The Rt Hon Andrew Stephenson CBE MP
Minister of State for Health and Secondary Care Department of Health and Social Care

27 March 2024

Dear Minister,

We are writing to seek an urgent meeting with you to discuss new treatments being developed by UK researchers for motor neuron disease (MND) and our concern that we will not be able to make them available to UK patients.

MND is a terminal illness which causes progressive paralysis affecting limbs, voice, eating and eventually breathing. One in every 300 people develops MND at some point, and people typically die within two years. Research is at an exciting stage: we are on the cusp of effective treatments. Much of this work is led by UK scientists, now working together through the UK MND Research Institute. The formation of the MND Research Institute in 2023, through £50 million funding from the UK Government, should herald a new era of investment by pharmaceutical companies and new scientific breakthroughs.

However, a specific case has prompted us to seek an urgent meeting with you now. Tofersen is a treatment developed and trialled at UK universities (Sheffield and London) alongside universities internationally, with investment from US pharmaceutical company, Biogen. It targets the 2% of people with a form of motor neuron disease caused by genetic variation in the SOD1 gene. The trial demonstrated that Tofersen greatly slows progression of the disease in those carrying this genetic change, with some even experiencing an apparent halt to their MND progression (some even possible reversal) and thriving long after they might have expected to have died. This remarkable result has understandably led to approval of Tofersen for people with SOD1 MND by the FDA and EMA.

Unfortunately, we have recently been informed that in the UK NICE will assess this treatment through its Single Technology Appraisal (STA) route rather than its Highly Specialised Technologies (HST) route. Effectively, NICE are assuming the treatment will be prescribed for all MND patients rather than the specific subset with the genetic mutation. Because of the impact of this mistaken route on the economic analysis, this approach not only makes it unlikely Tofersen will ever be approved, but completely undermines the concept of a precision medicine approach for MND. At a time when the UK is world-leading in MND research, this decision will make it more difficult for the UK and the MND Research Institute to attract investment from the pharmaceutical industry, reduce the likelihood of the UK being selected for future precision trials, and threaten the UK’s leadership in this field of research.

We would very much welcome the opportunity to discuss this pressing issue with you before the damage to UK patients and our research leadership is done.

Yours sincerely,

Ammar Al-Chalabi, PhD FRCP DipStat

Co-Director, UK MND Research Institute
NIHR Senior Investigator
Professor of Neurology and Complex Disease Genetics KCL

Christopher McDermott, PhD FRCP

Co-Director, UK MND Research Institute
NIHR Research Professor
Professor of Translational Neurology SITraN