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:
- Prioritised
- Prioritisation programme:
- Medicines evaluation
- 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
- Rationale:
- Prioritisation Board confirmed that all of the criteria for this to be considered as a highly specialised technology were met.
- 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
- Department of Health and Social Care
- Health Technology Wales
- NHS England
- Patient carer groups
- Motor Neurone Disease Association
- My Name’5 Doddie Foundation
- Professional groups
- Association of British Neurologists
- Royal College of Physicians
- Royal College of Radiologists
- UK Clinical Pharmacy Association
- Associated public health groups
- None
- Comparator companies
- Accord-UK (riluzole) (confidentiality agreement not signed, not participating)
- Glenmark Pharmaceuticals (riluzole) (confidentiality agreement not signed, not participating)
- Martindale Pharma (riluzole) (confidentiality agreement not signed, not participating)
- Ranbaxy (riluzole) (confidentiality agreement not signed, not participating)
- Sanofi (riluzole) (confidentiality agreement not signed, not participating)
- Sun Pharma UK (riluzole) (confidentiality agreement not signed, not participating)
- Zambon UK (riluzole) (confidentiality agreement not signed, not participating)
- General commentators
- All Wales Therapeutics and Toxicology Centre
- British National Formulary
- Department of Health - Northern Ireland
- Healthcare Improvement Scotland
- Medicines and Healthcare products Regulatory Agency
- NHS Wales Joint Commissioning Committee
- Scottish Medicines Consortium
- Welsh Government
- Relevant research groups
- None
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