Suggested remit: To appraise the clinical and cost effectiveness of tofersen within its marketing authorisation for treating amyotrophic lateral sclerosis caused by superoxide dismutase 1 (SOD1) gene mutations.
- Status:
- In progress
- Technology type:
- Medicine
- Decision:
- Selected
- Reason for decision:
- Anticipate the topic will be of importance to patients, carers, professionals, commissioners and the health of the public to ensure clinical benefit is realised, inequalities in use addressed, and help them make the best use of NHS resources
- Process:
- HST Standard
- ID number:
- 3767
Provisional Schedule
- Committee meeting: 1:
- 22 October 2026
- Expected publication:
- 17 March 2027
Project Team
- Project lead
- Thomas Feist
Email enquiries
If you have any queries please email HST@nice.org.uk
- External Assessment Group:
- BMJ Technology Assessment Group (BMJ-TAG), BMJ
Stakeholders
- Companies sponsors
- Biogen
- Others
- Barts and London MND Care Centre
- Birmingham MND Care Centre
- Bristol MND Care Centre
- Cambridge MND Care Centre
- Department of Health and Social Care
- Derby MND Care Centre
- King’s MND Care and Research Centre, King’s College Hospital
- Lancashire and South Cumbria MND Care Centre
- Leeds MND Care Centre
- Liverpool MND Care Centre
- Manchester MND Care Centre
- Middlesbrough MND Care Centre
- National MND Centre
- Newcastle MND Care and Research Centre
- NHS England
- Norfolk MND Care Network
- North Midlands MND Care Network
- Nottingham MND Care Centre
- Oxford MND Care Centre
- Sheffield MND Care Centre
- South West Peninsula MND Care Network
- Southampton MND Care Centre
- St Georges Hospital Care and Research Network
- Sussex MND Care and Research Network
- Willink Unit, Genetic Medicine, Central Manchester Foundation
- Patient carer groups
- Beacon
- Brain and Spine Foundation
- Brain Charity
- Gene People
- Genetic Alliance UK
- Motor Neurone Disease Association
- My Name’5 Doddie Foundation
- Neurological Alliance
- South Asian Health Foundation
- Specialised Healthcare Alliance
- Sue Ryder
- Professional groups
- Association of British Neurologists
- British Association of Neuroscience Nurses
- British Geriatrics Society
- British Neuropathological Society
- British Neuropsychiatry Association
- British Society of Rehabilitation Medicine
- British Thoracic Society
- Chartered Society of Physiotherapy
- Institute of Neurology
- Institute of Psychiatry
- Primary Care Neurology Society
- Royal College of General Practitioners
- Royal College of Nursing
- Royal College of Occupational Therapists
- Royal College of Pathologists
- Royal College of Physicians
- Royal College of Radiologists
- Royal College of Speech and Language Therapists
- Royal Pharmaceutical Society
- Royal Society of Medicine
- UK Clinical Pharmacy Association
- Associated public health groups
- Public Health Wales
- UK Health Security Agency
- Comparator companies
- Accord-UK (riluzole)
- Glenmark Pharmaceuticals (riluzole)
- Martindale Pharma (riluzole)
- Ranbaxy (riluzole)
- Sanofi (riluzole)
- Sun Pharma UK (riluzole)
- Zambon UK (riluzole)
- General commentators
- All Wales Therapeutics and Toxicology Centre
- Allied Health Professionals Federation
- Board of Community Health Councils in Wales
- British National Formulary
- Care Quality Commission
- Department of Health - Northern Ireland
- Healthcare Improvement Scotland
- Medicines and Healthcare products Regulatory Agency
- National Association of Primary Care
- National Pharmacy Association
- Neurological Alliance of Scotland
- NHS Confederation
- NHS Wales Joint Commissioning Committee
- Scottish Medicines Consortium
- Wales Neurological Alliance
- Welsh Government
- Relevant research groups
- Brain Research UK
- Cochrane Airways group
- Cochrane Cystic Fibrosis and Genetic Disorders Group
- Cochrane Movement Disorders Group
- Cochrane UK
- Genomics England
- MND Scotland
- MRC Clinical Trials Unit
- National Hospital for Neurology and Neurosurgery
- National Institute for Health Research
- Oxford Motor Neuron Disease Centre
- UCL Institute of Neurology
Timeline
Key events during the development of the guidance:
| Date | Update |
|---|---|
| 25 March 2026 | Invitation to participate |
| 24 February 2026 - 24 February 2026 | Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators |
| 14 January 2026 | Note - Note added to the project documents |
| 16 December 2025 | Note - Note added to the project documents |
| 18 November 2024 | Topic selection |
| 18 November 2024 | In progress. Topic routing was discussed at the NICE Prioritisation Board in October 2024. The Board concluded that the topic was suitable for a Highly Specialised Technology. Please see HST checklist on the project documents tab for further details. |
| 05 September 2024 | Suspended. The company has informed NICE that it will not provide an evidence submission for this appraisal at this time. Therefore, we are suspending the appraisal while we consider the next steps. |
| 26 March 2024 | Awaiting development. Status change linked to Topic Selection Decision being set to Selected |
| 22 February 2024 | Note added to the project documents |
| 15 September 2023 | Awaiting development. Status change linked to Topic Selection Decision being set to Selected |
| 15 September 2023 | Topic selection. Following a challenge from the company the routing was re-considered by the Topic Selection Oversight Panel (TSOP) in August 2023. The panel concluded that the topic should remain routed to the Technology Appraisal (TA) work programme. Please see project documents for further details. The evaluation timelines remain TBC pending an update from the company. |
| 01 June 2023 | Note - Note added to the project documents |
| 01 June 2023 | Topic selection |
| 16 March 2023 (10:00) | Scoping workshop |
| 16 January 2023 - 13 February 2023 | Consultation on suggested remit, draft scope and provisional matrix of consultees and commentators: 3767 |
| 16 January 2023 | In progress. In progress. Scoping commencing |
| 17 October 2022 | Awaiting development. Please note that following on from information received from the company this topic has now been scheduled into the work programme. We anticipate that it will begin during early-July 2023. |
| 11 November 2021 | Suspended. The National Institute for Health and Care Excellence (NICE) has been asked to conduct an evaluation of tofersen for treating amyotrophic lateral sclerosis caused by SOD1 gene mutations [ID3767]. We recently incorrectly invited stakeholders to respond to a written consultation on the draft scope and to attend a scoping workshop for this appraisal. To confirm NICE will not be progressing with the scoping exercise at this time. The consultation on the draft scope will now close and the workshop arranged for Friday 17 December 2021 has been cancelled. Please accept our apologies for any inconvenience. |
For further information on our processes and methods, please see our CHTE processes and methods manual