Is the UK government going to prevent me from continuing to receive a life saving treatment?
Last week, The National Institute for Health and Care Excellence (NICE) refused to accept an application from the pharmaceutical giant, Biogen, to follow the most suitable pathway for assessing the marketing/licensing of its proven, effective and life changing treatment, Tofersen, for motor neurone disease (MND) specifically caused by a mutation in the SOD1 gene.
Already approved in the USA and in Europe, the refusal is a body blow to patients and a potentially monumental setback for personalised medicine in the UK. Above all, it sends out the message that the UK is not open for business to the world’s leading pharmaceutical companies and their innovative and proven treatments. It is even more shocking given that leading UK research establishments have been at the heart of the trials for this treatment.
Eleanor Dalley, who lives with the specific genetic mutation and is accessing the drug via an early access program (EAP), funded by Biogen, says
I’m the fourth person in my family with SOD1 MND and the only one still living. This treatment was the miracle my family have prayed for but now it looks like it could be withdrawn. This decision makes me so angry. Are our lives worth so little? The UK system is failing us!
For NICE to ignore the results of the trial and the leading UK neuroscientists is unbelievable. It is the first treatment in 30 years shown to work by prolonging lives. It is likely to prevent people from developing SOD1 MND in future, including my daughter and other family members. If this lifeline drug does not get the necessary approvals, hope dies for helping future generations.
I am very lucky to be receiving Tofersen via the EAP and haven’t had any further progression since I began taking it. But what if Biogen stops the EAP? I am sick to my stomach worrying about it.
Eleanor Dalley
Nicola Waters from the advocacy group, Patients United 2 End MND, attended the public NICE meeting on 20th March and asked if the board would reconsider its decision.
The response was short and to the point. NICE will not reconsider the decision, saying that they had taken expert clinical advice. However, leading neuroscientists disagreed with this pointing out that their advice was entirely against the decision. They sent a strongly worded letter to the organisation.
SOD1 patients and Patients United have been buoyed with the support of Geoff Burrow father of rugby league player, Rob, who lives with the disease and does so much to raise awareness and funds for it.
Rob does not have the SOD1 gene, but the Burrow family is united with all of those living with MND. The fact that Tofersen works, even if only for a small percentage of patients, is a major breakthrough showing a pathway to discover other drugs targeted at specific genes known to be involved. We must change the NICE decision and give hope to the whole MND community
Geoff Burrow
Patients United are now seeking an urgent meeting with Health Ministers to ensure that the patient voice is heard and that the current decision is reversed.
We will continue to work with the charities and leading UK scientists in a joint effort to get the NICE decision changed. We are completely behind the UK MND Research Institute co-directors Professors Ammar Al-Chalabi and Chris McDermott in seeking a meeting with Minister of State with responsibility for NICE, the Rt Hon Andrew Stephenson MP
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