Suggested remit: To appraise the clinical and cost effectiveness of nusinersen and risdiplam within their marketing authorisations for treating spinal muscular atrophy.
Status
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In progress
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Technology type
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Medicine
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Decision
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Selected
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Reason for decision
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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
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Further information
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Scoping commenced.
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Process |
MTA Standard
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ID number |
6195
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Project Team
Project lead |
Louise Jafferally |
Email enquiries
External Assessment Group |
Newcastle NIHR TAR Team, Newcastle University |
Stakeholders
Companies sponsors |
Biogen (Nusinersen) |
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Roche (Risdiplam) |
Others |
Department of Health and Social Care |
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NHS England |
Patient carer groups |
Gene People |
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Muscular Dystrophy UK |
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Spinal Muscular Atrophy UK |
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TreatSMA |
Professional groups |
Adult SMA Reach |
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Association of British Neurologists |
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British Paediatric Respiratory Society |
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British Society of Physical and Rehabilitation Medicine |
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Royal College of Physicians |
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SMA Reach UK |
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UK National Screening Committee |
Comparator companies |
Novartis Gene Therapies, Inc. (onasemnogene abeparvovec) (confidentiality agreement signed, participating) |
General commentators |
All Wales Therapeutics and Toxicology Centre |
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British National Formulary |
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Department of Health, Social Services and Public Safety for Northern Ireland |
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Healthcare Improvement Scotland |
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Medicines and Healthcare products Regulatory Agency |
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Welsh Government |
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Welsh Health Specialised Services Committee |
Date
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Update
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11 November 2024
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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
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Invitation to participate |
28 July 2023 - 25 August 2023
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Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators |
28 July 2023
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In progress. Scoping commenced. |
For further information on our processes and methods, please see our CHTE processes and methods manual