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

Project Team

Project lead Emily Richards

Email enquiries

Timeline

Key events during the development of the guidance:

Date Update
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 Following a challenge received from the company in September 2023, NICE sought further clinical opinion regarding the topic routing. We have concluded that Tofersen for SOD1-ALS fails to meet HST criterion 1 “The disease is very rare defined by 1:50,000 in England, and the topic continues to be routed to the Technology Appraisal (TA) programme." Please note that following on from advice received from the company, further information regarding the timelines for this appraisal will be available in due course. In the meantime, NICE will continue to monitor the topic and will provide an update as and when the situation changes.
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 The topic routing was discussed at the Topic Selection Oversight Panel (TSOP) in April 2023. The panel concluded that the topic was suitable for a Technology Appraisal. Please see project documents for further details.
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