Suggested remit: To appraise the clinical and cost effectiveness of intrathecal onasemnogene abeparvovec within its marketing authorisation 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
- Process:
- STA Standard
- ID number:
- 6556
Provisional Schedule
- Committee meeting: 1:
- 07 July 2026
- Expected publication:
- 26 August 2026
- Expected publication:
- 28 October 2026
Project Team
- Project lead
- Leena Issa
Email enquiries
If you have any queries please email TACommC@nice.org.uk
- External Assessment Group:
- BMJ Technology Assessment Group (BMJ-TAG), BMJ
Stakeholders
- Companies sponsors
- Novartis Pharmaceuticals UK (onasemnogene abeparvovec)
- Others
- Department of Health and Social Care
- NHS England
- Patient carer groups
- ACE SMA
- Ally Cadence Trust for Spinal Muscular Atrophy
- Arthritis and Musculoskeletal Alliance
- Brain and Spine Foundation
- Brain Charity
- Gene People
- Genetic Alliance UK
- Jnetics
- Muscular Dystrophy UK
- Neurological Alliance
- Pathfinders Neuromuscular Alliance
- Rally Round Rupert
- South Asian Health Foundation
- Specialised Healthcare Alliance
- Spinal Muscular Atrophy UK
- The Annabelle Rose Foundation for Spinal Muscular Atrophy
- TREAT-SMA
- Professional groups
- Adult SMA Reach
- Association of Anaesthetists
- Association of British Neurologists
- Association of Chartered Physiotherapists in Neurology
- Association of Genetic Nurses and
- Counsellors
- Association of Paediatric Chartered Physiotherapists
- Association of Surgeons of Great Britain and Ireland
- British Dietetic Association
- British Geriatrics Society
- British Myology Society
- British Neuropathological Society
- British Orthopaedic Association
- British Paediatric Neurology Association
- British Society for Children's Orthopaedic Surgery
- British Society for Gene and Cell Therapy
- British Society for Genetic Medicine
- British Society for Rheumatology
- British Society of Physical and Rehabilitation Medicine
- Chartered Society of Physiotherapy
- Institute of Neurology
- Institute of Neurology and Neurosurgery
- National Neuroscience Advisory Group
- Neonatal and Paediatric Pharmacists Group
- Primary Care and Community Neurology Society
- Royal College of Anaesthetists
- Royal College of General Practitioners
- Royal College of Nursing
- Royal College of Paediatrics and Child Health
- Royal College of Pathologists
- Royal College of Physicians
- Royal College of Surgeons
- Royal Pharmaceutical Society
- Royal Society of Medicine
- SMA Reach UK
- Society for Endocrinology
- Treat-NMD
- UK Clinical Pharmacy Association
- Associated public health groups
- Public Health Wales
- UK Health Security Agency
- Comparator companies
- Biogen (nusinersen)
- Novartis Pharmaceuticals UK (onasemnogene abeparvovec) [intravenous infusion]
- Roche (risdiplam)
- 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
- Bone Research Society
- Brain Research UK
- Chronic Pain Policy Coalition
- Cochrane Musculoskeletal Group
- Genomics England
- John Walton Muscular Dystrophy Research Centre
- MRC Centre for Neuromuscular Diseases
- MRC Clinical Trials Unit
- National Hospital for Neurology and Neurosurgery
- National Institute for Health Research
- Orthopaedic Research UK
Timeline
Key events during the development of the guidance:
| Date | Update |
|---|---|
| 24 November 2025 | Invitation to participate |
| 02 October 2025 | To appraise the clinical and cost effectiveness of OAV101 IT within its marketing authorisation for treating spinal muscular atrophy ID6556 |
| 04 August 2025 - 02 September 2025 | Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators: 6556 |
| 04 August 2025 | In progress. Scoping commencing |
| 08 April 2025 | Awaiting development. Status change linked to Topic Selection Decision being set to Selected |
For further information on our processes and methods, please see our CHTE processes and methods manual