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Open letter to NICE and Biogen regarding tofersen

02 May 2024

My Name’5 Doddie Foundation has today (May 2) sent an open letter to Dr Sam Roberts, CEO and Helen Knight, Director of Medicines Evaluation at the National Institute for Health and Care Excellence (NICE) calling for the organisation to enter talks with biotechnology company Biogen to find a way to bring tofersen – a drug shown to slow the progress of a form of motor neuron disease –  to people with SOD1-MND in the UK via the NHS:

Dear Dr Roberts and Mrs Knight,

Re: Plea for NICE to enter into a dialogue with Biogen to find a way to bring tofersen to people with SOD1-MND in the UK

Motor neuron disease (MND) is a rapidly progressing terminal neurodegenerative disease, for which there are currently no effective approved treatments in the UK. Tofersen is the first treatment for MND that has demonstrated significant slowing of disease progression, but only in patients carrying a mutation in the SOD1 gene (“SOD1-MND”). Tofersen has been a great success story for the UK, which was instrumental in delivering its phase 3 clinical trial. However, with NICE’s decision to appraise tofersen through the Single Technology Appraisal (STA) route instead of the Highly Specialised Technologies (HST) route, patients with SOD-1 MND in the UK now risk not being able to access this life changing treatment through the NHS.

The problem with NICE’s routing decision

NICE has stated that it will not reconsider its routing decision as its Topic Selection Oversight Panel does not consider SOD1-MND as a clinically distinct subgroup of MND. Whether or not this is the case, the simple fact is that tofersen will only be prescribed to people with SOD1-MND. This equates to approximately 60-100 people in England at any time.

Whilst we completely agree with the need to ensure medicines are cost effective and affordable to the NHS, the decision to appraise tofersen via the STA route means that it is highly likely that it will be deemed not cost effective. Biogen, the company that makes tofersen, has conducted economic modelling that shows that even if tofersen was priced at £0, it would not be deemed cost effective through STA appraisal.

The implications of this decision beyond MND

Due to advances in genetics and our understanding of human disease, we are now in a new era of precision medicine, where therapies are being developed for patients dependent on their specific genetic makeup. Tofersen is an exemplar of a precision medicine, which is transforming the lives of people with SOD1-MND. Like many precision medicines, it is only suitable for the treatment of small subgroups of patients. For pharmaceutical companies, who we depend on to develop these life changing treatments, appropriate reimbursement models that balance both commercial needs and the needs of the NHS, are essential.

As more precision medicines come through the pipeline, NICE must have the processes in place to properly assess them and reach agreements regarding an appropriate price. NICE’s decision on the routing of tofersen is incredibly worrying and threatens the future ability of the UK to respond to this new era of precision medicine.

As a UK charity that funds research into the development of precision medicines, this is alarming. We will be raising this issue at the highest level with other funders and organisations working in this space and sharing this letter publicly with our supporters and the media.

What can NICE do to help?

We urge NICE to enter into a discussion with Biogen regarding how a fair decision can be reached on the appraisal and pricing of tofersen. We completely understand that negotiations will need to take place to reach a fair price. However, Biogen needs to receive assurance from NICE that these negotiations will take place if they apply through the STA route, otherwise it makes no commercial or financial sense for them to apply.

People with MND do not have time to wait. From date of diagnosis, one third of people with MND die within a year and half die within two years. Time is of the essence, lives are in your hands.

Please act now and start a dialogue with Biogen to agree a way forward.

Yours sincerely,

Nicola Roseman, CEO, My Name'5 Doddie Foundation

Jessica Lee, Director of Research, My Name'5 Doddie Foundation

Sean McGrath, Medical Affairs Lead, My Name'5 Doddie Foundation

Open letter to NICE and Biogen regarding tofersen
Open letter to NICE and Biogen regarding tofersen

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