The UK MND Clinical Studies Group has written to the National Institute for Health and Care Excellence (NICE), expressing concern over the decision to appraise the drug Tofersen through the Single Technology Appraisal route, and the implications this will have for people living with motor neuron disease in the UK. You can also view the letter here.

Dear Dr Roberts, Prof Benger, Ms Knight and Ms Nebhrajani

Re: Tofersen appraisal route decision

The UK Motor Neuron Disease Clinical Studies Group (UK MND CSG) is disappointed and perplexed by the decision taken by the National Institute for Health and Care Excellence (NICE) to appraise the drug Tofersen through the Single Technology Appraisal (STA) route rather than the Highly Specialised Technologies (HST) route. The UK MND CSG members include people living with MND, carers of people living with MND, all the major UK MND charities and representatives from 27 MND specialist clinics from around the UK.

Tofersen is a precision medicine treatment developed by Biogen for the 1-2% of people with MND who have the condition due to a change in the SOD1 gene. Patients from all over the UK travelled to Sheffield and London to take part in the clinical trials that assessed the treatment’s safety and efficacy. These trials were carried out in collaboration with NIHR Biomedical Research Centres and NIHR Clinical Research Facilities, with the UK MND Clinical trial network helping to identify patients for the trial. The clinical trials demonstrated that Tofersen slows down disease progression in SOD1 ALS.

The number of patients in the UK currently who could benefit from Tofersen is estimated to be no more than 100 at any one time. In England the standard practice is to appraise treatments that target rare diseases that affect less than 1 in 50,000 through a Highly Specialised Technology (HST) route. However, NICE has decided to review Tofersen through the Single Technology Appraisal (STA) route. NICE based this decision on its opinion that SOD1 MND is not a clinically distinct disease. This is an outdated concept in the current era when it is recognised that disease classifications based solely on consideration of clinical features are an inadequate way of making an accurate, and indeed even ‘precision’, diagnosis that lends itself to targeted ‘precision treatment’. It is completely at variance with the overwhelming consensus of clinicians and scientists working globally in neurodegenerative diseases that SOD1 is a distinct entity with a completely different pathological basis to the other 98% of MND. Biogen has requested an appraisal by NICE for SOD1 disease only and would seek marketing approval for SOD1 MND only.

This decision matters because it makes it highly unlikely that Tofersen will receive a positive appraisal from NICE. Even if Biogen was to give Tofersen away for free to the NHS, economic modelling indicates that it would fail the NICE Single Technology Appraisal. We understand as a result of this decision, Biogen is unlikely to submit an application to the MHRA for market approval or start the STA process. This means Tofersen will be unavailable to SOD1 MND patients in the UK. This is an unacceptable situation given the contribution UK patients and researchers have made to the development of this effective treatment, and given the approvals and availability of Tofersen for SOD1 patients in the United States and Europe.

Of further concern is the chilling impact this will have on pharmaceutical companies to bring their future innovative science and clinical trials to the UK, given the perception it will create that there is no effective route to market for precision medicine or rare disease therapies. This is completely contrary to the UK Government’s stated ambition for the Life Sciences sector [1].

We urgently request that NICE reconsider the appraisal routing decision to enable the 100 patients with SOD1 disease living in the UK to have access to Tofersen as soon as possible and to ensure that those already receiving it do not have their access to it stopped.

Yours Sincerely,

Chris McDermott, Chair UK MND Clinical Studies Group, Co-Director UK MND Research Institute
Ammar Al-Chalabi, UK MND Clinical Studies Group, Co-Director UK MND Research Institute
Brian Dickie, UK MND Clinical Studies Group, Motor Neurone Disease Association
John Ealing, UK MND Clinical Studies Group, Consultant Neurologist
Jane Haley, UK MND Clinical Studies Group, MND Scotland
Jessica Lee, UK MND Clinical Studies Group, My Name’5 Doddie Foundation
Karen Morrison, UK MND Clinical Studies Group, Consultant Neurologist
Ashwin Pinto, UK MND Clinical Studies Group, Consultant Neurologist
Aleksandar Radunovic, UK MND Clinical Studies Group, Consultant Neurologist
Pamela Shaw, UK MND Clinical Studies Group, Executive Board UK MND Research Institute
Kevin Talbot, UK MND Clinical Studies Group, Executive Board UK MND Research Institute
Martin Turner, UK MND Clinical Studies Group, Executive Board UK MND Research Institute
Tim Williams, UK MND Clinical Studies Group, Consultant Neurologist
Stacy Young, UK MND Clinical Studies Group

On behalf of the UK MND Clinical Studies Group and UK MND Research Institute

[1] https://www.great.gov.uk/international/content/investment/sectors/precision-medicine/