Suggested remit: To appraise the clinical and cost effectiveness of nusinersen and risdiplam within their marketing authorisations for treating spinal muscular atrophy.
- 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
- Further information:
- Scoping commenced.
- Process:
- MTA Standard
- ID number:
- 6195
Provisional Schedule
- Committee meeting:
- 11 November 2025
Project Team
- Project lead
- Kate Moore
Email enquiries
If you have any queries please email [email protected]
- External Assessment Group:
- Newcastle NIHR TAR Team, Newcastle University
Stakeholders
- Companies sponsors
- Biogen (Nusinersen)
- Roche (Risdiplam)
- Others
- Department of Health and Social Care
- NHS England
- Patient carer groups
- Gene People
- Muscular Dystrophy UK
- Spinal Muscular Atrophy UK
- TreatSMA
- Professional groups
- Adult SMA Reach
- Association of British Neurologists
- British Paediatric Respiratory Society
- British Society of Physical and Rehabilitation Medicine
- Royal College of Physicians
- SMA Reach UK
- UK National Screening Committee
- Comparator companies
- Novartis Gene Therapies, Inc. (onasemnogene abeparvovec) (confidentiality agreement signed, participating)
- General commentators
- All Wales Therapeutics and Toxicology Centre
- British National Formulary
- Department of Health, Social Services and Public Safety for Northern Ireland
- Healthcare Improvement Scotland
- Medicines and Healthcare products Regulatory Agency
- NHS Wales Joint Commissioning Committee
- Welsh Government
Timeline
Key events during the development of the guidance:
| Date | Update |
|---|---|
| 29 July 2025 | Assessment report sent for information |
| 07 May 2025 | There has been an update to the timelines for this appraisal following a request from the EAG for additional time to complete their report. As a result, the consultation on the EAG report will now take place between 21 July and 10 September, with the first committee meeting taking place on 11 November 2025. |
| 11 November 2024 | The committee meeting planned for 4 December 2024 will not go ahead. After considering the comments received on the External Assessment Group’s (EAG) report, NICE has decided to restart the evidence critique stage, and has asked Newcastle EAG to produce a new report. This work is expected to be completed by the end of May 2025. We therefore anticipate that a first committee discussion will now be September 2025. Access to treatments will continue while the appraisal is ongoing for people starting the treatments as well as those already taking the treatments. |
| 05 January 2024 | Invitation to participate |
| 28 July 2023 - 25 August 2023 | Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators |
| 28 July 2023 | In progress. Scoping commenced. |
For further information on our processes and methods, please see our CHTE processes and methods manual