatments for small groups of patients.

It should also be noted that tofersen has not yet received marketing authorisation in the UK and an application has not yet been submitted to the Medicines and Healthcare products Regulatory Agency (MHRA). As such, it is possible that the decision by NICE regarding the route of appraisal could affect whether an application is forthcoming.

People living with MND across the UK took part in the clinical trials of tofersen and are part of its success story, but NICE’s decision means that this may not now translate to access for people in England and Wales.

In Scotland, tofersen will be appraised by the Scottish Medicines Consortium which has yet to release details of how it plans to conduct its appraisal.

Biogen has confirmed the following: At the current time, the Early Access Programme and ongoing tofersen trials remain open to all eligible patients. No decision has been made to close these programmes.

Statement in response to NICE decision regarding tofersen
Statement in response to NICE decision regarding tofersen

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