Suggested remit: To appraise the clinical and cost effectiveness of olezarsen within its marketing authorisation for treating familial chylomicronaemia syndrome.
- 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:
- 6585
Provisional Schedule
- Committee meeting: 1:
- 25 June 2026
- Expected publication:
- 19 August 2026
Project Team
- Project lead
- Thomas Feist
Email enquiries
If you have any queries please email TAteam3@nice.org.uk
- External Assessment Group:
- Peninsula Technology Assessment Group (PenTAG), University of Exeter
Stakeholders
- Companies sponsors
- Sobi
- Others
- All Wales Inherited Metabolic Disease Service
- Central Manchester Foundation Trust, Willink Unit, Genetic Medicine
- Department of Health and Social Care
- Great Ormond Street Hospital Metabolic Unit
- Guys and St Thomas’ NHS Trust, Adult Inherited Metabolic Diseases
- Leeds Teaching Hospitals NHS Trust, St James’ Hospital, Diabetes
- NHS England
- NHS England National Programmes of Care, Internal Medicine: Hepatobiliary and Pancreas Clinical Reference Group (CRG)
- NHS Genomic Medicine Service
- Salford Royal NHS Foundation Trust Mark Holland Metabolic Unit
- Specialised Endocrinology CRG
- University Hospital Birmingham Foundation Trust, Department of Endocrinology
- University Hospitals of Bristol NHS Foundation Trust, Clinical Biochemistry
- Patient carer groups
- Action FCS
- Beacon
- Gene People
- Genetic Alliance UK
- Metabolic Support UK
- South Asian Health Foundation
- Specialised Healthcare Alliance
- Professional groups
- Association of Genetic Nurses and Counsellors
- British Dietetic Association
- British Geriatrics Society
- British Inherited Metabolic Disease Group
- British Society for Gene and Cell Therapy
- British Society for Genetic Medicine
- HEART UK
- National Metabolic Biochemistry Network
- Royal College of General Practitioners
- Royal College of Nursing
- Royal College of Pathologists
- Royal College of Physicians
- Royal Pharmaceutical Society
- Royal Society of Medicine
- Society for Endocrinology
- UK Clinical Pharmacy Association
- Associated public health groups
- Public Health Wales
- UK Health Security Agency
- Comparator companies
- Akcea Therapeutics (volanesorsen)
- Arrowhead Pharmaceuticals (plozasiran)
- 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
- Cell and Gene Therapy Catapult
- Department of Health - Northern Ireland
- Healthcare Improvement Scotland
- Medicines and Healthcare products Regulatory Agency
- National Association of Primary Care
- National Pharmacy Association
- National Services Division
- NHS Confederation
- NHS Wales Joint Commissioning Committee
- Scottish Medicines Consortium
- Welsh Government
- Relevant research groups
- Cochrane Cystic Fibrosis and Genetic Disorders Group
- Cochrane Metabolic and Endocrine Disorders Group
- Genomics England
- MRC Clinical Trials Unit
- National Institute for Health Research
- Society for The Study of Inborn Errors of Metabolism
Timeline
Key events during the development of the guidance:
| Date | Update |
|---|---|
| 12 November 2025 | Invitation to participate |
| 02 October 2025 | To appraise the clinical and cost effectiveness of Olezarsen within its marketing authorisation for treating familial chylomicronaemia syndrome ID6585 |
| 23 July 2025 - 20 August 2025 | Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators: 6585 |
| 23 July 2025 | In progress. Scoping commenced. |
| 30 January 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